Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)

• Register: ClinicalTrials.gov
• Last refreshed on: 9 October 2023
• Main ID: NCT02606136
• Date of registration: 04/11/2015
• Prospective Registration: Yes
• Primary sponsor: FibroGen
• Public title: Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) MISSION
• Scientific title: Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
• Date of first enrolment: January 4, 2016
• Target sample size: 21
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/ct2/show/NCT02606136
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

- Written consent/assent by participant and/or legal guardian as per regional and/or
institutional review board (IRB) requirements

- Non-ambulatory

- Brooke Score for Arms and Shoulders =5

- Diagnosis of DMD by medical history and confirmed Duchenne mutation in available
genetic testing using a validated genetic test

- Able to perform spirometry

- Able to undergo cardiac and extremity (upper arm) MRI

- Percent predicted FVC between 40 and 90, inclusive

- At least one historical ppFVC predicted value within 18 months of baseline

- Left ventricular ejection fraction = 45% as determined by cardiac MRI at screening or
within 3 months prior to Day 0

- Participants currently receiving heart failure cardiac medications (for example,
angiotensin converting enzyme inhibitors, angiotensin-receptor blockers, and
beta-blockers) must achieve a stable regimen for at least 3 months prior to screening

- On a stable dose of corticosteroids for a minimum of 6 months prior to screening with
no substantial change in dosage for a minimum of 3 months (except for adjustments for
changes in body weight) prior to screening and no foreseen change in corticosteroid
use during the course of study participation

- Received pneumococcal vaccine and is receiving annual influenza vaccinations

- Adequate renal function: cystatin C =1.4 mg/liter (L)

- Adequate hematological function

1. Platelets >100,000/microliter (µL)

2. Hemoglobin >12 grams (g)/deciliter (dL)

3. Absolute neutrophil count >1500/µL

- Adequate hepatic function

1. No history or evidence of liver disease

2. Gamma glutamyl transferase (GGT) =3 x upper limit of normal (ULN)

3. Total bilirubin =1.5 x ULN

- If sexually active, will use medically accepted contraceptives during participation in
the study and for 3 months after the last dose of study drug

Exclusion Criteria:

- Requires =16 hours continuous ventilation

- Prior or ongoing medical condition that, in the investigator's opinion, could
adversely affect the safety of the participant, makes it unlikely that the course of
156 weeks of treatment and follow-up would be completed, or could impair the
assessment of study results

- Anticipated spine surgery within 156 weeks

- Severe uncontrolled heart disease, including any of the following:

1. Need for intravenous diuretics or inotropic support within 3 months prior to
screening

2. Hospitalization for a heart failure exacerbation or arrhythmia in last 3 months

- Arrhythmia requiring anti-arrhythmic therapy

- Hospitalization due to respiratory failure in the last 6 weeks

- Poorly controlled asthma or underlying lung disease such as bronchopulmonary dysplasia

- Known or suspected active hepatitis B or C or history of human immunodeficiency virus
(HIV)

- Body mass index (BMI) =40 kilograms (kg)/square meter (m^2) or weight >117 kg

- Exposure to another investigational drug or another approved product for DMD (for
example, eteplirsen or golodirsen) within 28 days prior to start of study treatment

- Exposure to another investigational drug or another approved product for DMD (e.g.
eteplirsen) within 28 days prior to start of study treatment (or 5 half-lives of the
product whichever is longer) prior to first screening visit with the exception of
deflazacort. Use of deflazacort, if regarded by the principal investigator as standard
of care, is allowed.



Age minimum: 12 Years
Age maximum: N/A
Gender: Male

Health Condition(s) or Problem(s) studied

Duchenne Muscular Dystrophy

Intervention(s)

Drug: Pamrevlumab

Primary Outcome(s)

Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 [Time Frame: Baseline, Week 104]

Secondary Outcome(s)

Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104 [Time Frame: Baseline, Week 104]

Change From Baseline in Pinch Strength, as Measured by HHM at Week 104 [Time Frame: Baseline, Week 104]

Secondary ID(s)

2023-000321-80

FGCL-3019-079

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 30/12/2021
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02606136