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Clinical trial

A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid (ASA) in Two Siblings With Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.

NCT07412821 is registered in ClinicalTrials.gov with Cure ADSSL1 as lead sponsor.

NCT NCT07412821
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1b, Open Label, Single and Multiple Ascending Dose-escalation Study to Assess the Safety, Tolerability, Pharmacokinetics and Preliminary Efficacy of Subcutaneous Adenylosuccinic Acid (ASA) in Two Siblings With Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.
Lead sponsor
Cure ADSSL1
Phase
Phase 1
Overall status
Enrolling by invitation
Start date
March 26, 2026
Completion date
December 31, 2027
Enrollment
2 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

adenylosuccinate synthase 1 deficient myopathy