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Corticosteroid

Deflazacort for Limb-Girdle Muscular Dystrophy

Phase 3
Waitlist Available
Research Sponsored by PTC Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline up to week 52
Awards & highlights
No Placebo-Only Group
Pivotal 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).

Eligible Conditions
  • Limb-Girdle Muscular Dystrophy

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline up to week 52
This trial's timeline: 3 weeks for screening, Varies for treatment, and baseline up to week 52 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Secondary study objectives
Number of Participants With Adverse Events (AEs)

Side effects data

From 2019 Phase 3 trial • 196 Patients • NCT01603407
24%
Abnormal behavior
23%
Vomiting
18%
Fall
17%
Cough
17%
Naspharyngitis
15%
Pyrexia
15%
Diarrhea
15%
Abdominal pain upper
12%
Back pain
12%
Abdominal pain
12%
Ear infection
11%
Constipation
11%
Upper respiratory tract infection
9%
Headache
9%
Rhinitis
8%
Pain in extremity
8%
Abnormal weight gain
8%
Vitamin D deficiency
8%
Pollakiuria
6%
Psychomotor hyperactivity
5%
Muscle spasms
5%
Cushingoid
3%
Abdominal discomfort
3%
Arthralgia
3%
Rhinorrhoea
3%
Rash
3%
Malaise
3%
Influenza
3%
Nasopharyngitis
3%
Mood altered
3%
Aggression
3%
Insomnia
2%
Ligament sprain
2%
Head injury
2%
Epistaxis
2%
lower respiratory tract infection
2%
Rhabdomyolysis
2%
Gastrointestinal infection
2%
Circumsion
2%
Orchidopexy
2%
Tentomy
2%
Umbilical hernia repair
2%
Nausea
2%
Gastroenteritis
100%
80%
60%
40%
20%
0%
Study treatment Arm
Intermittent Prednisone
Daily Prednisone
Daily Deflazacort

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups
Experimental Treatment
Group I: DeflazacortExperimental Treatment1 Intervention
Participants 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.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Deflazacort
2014
Completed Phase 3
~330

Find a Location

Who is running the clinical trial?

PTC TherapeuticsLead Sponsor
74 Previous Clinical Trials
6,276 Total Patients Enrolled
Cristobal Passalacqua, MDStudy DirectorPTC Therapeutics
~2 spots leftby Dec 2025
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