Duchenne Muscular DystrophyPhase 2MISSIONNCT02606136
Trial of Pamrevlumab (FG-3019), in Non-Ambulatory Participants With Duchenne Muscular Dystrophy (DMD)
A Phase 2 study testing Pamrevlumab in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 10 sites in United States.
Trial 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. 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
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 10 sitesUnited States
- Study dates
- 4 Jan 2016to 9 Aug 2023
- Phase
- Phase 2
- 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
- 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
Show all 22Show fewer
- 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)
- Hemoglobin >12 grams (g)/deciliter (dL)
- Absolute neutrophil count >1500/μL
- Adequate hepatic function
- No history or evidence of liver disease
- Gamma glutamyl transferase (GGT) ≤3 x upper limit of normal (ULN)
- Total bilirubin ≤1.5 x ULN
- If sexually active, will use medically accepted contraceptives during participation in the study and for 3 months after the last dose of study drug
Who cannot join
- 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
Show all 13Show fewer
- 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.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
Rare Disease Research
Atlanta, Georgia, 30318
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Children's Medical Center Ambulatory Care Pavilion
Dallas, Texas, 75207
David Geffen School of Medicine at UCLA
Los Angeles, California, 90095
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Shriner's Hospital for Children - Portland
Portland, Oregon, 97239
University of California San Francisco - Benioff Children's Hospital
San Francisco, California, 94143
Washington University in St. Louis School of Medicine
St Louis, Missouri, 63110
About this study
Official title: Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
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.
Read the detailed description
The study will include a screening period, main study period, open-label extension (OLE) period, and follow-up period 4 weeks after the last dose. All participants who complete the main portion of the study for a minimum of 104 weeks (2 years) will be rolled over to an OLE for up to an additional 208 weeks (4 years).
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- Pamrevlumab
- Keywords
- Duchenne, muscular, dystrophy, DMD, non-ambulatory
- 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 Monoclonal Antibody to Connective tissue growth factor (CTGF), FG-3019
Pamrevlumab, 10 milligrams (mg)/milliliter (mL), single dose vials
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Pamrevlumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, Week 104
Secondary outcomes (9)
Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104
FEV1 is the amount of air which can be forcibly exhaled from the lungs in the first second of a forced exhalation. Predicted FEV1 is based on a formula using sex, age and height of a person, and is an estimate of healthy lung capacity. Percent of predicted FEV1= (observed value)/(predicted value) * 100%. Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104
Percent predicted PEF 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. Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104
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). Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104
The PUL module is an observer-administered performance battery of upper extremity mobility tasks for the shoulder (upper, 6 items: maximum score = 12), elbow (middle, 9 items: maximum score = 17) and wrist/hand (distal, 7 items: maximum score = 13). Higher scores of each item indicate higher level of function. Total score was calculated by adding the 3 level scores, with a maximum global score of 42 (total score range = 0-42; with higher score indicating better outcome). Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104
The HHM was used to measure distal upper arm strength (grip strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Pinch Strength, as Measured by HHM at Week 104
The HHM was used to measure distal upper arm strength (pinch strength). Data has been presented by dominant and non-dominant hand. Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104
Cardiac MRI was used to assess the cardiac fibrosis by detecting the presence of late gadolinium enhancement (LGE), a marker for myocardial fibrosis. Analysis was done using an 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.
Measured: Baseline, Week 104
Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104
T2-mapping with MRI was conducted to measure upper arm muscle fibrosis and fat in the biceps brachii muscle. Analysis was done using an 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. The visual score for muscle fat and fibrosis will be assessed using a modified 5-point Mercuri score in which 0 = normal muscle appearance and 5 = complete replacement of muscle by connective tissue and fat, where a lower score indicated visually healthier muscle. Change from baseline was calculated as the score at Week 104 - the score at baseline.
Measured: Baseline, Week 104
Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104
Analysis was done using an 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.
Measured: Baseline, Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2015 (estimated)
- Study start4 Jan 2016
- Primary completion7 May 2020
- Study completion9 Aug 2023
Why it stopped: Sponsor Decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- Kyntra Bio
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02606136
- Sponsor's protocol ID
- FGCL-3019-079
- Other IDs
- 2023-000321-80, 2023
- Acronym
- MISSION
- Status last verified
- Jul 2024
- First submitted
- 4 Nov 2015
- Last updated
- 27 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- Individual data sharing
- Undecided
Publications
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.
PubMed 35204752
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial of Pamrevlumab (FG-3019), a Monoclonal Antibody to Connective Tissue Growth Factor, in Non-Ambulatory Subjects With Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT02606136
- Acronym / sponsor ID
- MISSION / FGCL-3019-079
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- Duchenne, muscular, dystrophy, DMD, non-ambulatory
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor Decision
- Enrolment
- 21 participants (actual)
- Lead sponsor
- Kyntra BioIndustry (pharma, biotech, devices)
- Study start
- 4 Jan 2016 (actual)
- Primary completion
- 7 May 2020 (actual)
- Study completion
- 9 Aug 2023 (actual)
- First posted
- 17 Nov 2015 (estimated)
- Results
- Posted 30 Dec 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The study will include a screening period, main study period, open-label extension (OLE) period, and follow-up period 4 weeks after the last dose. All participants who complete the main portion of the study for a minimum of 104 weeks (2 years) will be rolled over to an OLE for up to an additional 208 weeks (4 years).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pamrevlumab | Experimental | Drug: PamrevlumabParticipants 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. |
Who may qualify
- Ages
- 12 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- 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
Key exclusion criteria (13 listed)
- 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:
- Arrhythmia requiring anti-arrhythmic therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PamrevlumabAlso: Monoclonal Antibody to Connective tissue growth factor (CTGF), FG-3019Pamrevlumab, 10 milligrams (mg)/milliliter (mL), single dose vials | Drug | Pamrevlumab |
Primary outcome
Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104
Time frame: Baseline, 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.
9 secondary outcomes
Change From Baseline in Percent Predicted Forced Expiratory Volume at 1 Second (ppFEV1) at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Percent Predicted Peak Expiratory Flow (PEF) at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Left Ventricular Ejection Fraction Percentage (LVEF%) at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Performance of Upper Limb (PUL) Total Score at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Grip Strength by Hand, as Measured by Hand Held Myometry (HHM) at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Pinch Strength, as Measured by HHM at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Cardiac Fibrosis (Scar Mass), as Measured by Magnetic Resonance Imaging (MRI) at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Upper Arm (Biceps Brachii MRI) Muscle Fat and Fibrosis Score, as Measured by MRI at Week 104
Time frame: Baseline, Week 104
Change From Baseline in Fat Fraction Percentage (%F), as Measured by MRI at Week 104
Time frame: Baseline, Week 104
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35204752Rayego-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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pamrevlumab in people with Duchenne Muscular Dystrophy, run by Kyntra Bio at 10 sites in United States.
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 13 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 27 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 30 Dec 2021). 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?
Annual Change From Baseline in Percent Predicted Forced Vital Capacity (ppFVC) at Week 104 (time frame: Baseline, Week 104). 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 NCT02606136, which also lists 1 linked publication.
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 27 Aug 2024.