Safety and Efficacy of Tideglusib in Congenital Myotonic Dystrophy

• Status: Enrolling by invitation
• Trial end date: 2023-03-28
• Target enrollment: 0
• Participant gender: All

Summary

This is an open-label extension phase 2/3 study for children and adolescents with Congenital Myotonic Dystrophy (Congenital DM1) who participated in and completed the preceding AMO-02-MD-2-003 study.

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: AMO Pharma Limited

Criteria

Inclusion Criteria:

1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11

2. Written or electronic, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or LAR provides consent, there must also be assent from the subject (as required by local regulations)

3. Subject's caregiver must be willing and able to support participation for duration of study

4. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol

Exclusion Criteria:

1. Subjects who discontinued prematurely from the antecedent AMO-02-MD-2-003 study

2. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²

3. New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit

4. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)

5. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)

6. Current enrollment in a clinical trial of an investigational drug or enrollment in a clinical trial of an investigational drug in the last 6 months other than the AMO-02- MD-2-003 study

7. Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or respiratory disease) that may impact the interpretability of the study results

8. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry