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

Conditions

• Adenylosuccinate Synthase 1 Deficient Myopathy

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

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

Study Details

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

Key Dates

First listed

Feb 17, 2026

Start date

Mar 26, 2026

Status verified

Apr 2026

Primary completion

Dec 31, 2027

Completion

Dec 31, 2027

Study Design

Enrollment

2 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Multiple ascending dose-escalation of ASA-001

Primary Outcome Measure

Incidence and severity of all adverse events (AEs), treatment emergent adverse events (TAEs) and serious adverse events (SAEs). [ Time Frame: Screening through to the last assessment at 10 months. ]

Locations (1)

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