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Limb-Girdle Muscular DystrophyPhase 3NCT03783923

A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

A Phase 3 study testing Deflazacort in people with Limb-Girdle Muscular Dystrophy, run by PTC Therapeutics at 18 sites in 8 countries.

Terminated

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

Official record
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Trial summary

This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment). Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Limb-Girdle Muscular Dystrophy
What is being tested?
Deflazacort
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
18 sitesin 8 countries
Study dates
31 Oct 2019to 1 Jan 2021
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP] gene).
  • Ability to ascend 4 stairs greater than or equal to (≥) 2.5 seconds and be able to complete the ascent and descent both at screening and baseline.
  • Ability to understand the nature of the study and the consent form and to comply with study related procedures.
  • Must weigh between 35 to 112.5 kilograms (kg).

Who cannot join

  • Received ≥4 weeks of continuous, systemic corticosteroid therapy within 3 months of study screening visit.
  • Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction less than (<) 30 percent [%]) at screening.
  • Requires fulltime ventilator support.
  • History of chronic systemic fungal or viral infections.
  • History of recent bacterial infection (including tuberculosis) per discretion of the Investigator.
  • Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated hemoglobin (HbA1c) ≥6.5% (based on historical or present diagnosis).
Show all 12Show fewer
  • History of immunosuppression or other contraindications to glucocorticosteroid therapy.
  • Requires concomitant use or greater than (>) 1 week of drugs or substances that are moderate to strong cytochrome P3A4 (CYP3A4) inhibitors (for example, clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4 inducers (that is, rifampin, efavirenz, carbamazepine, phenytoin) at baseline.
  • Participated in an interventional clinical trial within the last 3 months prior the baseline visit.
  • Unable or unwilling to comply with the contraceptive requirements of the protocol.
  • Female participants who are pregnant and/or breastfeeding.
  • Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, psychiatric, or allergic disease.

Trial locations and contacts

18 sites in 8 countries.

18 sites in 8 countries

United States8 sites

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30324

  • Hugo W Moser Research Institute at Kennedy Krieger Institute

    Baltimore, Maryland, 21205

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242

  • The University of Kansas Medical Center

    Kansas City, Kansas, 66160

  • University of Minnesota

    Minneapolis, Minnesota, 55455

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104

  • University of Washington

    Seattle, Washington, 98195

  • Washington University School of Medicine

    St Louis, Missouri, 63110

Other trials in United States

Canada2 sites

  • University of Alberta

    Edmonton, Alberta, T6G 2G3

  • Ottawa Hospital

    Ottawa, K1Y 4E9

Other trials in Canada

France2 sites

  • CHRU de NANCY Service de Neurologie

    France, 54035

  • University Hospital La Timone

    Marseille, 13385

Other trials in France

Russia2 sites

  • Pirogov Russian National Research Medical University

    Moscow, 125412

  • Saint-Petersburg State Pediatric Medical University

    Saint Petersburg, 194100

Other trials in Russia

Denmark1 site

  • Rigshospitalet, University of Copenhagen

    Copenhagen, 2200

Other trials in Denmark

Germany1 site

  • Ludwig-Maximilians University Munich, Friedrich-Baur-Institute

    Munich, 80801

Other trials in Germany

Norway1 site

  • Oslo University Hospital

    Oslo, 0424

Other trials in Norway

Sweden1 site

  • Sahlgrenska University Hospital

    Gothenburg, 41345

Other trials in Sweden

About this study

Official title: A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. Most participants enrolled will have a screening visit and 3 additional visits (after 1, 13, and 26 weeks of treatment).

Being tested
Deflazacort
Medical subject headings
Muscular Dystrophies, Limb-Girdle

What participants receive

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

  • DeflazacortDrug

    Also called Emflaza®

    Deflazacort tablet will be administered as per the dose and schedule specified in the arm.

Study groups in NCT03783923
Study groupRoleWhat they receive
DeflazacortExperimentalParticipants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally. The dose could be reduced in case of tolerability issues. Any participant assigned to placebo prior to the Version 4.0 amendment (prior to or after 01 February 2020) will have the option to be consented under Version 4.0 and will be switched to deflazacort for 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (prior to 01 February 2020) will have the option to re-consent under Protocol Version 4.0 and continue for an additional 26 weeks treatment. Any participant assigned to deflazacort prior to the Version 4.0 amendment (after 01 February 2020) will have the option to re-consent under Protocol Version 4.0 at their Week 13 Visit and continue treatment until Week 26. Any new participant enrolled until 31 May 2020 will receive deflazacort for 26 weeks.
Receives: Drug: Deflazacort

What is being measured

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

  1. Change From Baseline in Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

Secondary outcomes (14)

  1. Change From Baseline in Forced Vital Capacity (FVC) After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  2. Change From Baseline in 2-Minute Walk Test After 26 Weeks of Treatment of Deflazacort

    Measured: Baseline, Week 26

  3. Change From Baseline in Time to up and go After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  4. Change From Baseline in Time to Descend 4 Stairs After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  5. Change From Baseline in Time to Run/Walk 10 Meters After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  6. Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  7. Change From Baseline in Hand-Held Myometry After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  8. Change From Baseline in Global T2 Relaxation Time of Selected Upper and Lower Limb Muscles After 26 Weeks of Treatment With Deflazacort

    Measured: Baseline, Week 26

  9. Number of Participants With Adverse Events (AEs)

    An AE was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. Serious adverse event (SAE) was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs included both SAEs and non-serious AEs. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'. AEs were summarized separately for Stage 1 and for the overall ataluren experience.

    Measured: Baseline up to Week 52

  10. Area Under the Concentration Curve From Time Zero to t (AUC0-t) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort

    Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

  11. Area Under the Concentration Curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort

    Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

  12. Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort

    Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

  13. Time to Reach Cmax (Tmax) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort

    Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

  14. Half-Life (t1/2) of 21-desacetyl Deflazacort and 6β-hydroxy-21-desacetyl Deflazacort

    Measured: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13

Timeline

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

  1. First posted21 Dec 2018
  2. Study start31 Oct 2019
  3. Primary completion1 Jan 2021
  4. Study completion1 Jan 2021

Why it stopped: The study was terminated early due to low enrollment and missing efficacy assessment data due to missed visits related to COVID-19.

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
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03783923
Sponsor's protocol ID
PTCEMF-GD-004
Status last verified
May 2022
First submitted
19 Dec 2018
Last updated
27 Jun 2022
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Deflazacort in people with Limb-Girdle Muscular Dystrophy, run by PTC Therapeutics at 18 sites in 8 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 4 inclusion and 12 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 Jun 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 18 sites in United States, Canada, France, Russia, Denmark and 3 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. 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 27 Jun 2022). 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 Time to Climb 4 Stairs After 26 Weeks of Treatment With Deflazacort (time frame: Baseline, Week 26). 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 NCT03783923.

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 Jun 2022.

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