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Duchenne Muscular DystrophyPhase 3LELANTOS-1NCT04371666

Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)

A Phase 3 study testing Pamrevlumab and Corticosteroids in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 53 sites in 14 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older). Read more about this study

Who is it for?
Ages 12 years and older, men only
Conditions the study is for
Duchenne Muscular Dystrophy
What is being tested?
Pamrevlumab and Corticosteroids
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
98 participants
Where
53 sitesin 14 countries
Study dates
10 Aug 2020to 17 Aug 2023
Phase
Phase 3
Are there results?
Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 12 years and older
  • Men only
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Males at least 12 years of age, non-ambulatory at screening initiation
  • Written consent by participant and/or legal guardian as per regional/ country and/or Institutional Review Board (IRB)/Independent Ethics Committee (IEC) requirements
  • Male participants with partners of childbearing potential must use contraception during the conduct of the study, and for 12 weeks after the last dose of study drug.
  • Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test
  • Brooke Score for Arms and Shoulders ≤5
  • Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) and cardiac muscle
Show all 22Show fewer
  • Able to perform spirometry
  • Average (of Screening and Day 0) percent predicted forced vital capacity (FVC) between 45 and 85, inclusive
  • Left ventricular ejection fraction ≥50% as determined by local cardiac MRI read at screening or within 3 months prior to randomization (Day 0)
  • If participants have a history of cardiomyopathy, then participant must be on a stable dose of cardiomyopathy/ heart failure medications (for example, angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and betablockers) for at least 1 month prior to screening. If participants have no diagnosis of cardiomyopathy, then no dose of cardiomyopathy/heart failure medication is required for eligibility.
  • 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 (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.
  • Agreement to receive annual influenza vaccinations during the course of the study.
  • Adequate renal function: cystatin C ≤1.4 mg/liter (L)
  • Adequate hematology and electrolytes parameters:
  • Platelets >100,000/microliter (μL)
  • Hemoglobin >12 grams (g)/deciliter (dL)
  • Absolute neutrophil count >1500/μL
  • Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus (P) levels are within a clinically accepted range for DMD participants.
  • Adequate hepatic function:
  • No history or evidence of liver disease
  • Gamma glutamyl transferase (GGT) ≤3x upper limit of normal (ULN)
  • Total bilirubin ≤1.5xULN

Who cannot join

  • Previous exposure to pamrevlumab
  • BMI ≥40 kg/square meter (m^2) or weight >117 kg
  • History of:
  • allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies
  • hypersensitivity to study drug or any component of study drug
  • hypersensitivity reaction to Gadolinium-based Contrast Agents (GBCA) required for MRI acquisition
Show all 16Show fewer
  • Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (for example, eteplirsen [exondys 51], ataluren, golodirsen [vyondys 53], casimersen [amondys 45]) within 5 half-lives of screening, whichever is longer, with the exception of the systemic corticosteroids, including deflazacort
  • Severe uncontrolled heart failure (NYHA Classes III-IV), or renal dysfunction, including any of the following:
  • Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
  • Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening
  • Participants with glomerular filtration rate (GFR) of less than 30 mL/minute (min)/1.73 m^2 or with other evidence of acute kidney injury as determined by investigator
  • Arrhythmia requiring anti-arrhythmic therapy
  • Requires ≥16 hours continuous ventilation
  • Hospitalization due to respiratory failure within the 8 weeks prior to screening
  • 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
  • The Investigator judges that the participant 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

Trial locations and contacts

53 sites in 14 countries.

53 sites in 14 countries

United States24 sites

  • C.S. Mott Children's Hospital

    Ann Arbor, Michigan, 48109-4234

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30318

  • Children's Hospital Colorado

    Aurora, Colorado, 80045

  • Kennedy Krieger Institute

    Baltimore, Maryland, 21205

  • Carolinas HealthCare System Neurosciences Institute-Neurology - Charlotte

    Charlotte, North Carolina, 28207

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205-2664

  • Children's Health Dallas/UTSW

    Dallas, Texas, 75207

  • University of Kansas Medical Center

    Fairway, Kansas, 66205

  • Spectrum Health Hospitals Helen DeVos Children's Hospital

    Grand Rapids, Michigan, 49503

  • Penn State Health Children's Hospital

    Hershey, Pennsylvania, 17033

  • University of Iowa

    Iowa City, Iowa, 52242

  • Arkansas Children's

    Little Rock, Arkansas, 72202

  • University of California Los Angeles Medical Center

    Los Angeles, California, 90045

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232

  • Children's Specialty Group - Medical Center Office

    Norfolk, Virginia, 23507

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104

  • Shriners Hospital for Children

    Portland, Oregon, 97239

  • UC Davis Health

    Sacramento, California, 95817

  • University of Utah Health

    Salt Lake City, Utah, 84108

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Washington University School of Medicine in Saint Louis

    St Louis, Missouri, 63110

  • UMASS Med School

    Worcester, Massachusetts, 01655

Other trials in United States

Italy4 sites

  • Istituto di Ricovero e Cura a Carattere Scientifico Eugenio Medea - Lombardia

    Lecco, 23842

  • IRRCS Ospedale San Raffaele

    Milan, 20132

  • Fondazione Policlinico Universitario Agostino Gemelli

    Rome, 00168

  • Ospedale Pediatrico Bambino Gesù - Roma - Gianicolo

    Rome, 165

Other trials in Italy

Belgium3 sites

  • Universitair Ziekenhuis Gent

    Ghent, 9000

  • Universitair Ziekenhuis Leuven - Campus Gasthuisberg

    Leuven, 3000

  • Centre Hospitalier Régional de la Citadelle

    Liège, 4000

Other trials in Belgium

China3 sites

  • Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

    Beijing, 100730

  • West China Second University Hospital, Sichuan University

    Chengdu, Sichuan, 610041

  • Children's Hospital of Chongqing Medical University

    Chongqing, Chongqing Municipality, 400015

Other trials in China

France3 sites

  • CHU de Nantes - Hotel Dieu

    Nantes, 44093

  • Association Institut de Myologie

    Paris, 75012

  • Hopital Hautepierre

    Strasbourg, 67098

Other trials in France

Spain3 sites

  • Hospital General Universitario de Alicante

    Alicante, 3010

  • Hospital Universitari Vall d'Hebrón

    Barcelona, 08035

  • Hospital Universitari i Politecnic La Fe

    Valencia, 46026

Other trials in Spain

United Kingdom3 sites

  • Leeds Teaching Hospitals NHS Trust

    Leeds, LS1 3EX

  • University College London Hospitals NHS Foundation Trust

    London, WC1N 3BG

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, OX3 9DU

Other trials in United Kingdom

Czechia2 sites

  • Fakultní Nemocnice Brno - Dětská Nemocnice

    Brno, 613 00

  • Klinika dÄ>tské neurologie, Neuromuskulární centrum

    Prague, 150 06

Other trials in Czechia

Israel2 sites

  • The Chaim Sheba Medical Center

    Tel Aviv, 5265601

  • The Edith Wolfson Medical Center

    Tel Aviv, 5822012

Other trials in Israel

Netherlands2 sites

  • Leiden Universitair Medisch Centrum

    Leiden, 2333 ZA

  • Radboud Universitair Medisch Centrum

    Nijmegen, Gelderland, 6525

Other trials in Netherlands

Australia1 site

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052

Other trials in Australia

Austria1 site

  • Klinik Favoriten

    Vienna, 1100

Other trials in Austria

Canada1 site

  • London Health Sciences Centre

    London, Ontario, N6A 5W9

Other trials in Canada

Switzerland1 site

  • Inselspital Universitätsspital Bern

    Bern, 3010

Other trials in Switzerland

About this study

Official 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)

To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).

Read the detailed description

This is a global, Phase 3, randomized, double-blind trial of pamrevlumab or placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy, aged 12 years and older. Approximately 90 male participants will be randomized at a 1:1 ratio to Arm A (pamrevlumab + systemic corticosteroid) or Arm B (placebo+ systemic corticosteroid), respectively.

Participants must be fully informed of the potential benefits of approved products and make an informed decision that they prefer to participate in a clinical trial in which they could be randomized to placebo.

This trial has 3 study periods:

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

In the screening period, participants will be evaluated per the protocol inclusion/exclusion criteria to determine eligibility for participation in this trial.

During the treatment period, each participant will receive pamrevlumab or placebo at 35 mg/kg every 2 weeks for up to 52 weeks.

Participants who complete the 52-week study (either arm) may be eligible for rollover into an open-label extension treatment (OLE) with pamrevlumab + systemic corticosteroids.

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

Being tested
Pamrevlumab, Placebo, Corticosteroids
Keywords
Duchenne Muscular Dystrophy, DMD
Medical subject headings
Muscular Dystrophy, Duchenne

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • PamrevlumabDrug

    Also called FG-3019

    Pamrevlumab per dose and schedule specified in the arm description

  • PlaceboDrug

    Matching placebo per schedule specified in the arm description

  • CorticosteroidsDrug

    Systemic deflazacort or equivalent potency of corticosteroids administered orally

Study groups in NCT04371666
Study groupRoleWhat they receive
PamrevlumabExperimentalPamrevlumab 35 milligrams (mg)/kilogram (kg) intravenously (IV) every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Receives: Drug: Pamrevlumab; Drug: Corticosteroids
PlaceboPlacebo comparatorMatching placebo IV every 2 weeks + systemic deflazacort or equivalent potency of corticosteroids administered orally for up to 52 weeks
Receives: Drug: Placebo; Drug: Corticosteroids

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52

    The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items, 12 points), elbow (middle, 9 items, 17 points) and wrist/hand (distal, 7 items, 13 points). Higher scores indicate higher level of function. Total score ranges from 0-42 points and is the sum of the scores for the 3 subscales. Analysis was done using a random coefficient model (RCM), which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline ordinal PUL entry score as covariate.

    Measured: Baseline, Week 52

Secondary outcomes (4)

  1. Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 52, Assessed by Spirometry

    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 an RCM, which included fixed effects of time (as a continuous variable), treatment, and treatment-by-time interaction, with baseline value as covariate.

    Measured: Baseline, Week 52

  2. Change From Baseline in the Grip Strength of the Hands at Week 52, Assessed by Hand Held Myometry (HHM)

    The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Grip Strength was analyzed using a MMRM with fixed effects for treatment, visit (as a factor), treatment-by-visit interaction, and covariates (baseline values).

    Measured: Baseline, Week 52

  3. Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF %) at Week 52, Assessed by Magnetic Resonance Imaging (MRI)

    LVEF% is an important measure of cardiac function. LVEF is a fraction of blood (in percent) pumped out of the left ventricle of the heart (the main pumping chamber). The LVEF% was analyzed using an analysis of covariance (ANCOVA) model with treatment and baseline value.

    Measured: Baseline, Week 52

  4. Change From Baseline in Percent Predicted Peak Expiratory Flow (ppPEF) at Week 52, Assessed by Spirometry

    The ppPEF is a measure of the maximal or peak flow produced during an exhalation with maximal effort and, as such, is the most effort-dependent measure of lung function. The ppFEV1 was analyzed using an RCM including fixed effects of time, treatment, and treatment-by-time interaction, with baseline as covariate.

    Measured: Baseline, Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 May 2020
  2. Study start10 Aug 2020
  3. Primary completion13 Feb 2023
  4. Study completion17 Aug 2023

Why it stopped: Study did not meet its primary endpoint.

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
98 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT04371666
Sponsor's protocol ID
FGCL-3019-093
Other IDs
2020-000698-26
Acronym
LELANTOS-1
Status last verified
Feb 2024
First submitted
29 Apr 2020
Last updated
12 Mar 2024
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Undecided

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Pamrevlumab and Corticosteroids in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 53 sites in 14 countries.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 22 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 12 Mar 2024. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 53 sites in United States, Italy, Belgium, China, France and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Mar 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in the Total Score of Performance of Upper Limb (PUL) 2.0 Version at Week 52 (time frame: Baseline, Week 52). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT04371666.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 12 Mar 2024.

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