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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03692312




Registration number
NCT03692312
Ethics application status
Date submitted
16/03/2018
Date registered
2/10/2018
Date last updated
8/09/2023

Titles & IDs
Public title
Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy
Scientific title
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)
Secondary ID [1] 0 0
2016-004623-23
Secondary ID [2] 0 0
AMO-02-MD-2-003
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Congenital Myotonic Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Tideglusib
Treatment: Drugs - Placebo

Experimental: Tideglusib - Weight adjusted tideglusib, orally, once daily

Placebo comparator: Placebo - Matching placebo, orally, once daily


Treatment: Drugs: Tideglusib
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily

Treatment: Drugs: Placebo
Matching placebo formulation
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)
Timepoint [1] 0 0
22 weeks
Secondary outcome [1] 0 0
Change in Clinical Global Impression- Improvement Scale (CGI-I) scores
Timepoint [1] 0 0
22 weeks
Secondary outcome [2] 0 0
Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score
Timepoint [2] 0 0
22 Weeks
Secondary outcome [3] 0 0
Caregiver Completed Congenital DM1 Rating Scale (CC-CDM1-RS)
Timepoint [3] 0 0
22 weeks
Secondary outcome [4] 0 0
Clinical Global Impression - Severity Scale (CGI-S)
Timepoint [4] 0 0
22 weeks
Secondary outcome [5] 0 0
10-meter walk-run test
Timepoint [5] 0 0
22 weeks
Secondary outcome [6] 0 0
Incidence of Adverse events (AEs), including serious adverse events (SAEs), between Screening to end of study.
Timepoint [6] 0 0
22 to 28 weeks
Secondary outcome [7] 0 0
Incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, vital signs and bone mineral density) between Screening and end of study.
Timepoint [7] 0 0
22 to 28 weeks

Eligibility
Key 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
Minimum age
6 Years
Maximum age
16 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
3/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/04/2023
Sample size
Target
Accrual to date
Final
56
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
The Bright Alliance - Randwick
Recruitment postcode(s) [1] 0 0
2031 - Randwick
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
California
Country [3] 0 0
United States of America
State/province [3] 0 0
Illinois
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Pennsylvania
Country [7] 0 0
United States of America
State/province [7] 0 0
Utah
Country [8] 0 0
United States of America
State/province [8] 0 0
Virginia
Country [9] 0 0
Canada
State/province [9] 0 0
Ontario
Country [10] 0 0
New Zealand
State/province [10] 0 0
Auckland
Country [11] 0 0
United Kingdom
State/province [11] 0 0
Newcastle Upon Tyne

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
AMO Pharma Limited
Address
Country

Ethics approval
Ethics application status

Summary
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).
Trial website
https://clinicaltrials.gov/study/NCT03692312
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Joseph P Horrigan, MD
Address 0 0
AMO Pharma
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03692312