REACH CDM X: Safety and Efficacy of Tideglusib in Congenital Myotonic Dystrophy

Sponsor

AMO Pharma Limited (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05004129

Collaborator

(none)

Enrollment

Locations

Arm

19.1

Anticipated Duration (Months)

5.1

Patients Per Site

0.3

Patients Per Site Per Month

Study Details

Study Description

Brief 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.

Condition or Disease	Intervention/Treatment	Phase
Congenital Myotonic Dystrophy	Drug: Tideglusib	Phase 2/Phase 3

Detailed Description

This is an open-label extension study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks in children and adolescents with a diagnosis of Congenital DM1 who have completed the AMO-02-MD-2-003 study.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

56 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

A 52-Week, Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital DM1 (REACH CDM X)

Actual Study Start Date :

Aug 23, 2021

Anticipated Primary Completion Date :

Mar 28, 2023

Anticipated Study Completion Date :

Mar 28, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Tideglusib Weight adjusted tideglusib, orally, once daily	Drug: Tideglusib All subjects will receive weight-adjusted 1000 mg tideglusib following a titration period of 2 weeks at a weight-adjusted dose of 400 mg and 2 weeks at a weight-adjusted dose of 600 mg tideglusib.

Arm

Intervention/Treatment

Experimental: Tideglusib

Weight adjusted tideglusib, orally, once daily

Drug: Tideglusib

All subjects will receive weight-adjusted 1000 mg tideglusib following a titration period of 2 weeks at a weight-adjusted dose of 400 mg and 2 weeks at a weight-adjusted dose of 600 mg tideglusib.

Outcome Measures

Primary Outcome Measures

Safety (Adverse Events) [54 weeks]
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Enrolment to End of Treatment, and End of Treatment to the End of Follow-up period.

Secondary Outcome Measures

Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) [52 weeks]
The Clinician-completed Congenital DM1 Rating Scale is an 11-item rating scale completed by the clinician to score the symptom severity of domains that are clinically relevant in Congenital DM1.
Clinical Global Impressions Improvement Scale (CGI-I) [54 weeks]
The CGI-I requires the clinician to rate how much the subject's illness has changed (improved, worsened or stayed the same) relative to a baseline state.
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score [54 weeks]
The Top 3 concerns VAS allows caregivers to identify their main three causes of concern, related to the subject's myotonic dystrophy, rather than these being pre-specified within a scale and then rating how these concerns have changed at specific time-points during the study.
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) [52 weeks]
CC-CDM1-RS provides a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 symptoms that the caregiver is asked to rate from 0 to 4 based on overall severity.
Clinical Global Impressions Severity Scale (CGI-S) [54 weeks]
The CGI-S is a 7-point Likert type scale that requires the clinician to rate the severity of the subject's illness at the time of assessment, relative to the clinician's past experience with subjects who have the same diagnosis.
Autism Behavior Inventory- Clinician (ABI-C) [52 weeks]
ABI-C is a 14 item rating scale requires the clinician to assess the core features of Autism Spectrum Disorder as well as common associated behaviors.
Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview [52 weeks]
The Vineland Adaptive Behavior Scales require the clinician to measure personal and social skills needed for everyday living. It provides a targeted assessment of adaptive behavior via a semi-structured parent or caregiver interview.
10-meter walk-run test [52 weeks]
The 10-meter walk/run test is a performance measure used to assess walking speed in meters per second over a short distance. It can be used as an assessment of functional mobility.

Eligibility Criteria

Criteria

Ages Eligible for Study:

6 Years to 17 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Subjects 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 regulations)
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

Exclusion Criteria:

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Arkansas Children's Hospital	Little Rock	Arkansas	United States	72202
2	University of California, Los Angeles (UCLA)	Los Angeles	California	United States	90095
3	Stanford University	Palo Alto	California	United States	94304
4	University of Iowa Hospitals and Clinics	Iowa City	Iowa	United States	52242
5	University of Rochester - Medical Center	Rochester	New York	United States	14642
6	Children's Hospital of The King's Daughters	Norfolk	Virginia	United States	23507
7	Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program	Richmond	Virginia	United States	23219
8	The Bright Alliance	Randwick	New South Wales	Australia	2031
9	Children's Hospital London Health Sciences Centre (LHSC)	London	Ontario	Canada	N6A 4G5
10	Children's Hospital of Eastern Ontario	Ottawa	Ontario	Canada	K1H 8L1
11	New Zealand Clinical Research (NZCR)	Auckland		New Zealand	1010