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

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ClinicalTrials.gov Identifier: NCT03783923
Recruitment Status : Recruiting
First Posted : December 21, 2018
Last Update Posted : September 13, 2019
Sponsor:
Information provided by (Responsible Party):
PTC Therapeutics

Brief Summary:
This study is designed to evaluate the safety and efficacy of deflazacort in participants with LGMD2I. The study will include a 26-week double-blind, randomized, placebo-controlled period followed by a 26-week open-label extension period during which all participants will receive deflazacort.

Condition or disease Intervention/treatment Phase
Limb-Girdle Muscular Dystrophy Drug: Deflazacort Drug: Placebo Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 100 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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)
Actual Study Start Date : May 15, 2019
Estimated Primary Completion Date : August 31, 2020
Estimated Study Completion Date : August 31, 2020


Arm Intervention/treatment
Experimental: Deflazacort
Participants will receive deflazacort 0.6 milligrams per kilograms per day (mg/kg/day) orally for 26 weeks in the double-blind period, and for an additional 26 weeks in the open-label extension period. The initial dose of study drug in double-blind period can be reduced once after Week 13 to the next lower weight-band due to safety or tolerability considerations, regardless of any changes in weight. In the open-label extension period, dose adjustments are permitted at the discretion of the investigator.
Drug: Deflazacort
Deflazacort tablet will be administered as per the dose and schedule specified in the respective arms.
Other Name: Emflaza®

Placebo Comparator: Placebo
Participants will receive placebo matching deflazacort orally for 26 weeks in the double-blind period, and deflazacort 0.6 mg/kg/day for an additional 26 weeks in the open-label extension period. In the open-label extension period, dose adjustments are permitted at the discretion of the investigator.
Drug: Deflazacort
Deflazacort tablet will be administered as per the dose and schedule specified in the respective arms.
Other Name: Emflaza®

Drug: Placebo
Placebo matching deflazacort will be administered as per schedule specified in the respective arm.




Primary Outcome Measures :
  1. Change From Baseline in Time to Climb 4 Stairs at Week 26 [ Time Frame: Baseline, Week 26 ]

Secondary Outcome Measures :
  1. Change From Baseline in Forced Vital Capacity (FVC) at Week 26 [ Time Frame: Baseline, Week 26 ]
  2. Change From Baseline in 2-Minute Walk Test at Week 26 [ Time Frame: Baseline, Week 26 ]
  3. Change From Baseline in Time to up and go at Week 26 [ Time Frame: Baseline, Week 26 ]
  4. Change From Baseline in Time to Descent 4 Stairs at Week 26 [ Time Frame: Baseline, Week 26 ]
  5. Change From Baseline in Time to Run/Walk 10 Meters at Week 26 [ Time Frame: Baseline, Week 26 ]
  6. Change From Baseline in Maximal Inspiratory Pressure (MIP) and Maximal Expiratory Pressure (MEP) at Week 26 [ Time Frame: Baseline, Week 26 ]
  7. Change From Baseline in Hand-Held Myometry at Week 26 [ Time Frame: Baseline, Week 26 ]
  8. Number of Participants With Adverse Events [ Time Frame: Baseline to Week 52 ]
  9. Number of Participants With Laboratory Abnormalities [ Time Frame: Baseline to Week 52 ]
  10. Number of Participants With Electrocardiogram (ECG) Abnormalities [ Time Frame: Baseline to Week 52 ]
  11. Number of Participants With Ophthalmologic Abnormalities [ Time Frame: Baseline to Week 52 ]
  12. Area Under the Concentration curve From Time Zero to t (AUC0-t) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  13. Area Under the Concentration curve From Time Zero to Infinity (AUC0-inf) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  14. Maximum Observed Plasma Concentration (Cmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  15. Time to Reach Cmax (Tmax) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  16. Clearance (CL/F) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  17. Volume of Distribution (Vz/F) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  18. Terminal Elimination Rate Constant (Lambda z [λz]) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]
  19. Half-Life (t1/2) of 21-desacetyl deflazacort and 6β-hydroxy-21-desacetyl deflazacort [ Time Frame: Pre-dose, 0.5, 1, 2, 4, and 6 hours post-dose at Baseline and Week 13 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Genetic diagnosis of LGMD2I (confirmed mutation in the fukutin-related protein [FKRP] gene).
  • Ability to ascend 4 stairs greater than (>) 2.5 and less than (<) 8 seconds 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).

Exclusion Criteria:

  • Received greater than or equal to (>=) 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 <30 percent [%]) at screening.
  • Requires fulltime ventilator support.
  • History of chronic systemic fungal or viral infections.
  • History of acute bacterial infection (including tuberculosis) per discretion of the Investigator.
  • Diagnosis of diabetes mellitus (controlled and/or uncontrolled) defined as glycated hemoglobin (HbA1c) >=6.5%.
  • History of immunosuppression or other contraindications to glucocorticosteroid therapy.
  • Requires concomitant use or >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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03783923


Contacts
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Contact: Senior VP Corporate Relations 1-866-562-4620 medinfo@ptcbio.com
Contact: Medical Information 1-866-562-4620 medinfo@ptcbio.com

Locations
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United States, Georgia
Rare Disease Research, LLC Recruiting
Atlanta, Georgia, United States, 30324
Contact: Tu Tran    678-883-6897    tu.tran@rarediseaseresearch.com   
Principal Investigator: Han Phan, Dr.         
United States, Iowa
University of Iowa Hospitals and Clinics Not yet recruiting
Iowa City, Iowa, United States, 52242
Contact: Carrie Stephan    319-356-2673    carrie-stephan@uiowa.edu   
Principal Investigator: Katherine Mathews         
United States, Kansas
The University of Kansas Medical Center Not yet recruiting
Kansas City, Kansas, United States, 66160
Contact: Michaela Walker    913-945-9920    mwalker20@kumc.edu   
Principal Investigator: Jeffrey Statland         
United States, Maryland
Hugo W Moser Research Institute at Kennedy Krieger Institute Not yet recruiting
Baltimore, Maryland, United States, 21205
Contact: Genila Bibat    443-629-5371    bibat@kennedykrieger.org   
Principal Investigator: Kathryn Wagner         
United States, Minnesota
University of Minnesota Not yet recruiting
Minneapolis, Minnesota, United States, 55455
Contact: Peter Karachunski, Dr.    612-625-5000    karac001@umn.edu   
Principal Investigator: Peter Karachunski, Dr.         
United States, Missouri
Washington University School of Medicine Not yet recruiting
Saint Louis, Missouri, United States, 63110
Contact: Conrad Weihl, Dr.    314-362-5677    weihlc@wustl.edu   
Principal Investigator: Conrad Weihl, Dr.         
United States, Pennsylvania
University of Pennsylvania Not yet recruiting
Philadelphia, Pennsylvania, United States, 19104
Contact: Sara-Claude Michon    215-595-7468    claude.michon@pennmedicine.upenn.edu   
Contact: Kelsey Moulton    1-215-595-7468    kelsey.moulton@uphs.upenn.edu   
Principal Investigator: Lauren Elman, Dr.         
Canada, Alberta
University of Alberta Not yet recruiting
Edmonton, Alberta, Canada, T6G 2G3
Contact: Dorothy Miko    1-780-248-1798    dorothy.miko@ualberta.ca   
Principal Investigator: Cecile Phan, Dr.         
Canada
Ottawa Hospital Not yet recruiting
Ottawa, Canada, K1Y 4E9
Contact: Sergio Guber    16137985555    sguber@ohri.ca   
Principal Investigator: Hanns Lochmuller, Dr.         
Denmark
Rigshospitalet, University of Copenhagen Not yet recruiting
Copenhagen, Denmark, 2200
Contact: John Vissing    453-545-2562    john.vissing@regionh.dk   
Principal Investigator: John Vissing         
France
CHRU de NANCY Service de Neurologie Not yet recruiting
France, France, 54035
Contact: Chatelain Anne    +33(0) 3 83 85 16 88      
Principal Investigator: Maud Michaud, Dr.         
University Hospital La Timone Not yet recruiting
Marseille, France, 13385
Contact: Emmanuelle Salort-Campana    3349138433    emmanuelle.salort-campana@ap-hm.fr   
Principal Investigator: Shahram Attarian, Dr.         
Germany
Ludwig-Maximilians University Munich, Friedrich-Baur-Institute Not yet recruiting
Munich, Germany, 80801
Contact: Sabine Krause, Dr.    (089) 4400-57400    sabine.krause@med.uni-muenchen.de   
Principal Investigator: Maggie Walter, Prof.         
Norway
Oslo University Hospital Not yet recruiting
Oslo, Norway, 0424
Contact: Trine Popperud    4-72-307-0000    tripop@ous-hr.no   
Principal Investigator: Trine Popperud         
Russian Federation
Pirogov Russian National Research Medical University Not yet recruiting
Moscow, Russian Federation, 125412
Contact: Svetlana Artemyeva    79162078153    artemievasb@gmail.com   
Principal Investigator: Dmitry Vlodavets, Dr.         
Saint-Petersburg State Pediatric Medical University Not yet recruiting
Saint Petersburg, Russian Federation, 194100
Contact: Vasiliy Suslov    7-911-212-26-12    vms.92@mail.ru   
Principal Investigator: Dmitry Rudenko, Dr.         
Sweden
Sahlgrenska University Hospital Not yet recruiting
Gothenburg, Sweden, 41345
Contact: Christopher Lindberg, Dr.    46313421000    christopher.lindberg@vgregion.se   
Principal Investigator: Christopher Lindberg, Dr.         
Sponsors and Collaborators
PTC Therapeutics
Investigators
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Study Director: Cristobal Passalacqua, MD PTC Therapeutics

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Responsible Party: PTC Therapeutics
ClinicalTrials.gov Identifier: NCT03783923     History of Changes
Other Study ID Numbers: PTCEMF-GD-004-LGMD
First Posted: December 21, 2018    Key Record Dates
Last Update Posted: September 13, 2019
Last Verified: September 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Muscular Dystrophies, Limb-Girdle
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases
Genetic Diseases, Inborn
Deflazacort
Anti-Inflammatory Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs