Enrolling by invitationPhase 1NCT07412821

A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

Studying Adenylosuccinate synthetase-like 1-related distal myopathy

Last synced from ClinicalTrials.gov May 4, 2026

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Key facts

Sponsor: Cure ADSSL1
Principal Investigator: Perry B Shieh, M.D., Ph.D.
UCLA Medical Centre
Intervention: adenylosuccinic acid(drug)
Enrollment: 2 enrolled
Eligibility: 18 years · All sexes
Timeline: 2026 – 2027

Study locations (1)

University of California Los Angeles (UCLA), Los Angeles, California, United States

Collaborators

University of California, Los Angeles

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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