Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)

November 5, 2023 updated by: AMO Pharma Limited

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Tideglusib for the Treatment of Congenital or Childhood Onset DM1 (REACH CDM X)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

This is an open-label study of weight-adjusted 1000 mg tideglusib, once daily for 52 weeks with an open-ended optional extended access period in children and adolescents with a diagnosis of Congenital DM1 who participated in the AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Study Type

Interventional

Enrollment (Estimated)

76

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
  • Canada
    • Ontario
      • London, Ontario, Canada, N6A 4G5
        • Enrolling by invitation
        • Children's Hospital London Health Sciences Centre (LHSC)
      • Ottawa, Ontario, Canada, K1H 8L1
        • Recruiting
        • Children's Hospital of Eastern Ontario
        • Contact:
        • Contact:
        • Principal Investigator:
          • Hanns Lochmüller, MD
  • New Zealand
      • Auckland, New Zealand, 1010
        • Enrolling by invitation
        • New Zealand Clinical Research (NZCR)
  • United States
    • Arkansas
      • Little Rock, Arkansas, United States, 72202
    • California
      • Los Angeles, California, United States, 90095
        • Enrolling by invitation
        • University of California, Los Angeles (UCLA)
      • Palo Alto, California, United States, 94304
        • Enrolling by invitation
        • Stanford University
    • Illinois
      • Chicago, Illinois, United States, 60611
    • Iowa
      • Iowa City, Iowa, United States, 52242
    • New York
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • University of Pittsburgh Medical Center
        • Principal Investigator:
          • Hoda Abdel-Hamid, MD
        • Contact:
        • Contact:
    • Utah
      • Salt Lake City, Utah, United States, 84132
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Withdrawn
        • Children's Hospital of The King's Daughters
      • Richmond, Virginia, United States, 23219
        • Recruiting
        • Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
        • Contact:
        • Contact:
        • Principal Investigator:
          • Nicholas Johnson, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 years to 17 years (Child, Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Time Frame: 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.
52 Weeks
Safety (Adverse Events)
Time Frame: 52 Weeks
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from Screening to Enrolment (where applicable), from Enrolment to End of Treatment (52 Weeks), and End of Treatment to the End of Follow-up period.
52 Weeks
Safety (Adverse Events) - With Optional Expanded Access
Time Frame: Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
The incidence of AEs, including SAEs, and abnormal findings in objective assessments (e.g. laboratory values, ECGs and vital signs) from End of Treatment to End of Optional Extended Access, and End of Optional Extended Access to the End of Follow-up period.
Week 60 and every 8 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
Time Frame: 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.
54 weeks
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
Time Frame: 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.
52 weeks
Clinical Global Impressions Severity Scale (CGI-S)
Time Frame: 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.
54 weeks
Clinical Global Impressions Improvement Scale (CGI-I)
Time Frame: 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.
54 Weeks
Autism Behavior Inventory- Clinician (ABI-C)
Time Frame: 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.
52 Weeks
Socialization, Communication, Daily Living, and Adaptive Behavior Composite standard scores of the Vineland Adaptive Behavior Scale - Survey Interview
Time Frame: 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.
52 Weeks
10-meter walk-run test
Time Frame: 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.
52 Weeks
Plasma Troponin T levels
Time Frame: 52 Weeks
Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
52 Weeks
Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS) - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Improvement Scale (CGI-I) - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS) - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Clinical Global Impressions Severity Scale (CGI-S) - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
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.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Plasma Troponin T levels - With Optional Expanded Access
Time Frame: Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132
Troponin T can be used as a biomarker for cardiac dysfunction but can also be elevated in DM1 patients without evident cardiac dysfunction.
Week 68 and every 16 weeks thereafter up until discontinuation or study closure, assessed up to Week 132

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

AMO Pharma Limited

Investigators

  • Study Director: Joseph P Horrigan, MD, AMO Pharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 23, 2021

Primary Completion (Estimated)

March 28, 2025

Study Completion (Estimated)

March 28, 2025

Study Registration Dates

First Submitted

August 5, 2021

First Submitted That Met QC Criteria

August 5, 2021

First Posted (Actual)

August 13, 2021

Study Record Updates

Last Update Posted (Actual)

November 8, 2023

Last Update Submitted That Met QC Criteria

November 5, 2023

Last Verified

November 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • AMO-02-MD-2-004

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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