Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Cure ADSSL1
With
University of California, Los Angeles
Age
18–any
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Male and females, age 18 years and above and weighing between 60 and 85 kg
Patient(s) diagnosed with ADSS1 deficient myopathy with homozygous or compound heterozygous mutations in the ADSS1 gene.
Able to understand and comply with all the study requirements
Is willing and legally able to provide written informed consent.
Willing to use highly effective contraception
You may not be if
Any medical condition that could, in the Investigator's opinion, adversely affect the safety of the patient, make it unlikely that the course of treatment would be completed, or impair the assessment of study results.
Any patient who, in the Investigator's opinion, seems unable/unwilling to comply with the study procedures.
Women who are pregnant or breastfeeding, or planning to become pregnant
As judged by the investigator, clinical features are present at the time of screening / baseline assessments indicating that safe travel and completion of the study and its assessments are unlikely
Other severe systemic illness or disease
Participation in another treatment clinical study within thirty (30) days or 5 half-lives of the investigational product, whichever is longer, prior to signing and dating of Informed Consent Form for this study.
Known hypersensitivity to any of the components/excipients in ASA-001
Serologic evidence of hepatitis B, C, or HIV
Ongoing/active infection (including current COVID-19 infection)
Presence of clinically significant liver or renal abnormalities
Clinical chemistry and hematology outside the limits acceptable for this patient population
History of anaphylaxis or severe allergic reaction to drug therapy or foods.
Concomitant medications to manage chronic condition(s) must not interfere with the mechanism of action for ASA-001 in the opinion of the Investigator and dose(s) must not alter for at least 4 weeks before screening through to dosing (Day 1).
Clareo Health | A Phase 1b Study of Adenylosuccinic Acid (ASA-001) for Adenylosuccinate Synthase 1 (ADSS1) Deficient Myopathy.
