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

NCT02606136 | Terminated Phase 2 Results DMC FDA Drug

• 2 other identifiers: FGCL-3019-0792023-000321-80
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 10

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 participants with DMD.

Conditions

Duchenne Muscular Dystrophy

Keywords

Duchenne; muscular; dystrophy; DMD; non-ambulatory

Outcome Measures

Primary Outcomes (1)

• Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104

  FVC is a standard pulmonary function test used to quantify respiratory muscle weakness. FVC was the volume of air that can forcibly be blown out after full inspiration in the upright position, measured in liters. Predicted FVC is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FVC = (observed value)/(predicted value) \* 100%. Analysis was done using a random coefficient model (RCM), which included visit in years (as a continuous variable), and baseline efficacy as fixed effects, the intercept and the linear slope of visit as random effect, and individual participants as participant effect.

  Baseline, Week 104

Secondary Outcomes (9)

• Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104

  Baseline, Week 104

• Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104

  Baseline, Week 104

• Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104

  Baseline, Week 104

• Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104

  Baseline, Week 104

• Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104

  Baseline, Week 104

Study Arms (1)

Pamrevlumab

EXPERIMENTAL

Participants will receive pamrevlumab 35 milligrams (mg)/kilogram (kg) by intravenous (IV) infusion every 2 weeks for a minimum of 104 weeks. Participants who complete the main study, will continue to receive pamrevlumab 35 mg/kg by IV infusion every 2 weeks for a minimum of up to 208 weeks in the OLE.

Drug: Pamrevlumab

Interventions

Pamrevlumab DRUG

Pamrevlumab, 10 milligrams (mg)/milliliter (mL), single dose vials

Also known as: Monoclonal Antibody to Connective tissue growth factor (CTGF), FG-3019

Pamrevlumab

Eligibility Criteria

• Age: 12 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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
• Platelets \>100,000/microliter (μL)

You may not qualify if:

• 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:
• 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 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.

Sponsors & Collaborators

• Kyntra Bio: lead

Study Sites (10)

David Geffen School of Medicine at UCLA

Los Angeles, California, 90095, United States

Location

University of California San Francisco - Benioff Children's Hospital

San Francisco, California, 94143, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Rare Disease Research

Atlanta, Georgia, 30318, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University in St. Louis School of Medicine

St Louis, Missouri, 63110, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Shriner's Hospital for Children - Portland

Portland, Oregon, 97239, United States

Location

The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Children's Medical Center Ambulatory Care Pavilion

Dallas, Texas, 75207, United States

Location

Related Publications (1)

• Rayego-Mateos S, Morgado-Pascual JL, Lavoz C, Rodrigues-Diez RR, Marquez-Exposito L, Tejera-Munoz A, Tejedor-Santamaria L, Rubio-Soto I, Marchant V, Ruiz-Ortega M. CCN2 Binds to Tubular Epithelial Cells in the Kidney. Biomolecules. 2022 Feb 3;12(2):252. doi: 10.3390/biom12020252.

  PMID: 35204752 DERIVED

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

pamrevlumab

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Results Point of Contact

• Title: Clinical Trial Information Desk
• Organization: FibroGen, Inc.

FG3019-079DMDStudy@fibrogen.com

415-978-1441

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 4, 2015
• First Posted: November 17, 2015
• Study Start: January 4, 2016
• Primary Completion: May 7, 2020
• Study Completion: August 9, 2023
• Last Updated: August 27, 2024
• Results First Posted: December 30, 2021

Record last verified: 2024-07

Locations

US (10)