A clinical study to evaluate how safe and effective the drug Deflazacort (EMFLAZA®) compared to placebo is in subjects with Limb-Girdle Muscular Dystrophy Type 2I

A MULTICENTER RANDOMIZED PLACEBO-CONTROLLED PHASE 3 STUDY ON THE SAFETY AND EFFICACY OF DEFLAZACORT (EMFLAZA®) IN SUBJECTS WITH LIMB-GIRDLE MUSCULAR DYSTROPHY 2I (LGMD2I

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-004740-36-FR

Enrollment

100

Registered

2019-06-04

Start date

2019-08-19

Completion date

Unknown

Last updated

2021-04-05

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Limb-Girdle Muscular Dystrophy Type 2I (LGMD2I) MedDRA version: 20.0 Level: PT Classification code 10028356 Term: Muscular dystrophy System Organ Class: 10010331 - Congenital, familial and genetic disorders

Interventions

Trade Name: EMFLAZA® (DEFLAZACORT) Product Name: EMFLAZA® (deflazacort) Product Code: EMFLAZA® (deflazacort) Pharmaceutical Form: Tablet INN or Proposed INN: Deflazacort CAS Number: 14484-47-0 Current

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Genetic diagnosis of LGMD2I (confirmed mutation in the FKRP gene) 2. Male or female subjects aged =18 years 3. Ability to ascend 4 stairs >2.5 and =65 years) yes F.1.3.1 Number of subjects for this age range 10

Exclusion criteria

Exclusion criteria: 1. Received =4 weeks of continuous, systemic corticosteroid therapy within 3 months of study Screening Visit. 2. Presence of significant cardiomyopathy as defined by echocardiogram (left ventricular ejection fraction 1 week of drugs or substances that are moderate to strong CYP3A4 inhibitors (ie, clarithromycin, fluconazole, diltiazem, verapamil, grapefruit juice) or moderate or strong CYP3A4 inducers (ie, rifampin, efavirenz, carbamazepine, phenytoin) at baseline. 9. Participated in an interventional clinical trial within the last months prior the baseline Visit 10. Unable or unwilling to comply with the contraceptive requirements of the protocol 11. Female subjects who are pregnant and/or breastfeeding 12. Evidence or history of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, neurologic, psychiatric, or allergic disease

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate the efficacy of oral deflazacort as measured by muscle function in subjects with LGMD2I;Secondary Objective: • To evaluate the effects of oral deflazacort on pulmonary function in subjects with LGMD2I • To evaluate the pharmacokinetic (PK) profile of oral deflazacort in subjects with LGMD2I • To evaluate safety of deflazacort in subjects with LGMD2I;Primary end point(s): Change from baseline in time to climb 4 stairs at Week 26;Timepoint(s) of evaluation of this end point: Baseline, Week 26	—

Measure

Time frame

Main Objective: To evaluate the efficacy of oral deflazacort as measured by muscle function in subjects with LGMD2I;Secondary Objective: • To evaluate the effects of oral deflazacort on pulmonary function in subjects with LGMD2I • To evaluate the pharmacokinetic (PK) profile of oral deflazacort in subjects with LGMD2I • To evaluate safety of deflazacort in subjects with LGMD2I;Primary end point(s): Change from baseline in time to climb 4 stairs at Week 26;Timepoint(s) of evaluation of this end point: Baseline, Week 26

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Secondary

Measure	Time frame
Secondary end point(s): • Key secondary endpoints o Change from baseline in forced vital capacity (FVC) at Week 26 o Change from baseline in 2-minute walk test at Week 26 • Secondary endpoints o Change from baseline in time to up and go at Week 26 o Change from baseline to time to descent 4 stairs at Week 26 o Change from baseline in time to run/walk 10 meters at Week 26 o Change from baseline in maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) at Week 26 o Change from baseline in hand-held myometry at Week 26 • • Safety profile characterized by type frequency, severity, timing, and relationship to study drug of any adverse events, laboratory abnormalities, electrocardiogram (ECG) abnormalities, ophthalmologic abnormalities, dual-energy X-ray absorptiometry (DEXA) to evaluate bone density and/or X-ray to assess spine fracture • Pharmacokinetic assessments at baseline and Week 13;Timepoint(s) of evaluation of this end point: Baseline, Week 26 PK: Baseline, Week 13	—

Measure

Time frame

Secondary end point(s): • Key secondary endpoints o Change from baseline in forced vital capacity (FVC) at Week 26 o Change from baseline in 2-minute walk test at Week 26 • Secondary endpoints o Change from baseline in time to up and go at Week 26 o Change from baseline to time to descent 4 stairs at Week 26 o Change from baseline in time to run/walk 10 meters at Week 26 o Change from baseline in maximal inspiratory pressure (MIP) and maximal expiratory pressure (MEP) at Week 26 o Change from baseline in hand-held myometry at Week 26 • • Safety profile characterized by type frequency, severity, timing, and relationship to study drug of any adverse events, laboratory abnormalities, electrocardiogram (ECG) abnormalities, ophthalmologic abnormalities, dual-energy X-ray absorptiometry (DEXA) to evaluate bone density and/or X-ray to assess spine fracture • Pharmacokinetic assessments at baseline and Week 13;Timepoint(s) of evaluation of this end point: Baseline, Week 26 PK: Baseline, Week 13

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Countries

Canada, Denmark, France, Germany, Norway, Sweden, United States

Contacts

Public ContactRochelle Greenbaum

PTC Therapeutics, Inc.

rgreenbaum@ptcbio.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026