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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05004129
Recruitment Status : Enrolling by invitation
First Posted : August 13, 2021
Last Update Posted : November 16, 2021
Sponsor:
Information provided by (Responsible Party):
AMO Pharma Limited

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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 : August 23, 2021
Estimated Primary Completion Date : March 28, 2023
Estimated Study Completion Date : March 28, 2023


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.




Primary Outcome Measures :
  1. Safety (Adverse Events) [ Time Frame: 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 :
  1. 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.

  2. 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.

  3. 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.

  4. 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.

  5. 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.

  6. 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.

  7. 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.

  8. 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.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   6 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  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

Exclusion Criteria:

  1. Subjects who discontinued prematurely from the antecedent AMO-02-MD-2-003 study
  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 Eligibility/Baseline Visit
  4. Use within 4 weeks prior to Eligibility/Baseline Visit of strong CYP3A4 inhibitors (eg.clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir)
  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 other than the AMO-02- MD-2-003 study
  7. 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
  8. Hypersensitivity to tideglusib or any components of its formulation including allergy to strawberry

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05004129


Locations
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United States, Arkansas
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72202
United States, California
University of California, Los Angeles (UCLA)
Los Angeles, California, United States, 90095
Stanford University
Palo Alto, California, United States, 94304
United States, Iowa
University of Iowa Hospitals and Clinics
Iowa City, Iowa, United States, 52242
United States, New York
University of Rochester - Medical Center
Rochester, New York, United States, 14642
United States, Virginia
Children's Hospital of The King's Daughters
Norfolk, Virginia, United States, 23507
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research Program
Richmond, Virginia, United States, 23219
Australia, New South Wales
The Bright Alliance
Randwick, New South Wales, Australia, 2031
Canada, Ontario
Children's Hospital London Health Sciences Centre (LHSC)
London, Ontario, Canada, N6A 4G5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, Canada, K1H 8L1
New Zealand
New Zealand Clinical Research (NZCR)
Auckland, New Zealand, 1010
Sponsors and Collaborators
AMO Pharma Limited
Investigators
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Study Director: Joseph P Horrigan, MD AMO Pharma
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Responsible Party: AMO Pharma Limited
ClinicalTrials.gov Identifier: NCT05004129    
Other Study ID Numbers: AMO-02-MD-2-004
First Posted: August 13, 2021    Key Record Dates
Last Update Posted: November 16, 2021
Last Verified: November 2021

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by AMO Pharma Limited:
Tideglusib
AMO-02-MD-2-004
Congenital Myotonic Dystrophy
Myotonic Dystrophy
Dystrophia Myotonica
Myotonia Atrophica
Myotonia Dystrophica
Myotonic Dystrophy, Congenital
Steinert Disease
Steinert Myotonic Dystrophy
Steinert's Disease
Additional relevant MeSH terms:
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Myotonic Dystrophy
Muscular Dystrophies
Muscular Disorders, Atrophic
Muscular Diseases
Musculoskeletal Diseases
Myotonic Disorders
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Genetic Diseases, Inborn