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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 : January 25, 2019
Sponsor:
Information provided by (Responsible Party):
FibroGen

Tracking Information
First Submitted Date  ICMJE November 4, 2015
First Posted Date  ICMJE November 17, 2015
Last Update Posted Date January 25, 2019
Actual Study Start Date  ICMJE November 2015
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: September 11, 2017)
Annual change in percent predicted annual forced vital capacity (FVC) during treatment with pamrevlumab. [ Time Frame: From baseline to 104 weeks ]
Original Primary Outcome Measures  ICMJE
 (submitted: November 13, 2015)
Difference in annual forced vital capacity (FVC) decline during treatment of FG-3019 compared with the estimated annual decline prior to FG-3019 treatment. [ Time Frame: From baseline to 2 years ]
Change History Complete list of historical versions of study NCT02606136 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: September 11, 2017)
  • 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 ]
Original Secondary Outcome Measures  ICMJE
 (submitted: November 13, 2015)
  • Change in forced expiratory volume (FEV1) [ Time Frame: From baseline to 1 year ]
  • Change in maximum inspiratory pressure (MIP) [ Time Frame: From baseline to 1 year ]
  • Change in maximum expiratory pressure (MEP) [ Time Frame: From baseline to 1 year ]
  • Change in peak expiratory flow (PEF) [ Time Frame: From baseline to 1 year ]
  • Change in peak cough flow [ Time Frame: From baseline to 1 year ]
  • Change in left ventricular ejection fraction (LVEF) [ Time Frame: From baseline to 1 year ]
  • Change in Performance of Upper Limb (PUL) Score [ Time Frame: From baseline to 1 year ]
  • Change in grip strength [ Time Frame: From baseline to 1 year ]
  • Change in pinch strength [ Time Frame: From baseline to 1 year ]
  • Change in Brooke scale for upper extremity [ Time Frame: From baseline to 1 year ]
  • Change in cardiac fibrosis score assessed by MRI [ Time Frame: From baseline to 1 year ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
Official Title  ICMJE Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
Brief Summary 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.
Detailed Description Each subject will receive pamrevlumab (35 mg/kg) every two weeks by intravenous infusion for up to 156 weeks. After at least 10 to 12 subjects complete one year of treatment interim analysis may increase total subjects enrolled to approximately 32. All subjects will be closely monitored for safety. Efficacy assessments will be performed routinely over the course of the study.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Duchenne Muscular Dystrophy
Intervention  ICMJE Drug: pamrevlumab (FG-3019)
pamrevlumab (FG-3019), 10 mg/mL, single dose vials
Other Name: Monoclonal Antibody to CTGF
Study Arms  ICMJE Experimental: pamrevlumab (FG-3019)
Each subject will receive pamrevlumab (FG-3019) (35 mg/kg, every 2 weeks) for up to 156 weeks.
Intervention: Drug: pamrevlumab (FG-3019)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Active, not recruiting
Estimated Enrollment  ICMJE
 (submitted: November 13, 2015)
22
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE April 2021
Estimated Primary Completion Date April 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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 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

    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.
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02606136
Other Study ID Numbers  ICMJE FGCL-3019-079
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
IPD Sharing Statement  ICMJE
Plan to Share IPD: Undecided
Responsible Party FibroGen
Study Sponsor  ICMJE FibroGen
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account FibroGen
Verification Date January 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP