Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

AMO Pharma

Status and phase: Completed; Phase 3; Phase 2

Conditions: Congenital Myotonic Dystrophy

Treatments: Drug: Tideglusib; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT03692312

AMO-02-MD-2-003

2016-004623-23 (EudraCT Number)

Details and patient eligibility

About

This is a randomized, multicenter, double-blind, placebo-controlled, Phase 2/3 study of patients (aged 6 to 16 years) diagnosed with Congenital Myotonic Dystrophy (Congenital DM1).

Full description

This is a randomized, double-blind, placebo controlled study of weight adjusted dose 1000 mg/day tideglusib versus placebo in the treatment of children and adolescents 6-16 years of age with Congenital DM1.

Enrollment: 56 patients

Sex: All

Ages: 6 to 16 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Male or female children and adolescents aged ≥6 years and ≤16 years

2. Diagnosis of Congenital DM1 (also known as Steinert's disease)

   • Diagnosis must be genetically confirmed

   • One or more of the following clinically relevant (e.g. requiring medical intervention) signs or symptoms was evident within the first month after birth:

     • Hypotonia
     • Generalized weakness
     • Respiratory insufficiency
     • Feeding difficulties
     • Clubfoot or another musculoskeletal deformity

3. Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)

4. 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

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

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

Exclusion criteria

1. Not able to walk; (full time wheel chair use)
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 Screening
4. Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
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
7. Existing or historical medical conditions or complications (e.g. neurological, cardiovascular, renal, hepatic, endocrine, gastrointestinal or respiratory disease) which would cause the investigator to conclude that the subject will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment
8. Hypersensitivity to tideglusib and its excipients including allergy to strawberry

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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56 participants in 2 patient groups, including a placebo group

Tideglusib

Experimental group

Description:

Weight adjusted tideglusib, orally, once daily

Treatment:

Drug: Placebo

Drug: Tideglusib

Placebo

Placebo Comparator group

Description:

Matching placebo, orally, once daily

Treatment:

Drug: Placebo