Deflazacort

Generic Name: Deflazacort

Brand Names: Emflaza

Drug Type: Small Molecule

Chemical Formula: C_₂₅H_₃₁NO_₆

CAS Number: 14484-47-0

Unique Ingredient Identifier: KR5YZ6AE4B

Background: Deflazacort, also known as Emflaza, is a corticosteroid prodrug used as an agent to manage Duchenne Muscular Dystrophy (DMD). It is marketed by Marathon Pharmaceuticals and was approved in February 2017 by the FDA.
...

Indication: ⑴用于原发性肾上腺皮质功能减退、风湿病、胶原性疾病、皮肤病、变态反应性疾病、眼科疾病、暴发性和播散性肺结核、造血系统疾患、溃疡性结肠炎、特发性肾病综合征等。

⑵用于治疗杜氏肌营养不良（DMD）。

Associated Conditions: Duchenne Muscular Dystrophy (DMD)

Associated Therapies: -

Clinical Trials

Study of the Excretion of Orally Administered Corticosteroids for the Improval of the Detection of Said Substances in Anti-doping Controls

Phase 1

Conditions: Healthy Volunteers

Interventions: Drug: Methylprednisone
Drug: Deflazacort
Drug: Dexamethasone

First Posted Date: 2021-03-10

Last Posted Date: 2021-04-01

Lead Sponsor: Parc de Salut Mar

Target Recruit Count: 50

Registration Number: NCT04791345

Locations: 🇪🇸
IMIM (Hospital del Mar Medical Research Institute), Barcelona, Spain

Deflazacort vs. Prednisolone in Acute-stage ABPA

Phase 2

Active, not recruiting

Conditions: Allergic Bronchopulmonary Aspergillosis

Interventions: Drug: Deflazacort
Drug: Prednisolone

First Posted Date: 2020-01-13

Last Posted Date: 2023-11-27

Lead Sponsor: Post Graduate Institute of Medical Education and Research, Chandigarh

Target Recruit Count: 150

Registration Number: NCT04227483

Locations: 🇮🇳
Chest Clinic, Dept. of Pulmonary Medicine, Chandigarh, India

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

Phase 3

Terminated

Conditions: Limb-Girdle Muscular Dystrophy

Interventions: Drug: Deflazacort

First Posted Date: 2018-12-21

Last Posted Date: 2022-06-27

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 11

Registration Number: NCT03783923

Locations: 🇺🇸
Washington University School of Medicine, Saint Louis, Missouri, United States
🇨🇦
University of Alberta, Edmonton, Alberta, Canada
🇩🇰
Rigshospitalet, University of Copenhagen, Copenhagen, Denmark

and more 15 locations

A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

Phase 3

Withdrawn

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Deflazacort

First Posted Date: 2018-08-22

Last Posted Date: 2019-06-21

Lead Sponsor: PTC Therapeutics

Registration Number: NCT03642145

Deflazacort Expanded Access Program for Children, Adolescents and Adults With Duchenne Muscular Dystrophy

Conditions: Duchenne Muscular Dystrophy

First Posted Date: 2015-10-30

Last Posted Date: 2018-02-28

Lead Sponsor: PTC Therapeutics

Registration Number: NCT02592941

Locations: 🇺🇸
UCLA Health, Los Angeles, California, United States
🇺🇸
Children's Healthcare of Atlanta- Scottish Rite, Atlanta, Georgia, United States
🇺🇸
Children's Medical Center, Dallas, Texas, United States

and more 39 locations

Food Effect and Bioavailability of Deflazacort Formulations in Healthy Volunteers

Phase 1

Completed

Conditions: Healthy Volunteers

Interventions: Drug: Deflazacort

First Posted Date: 2015-06-30

Last Posted Date: 2017-08-18

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 45

Registration Number: NCT02485431

Locations: 🇺🇸
ICON Early Phase Services, San Antonio, Texas, United States

An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerability Deflazacort

Phase 1

Completed

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Deflazacort

First Posted Date: 2014-11-20

Last Posted Date: 2017-12-08

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 24

Registration Number: NCT02295748

Locations: 🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸
Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸
University of Rochester, Rochester, New York, United States

and more 1 locations

A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Deflazacort Pharmacokinetics

Phase 1

Completed

Conditions: Hepatic Impairment

Interventions: Drug: Deflazacort

First Posted Date: 2014-11-10

Last Posted Date: 2017-08-18

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 16

Registration Number: NCT02286609

Locations: 🇺🇸
University of Miami Division of Clinical Pharmacology, Miami, Florida, United States
🇺🇸
Orlando Clinical Research Center, Orlando, Florida, United States

A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Pharmacokinetics

Phase 1

Completed

Conditions: Renal Impairment

Interventions: Drug: Deflazacort

First Posted Date: 2014-11-10

Last Posted Date: 2017-08-18

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 16

Registration Number: NCT02286622

Locations: 🇺🇸
University of Miami Division of Clinical Pharmacology, Miami, Florida, United States
🇺🇸
Orlando Clinical Research Center, Orlando, Florida, United States

A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects With Duchenne Muscular Dystrophy

Phase 1

Completed

Conditions: Duchenne Muscular Dystrophy

Interventions: Drug: Deflazacort

First Posted Date: 2014-09-29

Last Posted Date: 2017-08-18

Lead Sponsor: PTC Therapeutics

Target Recruit Count: 24

Registration Number: NCT02251600

Locations: 🇺🇸
Lurie Children's Hospital, Chicago, Illinois, United States
🇺🇸
UCLA, Los Angeles, California, United States
🇺🇸
University of Utah, Salt Lake City, Utah, United States

and more 1 locations

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