Trial Outcomes & Findings for An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy (NCT NCT03532542)
NCT ID: NCT03532542
Last Updated: 2024-09-19
Results Overview
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
171 participants
Primary outcome timeframe
Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
Results posted on
2024-09-19
Participant Flow
Participants amenable to exon 53 or exon 45 skipping were enrolled into this study from Study 4045-101 (NCT02530905), Study 4053-101 (NCT02310906), and Study 4045-301 (NCT02500381).
Participant milestones
| Measure |
Golodirsen
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen intravenous (IV) infusions, weekly, at 30 milligrams/kilogram (mg/kg) for up to 144 weeks.
|
Casimersen
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
|---|---|---|
|
Overall Study
STARTED
|
74
|
97
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
74
|
97
|
|
Overall Study
COMPLETED
|
26
|
27
|
|
Overall Study
NOT COMPLETED
|
48
|
70
|
Reasons for withdrawal
| Measure |
Golodirsen
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen intravenous (IV) infusions, weekly, at 30 milligrams/kilogram (mg/kg) for up to 144 weeks.
|
Casimersen
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
|---|---|---|
|
Overall Study
Death
|
1
|
0
|
|
Overall Study
Study Terminated by Sponsor
|
41
|
65
|
|
Overall Study
Withdrawal by Subject
|
6
|
5
|
Baseline Characteristics
An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
Baseline characteristics by cohort
| Measure |
Golodirsen
n=74 Participants
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
Casimersen
n=97 Participants
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
Total
n=171 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
11.7 years
STANDARD_DEVIATION 2.14 • n=99 Participants
|
12.2 years
STANDARD_DEVIATION 2.57 • n=107 Participants
|
12.0 years
STANDARD_DEVIATION 2.40 • n=206 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
74 Participants
n=99 Participants
|
97 Participants
n=107 Participants
|
171 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
10 Participants
n=99 Participants
|
6 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
54 Participants
n=99 Participants
|
89 Participants
n=107 Participants
|
143 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
10 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
12 Participants
n=206 Participants
|
|
Race/Ethnicity, Customized
White
|
65 participants
n=99 Participants
|
86 participants
n=107 Participants
|
151 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Black
|
1 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Asian
|
4 participants
n=99 Participants
|
7 participants
n=107 Participants
|
11 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
American Indian or Alaska Native
|
1 participants
n=99 Participants
|
0 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Native Hawaiian or Other Pacific Islander
|
0 participants
n=99 Participants
|
1 participants
n=107 Participants
|
1 participants
n=206 Participants
|
|
Race/Ethnicity, Customized
Other
|
3 participants
n=99 Participants
|
3 participants
n=107 Participants
|
6 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)Population: Safety Analysis Set: All enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
A treatment-emergent adverse event (TEAE) was any untoward medical occurrence in a clinical study participant that did not necessarily have a causal relationship with the study drug. A TEAE could, therefore, be any unfavorable and unintended symptom, sign, disease, condition, or test abnormality that occurred during or after administration of the study drug, whether or not considered related to the study drug. A TESAE was any TEAE that resulted in any of the following outcomes: death, a life-threatening event, required or prolonged inpatient hospitalization, persistent or significant disability/incapacity, or an important medical event (that is, may have jeopardized the participant and may have required medical or surgical intervention to prevent one of the previously mentioned outcomes). A summary of serious and all other non-serious TEAEs regardless of causality is located in the Reported Adverse Events module.
Outcome measures
| Measure |
Golodirsen
n=74 Participants
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
Casimersen
n=97 Participants
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
|---|---|---|
|
Number of Participants With Treatment-emergent Serious Adverse Events (TESAEs)
|
12 Participants
|
22 Participants
|
Adverse Events
Golodirsen
Serious events: 12 serious events
Other events: 63 other events
Deaths: 1 deaths
Casimersen
Serious events: 22 serious events
Other events: 93 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Golodirsen
n=74 participants at risk
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
Casimersen
n=97 participants at risk
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
|---|---|---|
|
Injury, poisoning and procedural complications
Femur fracture
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
8.2%
8/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Traumatic fracture
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Head injury
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Humerus fracture
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Near drowning
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Cellulitis
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Gastroenteritis
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Gastroenteritis viral
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Appendicitis
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
COVID-19 pneumonia
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Device related infection
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Otitis media chronic
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Rotavirus infection
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Sepsis
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Septic shock
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Vascular device infection
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Renal and urinary disorders
Haematuria
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Renal and urinary disorders
Nephrolithiasis
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Renal and urinary disorders
Renal embolism
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
2.1%
2/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Neuromuscular scoliosis
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Tendinous contracture
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Congenital, familial and genetic disorders
Cryptorchism
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Nervous system disorders
Idiopathic intracranial hypertension
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Nervous system disorders
Headache
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary embolism
|
1.4%
1/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
0.00%
0/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Acute pulmonary oedema
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Nocturnal dyspnoea
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Catheter site pain
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Chest pain
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Systemic inflammatory response syndrome
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Cardiac disorders
Cardiogenic shock
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Eye disorders
Papilloedema
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Abdominal pain
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Metabolism and nutrition disorders
Hypervolaemia
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Product Issues
Device dislocation
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Vascular disorders
Distributive shock
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Vascular disorders
Haemorrhage
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Vascular disorders
Hypovolaemic shock
|
0.00%
0/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
Other adverse events
| Measure |
Golodirsen
n=74 participants at risk
Participants amenable to exon 53 skipping who have completed a clinical trial evaluating golodirsen received open-label golodirsen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
Casimersen
n=97 participants at risk
Participants amenable to exon 45 skipping who have completed a clinical trial evaluating casimersen received open-label casimersen IV infusions, weekly, at 30 mg/kg for up to 144 weeks.
|
|---|---|---|
|
Infections and infestations
COVID-19
|
13.5%
10/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
27.8%
27/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Nasopharyngitis
|
35.1%
26/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
21.6%
21/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Upper respiratory tract infection
|
9.5%
7/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
15.5%
15/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Influenza
|
8.1%
6/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
7.2%
7/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Gastroenteritis
|
6.8%
5/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Infections and infestations
Rhinitis
|
10.8%
8/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Fall
|
25.7%
19/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
17.5%
17/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Contusion
|
10.8%
8/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
13.4%
13/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Back injury
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
9.3%
9/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Spinal compression fracture
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
7.2%
7/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Femur fracture
|
6.8%
5/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
12.2%
9/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Limb injury
|
8.1%
6/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
4.1%
4/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Joint injury
|
6.8%
5/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
2.1%
2/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Injury, poisoning and procedural complications
Tibia fracture
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
23.0%
17/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
17.5%
17/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
10.8%
8/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
14.4%
14/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
14.9%
11/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
13.4%
13/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Osteoporosis
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
2.1%
2/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Vomiting
|
29.7%
22/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
15.5%
15/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Diarrhoea
|
17.6%
13/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
11.3%
11/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Constipation
|
6.8%
5/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
7.2%
7/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Nausea
|
9.5%
7/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Gastrointestinal disorders
Gastrooesophageal reflux disease
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
4.1%
4/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Pyrexia
|
17.6%
13/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
22.7%
22/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Pain
|
2.7%
2/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
6.2%
6/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Adverse drug reaction
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
General disorders
Gait inability
|
6.8%
5/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
1.0%
1/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Nervous system disorders
Headache
|
29.7%
22/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
25.8%
25/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Nervous system disorders
Dizziness
|
9.5%
7/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
3.1%
3/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
16.2%
12/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
19.6%
19/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
12.2%
9/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
10.3%
10/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
4.1%
3/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
8.1%
6/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
3.1%
3/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
8.2%
8/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Skin and subcutaneous tissue disorders
Ingrowing nail
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
3.1%
3/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Investigations
Urine protein/creatinine ratio increased
|
9.5%
7/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
8.2%
8/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Renal and urinary disorders
Haematuria
|
8.1%
6/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Renal and urinary disorders
Proteinuria
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
2.1%
2/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Eye disorders
Cataract
|
8.1%
6/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
5.2%
5/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
|
Vascular disorders
Haematoma
|
5.4%
4/74 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
4.1%
4/97 • Up to 33 days after the last infusion of study drug (up to approximately 149 weeks)
All reported adverse event data based upon Safety Analysis Set: all enrolled participants who received at least 1 dose of study drug (golodirsen or casimersen).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER