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Phase 2 Clinical Trial of SGS-742 Therapy in Succinic Semialdehyde Dehydrogenase Deficiency

NCT02019667 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 19

Last updated 2020-02-24

Study results available
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Summary

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.

Outcome Measures:

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

Conditions

Interventions

DRUG

SGS-742

DRUG

Placebo

Sponsors & Collaborators

  • National Institute of Neurological Disorders and Stroke (NINDS)

    lead NIH

Principal Investigators

  • Sara K Inati, M.D. · National Institute of Neurological Disorders and Stroke (NINDS)

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Model
CROSSOVER

Eligibility

Min Age
4 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2014-03-31
Primary Completion
2019-01-31
Completion
2019-04-03
FDA Drug
Yes

Countries

  • United States

Study Locations

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Drugs
Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02019667 on ClinicalTrials.gov