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Trial record 3 of 3 for:    pamrevlumab | dmd

A Phase 3 Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids, in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)

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ClinicalTrials.gov Identifier: NCT04632940
Recruitment Status : Recruiting
First Posted : November 17, 2020
Last Update Posted : March 15, 2021
Sponsor:
Information provided by (Responsible Party):
FibroGen

Brief Summary:
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every two weeks in ambulatory subjects with Duchenne muscular dystrophy (age 6 to <12 years).

Condition or disease Intervention/treatment Phase
Duchenne Muscular Dystrophy Drug: Pamrevlumab Drug: Placebo Phase 3

Detailed Description:

This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy, aged 6 to <12 years (ambulatory subjects only). Approximately 70 subjects will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic deflazacort or equivalent potency of corticosteroids administered orally) or Arm B (placebo+ systemic deflazacort or equivalent potency of corticosteroids administered orally), respectively.

Subjects must be fully informed of the potential benefits of approved products and make an informed decision when participating in a clinical trial in which they could be randomized to placebo.

Subjects will be randomized in a 1:1 ratio to one of the two study treatment arms; pamrevlumab or placebo. Randomization will be stratified by exon 44 deletion.

The main study has three study periods:

  • Screening period: Up to 4 weeks
  • Treatment period: 52 weeks
  • Safety Follow-up period/final assessment: A visit 28 days (+/- 3 Days) and a final safety follow-up phone call 60 days (+ 3 Days) after the last dose

Each subject will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Subjects who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab.

Subjects who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 70 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination With Systemic Corticosteroids in Ambulatory Subjects With Duchenne Muscular Dystrophy (DMD)
Actual Study Start Date : December 11, 2020
Estimated Primary Completion Date : January 2023
Estimated Study Completion Date : March 2023


Arm Intervention/treatment
Experimental: Arm A
pamrevlumab 35 mg/kg IV Q2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally
Drug: Pamrevlumab
pamrevlumab 35 mg/kg IV Q2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally
Other Name: FG-3019

Placebo Comparator: Arm B
matching placebo IV Q2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally
Drug: Placebo
matching placebo IV Q2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally




Primary Outcome Measures :
  1. Ambulatory function assessment: [ Time Frame: baseline to Week 52 ]
    Change in NorthStar Ambulatory Assessment (NSAA) Linearized total score


Secondary Outcome Measures :
  1. Other Muscle function assessments: [ Time Frame: baseline to Week 52 ]
    Change in 4-stair climb Velocity (4SCV) assessment

  2. Other Muscle function assessments: [ Time Frame: baseline to Week 52 ]
    Change in the 10-meter walk/run test

  3. Other Muscle function assessments: [ Time Frame: baseline to Week 52 ]
    Changes in Time to Stand (TTSTAND)

  4. Other Muscle function assessments: [ Time Frame: baseline to Week 52 ]
    Time to Loss of Ambulation (LoA)



Information from the National Library of Medicine

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.


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Ages Eligible for Study:   6 Years to 11 Years   (Child)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Age, and consent:

  1. Males at least 6 to <12 years of age at screening initiation
  2. Written consent by legal guardian as per regional/ country and/or IRB/IEC requirements

    DMD diagnosis:

  3. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.

    Pulmonary criteria:

  4. Average (of screening and day 0) percent predicted FVC above 45%
  5. On a stable dose of systemic corticosteroids for a minimum of 6 months, with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (e.g. prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) or stable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.

    Performance criteria:

  6. Able to complete 6MWD test with a distance of at least 270M but no more than 450M on two occasions within 3 months prior to Randomization with ≤10% variation between these two tests.
  7. Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses) at screening visit.
  8. Able to undergo MRI test for the lower extremities vastus lateralis muscle.

    Vaccination:

  9. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharide vaccine as per national recommendations) and is receiving annual influenza vaccinations.

    Laboratory criteria:

  10. Adequate renal function: cystatin C ≤1.4 mg/L
  11. Adequate hematology and electrolytes parameters:

    1. Platelets >100,000/mcL
    2. Hemoglobin >12 g/dL
    3. Absolute neutrophil count >1500 /μL
    4. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range
  12. Adequate hepatic function:

    1. No history or evidence of liver disease
    2. Gamma glutamyl transferase (GGT) ≤3x upper limit of normal (ULN)
    3. Total bilirubin ≤1.5xULN

Exclusion Criteria:

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 allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
  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, ataluren, golodirsen) within 5 half-lives of screening, whichever is longer with the exception of the systemic corticosteroids, including deflazacort

    Pulmonary 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), including any of the following:

    1. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
    2. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening
  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

Information from the National Library of Medicine

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): NCT04632940


Contacts
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Contact: Jessica Charpentier 415.978.1346 LELANTOS@fibrogen.com

Locations
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United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 72202
United States, Georgia
Rare Disease Research Center Recruiting
Atlanta, Georgia, United States, 30318
United States, Massachusetts
University of Massachusetts Memorial Medical Center Recruiting
Worcester, Massachusetts, United States, 01655
Sponsors and Collaborators
FibroGen
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Responsible Party: FibroGen
ClinicalTrials.gov Identifier: NCT04632940    
Other Study ID Numbers: FGCL-3019-094
First Posted: November 17, 2020    Key Record Dates
Last Update Posted: March 15, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by FibroGen:
Duchenne Muscular Dystrophy, DMD
Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Additional relevant MeSH terms:
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Muscular Dystrophies
Muscular Dystrophy, Duchenne
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked