Pentoxifylline in Duchenne Muscular Dystrophy

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT00102453
• Date of registration: 29/01/2005
• Prospective Registration: No
• Primary sponsor: Cooperative International Neuromuscular Research Group
• Public title: Pentoxifylline in Duchenne Muscular Dystrophy
• Scientific title: An Open-Label Pilot Study of Pentoxifylline in Steroid-naive Duchenne Muscular Dystrophy
• Date of first enrolment: March 2002
• Target sample size: 17
• Recruitment status: Completed
• URL: http://clinicaltrials.gov/show/NCT00102453
• Study type: Interventional
• Study design: Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Diana Escolar, MD
• Affiliation: Children's Research Institute

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Male

2. Age 4 to 7 years

3. Ambulant independently. Subjects may use a wheelchair occasionally, but only for long
distances

4. Diagnosis of DMD confirmed by at least one of the following:

- Dystrophin immunofluorescence and/or immunoblot showing complete dystrophin
deficiency, and clinical picture consistent with typical DMD OR

- Gene deletion test positive (missing one or more exons) in the central rod
domain (exons 25-60) of dystrophin, where reading frame can be predicted as
'out-of-frame',

- and clinical picture consistent with typical DMD.

- Complete dystrophin gene sequencing showing an alteration (point mutation,
duplication, or other mutation resulting in a stop codon mutation) that can be
definitely associated with DMD, with a typical clinical picture of DMD.

5. Positive family history of DMD confirmed by one of the criteria listed above in a
sibling or maternal uncle, and clinical picture typical of DMD.

6. Glucocorticosteroid - naïve (i.e. has not been treated with prednisone or Deflazacort
within 1 year before onset of the study)

7. Has not participated in other therapeutic research protocol within the last 6 months.

8. Evidence of muscle weakness by MRC score or clinical functional evaluation

9. Ability to provide reproducible repeat QMT bicep score of either the right or left
arm within 15% of first assessment score.

Exclusion Criteria:

1. Symptomatic DMD carrier

2. Use of any medication, nutritional supplement or herb for treatment of DMD within the
last 3 months.

3. Symptomatic cardiomyopathy or ventricular arrhythmias

4. History of significant concomitant illness, impairment of blood clotting ability (as
evidenced by increased PT/PTT or bleeding time over the upper limit of normal (ULN)),
recent cerebral or retinal hemorrhage, bleeding diathesis, gastric ulcer, hypotension
or significant impairment of renal or hepatic function (defined as serum creatinine
and GGT respectively, greater than 1.5 times normal upper limit for age and gender).



Age minimum: 4 Years
Age maximum: 7 Years
Gender: Male

Health Condition(s) or Problem(s) studied

Muscular Dystrophy, Duchenne

Intervention(s)

Drug: Pentoxifylline

Primary Outcome(s)

QMT measurements [Time Frame: Each study visit]

Secondary Outcome(s)

Change in manual muscle test (MMT) at 12 months [Time Frame: Each study visit]

Secondary ID(s)

CNMC0302

Source(s) of Monetary Support

Please refer to primary and secondary sponsors