Marathon 022OLE

Principal Investigator: Russell Butterfield
Keywords: DUCHENNE MUSCULAR DYSTROPHY , INVESTIGATIONAL DRUG STUDY , MUSCULAR DYSTROPHY , DUCHENNE , DMD , DEFLAZACORT , GLUCOCORTICOSTEROID Department: Pediatric Administration
IRB Number: 00080246 Co Investigator: Nicholas Johnson
Specialty: Neurology
Sub Specialties: Muscular Dystrophy
Recruitment Status: Completed

Contact Information

Brianna Jensen
briaj@genetics.utah.edu
801-585-9399

Brief Summary

  The objective of the study is to assess the safety and tolerability of long-term use of deflazacort in DMD subjects that were previously enrolled in the MP-104-CL-005 Pharmacokinetic (PK) study.

Inclusion Criteria

1. In the opinion of the Investigator, the subject is capable of understanding and complying

with protocol requirements.

2. 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.

3. 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.

Subjects under age 7 at the time of study entry who turn age 7 will sign and date a written

informed assent form (IAF) at the visit following their 7th birthday, if required by the site’s

IRB.

4. Subject participated in and received at least one dose of study medication in the MP-104-

CL-005 protocol.

5. 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 all of the

following clinical signs of DMD:

a. onset of weakness before 5 years of age;

b. proximal muscle weakness;

c. increased serum creatine kinase more than 10 times the upper limit of normal

(ULN) at some stage in their course of disease.

6. The subject weighs at least 13 kg and has a body mass index (BMI) of ≤ 40 kg/m2.

7. Willingness and ability to comply with scheduled visits, oral drug administration, and study

procedures including blood sample draws for safety labs.

8. Up to date on all childhood vaccinations, including varicella vaccine (chicken pox).

9. 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.

10. Continuous non-smoker who has not used nicotine-containing products for at least

3 months prior to the first dose.

11. The subject is able to take tablets.

Exclusion Criteria

Subjects must not be enrolled in the study if they meet any of the following criteria:

1. 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 MP-104-CL-005,

observational cohort studies or non-interventional studies.

2. The subject discontinued previous deflazacort therapy, at any time, due to a severe or

intolerable adverse reaction.

3. 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.

4. Any significant finding on the Columbia-suicide severity rating scale (C-SSRS) for subjects

(ages 12-16, inclusive), in the opinion of the PI, warrants exclusion from this study.

5. The subject has, in the judgment of the, clinically significant abnormal clinical chemistry

laboratory parameters that may affect safety at Day 0.

6. The subject has, in the judgment of the Investigator, a history or current medical condition

that could affect safety including, but not limited to:

a. Major renal or hepatic impairment

b. Immunosuppression or other contraindications for corticosteroid treatment

c. History of chronic systemic fungal or viral infections

d. Diabetes mellitus

e. Idiopathic hypocalcuria

f. Symptomatic cardiomyopathy at Day 0

7. The subject has a history of hypersensitivity or allergic reaction to steroids or their

formulations including, but not limited to lactose, sucrose, etc.

8. Inability to take tablets as assessed by site investigator.

9. Subject is mentally or legally incapacitated or has significant emotional problems at the

time of screening visit or expected during the conduct of the study.

10. History of any illness that, in the opinion of the PI, might confound the results of the study

or poses an additional risk to the subject by their participation in the study.

11. Positive urine drug or alcohol results at Day 0.

12. Clinically significant hemoglobin level below the lower limit of normal at Day 0 that may

affect the safety of the patient. Sites may obtain hematology results locally at Day 0 in

lieu of waiting for return of central laboratory results if deemed appropriate or if low

hemoglobin is of particular concern for a patient; however review of Day 0 laboratory

results prior to study enrollment is not required.