A Phase 3 interventional study of Pamrevlumab and Placebo in Duchenne Muscular Dystrophy, sponsored by FibroGen. Terminated at 52 sites in 11 countries. Open to male participants aged 6 Years to 11 Years. Per ClinicalTrials.gov, last updated 2024-08-26.
Sponsored by FibroGen · Phase 3, Interventional, and Treatment
Among 232 studies in Muscular Dystrophy, Duchenne with a site in United States — 35 open to participants now, 60 under way, 8 of them phase 3. Muscular Dystrophy, Duchenne · United States.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, this one is the 41st largest by enrollment of the 171 interventional ones that give a count, is one of 12 that started in 2021, is one of 32 in phase 3, and was the 165th registered.
481 studies on the registry are indexed under Muscular Dystrophy, Duchenne; 107 are open to participants now.
Browse Muscular Dystrophy, Duchenne studies →35 of the 232 studies in Muscular Dystrophy, Duchenne with a site in United States are open now. Recruiting first, the newest first:
Vanderbilt University Medical Center · Sites in United States
Ages 18+ · Men only
Sarepta Therapeutics, Inc. · Sites in United States
Ages 4+ · Men only
University of Florida · Sites in United States
Ages 6+ · Men only
Vanderbilt University Medical Center · Sites in United States
Ages 8–18 · Men only
University of Rochester · Sites in United States
Ages 0 days–3 yrs · Men only
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 66 Muscular Dystrophy, Duchenne studies recruiting now, 30 (45%) set no upper age limit and 34 (52%) stop at 75 or younger; 59 take children under 18; 12 take healthy volunteers.
This study: 6 Years to 11 Years · male · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
232 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 57 · Phase 2 90 · Phase 3 40 · Phase 4 5
First in 2000; this study’s year is marked. 12 started in 2021.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 73 is above the median of 26 across 332 interventional studies indexed under Muscular Dystrophy, Duchenne.
Median enrollment 25 across the 171 interventional studies, 75 across the 55 observational. Of the 160 that completed or were terminated, 78 (49%) have results posted.
This tab as a report to print or save as PDF → With an account.
130 of 232 have an industry lead sponsor. The 10 most frequent:
This study is registered under FibroGen, one of the names counted as Kyntra Bio. Under that name alone:
FibroGen is the lead sponsor of 39 studies on the registry; none are open to participants now.
Of its 14 completed or terminated interventional studies of FDA-regulated products, 14 (100%) have results posted.
Kyntra Bio has 3 studies filed under Muscular Dystrophy, Duchenne — fewer than ten, so there is no comparison to draw.
19 of the studies in Muscular Dystrophy, Duchenne with a site in United States that are under way reach primary completion within a year, 1 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05881408Phase 3Sarepta Therapeutics, Inc.A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) | 2027-05-31 |
| NCT06290713University of FloridaVasodilator and Exercise Study for DMD (VASO-REx) | 2026-11 |
| NCT06450639Hoffmann-La RocheA Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD) | 2026-11-18 |
| NCT03368742Solid Biosciences Inc.Microdystrophin Gene Transfer Study in Adolescents and Children With DMD | 2026-11-30 |
| NCT03655223RTI InternationalEarly Check: Expanded Screening in Newborns | 2026-11-30 |
Add all 19 to your calendar (.ics) → With an account.
Among the 232 studies in Muscular Dystrophy, Duchenne with a site in United States, from their registry updates:
This tab as a report to print or save as PDF → With an account.
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids administered every 2 weeks in ambulatory participants with Duchenne muscular dystrophy (DMD) (age 6 to \<12 years).
This is a global, randomized, double-blind, trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with DMD, aged 6 to \<12 years (ambulatory participants only). Approximately 70 participants 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. Randomization will be stratified by exon 44 deletion for analysis. Stratification has no impact upon treatment assignment nor dosage.
Participants 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.
The main study has 3 study periods:
Each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks. Participants who complete 52 weeks of treatment may be eligible for an open-label extension (OLE), offering extended treatment with pamrevlumab.
Participants who discontinue study treatment for any reason should be encouraged to return to the investigative site to complete final safety and efficacy assessments.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Age, and consent:
Written consent by participant and/or legal guardian as per regional/ country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
DMD diagnosis:
Medical history includes diagnosis of DMD and confirmed Duchenne mutation, including status of exon 44 using a validated genetic test.
Pulmonary criteria:
On a stable dose of systemic 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 (for example, 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:
Able to undergo magnetic resonance imaging (MRI) test for the lower extremities vastus lateralis muscle.
Vaccination:
Agreement to receive annual influenza vaccinations during the conduct of the study.
Laboratory criteria:
Adequate hematology and electrolytes parameters:
Adequate hepatic function:
Exclusion Criteria:
General Criteria:
History of
Exposure to any drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (for example, eteplirsen, ataluren, golodirsen, casimersen) within 5 half-lives of screening, whichever is longer with the exception of the systemic , including deflazacort
Pulmonary and Cardiac criteria:
Severe uncontrolled heart failure ( [NYHA] Classes III-IV) or renal dysfunction, including any of the following:
Any other evidence of structural or functional heart abnormality
Clinical judgment:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
BMI (body mass index)
Weight in kilograms divided by height in metres squared. Example: 70 kg and 1.75 m gives 70 ÷ (1.75 × 1.75) ≈ 22.9.
What counts as overweight or obesity differs between countries (some use lower cut-offs), so go by the range the study states.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Pamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Drug: Pamrevlumab · Drug: Corticosteroids
Matching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Drug: Placebo · Drug: Corticosteroids
Pamrevlumab will be administered per dose and schedule specified in the arm description.
Also known as: FG-3019
Placebo will be administered per schedule specified in the arm description.
Systemic deflazacort or equivalent potency of corticosteroids administered orally
Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52
The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).
Time frame: Baseline, Week 52
Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52
The 4SCV (centimeters \[cm\]/second \[sec\]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb.
Time frame: Baseline, Week 52
Change From Baseline in the 10-Meter Walk/Run Test at Week 52
The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec).
Time frame: Baseline, Week 52
Change From Baseline in Time to Stand (TTSTAND) at Week 52
The time (in sec) required for a participant to stand from supine position has been reported. A longer time taken reflected a worse outcome.
Time frame: Baseline, Week 52
Time to Loss of Ambulation (LoA) From Baseline to Week 52
Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates.
Time frame: Baseline to Week 52
| Milestone | Pamrevlumab | Placebo |
|---|---|---|
| Started | 37 | 36 |
| Received at least 1 dose of study drug | 36 | 36 |
| Completed | 35 | 36 |
| Not completed | 2 | 0 |
| Withdrew: Participant/legal guardian decision | 2 | 0 |
| Milestone | Pamrevlumab | Placebo |
|---|---|---|
| Started | 34 | 35 |
| Received at least 1 dose of study drug | 34 | 34 |
| Completed | 0 | 0 |
| Not completed | 34 | 35 |
| Withdrew: Adverse event | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Physician decision | 1 | 0 |
| Withdrew: Participant/legal guardian decision | 11 | 13 |
| Withdrew: Sponsor decision to terminate study | 21 | 19 |
| Withdrew: Other than specified | 0 | 1 |
| Withdrew: Entered into ole but not treated | 0 | 1 |
The NSAA consisted of 17 activities, each scored as 0 (activity could not be performed), 1 (modified method but achieved goal without physical assistance from another), or 2 (normal, achieved goal without assistance). The sum of these 17 scores was used to form a total score ranging from 0 (worst) to 34 (fully independent function). If fewer than 15 of the 17 activities were performed, the total score was considered missing. If 15 to 16 activities were performed, the total score was calculated by multiplying the sum of the scores in the x activities that were performed by 17/x. If an activity could not be performed due to disease progression/loss of ambulation, a score of 0 was assigned. Higher scores indicated better functioning. Least square (LS) mean and standard error (SE) were analyzed using a mixed model for repeated measure (MMRM).
| units on a scale | Pamrevlumab | Placebo |
|---|---|---|
| Change From Baseline in North Star Ambulatory Assessment (NSAA) Total Score at Week 52 | -3.022 ± 0.5505 | -2.494 ± 0.6962 |
The 4SCV (centimeters \[cm\]/second \[sec\]) was calculated as the ratio of the total height (cm) of stairs climbed divided by the number of seconds taken to complete the 4-stair climb.
| cm/sec | Pamrevlumab | Placebo |
|---|---|---|
| Change From Baseline in 4-Stair Climb Velocity (4SCV) Assessment at Week 52 | -1.858 ± 4.5459 | -3.797 ± 5.1899 |
The time (in sec) required for a participant to run or walk a distance of 10 meters as quickly as possible was calculated as velocity (meters/sec).
| meters/sec | Pamrevlumab | Placebo |
|---|---|---|
| Change From Baseline in the 10-Meter Walk/Run Test at Week 52 | -0.176 ± 0.2193 | -0.196 ± 0.3552 |
The time (in sec) required for a participant to stand from supine position has been reported. A longer time taken reflected a worse outcome.
| sec | Pamrevlumab | Placebo |
|---|---|---|
| Change From Baseline in Time to Stand (TTSTAND) at Week 52 | 2.24 ± 3.353 | 1.94 ± 3.688 |
Time (days) to LoA was defined as the number of days from randomization to the date of LoA, or all-cause death based on observed data, whichever occurred earlier during the on-study period. Median time (days) to LoA was calculated using Kaplan Meier Survival Estimates.
| days | Pamrevlumab | Placebo |
|---|---|---|
| Time to Loss of Ambulation (LoA) From Baseline to Week 52 | NA (NA to NA) | NA (NA to NA) |
Collected over Baseline up to Week 129. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| DB Period: Pamrevlumab | 0/37 (0%) | 3/36 (8.3%) | 35/36 (97.2%) |
| DB Period: Placebo | 0/36 (0%) | 1/36 (2.8%) | 33/36 (91.7%) |
| OLE Period: Pamrevlumab | 0/69 (0%) | 0/68 (0%) | 45/68 (66.2%) |
| Event | DB Period: Pamrevlumab | DB Period: Placebo | OLE Period: Pamrevlumab |
|---|---|---|---|
| MyocarditisCardiac disorders | 1/36 | 0/36 | 0/68 |
| Upper respiratory tract infectionInfections and infestations | 1/36 | 0/36 | 0/68 |
| Knee deformityMusculoskeletal and connective tissue disorders | 1/36 | 0/36 | 0/68 |
| RhabdomyolysisMusculoskeletal and connective tissue disorders | 0/36 | 1/36 | 0/68 |
| Event | DB Period: Pamrevlumab | DB Period: Placebo | OLE Period: Pamrevlumab |
|---|---|---|---|
| COVID-19Infections and infestations | 16/36 | 8/36 | 4/68 |
| HeadacheNervous system disorders | 14/36 | 5/36 | 5/68 |
| VomitingGastrointestinal disorders | 10/36 | 7/36 | 12/68 |
| NasopharyngitisInfections and infestations | 10/36 | 7/36 | 11/68 |
| PyrexiaGeneral disorders | 9/36 | 7/36 | 10/68 |
| DiarrhoeaGastrointestinal disorders | 8/36 | 3/36 | 6/68 |
| Upper respiratory tract infectionInfections and infestations | 4/36 | 8/36 | 6/68 |
| FallInjury, poisoning and procedural complications | 7/36 | 2/36 | 3/68 |
| CoughRespiratory, thoracic and mediastinal disorders | 6/36 | 4/36 | 10/68 |
| Nasal congestionRespiratory, thoracic and mediastinal disorders | 3/36 | 6/36 | 1/68 |
The intent-to-treat (ITT) set included all randomized participants.
| Age, Continuous(years) | Pamrevlumab | Placebo | Total |
|---|---|---|---|
| Mean | 9.1 ± 1.46 | 9.0 ± 1.56 | 9.0 ± 1.50 |
| Sex: Female, Male(Participants) | Pamrevlumab | Placebo | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 37 | 36 | 73 |
| Ethnicity (NIH/OMB)(Participants) | Pamrevlumab | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 1 | 5 | 6 |
| Not Hispanic or Latino | 34 | 28 | 62 |
| Unknown or Not Reported | 2 | 3 | 5 |
| Race (NIH/OMB)(Participants) | Pamrevlumab | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 10 | 9 | 19 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 1 | 1 | 2 |
| White | 23 | 22 | 45 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 3 | 4 | 7 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Undecided
Ran from Mar 2021 to primary completion in Jun 2023, 27 months.
Results were first posted in Aug 2024, 1 year 2 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jul 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Pamrevlumab
Muscular Dystrophy, Duchenne →