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

NCT07412821 · Status: ENROLLING_BY_INVITATION · Phase: PHASE1 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2026-04-07

No results posted yet for this study

Summary

The goal of this clinical trial is to evaluate the safety, tolerability and preliminary efficacy of ASA-001 in two adults diagnosed with ADSS1 deficient myopathy. The main questions it aims to answer are:

* Whether ASA-001 can be safely administered to ADSS1 deficient myopathy patients;
* Whether daily treatment with ASA-001 provides benefit or slows progression of disease.

Participants will:

* Take ASA-001 every day for 8 months;
* Visit the clinic once every 2 weeks for check-ups and tests

Conditions

  • Adenylosuccinate Synthase 1 Deficient Myopathy

Interventions

DRUG

adenylosuccinic acid

ASA-001 will be administered as a sterile solution (500-2500 mg/day) by sub-cutaneous infusion pump.

Sponsors & Collaborators

Principal Investigators

  • Perry B Shieh, M.D., Ph.D. · UCLA Medical Centre

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
18 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-03-26
Primary Completion
2027-12-31
Completion
2027-12-31
FDA Drug
Yes

Countries

  • United States

Study Locations

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Entities

Companies

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 NCT07412821 on ClinicalTrials.gov