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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 23 November 2020
Main ID:  EUCTR2020-000698-26-BE
Date of registration: 21/09/2020
Prospective Registration: Yes
Primary sponsor: FibroGen, Inc.
Public title: Evaluation of Pamrevlumab for the Treatment of Male Patients Affected by Non-ambulatory Duchenne Muscular Dystrophy and just Treated with Corticosteroids
Scientific title: A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Subjects with Non-ambulatory Duchenne Muscular Dystrophy (DMD)
Date of first enrolment:
Target sample size: 90
Recruitment status: NA
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2020-000698-26
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: yes
Randomised: yes
Open: no
Single blind: no
Double blind: yes
Parallel group: yes
Cross over: no
Other: yes
Other trial design description: Open-label extension
If controlled, specify comparator, Other Medicinial Product: no
Placebo: yes
Other: no
Number of treatment arms in the trial: 2
 
Phase:  Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Austria Belgium Canada Czech Republic France Israel Italy
Netherlands Slovakia Spain Switzerland Turkey United Kingdom United States
Contacts
Name: Clinical Trials Information   
Address:  409 Illinois Street 94158 San Francisco United States
Telephone:
Email: FG3019_093@Fibrogen.com
Affiliation:  FibroGen, Inc.
Name: Clinical Trials Information   
Address:  409 Illinois Street 94158 San Francisco United States
Telephone:
Email: FG3019_093@Fibrogen.com
Affiliation:  FibroGen, Inc.
Key inclusion & exclusion criteria
Inclusion criteria:
Double-blind phase:
Age, Consent, and Contraception
1. Males at least 12 years of age, non-ambulatory at screening initiation;
2. Written consent by patient and/or legal guardian as per regional/ country and/or IRB/IEC requirements;
3. Male subjects with partners of childbearing potential must use contraception during the conduct of the study, and for 3 months after the last dose of study drug.

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

Performance criteria:
5. Brooke Score for Arms and Shoulders =5:
6. Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) andcardiac muscle;
7. Able to perform spirometry.

Pulmonary and Cardiac criteria:
8. Reproducible (+/- 5% difference between screening and baseline) percent predicted FVC between 45 and 85, inclusive;
9. Left ventricular ejection fraction =50% as determined by cardiac MRI at screening or within 3 months prior to randomization (Day 0);
10. Prior diagnosis of cardiomyopathy, subjects must be on a stable regimen dose for cardiomyopathy/ heart failure medications (e.g., angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and beta-blockers) for at least 1 month prior to screening;
11. On a stable dose of systemic corticosteroids for a minimum of 6 months, with nos ubstantial 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) orstable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.

Vaccination:
12. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharidevaccine as per national recommendations) and is receiving annual influenza vaccinations.

Laboratory criteria:
13. Adequate renal function: cystatin C =1.4 mg/L;
14. Adequate hematology and electrolytes parameters:
a. Platelets >100,000/mcL
b. Hemoglobin >12 g/dL
c. Absolute neutrophil count >1500 /µL
d. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus(P) levels are within a clinically accepted range;
15. Adequate hepatic function:
a. No history or evidence of liver disease
b. Gamma glutamyl transferase (GGT) =3x upper limit of normal (ULN)
c. Total bilirubin =1.5xULN.

Open-label extension phase:
All patients must have completed treatment through the primary endpoint and completed the Week 52 visit on the double-blind phase. The study investigator must consider the subject medically stable for continued treatment. Written consent/assent by the patients and/or their legal guardian must be obtained prior to the patient’s participation in the open-label extension phase.
Are the trial subjects under 18? yes
Number of subjects for this age range: 30
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 55
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 5

Exclusion criteria:
Both phases:
General criteria:
1. Previous exposure to pamrevlumab;
2. BMI =40 kg/m2 or weight >117 kg;
3. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies;
4. 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) within 5 half-lives of screening, whichever is longer, with the exception of the systemic corticosteroids,including deflazacort.

Cardiac and Pulmonary assessment:
5.Severe uncontrolled heart failure (NYHA Classes III-IV), including any of the following:
a. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
b. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening;
6. Arrhythmia requiring anti-arrhythmic therapy;
7. Requires =16 hours continuous ventilation;
8. Hospitalization due to respiratory failure within the 8 weeks prior to screening;
9. 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.

Clinical judgments:
10.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 or psychiatric conditions which could confound efficacy assessment and/or safety assessment.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes
Health Condition(s) or Problem(s) studied
Non-ambulatory Duchenne Muscular Dystrophy
MedDRA version: 20.0 Level: PT Classification code 10013801 Term: Duchenne muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Intervention(s)

Product Name: Pamrevlumab
Product Code: FG-3019
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: PAMREVLUMAB
CAS Number: 946415-13-0
Current Sponsor code: FG-3019
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Solution for infusion
Route of administration of the placebo: Intravenous use

Primary Outcome(s)
Main Objective: Evaluation of the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in subjects with Duchenne muscular dystrophy (non-ambulatory, age 12 years and older).
Primary end point(s): Double-blind phase:
Function assessment:
• Change in the total score of Performance of Upper Limb Version 2.0

Open-label extension phase:
Function assessment:
• Annual mean change in the total score of Performance of Upper Limb Version 2.0.

Pulmonary assessment:
• Annual mean change in percent predicted forced vital capacity, assessed by spirometry;
• Annual mean change in percent predicted peak expiratory flow, assessed by spirometry.

Performance assessment:
• Annual mean change in the Grip strength of the hands extremities, assessed by Hand Held Myometry.

Cardiac assessment:
• Annual mean change in Left Ventricular Ejection Fraction percentage (LVEF %),assessed by MRI.

Fibrosis/MRI assessments:
• Annual mean change in fibrosis score of the biceps brachii, assessed by MRI;
• Annual mean change in Cardiac fibrosis score, assessed by Late Gadolinium Enhancement;
• Annual mean change in Myocardial Circumferential Strain percentage, assessed by cardiac MRI.

Both phases:
Safety assessments
• Treatment-emergent adverse events (TEAEs), treatment-emergent serious adverse events (TESAEs), clinically significant laboratory test abnormalities, and discontinuation of treatment due to TEAEs serve as the safety assessments for the open-label extension phase.
Secondary Objective: Not applicable
Timepoint(s) of evaluation of this end point: Double-blind phase:
Baseline
Week 52

Open-label extension phase:
Week 52
Week 104
Week 156
Week 208

Safety:
From screening throughout week 208
Secondary Outcome(s)
Secondary end point(s): Double-blind phase:
Pulmonary assessment:
• Change in percent predicted forced vital capacity,assessed by spirometry;
• Change in percent predicted peak expiratory flow,assessed by spirometry.

Performance assessment:
• Change in the Grip strength of the hands from baseline to Week 52, assessed by Hand Held Myometry.

Cardiac assessment:
• Change in Left Ventricular Ejection Fraction percentage (LVEF %), assessed by MRI.

Open-label extension phase:
Not applicable
Timepoint(s) of evaluation of this end point: Baseline
Week 52
Secondary ID(s)
126630
2020-000698-26-AT
FGCL-3019-093
NCT04371666
Source(s) of Monetary Support
FibroGen, Inc.
Secondary Sponsor(s)
Ethics review
Status:
Approval date:
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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