A Randomized, Double-Blind Study to Evaluate the Efficacy and Safety of Tideglusib Versus Placebo for the Treatment of Children and Adolescents With Congenital Myotonic Dystrophy (REACH CDM)

AMO Pharma Limited14 sites in 5 countries56 target enrollmentMarch 3, 2021

ConditionsCongenital Myotonic Dystrophy

InterventionsPlacebo Tideglusib

DrugsTideglusib Placebo

Overview

Phase: Phase 2
Intervention: Placebo
Conditions: Congenital Myotonic Dystrophy
Sponsor: AMO Pharma Limited
Enrollment: 56
Locations: 14
Primary Endpoint: Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Status: Completed
Last Updated: 6 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

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).

Detailed 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.

Registry

clinicaltrials.gov

Start Date

March 3, 2021

End Date

April 4, 2023

Last Updated

6 months ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

AMO Pharma Limited

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Male or female children and adolescents aged ≥6 years and ≤16 years
•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
•Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)

Exclusion Criteria

•Not able to walk; (full time wheel chair use)
•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 Screening
•Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
•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 (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
•Hypersensitivity to tideglusib and its excipients including allergy to strawberry

Arms & Interventions

Placebo

Matching placebo, orally, once daily

Intervention: Placebo

Tideglusib

Weight adjusted tideglusib, orally, once daily

Intervention: Tideglusib

Tideglusib

Weight adjusted tideglusib, orally, once daily

Intervention: Placebo

Outcomes

Primary Outcomes

Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)

Time Frame: Baseline and week 20

The Clinician-Completed Congenital DM1 Scale is an 11-item rating scale completed by the clinician that scores the symptom severity of domains that are clinically relevant in Congenital DM1. The severity of the clinician's concern in each domain is scored by using a 5-point Likert Scale. Scores range from 0 = Not present to 4 = Very severe.

Secondary Outcomes

10-meter Walk-run Test(20 weeks)
Change in Clinical Global Impression- Improvement Scale (CGI-I) Scores(Baseline and week 20)
Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) Score(Baseline and week 20)
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)(Baseline and week 20)
Clinical Global Impression - Severity Scale (CGI-S)(Baseline and week 20)
Number of Adverse Events (AEs), Including Serious Adverse Events (SAEs), Between Screening to End of Study.(Between Screening to End of Study, up to 28 weeks)
Number of Abnormal Findings in Objective Assessments (e.g. Laboratory Values, ECGs, Vital Signs and Bone Mineral Density) Between Screening and End of Study.(Between Screening to End of Study, up to 28 weeks)
CDM1-RS Independent Central Rater Score (CDM1-RS)(Baseline to week 20)
CGI-I Independent Central Rater Score (CGI-I)(Baseline and week 20)
Independent Rater Clinical Global Impression - Severity Scale (CGI-S)(Baseline and week 20)