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NIH Phase 2 Clinical Trial
SGS-742 Therapy in SSADH
9/26/16 Update

The initial goal of the NIH SGS-742 Medication Trial for SSADH patients that started in 2013 was designed to test a total of 22 patients.  However, struggling to meet that 22 patient goal, the NIH lowered the age restriction from 8 to 4 years of age in 2016.  The NIH also agreed to allow non-US patients to participate and extended the study through March of 2018. 

We are grateful to those who have participated in the trial and have gone to great measures to get the concessions in place with the NIH in hopes of having 22 patients complete the trail.  Please consider participating as this trial is not just imperative for this medication, but for all future NIH sponsored research opportunities for SSADH patients.

Understanding the critical importance of this effort, the SSADH Association has committed to financially support non-US patients and their caregivers to travel to the United States to complete the SGS-742 Medication Trial.  While the exact amount is not known at this time, an estimate of funds could be as high as $20,000Click here for details about the SGS trial from the NIH Website.


NIH Contact Information:
Tamika N. Mason [C]
Patient Care and Recruitment Specialist, Clinical Epilepsy Section
NINDS/NIH Building 10
10 Center Drive, Rm 7-5644 SW
Bethesda, MD 20892
Phone: 301-496-1923
Fax: 301-480-8383
tamika.mason@nih.gov


Please email Carolyn Hoffman at choffmanwi@aol.com if you are interested in being part of the trial or if you have contacted the NIH and have not heard back.


Details for the NIH SGS-742 Trial:

Additional evaluations will include neurological and neuropsychological examinations, magnetic resonance spectroscopy and CSF collection to measure GABA levels.

The trial will have a baseline phase in which each patient will undergo a neurological examination and a neuropsychological evaluation. During the subsequent treatment phase, patients will be randomized to SGS-742, supplied by IRIX Pharmaceuticals, 600 mg t.i.d. given orally, or placebo, each for 6 months. Patients will then have repeat TMS, neurological and neuropsychological evaluations, followed by cross-over to the alternate treatment arm, and re-evaluation after 6 months.

Outcome Measures: The primary outcome measures for drug efficacy will be TMS parameters of cortical excitation and inhibition. The secondary outcome measure will be performance on neuropsychological testing. The outcome measures for safety will include clinical examination and neuropsychological tests.

Detailed Description:
Objective: To perform a clinical trial assessing the safety, tolerability and efficacy of the GABA(B) receptor antagonist SGS-742 in patients with SSADH deficiency.
Study Population: Twenty-two children and adults with SSADH deficiency.

Design: Double-blind, cross-over, phase II clinical trial. SGS-742 is a GABA (B) receptor antagonist that has shown to be safe and well-tolerated in clinical trials in adults with cognitive impairment. In addition, preliminary data in the SSADH knockout mouse model suggest efficacy in this specific syndrome. The primary outcome measure will be a change in the Auditory Comprehension subtest of the Neuropsychological Assessment Battery Language Module score; the secondary outcome measure will be a change in cortical excitation and inhibition measured by transcranial magnetic stimulation (TMS).

Click here to link to the NIH Web Site.

 

We are encouraging families to participate in the SGS-742 Trial.

If you are willing to be part of the study or want more information about what is involved, please contact:

Tamika N. Mason [C]
Patient Care and Recruitment Specialist, Clinical Epilepsy Section
NINDS/NIH Building 10
10 Center Drive, Rm 7-5644 SW
Bethesda, MD 20892
Phone: 301-496-1923
Fax: 301-480-8383
tamika.mason@nih.gov