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

This study is currently recruiting participants.
See Contacts and Locations
Verified August 2017 by FibroGen
Sponsor:
Information provided by (Responsible Party):
FibroGen
ClinicalTrials.gov Identifier:
NCT02606136
First received: November 4, 2015
Last updated: August 15, 2017
Last verified: August 2017
  Purpose
This is a Phase 2, open-label, single arm trial of pamrevlumab (FG-3019) to estimate pamrevlumab's safety and efficacy in non-ambulatory subjects with DMD.

Condition Intervention Phase
Duchenne Muscular Dystrophy Drug: pamrevlumab (FG-3019) Phase 2

Study Type: Interventional
Study Design: 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

Resource links provided by NLM:


Further study details as provided by FibroGen:

Primary Outcome Measures:
  • Change in annual forced vital capacity (FVC) (% predicted) during treatment of pamrevlumab compared with the estimated annual change prior to pamrevlumab treatment. [ Time Frame: From baseline to 52 weeks ]

Secondary Outcome Measures:
  • Change in forced expiratory volume (FEV1) [ Time Frame: From baseline to 52 weeks ]
  • Change in maximum inspiratory pressure (MIP) [ Time Frame: From baseline to 52 weeks ]
  • Change in maximum expiratory pressure (MEP) [ Time Frame: From baseline to 52 weeks ]
  • Change in peak expiratory flow (PEF) [ Time Frame: From baseline to 52 weeks ]
  • Change in peak cough flow [ Time Frame: From baseline to 52 weeks ]
  • Change in left ventricular ejection fraction (LVEF) [ Time Frame: From baseline to 52 weeks ]
  • Change in Performance of Upper Limb (PUL) Score [ Time Frame: From baseline to 52 weeks ]
  • Change in grip strength [ Time Frame: From baseline to 52 weeks ]
  • Change in pinch strength [ Time Frame: From baseline to 52 weeks ]
  • Change in Brooke scale for upper extremity [ Time Frame: From baseline to 52 weeks ]
  • Change in cardiac fibrosis score assessed by MRI [ Time Frame: From baseline to 52 weeks ]
  • Change in upper arm (bicep) muscle fat and fibrosis assessed by MRI [ Time Frame: From baseline to 52 weeks ]

Estimated Enrollment: 22
Actual Study Start Date: November 2015
Estimated Study Completion Date: July 2019
Estimated Primary Completion Date: January 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: pamrevlumab (FG-3019)
Each subject will receive pamrevlumab (FG-3019) (35 mg/kg, every 2 weeks) for at least one year with the possibility for an additional 6 months of treatment if their pulmonary function tests show stabilization or improvement in the first 52 weeks of treatment.
Drug: pamrevlumab (FG-3019)
pamrevlumab (FG-3019), 10 mg/mL, single dose vials
Other Name: Monoclonal Antibody to CTGF

Detailed Description:
Each subject will receive pamrevlumab (35 mg/kg) every two weeks by intravenous infusion for at least one year. Subjects may continue on the study for an additional 6 months (for a total of a year and a half) if their pulmonary function tests show stabilization or improvement in the first 52 weeks of treatment and the FibroGen Medical Monitor approves of their continuing. All subjects will be closely monitored for safety. Efficacy assessments will be performed routinely over the course of the study.
  Eligibility

Ages Eligible for Study:   12 Years and older   (Child, Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  • Adequate hematological function

    1. Platelets >100,000/mcL
    2. Hemoglobin >12 g/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 ≤1.5xULN
  • 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 78 weeks of treatment and follow-up would be completed, or could impair the assessment of study results
  • Anticipated spine surgery within 78 weeks
  • Severe uncontrolled heart disease

    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 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.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02606136

Contacts
Contact: Gustavo Lorente 415-978-1441 glorente@fibrogen.com

Locations
United States, California
David Geffen School of Medicine at UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Andres Vargas    310-825-3264      
Principal Investigator: Perry Shieh, MD         
University of California San Francisco - Benioff Children's Hospital Recruiting
San Francisco, California, United States, 94143
Contact: Maria Kucherzki    415-502-7298      
Principal Investigator: Jonathan Strober, MD         
United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045
Contact: Lauri Filar    720-777-5173      
Principal Investigator: Joanne Janas, MD         
United States, Iowa
University of Iowa Children's Hospital Recruiting
Iowa City, Iowa, United States, 52242
Contact: Diane Recker    319-335-6073      
Principal Investigator: Katherine Mathews, MD         
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02115
Contact: Timothy Harrington    857-217-4677      
Principal Investigator: Basil Darras, MD         
United States, Missouri
Washington University in St. Louis School of Medicine Recruiting
Saint Louis, Missouri, United States, 63110
Contact: Alyssa Sonsoucie    513-636-3202      
Principal Investigator: Anne Connolly, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Ray Hu    513-803-1875      
Principal Investigator: Cuixia Tian, MD         
United States, Oregon
Shriner's Hospital for Children - Portland Recruiting
Portland, Oregon, United States, 97239
Contact: Coleman Hilton    971-282-4613      
Principal Investigator: Erika L Finanger, MD         
United States, Pennsylvania
The Children's Hospital of Philadelphia Recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Michele Bergman    215-425-2111      
Principal Investigator: John Brandsema, MD         
United States, Texas
Children's Medical Center Ambulatory Care Pavilion Recruiting
Dallas, Texas, United States, 75207
Contact: Holly Lawrence    214-456-2463      
Principal Investigator: Diana Castro, MD         
Sponsors and Collaborators
FibroGen
  More Information

Responsible Party: FibroGen
ClinicalTrials.gov Identifier: NCT02606136     History of Changes
Other Study ID Numbers: FGCL-3019-079
Study First Received: November 4, 2015
Last Updated: August 15, 2017
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

ClinicalTrials.gov processed this record on August 18, 2017