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Tests treatment safety and results for Congenital Myotonic Dystrophy
Official title Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy
ClinicalTrials.gov ID: NCT05004129
What this study is testing
What is Tideglusib?
Tideglusib is an investigational medicine, being studied as a potential treatment for congenital myotonic dystrophy.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 6 to 45
You may be able to join if
- people who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. people who did not complete AMO-02-MD-2-003, people who...
- people under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
- Diagnosis must be genetically confirmed
- people must be male or female aged ≥6 years to ≤45 years at Screening
- people must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
You likely can't join if
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
- Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole...
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- 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...
- Existing or historical medical conditions or complications (eg. neurological, cardiovascular, renal, hepatic, gastrointestinal, endocrine or...
See the full eligibility criteria
- people who do not enter this study directly from completing the AMO-02-MD-2-003 study (i.e. people who did not complete AMO-02-MD-2-003, people who completed AMO-02-MD-2-003 but did not directly rollover or people who...
- people under study must be individuals with a diagnosis of Congenital or Childhood Onset DM1.
- Diagnosis must be genetically confirmed
- people must be male or female aged ≥6 years to ≤45 years at Screening
- people must have a Clinical Global Impression - Severity (CGI-S) score of 3 or greater at Screening (V-1)
- 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's caregiver must be willing and able to support participation for duration of study
- Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol people entering directly from completing the antecedent AMO-02-MD-2-003 study will not be considered...
- people who have completed the antecedent AMO-02-MD-2-003 study through V11
- 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...
- Subject's caregiver must be willing and able to support participation for duration of study
- Subject must be willing and able to comply with the required food intake restrictions as outlined per protocol Key
- Body mass index (BMI) less than 13.5 kg/m² or greater than 40 kg/m²
- New or change in medications/therapies within 4 weeks prior to Eligibility/Baseline Visit
- Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
- Concurrent use of drugs metabolized by CYP3A4 with a narrow therapeutic window (e.g. warfarin and digitoxin)
- 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
- 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
- Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry
The study team makes the final eligibility decision.
Where it's taking place
- Little Rock, Arkansas, United States
- Los Angeles, California, United States
- Palo Alto, California, United States
- Chicago, Illinois, United States
- Iowa City, Iowa, United States
- Rochester, New York, United States
- Pittsburgh, Pennsylvania, United States
- Salt Lake City, Utah, United States
- Norfolk, Virginia, United States
- Richmond, Virginia, United States
- Randwick, New South Wales, Australia
- London, Ontario, Canada
- Ottawa, Ontario, Canada
- Auckland, New Zealand
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 6 years to 45 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Little Rock, Arkansas, United States; Los Angeles, California, United States; Palo Alto, California, United States; Chicago, Illinois, United States; Iowa City, Iowa, United States; Rochester, New York, United States and 8 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.