Trial Outcomes & Findings for Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System (NCT NCT03643562)
NCT ID: NCT03643562
Last Updated: 2024-01-17
Results Overview
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Recruitment status
TERMINATED
Study phase
PHASE3
Target enrollment
27 participants
Primary outcome timeframe
Baseline up to Week 134
Results posted on
2024-01-17
Participant Flow
Participant milestones
| Measure |
Treatment Naive
Treatment-naive participants received prescribed adrabetadex by intra-thecal (IT) injection via lumbar puncture (LP) every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
18
|
|
Overall Study
Received at Least 1 Dose of Study Drug
|
9
|
18
|
|
Overall Study
COMPLETED
|
1
|
2
|
|
Overall Study
NOT COMPLETED
|
8
|
16
|
Reasons for withdrawal
| Measure |
Treatment Naive
Treatment-naive participants received prescribed adrabetadex by intra-thecal (IT) injection via lumbar puncture (LP) every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
|---|---|---|
|
Overall Study
Adverse Event
|
1
|
0
|
|
Overall Study
Lack of Efficacy
|
0
|
1
|
|
Overall Study
Physician Decision
|
0
|
1
|
|
Overall Study
Study Terminated by Sponsor
|
2
|
10
|
|
Overall Study
Withdrawal by Subject
|
2
|
2
|
|
Overall Study
Other than specified
|
3
|
2
|
Baseline Characteristics
Niemann-Pick Type C Treatment With Adrabetadex for Symptoms of Brain and Nervous System
Baseline characteristics by cohort
| Measure |
Treatment Naive
n=9 Participants
Treatment-naive participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
n=18 Participants
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Total
n=27 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
12.33 years
STANDARD_DEVIATION 8.718 • n=5 Participants
|
17.94 years
STANDARD_DEVIATION 13.705 • n=7 Participants
|
16.07 years
STANDARD_DEVIATION 12.388 • n=5 Participants
|
|
Sex: Female, Male
Female
|
4 Participants
n=5 Participants
|
7 Participants
n=7 Participants
|
11 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=5 Participants
|
11 Participants
n=7 Participants
|
16 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
8 Participants
n=5 Participants
|
18 Participants
n=7 Participants
|
26 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
7 Participants
n=5 Participants
|
17 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline up to Week 134Population: All enrolled participants
An adverse event (AE) was defined as any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse events (SAEs) were defined as death, a life-threatening AE, inpatient hospitalization or prolongation of existing hospitalization, persistent or significant disability or incapacity, a congenital anomaly or birth defect, or an important medical event that jeopardized participant and required medical intervention to prevent 1 of the outcomes listed in this definition. A TEAE was defined as an AE with onset on or after the start of adrabetadex treatment. A summary of other non-serious AEs and all serious AEs, regardless of causality is located in Reported AE section.
Outcome measures
| Measure |
Treatment Naive
n=9 Participants
Treatment-naive participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
n=18 Participants
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
|---|---|---|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
SAEs
|
5 Participants
|
9 Participants
|
|
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
All TEAEs
|
9 Participants
|
17 Participants
|
Adverse Events
Treatment Naive
Serious events: 5 serious events
Other events: 9 other events
Deaths: 0 deaths
Previously Treated
Serious events: 9 serious events
Other events: 17 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Treatment Naive
n=9 participants at risk
Treatment-naive participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
n=18 participants at risk
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
|---|---|---|
|
Cardiac disorders
Ventricular extrasystoles
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Ear and labyrinth disorders
Deafness
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Constipation
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Crohn's disease
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Hepatobiliary disorders
Cholangitis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Hepatobiliary disorders
Cholangitis acute
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Meningitis
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Staphylococcal bacteraemia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Urosepsis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Femur fracture
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Investigations
Norovirus test positive
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Ataxia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Lennox-Gastaut syndrome
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Sedation
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Seizure
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
16.7%
3/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Mental status changes
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Staring
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Renal and urinary disorders
Acute kidney injury
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Renal and urinary disorders
Urinary tract obstruction
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Acute respiratory failure
|
22.2%
2/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory distress
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
Other adverse events
| Measure |
Treatment Naive
n=9 participants at risk
Treatment-naive participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
Previously Treated
n=18 participants at risk
Previously-treated participants received prescribed adrabetadex by IT injection via LP every 2 weeks. Treatment with adrabetadex continued until the clinician considered adrabetadex to be no longer beneficial to the participant, the participant withdrew from the study, adrabetadex received marketing authorization in the United States, or the development program was discontinued.
|
|---|---|---|
|
Cardiac disorders
Tachycardia
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Ear and labyrinth disorders
Deafness
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Ear and labyrinth disorders
Deafness neurosensory
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Ear and labyrinth disorders
Hypoacusis
|
22.2%
2/9 • Baseline up to Week 134
All enrolled participants
|
22.2%
4/18 • Baseline up to Week 134
All enrolled participants
|
|
Ear and labyrinth disorders
Middle ear effusion
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Eye disorders
Gaze palsy
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Constipation
|
22.2%
2/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Dental caries
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Diarrhoea
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
16.7%
3/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Dysphagia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Haematochezia
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Nausea
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Gastrointestinal disorders
Vomiting
|
44.4%
4/9 • Baseline up to Week 134
All enrolled participants
|
22.2%
4/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Adverse drug reaction
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Complication associated with device
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Fatigue
|
66.7%
6/9 • Baseline up to Week 134
All enrolled participants
|
38.9%
7/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Gait disturbance
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Gait inability
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Medical device site injury
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Procedural failure
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Pyrexia
|
33.3%
3/9 • Baseline up to Week 134
All enrolled participants
|
16.7%
3/18 • Baseline up to Week 134
All enrolled participants
|
|
General disorders
Unevaluable event
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Cellulitis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Gastroenteritis viral
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Impetigo
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Lip infection
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Otitis media
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Pneumonia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Sinusitis
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Tinea pedis
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Tooth infection
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Upper respiratory tract infection
|
33.3%
3/9 • Baseline up to Week 134
All enrolled participants
|
44.4%
8/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Urinary tract infection
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Infections and infestations
Viral infection
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Ankle fracture
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Bite
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Fall
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Gastrostomy tube site complication
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Intentional product use issue
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Joint dislocation
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Lip injury
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Post procedural haemorrhage
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Post procedural swelling
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Skin abrasion
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Skin laceration
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Upper limb fracture
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Investigations
Blood magnesium decreased
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Investigations
Blood urine present
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Investigations
Coronavirus test positive
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Investigations
Oxygen saturation decreased
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Metabolism and nutrition disorders
Decreased appetite
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Metabolism and nutrition disorders
Dehydration
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
33.3%
3/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Bursitis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Foot deformity
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Musculoskeletal and connective tissue disorders
Osteopenia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Ataxia
|
55.6%
5/9 • Baseline up to Week 134
All enrolled participants
|
33.3%
6/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Balance disorder
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Cataplexy
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Cognitive disorder
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Drooling
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Dysarthria
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Generalised tonic-clonic seizure
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Headache
|
33.3%
3/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Hypersomnia
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Memory impairment
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Motor dysfunction
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Myoclonus
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Petit mal epilepsy
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Primary headache associated with sexual activity
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Sedation
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Seizure
|
22.2%
2/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Somnolence
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
22.2%
4/18 • Baseline up to Week 134
All enrolled participants
|
|
Nervous system disorders
Tremor
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Bipolar disorder
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Communication disorder
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Emotional disorder
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Hallucination
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Insomnia
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Psychiatric disorders
Irritability
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Renal and urinary disorders
Bladder hypertrophy
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Renal and urinary disorders
Urinary incontinence
|
22.2%
2/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Increased upper airway secretion
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Pneumonia aspiration
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Respiratory tract congestion
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
11.1%
1/9 • Baseline up to Week 134
All enrolled participants
|
0.00%
0/18 • Baseline up to Week 134
All enrolled participants
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Skin and subcutaneous tissue disorders
Acne cystic
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Skin and subcutaneous tissue disorders
Henoch-Schonlein purpura
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
5.6%
1/18 • Baseline up to Week 134
All enrolled participants
|
|
Skin and subcutaneous tissue disorders
Rash
|
0.00%
0/9 • Baseline up to Week 134
All enrolled participants
|
11.1%
2/18 • Baseline up to Week 134
All enrolled participants
|
Additional Information
Executive Vice President, Regulatory Affairs
Mandos, LLC
Phone: 619-905-0489
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place