Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Launched by AMO PHARMA LIMITED · Aug 5, 2021

Keywords

ClinConnect Summary

This clinical trial is studying a medication called tideglusib to see how safe it is and how well it works for people with Congenital Myotonic Dystrophy (Congenital DM1). The trial is open to individuals aged 6 to 45 years who have a confirmed diagnosis of this condition, either those who have previously participated in another related study or those who are new to the treatment. Participants will need to have a certain level of severity in their condition and must have a caregiver who can help them through the study process.

If you or a family member are interested in participating, it’s important to know that there are some requirements and restrictions. For example, participants must not have certain health issues or be taking specific medications that could affect the study results. Throughout the trial, participants will be monitored closely, and they will need to follow specific dietary guidelines related to the treatment. This study could help improve understanding and treatment of Congenital Myotonic Dystrophy, making it an important opportunity for those eligible.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• Subjects who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. subjects who did not complete AMO-02-MD-2-003, subjects who completed AMO-02-MD-2-003 but did not directly rollover or subjects who are re-entering AMO-02-MD-2-004), will not be considered eligible for the study without meeting all of the criteria below:
• • 1. Subjects under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
• • 2. Diagnosis must be genetically confirmed
• • 3. Subjects must be male or female aged ≥6 years to ≤45 years at Screening
• • 4. Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
• • 5. Written, voluntary informed consent must be obtained before any study related procedures are conducted. Where a parent or legally authorized representative (LAR) provides consent, there must also be assent from the subject (as required by local regulations)
• • 6. Subject's caregiver must be willing and able to support participation for duration of study
• • 7. Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol
• Subjects entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered eligible for the study without meeting all of the criteria below:
• • 1. Subjects who have completed the antecedent AMO-02-MD-2-003 study through V11
• • 2. Written, 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
• Key Exclusion Criteria:
• • 1. Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
• • 2. New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
• • 3. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
• • 4. Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
• • 5. 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
• • 6. 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
• • 7. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry