Trial Outcomes & Findings for Safety, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 in Mucopolysaccharidosis III, Type B (MPS IIIB) (NCT NCT02324049)
NCT ID: NCT02324049
Last Updated: 2018-08-21
Results Overview
TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A). A severe AE was defined as an AE that was incapacitating and required medical intervention. TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Recruitment status
COMPLETED
Study phase
PHASE1/PHASE2
Target enrollment
11 participants
Primary outcome timeframe
Baseline to Week 142
Results posted on
2018-08-21
Participant Flow
Diagnosis of mucopolysaccharidosis III, type B (MPS IIIB), determined by either documented deficiency in Alpha-N-acetylglucosaminidase (NAGLU) enzyme activity ≤10% of the mean value in normal individuals at Screening OR documented functionally-relevant mutations in both alleles of the NAGLU gene based on historical or Screening laboratory results.
Participant milestones
| Measure |
SBC-103
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 milligrams (mg) per kilogram (kg) every other week (QOW) for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered intravenous (IV) QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
Part A
STARTED
|
11
|
|
Part A
Safety Analysis Set
|
11
|
|
Part A
COMPLETED
|
11
|
|
Part A
NOT COMPLETED
|
0
|
|
Part B
STARTED
|
11
|
|
Part B
Safety Analysis Set
|
11
|
|
Part B
COMPLETED
|
11
|
|
Part B
NOT COMPLETED
|
0
|
|
Part C
STARTED
|
11
|
|
Part C
Safety Analysis Set
|
11
|
|
Part C
COMPLETED
|
0
|
|
Part C
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
SBC-103
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 milligrams (mg) per kilogram (kg) every other week (QOW) for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered intravenous (IV) QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
Part C
Study terminated
|
11
|
Baseline Characteristics
There were 5 participants in the 5.0 mg/kg group and 6 participants in the 10.0 mg/kg group.
Baseline characteristics by cohort
| Measure |
SBC-103
n=11 Participants
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
Age, Categorical
<=18 years
|
11 Participants
n=11 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=11 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=11 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=11 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
10 Participants
n=11 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=11 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=11 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
1 Participants
n=11 Participants
|
|
Overall Age at Diagnosis
5.0 mg/kg
|
48.0 Months
n=5 Participants • There were 5 participants in the 5.0 mg/kg group and 6 participants in the 10.0 mg/kg group.
|
|
Overall Age at Diagnosis
10.0 mg/kg
|
25.0 Months
n=6 Participants • There were 5 participants in the 5.0 mg/kg group and 6 participants in the 10.0 mg/kg group.
|
|
Overall Age at Diagnosis
Overall
|
37.0 Months
n=11 Participants • There were 5 participants in the 5.0 mg/kg group and 6 participants in the 10.0 mg/kg group.
|
PRIMARY outcome
Timeframe: Baseline to Week 142Population: Safety Population: all participants for whom informed consent had been obtained, who had a confirmed diagnosis of MPS IIIB, and who had received any amount of SBC-103.
TEAEs were defined as any adverse event (AE) that occurred after administration of the first dose of study drug on Day 1 (Part A). A severe AE was defined as an AE that was incapacitating and required medical intervention. TEAEs were summarized cumulatively over the entire study and separately for Part C, data for all severe TEAEs throughout the entire study is presented. A summary of serious and all other non-serious AEs regardless of causality is located in the Reported Adverse Events module.
Outcome measures
| Measure |
SBC-103
n=11 Participants
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
5.0 mg/kg
|
1 Participants
|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
10.0 mg/kg
|
0 Participants
|
|
Number Of Participants Who Experienced Severe Treatment-emergent Adverse Events (TEAEs)
Overall
|
1 Participants
|
Adverse Events
SBC-103
Serious events: 3 serious events
Other events: 11 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SBC-103
n=11 participants at risk
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
Infections and infestations
Bacteraemia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Pneumonia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Staphylococcal bacteraemia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Vascular disorders
Flushing
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Cardiac disorders
Cyanosis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
General disorders
Pyrexia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Blood pressure increased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
Other adverse events
| Measure |
SBC-103
n=11 participants at risk
Part A (Initial therapy): Participants received SBC-103, 0.3, 1.0, or 3.0 mg/kg QOW for 24 weeks, followed by a ≥ 4-week treatment break. Participants enrolled in the lowest dosage first.
Part B: Participants were escalated to the next highest dose that was considered safe (1.0 or 3.0 mg/kg QOW) for ≥ 8 weeks. Participants who received doses of 0.3 mg/kg in Part A were considered for a second dose escalation to 3.0 mg/kg at any time during Part B provided that they tolerated at least 2 doses of 1.0 mg/kg in Part B. Participants who received and tolerated at least 4 doses of SBC-103 QOW at 3.0 mg/kg were considered for participation in Part C.
Part C: Participants received SBC-103 5.0 or 10.0 mg/kg administered IV QOW. Dosing in Part C began at the 5.0 mg/kg dose level. The decision to begin dosing the first participant at 10.0 mg/kg was based on the review of safety data at 5.0 mg/kg.
|
|---|---|
|
General disorders
Pyrexia
|
81.8%
9/11 • Day 1 postdose up to Week 142
|
|
General disorders
Catheter site erythema
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
General disorders
Catheter site rash
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
General disorders
Catheter site swelling
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
General disorders
Influenza like illness
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
General disorders
Local swelling
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
General disorders
Pain
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Nasopharyngitis
|
45.5%
5/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Upper respiratory tract infection
|
36.4%
4/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Lower respiratory tract infection
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Rhinitis
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Gastroenteritis viral
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Ear infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Fungal skin infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Hordeolum
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Pharyngitis streptococcal
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Candida nappy rash
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Conjunctivitis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Eye infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Eye infection bacterial
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Folliculitis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Fungal infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Influenza
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Localised infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Oral candidiasis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Oral herpes
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Otitis externa
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Otitis media
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Paronychia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Respiratory tract infection viral
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Rotavirus infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Tonsillitis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Infections and infestations
Viral infection
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Rash
|
36.4%
4/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Dermatitis diaper
|
36.4%
4/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Erythema
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Rash erythematous
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Dermatitis allergic
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Dermatitis contact
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Eczema
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Miliaria
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Papule
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Seborrhoeic dermatitis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Skin discolouration
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Skin plaque
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Vomiting
|
63.6%
7/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Diarrhoea
|
54.5%
6/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Salivary hypersecretion
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Constipation
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Breath odour
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Eructation
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Lip swelling
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Nausea
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Pancreatic enlargement
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Gastrointestinal disorders
Swollen tongue
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
45.5%
5/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
27.3%
3/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
27.3%
3/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Wheezing
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Bronchial secretion retention
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Head injury
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Fall
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Infusion related reaction
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Anaesthetic complication neurological
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Contusion
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Eye contusion
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Eyelid injury
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Foreign body
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Lip injury
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Mouth injury
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Cardiac murmur
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Investigations
Body temperature increased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
CSF protein increased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Electrocardiogram Q wave abnormal
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Liver palpable
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Mean platelet volume increased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Oxygen saturation decreased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Investigations
Serum ferritin decreased
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Ear and labyrinth disorders
Deafness
|
36.4%
4/11 • Day 1 postdose up to Week 142
|
|
Ear and labyrinth disorders
Ear pain
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Ear and labyrinth disorders
Deafness bilateral
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Ear and labyrinth disorders
Hypoacusis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Ear and labyrinth disorders
Middle ear effusion
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Vascular disorders
Flushing
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Vascular disorders
Hyperaemia
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Vascular disorders
Hypertension
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Headache
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Hyperreflexia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Hypertonia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Lethargy
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Psychomotor hyperactivity
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Seizure
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Speech disorder
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Nervous system disorders
Syncope
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Blood and lymphatic system disorders
Anaemia
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Cardiac disorders
Left ventricular hypertrophy
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Cardiac disorders
Sinus bradycardia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Cardiac disorders
Tachycardia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Eye disorders
Mydriasis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Eye disorders
Eye swelling
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Eye disorders
Ocular hyperaemia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Musculoskeletal and connective tissue disorders
Coccydynia
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Musculoskeletal and connective tissue disorders
Muscle mass
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Metabolism and nutrition disorders
Decreased appetite
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Metabolism and nutrition disorders
Iron deficiency
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Renal and urinary disorders
Proteinuria
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Congenital, familial and genetic disorders
Dysmorphism
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Endocrine disorders
Precocious puberty
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Product Issues
Device occlusion
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Injury, poisoning and procedural complications
Periorbital haemorrhage
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Skin and subcutaneous tissue disorders
Swelling face
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Psychiatric disorders
Hypervigilance
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Psychiatric disorders
Insomnia
|
18.2%
2/11 • Day 1 postdose up to Week 142
|
|
Psychiatric disorders
Aggression
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Psychiatric disorders
Agitation
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
|
Psychiatric disorders
Anxiety
|
9.1%
1/11 • Day 1 postdose up to Week 142
|
Additional Information
Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc.
Phone: 475-230-2596
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place