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

NCT02251600 | Completed Phase 1

• 1 other identifier: MP-104-CL-005
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 4

Brief Summary

Study to characterize the single-state and steady-state dosing of oral deflazacort in pediatric and adolescents subjects.

Conditions

Duchenne Muscular Dystrophy

Keywords

Pediatric; Adolescent; Deflazacort

Outcome Measures

Primary Outcomes (2)

• The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite

  The area under the plasma concentration time curve, from time 0 to the last measurable concentration non-zero, for single-state pharmacokinetics on Day 1 of deflazacort and 21-desacetyl-DFZ, the active metabolite

  Day 1, Day 8

• The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active metabolite

  The area under the plasma concentration versus time curve over the final dosing interval for steady state pharmacokinetics on Day 8 of deflazacort and 21-desacetyl-DFZ, the active

  Day 8

Secondary Outcomes (1)

• Number of participants with adverse events as a measure of safety and tolerability

  Day 1-8

Study Arms (1)

Deflazacort

EXPERIMENTAL

This is an open label and single period study , dosed with 0.9mg/kg Deflazacort.

Drug: Deflazacort

Interventions

Deflazacort DRUG

Oral deflazacort administered once daily at 0.9 mg/kg for eight days

Also known as: DFZ

Deflazacort

Eligibility Criteria

• Age: 4 Years - 16 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• In the opinion of the investigator, the subject is capable of understanding and complying with protocol requirements.
• The subject or, when applicable, the subject's legally acceptable representative signs and dates a written, informed consent form and any required privacy authorization prior to the initiation of any study procedures.
• If above the age of 7, the subject signs and dates a written, informed assent form (IAF) and any required privacy authorization prior to the initiation of any study procedures.
• The subject must have confirmed diagnosis of Duchenne Muscular Dystrophy defined as muscle biopsy and dystrophin analyses consistent with DMD or DNA mutation and analysis by PCR or Southern blot techniques to detect gene deletions as well as:
• onset of weakness before 5 years of age;
• proximal muscle weakness;
• increased serum creatine kinase more than 10 times the upper limit of normal (ULN);
• The subject is male and aged 4 to 16 years, inclusive.
• The subject weighs at least 13 kg and has a Body Mass Index (BMI) of ≤ 40.
• Willingness and ability to comply with scheduled visits, oral drug administration, and study procedures including blood sample draws (total blood volume collected not to exceed 50 mL for the study duration or 25 mL on any single study day \[≤ 3 mL/kg\])
• The subject has a life expectancy of \>1 year.
• Up to date on all childhood vaccinations, specifically varicella vaccine (chicken pox).
• Baseline health is judged to be stable based on medical history, physical examination, laboratory profiles, vital signs, or ECGs at screening, as deemed by the Investigator.
• Continuous non smoker who has not used nicotine containing products for at least 3 months prior to the first dose.
• The subject is able to take tablets.

You may not qualify if:

• The subject has received any investigational compound and/or has participated in another clinical study within 90 days prior to study treatment with the exception of observational cohort studies or non-interventional studies.
• The subject has received deflazacort within 30 days or previous discontinued deflazacort due to an intolerable reaction.
• The subject is an immediate family member, study site employee, or is in a dependent relationship with a study site employee who is involved in the conduct of this study (e.g. spouse, parent, child, sibling) or may consent under duress.
• The subject has, in the judgment of the investigator, clinically significant abnormal clinical chemistry laboratory parameters that may affect safety at Screening.
• The subject has, in the judgment of the investigator, a history or current medical condition that could affect safety including, but not limited to:
• Major renal or hepatic impairment
• Immunosuppression or other contraindications for corticosteroid treatment
• History of chronic systemic fungal or viral infections
• Diabetes mellitus
• Idiopathic hypocalcuria
• Symptomatic cardiomyopathy at screening
• The subject has a history of hypersensitivity or allergic reaction to steroids or their formulations including, but not limited to lactose, sucrose, etc.
• Inability to take tablets as assessed by site investigator.
• Unable to refrain from or anticipates the use of:
• Any medications at least 4 hours before and after dosing on PK Days 1 and 8.

Sponsors & Collaborators

• PTC Therapeutics: lead

Study Sites (4)

UCLA

Los Angeles, California, 90095, United States

Location

Lurie Children's Hospital

Chicago, Illinois, 60611, United States

Location

University of Rochester Medical Center

Rochester, New York, 14642, United States

Location

University of Utah

Salt Lake City, Utah, 84112, United States

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

deflazacort

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Katherine Smith, MD

  Drug Safety Solutions/Celerion

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2014
• First Posted: September 29, 2014
• Study Start: December 1, 2014
• Primary Completion: October 1, 2015
• Study Completion: October 1, 2015
• Last Updated: August 18, 2017

Record last verified: 2017-08

Locations

US (4)