Trial Outcomes & Findings for Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53) (NCT NCT04956289)
NCT ID: NCT04956289
Last Updated: 2024-08-20
Results Overview
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
COMPLETED
PHASE2
20 participants
baseline to up to 48 weeks of treatment
2024-08-20
Participant Flow
Participants were enrolled in the study from July 1, 2021 to July 22, 2022 at 8 sites in the United States, Italy, Russia, Spain, and China.
A total of 27 participants were screened, but 3 participants did not meet inclusion or exclusion criteria and 4 participants failed to screen for other reasons.
Participant milestones
| Measure |
Viltolarsen 80mg/kg
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Overall Study
STARTED
|
20
|
|
Overall Study
Ambulant
|
10
|
|
Overall Study
Non-ambulant
|
10
|
|
Overall Study
COMPLETED
|
20
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
Baseline characteristics by cohort
| Measure |
Viltolarsen 80mg/kg
n=20 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Age, Continuous
|
12.8 years
STANDARD_DEVIATION 5.47 • n=99 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
20 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
20 Participants
n=99 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
5 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10).
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=20 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Treatment Emergent Adverse Events
|
19 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Treatment-Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=19 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity
Mild
|
7 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity
Moderate
|
12 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Maximum Severity
Severe
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Treatment-Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=19 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Dose not changed
|
16 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Dose reduced
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Drug interrupted
|
2 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Drug withdrawn
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Not applicable
|
1 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Investigational Product-related Treatment Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=19 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Recovered or resolved
|
17 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Recovered or resolved with sequelae
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Recovering or resolving
|
0 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Not recovered or resolved
|
2 Participants
|
|
Number of Participants With Treatment Emergent Adverse Events by Worst Outcome
Fatal
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10).
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=20 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events
|
4 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Investigational Product-related Treatment Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=4 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity
Mild
|
3 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity
Moderate
|
1 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Maximum Severity
Severe
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Investigational Product-related Treatment Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=4 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Dose not changed
|
3 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Dose reduced
|
0 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Drug interrupted
|
1 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Highest Intervention Level Regarding Investigational Product
Drug withdrawn
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10). Only participants with Investigational Product-related Treatment Emergent Adverse Events were evaluated.
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=4 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Recovered or resolved
|
4 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Recovering or resolving
|
0 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Not recovered or resolved
|
0 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Fatal
|
0 Participants
|
|
Number of Participants With Investigational Product-related Treatment Emergent Adverse Events by Worst Outcome
Recovered or resolved with sequelae
|
0 Participants
|
PRIMARY outcome
Timeframe: baseline to up to 48 weeks of treatmentPopulation: The Safety Population consisted of all patients who received at least 1 dose of IP (Investigational Product). This was the primary analysis population for the evaluation of safety. It includes all patients treated with viltolarsen both ambulant (10) and non-ambulant (10).
No statistical analysis was performed for this endpoint. The information has been introduced in the section "Adverse Events".
Outcome measures
| Measure |
Viltolarsen 80mg/kg
n=20 Participants
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Number of Participants With Adverse Event of Special Interest
|
5 Participants
|
Adverse Events
Viltolarsen 80mg/kg
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Viltolarsen 80mg/kg
n=20 participants at risk
Participants who confirmed DMD with genetic deletions amenable to exon 53 skipping were administered an intravenous infusion of Viltolarsen 80 mg/kg once a week for up to 48 weeks.
|
|---|---|
|
Infections and infestations
COVID-19
|
30.0%
6/20 • Number of events 6 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Nasopharyngitis
|
15.0%
3/20 • Number of events 3 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Upper respiratory tract infection
|
15.0%
3/20 • Number of events 3 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Influenza
|
10.0%
2/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Rhinitis
|
10.0%
2/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Otitis media
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Infections and infestations
Pharyngitis
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Diarrhoea
|
25.0%
5/20 • Number of events 13 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Food poisoning
|
10.0%
2/20 • Number of events 8 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Abdominal pain upper
|
10.0%
2/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Toothache
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Gastrointestinal disorders
Vomiting
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Nervous system disorders
Headache
|
20.0%
4/20 • Number of events 4 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Nervous system disorders
Tension headache
|
5.0%
1/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Pyrexia
|
10.0%
2/20 • Number of events 3 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Gait inability
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
General disorders
Non-cardiac chest pain
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Injury, poisoning and procedural complications
Joint injury
|
10.0%
2/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Injury, poisoning and procedural complications
Contusion
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Renal and urinary disorders
Haematuria
|
20.0%
4/20 • Number of events 6 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Renal and urinary disorders
Nephrolithiasis
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
10.0%
2/20 • Number of events 2 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Cardiac disorders
Angina pectoris
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Cardiac disorders
Tachycardia
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Protein urine
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Investigations
Urine cytology abnormal
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Blister
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Immune system disorders
Drug hypersensitivity
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
|
Vascular disorders
Hypertension
|
5.0%
1/20 • Number of events 1 • Up to 48 weeks
Number of participants with Treatment Emergent Adverse Events as assessed by CTCAE v4.03
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee The most restrictive relevant agreement on the PI provides that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is not less than 30 days from the time submitted to the sponsor for review.
- Publication restrictions are in place
Restriction type: OTHER