Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy (STOPSMA)

This study has been completed.
Sponsor:
Collaborator:
Information provided by (Responsible Party):
Kathryn Swoboda, University of Utah
ClinicalTrials.gov Identifier:
NCT00528268
First received: September 10, 2007
Last updated: June 14, 2015
Last verified: June 2015
Results First Received: June 14, 2015  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Spinal Muscular Atrophy
Intervention: Drug: Sodium phenylbutyrate (NaPB)

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Cohort 1

Family history of SMA type I 0-3 months old Confirmation of no more than 3 SMN2 copies

Sodium phenylbutyrate (NaPB): The powder form of the drug will be dispensed. The target NaPB dosing is 450-600 mg/kg/day, divided into four doses. For cohort 1, we propose to continue treatment for 18 months. For cohort 2, we propose to continue treatment for 24 months.

Cohort 2

Family history of SMA type II 0-6 months old Confirmation of no more than 4 SMN2 copies

Sodium phenylbutyrate (NaPB): The powder form of the drug will be dispensed. The target NaPB dosing is 450-600 mg/kg/day, divided into four doses. For cohort 1, we propose to continue treatment for 18 months. For cohort 2, we propose to continue treatment for 24 months.


Participant Flow:   Overall Study
    Cohort 1     Cohort 2  
STARTED     6     8  
COMPLETED     5     7  
NOT COMPLETED     1     1  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Cohort 1

Family history of SMA type I 0-3 months old Confirmation of no more than 3 SMN2 copies

Sodium phenylbutyrate (NaPB): The powder form of the drug will be dispensed. The target NaPB dosing is 450-600 mg/kg/day, divided into four doses. For cohort 1, we propose to continue treatment for 18 months. For cohort 2, we propose to continue treatment for 24 months.

Cohort 2

Family history of SMA type II 0-6 months old Confirmation of no more than 4 SMN2 copies

Sodium phenylbutyrate (NaPB): The powder form of the drug will be dispensed. The target NaPB dosing is 450-600 mg/kg/day, divided into four doses. For cohort 1, we propose to continue treatment for 18 months. For cohort 2, we propose to continue treatment for 24 months.

Total Total of all reporting groups

Baseline Measures
    Cohort 1     Cohort 2     Total  
Number of Participants  
[units: participants]
  6     8     14  
Age  
[units: participants]
     
<=18 years     6     8     14  
Between 18 and 65 years     0     0     0  
>=65 years     0     0     0  
Gender  
[units: participants]
     
Female     2     5     7  
Male     4     3     7  
Region of Enrollment  
[units: participants]
     
United States     6     8     14  



  Outcome Measures

1.  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.   [ Time Frame: 24 months ]

2.  Secondary:   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 ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.   Safety Issue:   No


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: Dr. Kathryn Swoboda
Organization: University of Utah
phone: 801-585-9717
e-mail: swoboda@genetics.utah.edu


No publications provided


Responsible Party: Kathryn Swoboda, University of Utah
ClinicalTrials.gov Identifier: NCT00528268     History of Changes
Other Study ID Numbers: 22183, 1R01HD054599-01
Study First Received: September 10, 2007
Results First Received: June 14, 2015
Last Updated: June 14, 2015
Health Authority: United States: Food and Drug Administration