|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
19 October 2017 |
|
Main ID: |
NCT00528268 |
|
Date of registration:
|
10/09/2007 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
STOPSMA |
|
Scientific title:
|
Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy |
|
Date of first enrolment:
|
July 2007 |
|
Target sample size:
|
14 |
|
Recruitment status: |
Completed |
|
URL:
|
https://clinicaltrials.gov/show/NCT00528268 |
|
Study type:
|
Interventional |
|
Study design:
|
|
|
Phase:
|
Phase 1/Phase 2
|
|
|
Countries of recruitment
|
|
United States
| | | | | | | |
|
Contacts
|
|
Name:
|
Kathryn Swoboda, MD |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University of Utah |
| | |
|
Key inclusion & exclusion criteria
|
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.
Age minimum:
N/A
Age maximum:
6 Months
Gender:
All
|
|
Health Condition(s) or Problem(s) studied
|
|
Spinal Muscular Atrophy
|
|
Intervention(s)
|
|
Drug: Sodium phenylbutyrate (NaPB)
|
|
Primary Outcome(s)
|
|
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.
[Time Frame: 24 months]
|
|
Secondary Outcome(s)
|
|
The Study Will Determine Potential Benefit of NaPB on Lean Body Mass; Overall Motor Function; Potential Cellular Response to NaPB; and Drug Compliance.
[Time Frame: 24 months]
|
|
Secondary ID(s)
|
|
1R01HD054599-01
|
|
22183
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|