Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD) (DMD)
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ClinicalTrials.gov Identifier: NCT02606136 |
Recruitment Status
:
Active, not recruiting
First Posted
: November 17, 2015
Last Update Posted
: April 18, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Duchenne Muscular Dystrophy | Drug: pamrevlumab (FG-3019) | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 22 participants |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy |
Actual Study Start Date : | November 2015 |
Estimated Primary Completion Date : | April 2020 |
Estimated Study Completion Date : | April 2021 |

Arm | Intervention/treatment |
---|---|
Experimental: pamrevlumab (FG-3019)
Each subject will receive pamrevlumab (FG-3019) (35 mg/kg, every 2 weeks) for up to 156 weeks.
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Drug: pamrevlumab (FG-3019)
pamrevlumab (FG-3019), 10 mg/mL, single dose vials
Other Name: Monoclonal Antibody to CTGF
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- Annual change in percent predicted annual forced vital capacity (FVC) during treatment with pamrevlumab. [ Time Frame: From baseline to 104 weeks ]
- Change in forced expiratory volume (FEV1) [ Time Frame: From baseline to 104 weeks ]
- Change in maximum inspiratory pressure (MIP) [ Time Frame: From baseline to 104 weeks ]
- Change in maximum expiratory pressure (MEP) [ Time Frame: From baseline to 104 weeks ]
- Change in peak expiratory flow (PEF) [ Time Frame: From baseline to 104 weeks ]
- Change in peak cough flow [ Time Frame: From baseline to 104 weeks ]
- Change in left ventricular ejection fraction (LVEF) [ Time Frame: From baseline to 104 weeks ]
- Change in Performance of Upper Limb (PUL) Score [ Time Frame: From baseline to 104 weeks ]
- Change in grip strength [ Time Frame: From baseline to 104 weeks ]
- Change in pinch strength [ Time Frame: From baseline to 104 weeks ]
- Change in Brooke scale for upper extremity [ Time Frame: From baseline to 104 weeks ]
- Change in cardiac fibrosis score assessed by MRI [ Time Frame: From baseline to 104 weeks ]
- Change in upper arm (bicep) muscle fat and fibrosis assessed by MRI [ Time Frame: From baseline to 104 weeks ]

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 12 Years and older (Child, Adult, Senior) |
Sexes Eligible for Study: | Male |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- At least 12 years of age
- Written consent/assent by patient and/or legal guardian as per regional and/or 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 FVC% 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
- Subjects currently receiving heart failure cardiac medications (e.g. 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/L
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Adequate hematological function
- Platelets >100,000/mcL
- Hemoglobin >12 g/dL
- Absolute neutrophil count >1500/μL
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Adequate hepatic function
- No history or evidence of liver disease
- Gamma glutamyl transferase (GGT) ≤3 x upper limit of normal (ULN)
- Total bilirubin ≤1.5xULN
- 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 subject, 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
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Severe uncontrolled heart disease
- Need for intravenous diuretics or inotropic support within 3 months prior to screening
- 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 HIV
- BMI ≥40 kg/m^2 or weight >117 kg
- Exposure to another investigational drug 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02606136
United States, California | |
David Geffen School of Medicine at UCLA | |
Los Angeles, California, United States, 90095 | |
University of California San Francisco - Benioff Children's Hospital | |
San Francisco, California, United States, 94143 | |
United States, Colorado | |
Children's Hospital Colorado | |
Aurora, Colorado, United States, 80045 | |
United States, Georgia | |
Rare Disease Research | |
Atlanta, Georgia, United States, 30318 | |
United States, Massachusetts | |
Boston Children's Hospital | |
Boston, Massachusetts, United States, 02115 | |
United States, Missouri | |
Washington University in St. Louis School of Medicine | |
Saint Louis, Missouri, United States, 63110 | |
United States, Ohio | |
Cincinnati Children's Hospital Medical Center | |
Cincinnati, Ohio, United States, 45229 | |
United States, Oregon | |
Shriner's Hospital for Children - Portland | |
Portland, Oregon, United States, 97239 | |
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19104 | |
United States, Texas | |
Children's Medical Center Ambulatory Care Pavilion | |
Dallas, Texas, United States, 75207 |
Responsible Party: | FibroGen |
ClinicalTrials.gov Identifier: | NCT02606136 History of Changes |
Other Study ID Numbers: |
FGCL-3019-079 |
First Posted: | November 17, 2015 Key Record Dates |
Last Update Posted: | April 18, 2018 |
Last Verified: | April 2018 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Undecided |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Keywords provided by FibroGen:
Duchenne muscular dystrophy DMD non-ambulatory |
Additional relevant MeSH terms:
Muscular Dystrophies Muscular Dystrophy, Duchenne Muscular Disorders, Atrophic Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Nervous System Diseases |
Genetic Diseases, Inborn Genetic Diseases, X-Linked Antibodies Antibodies, Monoclonal Immunologic Factors Physiological Effects of Drugs |