Duchenne disease Duchenne Muscular Dystrophy
Conditions
Interventions
The duration of total involvement in the study will be approximately 64 weeks
(4 weeks in screening, 52 weeks of treatment, a follow-up visit 4 weeks later
and a final follow-up phone call 60 days a
Sponsors
FibroGen, Inc.
Eligibility
Inclusion criteria
Inclusion criteria: Subjects must meet all of the following criteria in order to be eligible for the study: Age, and consent: 1. Males at least 6 to 100,000/mcL b. Hemoglobin >12 g/dL c. Absolute neutrophil count >1500 /µL d. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range for DMD patients 12. Adequate hepatic function: a. No history or evidence of liver disease b. Gamma glutamyl transferase (GGT) :S3x upper limit of normal (ULN) c. Total bilirubin :S1.5xULN
Exclusion criteria
Exclusion criteria: Subjects must not meet any of the following criteria in order to be eligible: General Criteria: 1. Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function 2. Severe intellectual impairment (eg, severe autism, severe cognitive impairment, severe behavioral disturbances) preventing the ability to perform study assessments in the Investigator*s judgment 3. Previous exposure to pamrevlumab 4. BMI >40 kg/m2 or weight >117 kg 5. History of : a) allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies b) hypersensitivity to study drug or any component of study drug 6. Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (e.g., eteplirsen (exondys 51), ataluren, golodirsen (vyondys 53), casimersen (amondys 45)) within 5 half-lives of screening, whichever is longer with the exception of the systemic corticosteroids, including deflazacort Pulmonary, Renal and Cardiac criteria: 7. Requires >=16 hours continuous ventilation 8. Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the investigator might impact respiratory function 9. Hospitalization due to respiratory failure within the 8 weeks prior to screening 10. Severe uncontrolled heart failure (NYHA Classes III-IV), or renal dysfunction, including any of the following: a. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening b. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening c. Patients with glomerular filtration rate (GFR) of less than 30 ml/min/1,73 m² or with other evidence of acute kidney injury as determined by investigator 11. Arrhythmia requiring anti-arrhythmic therapy 12. Any other evidence of clinically significant structural or functional heart abnormality Clinical judgment: 13. The Investigator judges that the subject will be unable to fully participate in the study and complete it for any reason, including inability to comply with study procedures and treatment, or any other relevant medical, surgical or psychiatric conditions
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Ambulatory functional assessment: • Change in NorthStar Ambulatory Assessment (NSAA) total score from baseline to Week 52. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary Endpoints: Other Muscle function assessments: • Change in 4-stair climb Velocity (4SCV) assessment from baseline to Week 52. • Change in the 10-meter walk/run test from baseline to Week 52. • Changes in Time to Stand (TTSTAND) from baseline to Week 52. • Time to Loss of Ambulation (LoA) from baseline to Week 52 Exploratory Endpoint: • Change in Duchenne Video Assessment severity percentage from baseline to Week 52. • Change in ppFVC and ppPEF assessed by spirometry, from baseline to Week 52. MRI Assessment: • Changes in lower extremities vastus lateralis muscle fibrosis score from baseline to Week 52, assessed by MRI. Safety Assessments • All treatment emergent adverse events (TEAEs), serious adverse events (SAEs), clinically significant laboratory test abnormalities, discontinuation of treatment due to treatment-related AEs and hypersensitivity/anaphylactic reactions. • Number and percentage of subjects with hospitalizations due to any serious adverse events with pulmonary and/or cardiac cause(s). • Number and percentage of subjects with bone fractures • Annualized height velocity, HV (cm/year) from Baseline to Week 52 Pharmacokinetics/pharmacodynamics (PK/PD) assessment • Population PK/PD analysis. | — |
Countries
Netherlands
Outcome results
None listed