Trial Outcomes & Findings for Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies (NCT NCT04033159)

NCT ID: NCT04033159

Last Updated: 2023-06-27

Results Overview

Number of participants with drug-related TEAEs during the study period.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

14 participants

Primary outcome timeframe

Baseline until Study termination, up to 28 months

Results posted on

2023-06-27

Participant Flow

No participant was enrolled in the "Cohort 3 - High Dose" Arm/Group

Participant milestones

Participant milestones
Measure
Cohort 1 - Low Dose
DYN101 in a low dose (1.5 mg/kg weekly IV)
Cohort 2 - Middle Dose
DYN101 in a middle dose (4.5 mg/kg weekly IV)
Cohort 3 - High Dose
DYN101 in a high dose (9 mg/kg weekly IV)
Single Ascending Dose (Day 1)
STARTED
6
8
0
Single Ascending Dose (Day 1)
COMPLETED
6
8
0
Single Ascending Dose (Day 1)
NOT COMPLETED
0
0
0
Washout Period
STARTED
6
8
0
Washout Period
COMPLETED
6
0
0
Washout Period
NOT COMPLETED
0
8
0
Multiple Ascending Dose Period
STARTED
6
0
0
Multiple Ascending Dose Period
COMPLETED
0
0
0
Multiple Ascending Dose Period
NOT COMPLETED
6
0
0

Reasons for withdrawal

Reasons for withdrawal
Measure
Cohort 1 - Low Dose
DYN101 in a low dose (1.5 mg/kg weekly IV)
Cohort 2 - Middle Dose
DYN101 in a middle dose (4.5 mg/kg weekly IV)
Cohort 3 - High Dose
DYN101 in a high dose (9 mg/kg weekly IV)
Washout Period
Physician Decision
0
1
0
Washout Period
Study terminated by Sponsor
0
7
0
Multiple Ascending Dose Period
Study terminated by Sponsor
6
0
0

Baseline Characteristics

Early Phase Human Drug Trial to Investigate Dynamin 101 (DYN101) in Patients ≥ 16 Years With Centronuclear Myopathies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Cohort 1 - Low Dose
n=6 Participants
DYN101 in a low dose (1.5 mg/kg)
Cohort 2 - Middle Dose
n=8 Participants
DYN101 in a middle dose (4.5 mg/kg)
Total
n=14 Participants
Total of all reporting groups
Age, Continuous
39.8 years
STANDARD_DEVIATION 16.49 • n=5 Participants
37.4 years
STANDARD_DEVIATION 16.80 • n=7 Participants
38.4 years
STANDARD_DEVIATION 16.07 • n=5 Participants
Sex: Female, Male
Female
2 Participants
n=5 Participants
4 Participants
n=7 Participants
6 Participants
n=5 Participants
Sex: Female, Male
Male
4 Participants
n=5 Participants
4 Participants
n=7 Participants
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
6 Participants
n=5 Participants
8 Participants
n=7 Participants
14 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
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
White
5 Participants
n=5 Participants
7 Participants
n=7 Participants
12 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
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Mutation
DNM2
3 Participants
n=5 Participants
5 Participants
n=7 Participants
8 Participants
n=5 Participants
Mutation
MTM1
3 Participants
n=5 Participants
3 Participants
n=7 Participants
6 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline until Study termination, up to 28 months

Number of participants with drug-related TEAEs during the study period.

Outcome measures

Outcome measures
Measure
Single Dose Cohort 1 - Low Dose
n=6 Participants
DYN101 in a low dose (1.5 mg/kg), single administration
Single Dose Cohort 2 - Middle Dose
n=8 Participants
DYN101 in a middle dose (4.5 mg/kg), single administration
Multiple Dose Cohort 1 - Low Dose
n=6 Participants
DYN101 in a low dose (1.5 mg/kg), weekly administration
Number of Participants With Drug-related Treatment Emergent Adverse Events (TEAEs)
1 Participants
5 Participants
6 Participants

Adverse Events

Single Dose Cohort 1 - Low Dose

Serious events: 3 serious events
Other events: 6 other events
Deaths: 0 deaths

Single Dose Cohort 2 - Middle Dose

Serious events: 2 serious events
Other events: 8 other events
Deaths: 0 deaths

Multiple Dose Cohort 1 - Low Dose

Serious events: 0 serious events
Other events: 6 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Single Dose Cohort 1 - Low Dose
n=6 participants at risk
DYN101 in a low dose (1.5 mg/kg), single administration
Single Dose Cohort 2 - Middle Dose
n=8 participants at risk
DYN101 in a middle dose (4.5 mg/kg), single administration
Multiple Dose Cohort 1 - Low Dose
n=6 participants at risk
DYN101 in a low dose (1.5 mg/kg), weekly administration
Respiratory, thoracic and mediastinal disorders
COVID-19
16.7%
1/6 • Number of events 1 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Ear and labyrinth disorders
Cholesteatoma
16.7%
1/6 • Number of events 1 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Breast mass
16.7%
1/6 • Number of events 1 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Tibia fracture
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Number of events 1 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Respiratory, thoracic and mediastinal disorders
Respiratory muscle weakness
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Number of events 1 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months

Other adverse events

Other adverse events
Measure
Single Dose Cohort 1 - Low Dose
n=6 participants at risk
DYN101 in a low dose (1.5 mg/kg), single administration
Single Dose Cohort 2 - Middle Dose
n=8 participants at risk
DYN101 in a middle dose (4.5 mg/kg), single administration
Multiple Dose Cohort 1 - Low Dose
n=6 participants at risk
DYN101 in a low dose (1.5 mg/kg), weekly administration
Blood and lymphatic system disorders
Anaemia
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Blood and lymphatic system disorders
Thrombocytopenia
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
83.3%
5/6 • Baseline until Study termination, up to 28 months
Cardiac disorders
Arrhythmia
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Cardiac disorders
Palpitations
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Ear and labyrinth disorders
Vertigo
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Abdominal pain
33.3%
2/6 • Baseline until Study termination, up to 28 months
50.0%
4/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Abdominal pain upper
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Nausea
16.7%
1/6 • Baseline until Study termination, up to 28 months
37.5%
3/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Oral mucosal discolouration
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Palatal disorder
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Toothache
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
General disorders
Chest pain
33.3%
2/6 • Baseline until Study termination, up to 28 months
25.0%
2/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
General disorders
Eye complication associated with device
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
General disorders
Fatigue
0.00%
0/6 • Baseline until Study termination, up to 28 months
25.0%
2/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
General disorders
Vessel puncture site bruise
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Hepatobiliary disorders
Hepatic fibrosis
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Immune system disorders
Immunisation reaction
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Infections and infestations
COVID-19
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Infections and infestations
Cystitis
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Infections and infestations
Nasopharyngitis
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Infections and infestations
Pyelonephritis
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Infections and infestations
Sinusitis
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Cardiac disorders
Tachycardia
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Ankle fracture
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Fall
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Foot fracture
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Post procedural haematoma
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Procedural pain
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Blood bilirubin increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Investigations
C-reactive protein increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Investigations
Hepatic enzyme increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
25.0%
2/8 • Baseline until Study termination, up to 28 months
33.3%
2/6 • Baseline until Study termination, up to 28 months
Investigations
Red blood cells urine positive
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Investigations
Transaminases increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Arthralgia
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Back pain
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Joint swelling
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Myalgia
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Pain in extremity
33.3%
2/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Disturbance in attention
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Headache
33.3%
2/6 • Baseline until Study termination, up to 28 months
37.5%
3/8 • Baseline until Study termination, up to 28 months
33.3%
2/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Hypoaesthesia
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Neuralgia
33.3%
2/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Anxiety
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Sleep disorder
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Renal and urinary disorders
Haematuria
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Skin and subcutaneous tissue disorders
Hyperhidrosis
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Skin and subcutaneous tissue disorders
Rash
16.7%
1/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Vascular disorders
Circulatory collapse
0.00%
0/6 • Baseline until Study termination, up to 28 months
12.5%
1/8 • Baseline until Study termination, up to 28 months
0.00%
0/6 • Baseline until Study termination, up to 28 months
Blood and lymphatic system disorders
Leukopenia
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Hypoaesthesia oral
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
33.3%
2/6 • Baseline until Study termination, up to 28 months
Gastrointestinal disorders
Diarrhoea
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
33.3%
2/6 • Baseline until Study termination, up to 28 months
Infections and infestations
Viral infection
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Injury, poisoning and procedural complications
Upper limb fracture
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Alanine aminotransferase increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Aspartate aminotransferase increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Cystatin C increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Gamma-glutamyltransferase increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Liver function test increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
50.0%
3/6 • Baseline until Study termination, up to 28 months
Investigations
Total bile acids increased
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
33.3%
2/6 • Baseline until Study termination, up to 28 months
Musculoskeletal and connective tissue disorders
Neck pain
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Investigations
Dizziness
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Paraesthesia
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Nervous system disorders
Tension headache
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Psychiatric disorders
Depression
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months
Renal and urinary disorders
Nephrolithiasis
0.00%
0/6 • Baseline until Study termination, up to 28 months
0.00%
0/8 • Baseline until Study termination, up to 28 months
16.7%
1/6 • Baseline until Study termination, up to 28 months

Additional Information

Dynacure

Dynacure

Phone: +33 3 74 95 20 50

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place

Restriction type: LTE60