Spinal Muscular Atrophy Clinical Trial
— STOPSMAOfficial title:
Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
Verified date | June 2015 |
Source | University of Utah |
Contact | n/a |
Is FDA regulated | No |
Health authority | United States: Food and Drug Administration |
Study type | Interventional |
In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants. A variety of outcome measures will be performed at each study visit to follow the course of the disease. Total duration of the study for type I infants will be 18 months, for type II infants, 24 months.
Status | Completed |
Enrollment | 14 |
Est. completion date | December 2013 |
Est. primary completion date | December 2013 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | N/A to 6 Months |
Eligibility |
Inclusion Criteria: - Laboratory documentation of homozygous absence of SMN1 exon 7. - Confirmation of no more than 3 SMN2 copies for cohort 1; no more than 4 copies for cohort 2. - Family history of affected sibling with SMA type I for cohort 1 and SMA type II for cohort 2. - Age = 3 months, cohort 1; Age = 6 months, cohort 2. - Written informed consent of parents/guardian. - Laboratory results demonstrating normal values for age. Exclusion Criteria: -Evidence of hepatic insufficiency, renal insufficiency, edema with sodium retention, known seizure disorder, urea cycle disorder, cardiac arrhythmia, congenital heart defect, hypertension, significant central nervous system (CNS) impairment, or neurodegenerative or neuromuscular disease other than SMA. History of allergy/sensitivity to sodium phenylbutyrate (NaPB). - Use of NaPB within 30 days of study entry. - Serious illness requiring hospitalization = 14 days prior to study entry. - Use of medications intended for the treatment of SMA including riluzole, valproic acid, hydroxyurea, oral use of albuterol, NaPB, butyrate derivatives, creatine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition within 30 days prior to study entry. - Unwillingness to travel for study assessments. |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | University of Utah | Salt Lake City | Utah |
Lead Sponsor | Collaborator |
---|---|
University of Utah | Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The Study Will Assess the Safety, Tolerability and Potential Efficacy of Sodium Phenylbutyrate (NaPB) in Presymptomatic Infants Genetically Confirmed to Have SMA. It Will Also Determine Selected Pharmacokinetic Parameters. | Number of participants with SAE's related to research. | 24 months | Yes |
Secondary | The Study Will Determine Potential Benefit of NaPB on Lean Body Mass; Overall Motor Function; Potential Cellular Response to NaPB; and Drug Compliance. | 24 months | No |
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