Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

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Collaborators:

Information provided by (Responsible Party):

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Tracking Information

First Submitted Date ^ICMJE

March 16, 2018

First Posted Date ^ICMJE

October 2, 2018

Last Update Posted Date

October 2, 2018

Actual Study Start Date ^ICMJE

October 2018

Estimated Primary Completion Date

October 2019 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures ^ICMJE

Change in Clinician-Completed Congenital DM1 Rating Scale (CDM1-RS)[ Time Frame: 22 weeks ]

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.

Original Primary Outcome Measures ^ICMJE

Same as current

Current Secondary Outcome Measures ^ICMJE

Change in Clinical Global Impression- Improvement Scale (CGI-I) scores[ Time Frame: 22 weeks ]
The clinician administered CGI-I rates how much the subject's illness has improved or worsened relative to a baseline state.
Change in Top 3 Caregiver Concerns Visual Analogue Scale (VAS) score[ Time Frame: 22 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)[ Time Frame: 22 weeks ]
The Caregiver-Completed Congenital DM1 Scale is a caregiver assessment of the subject on symptoms that may occur in individuals with CDM1. There are a total of 11 clinically relevant symptoms that the caregiver is asked to rate the severity of.
Clinical Global Impression - Severity Scale (CGI-S)[ Time Frame: 22 weeks ]
The Clinical Global Impression - Severity Scale (CGI-S) is a 7-point 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.
Incidence of Adverse events (AEs), including serious adverse events (SAEs), between Screening to end of study.[ Time Frame: 22 to 28 weeks ]
Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.
Incidence of abnormal findings in objective assessments (e.g. laboratory values, ECGs, vital signs and bone mineral density) between Screening and end of study.[ Time Frame: 22 to 28 weeks ]
Abnormal laboratory findings (e.g. hematology, liver function, biochemistry, urinalysis) or other abnormal assessments (e.g. ECGs, vital signs) that are judged by the Investigator as clinically significant will be recorded as AEs or SAEs if they meet the definition of an AE. The Investigator will exercise his or her medical and scientific judgment in deciding whether an abnormal laboratory finding or other abnormal assessment is clinically significant.

Descriptive Information

Brief Title ^ICMJE

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Official Title ^ICMJE

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

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.

Study Type ^ICMJE

Interventional

Study Phase

Phase 2/Phase 3

Study Design ^ICMJE

Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
Masking: Interventional
Masking Description:
Primary Purpose: Treatment

Condition ^ICMJE

Intervention ^ICMJE

Drug: Tideglusib
Tideglusib for oral suspension, weight-adjusted at 400mg, 600mg or 1000 mg dose levels, once daily
Drug: Placebo
Matching placebo formulation

Study Arms

Experimental: Tideglusib
Weight adjusted tideglusib, orally, once daily
Placebo Comparator: Placebo
Matching placebo, orally, once daily

Recruitment Information

Recruitment Status ^ICMJE

Not yet recruiting

Estimated Enrollment ^ICMJE

Original Estimated Enrollment ^ICMJE

Same as current

Estimated Study Completion Date

November 2019

Estimated Primary Completion Date

October 2019 (Final data collection date for primary outcome measure)

Eligibility Criteria ^ICMJE

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 week 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 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

Sex/Gender

Sexes Eligible for Study:

All

Ages

6 Years and older (Adult, Older Adult)

Accepts Healthy Volunteers

Listed Location Countries ^ICMJE

Canada|United States

Removed Location Countries

Administrative Information

Has Data Monitoring Committee

Yes

U.S. FDA-regulated Product

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

Plan to Share IPD:

Responsible Party

，

Study Sponsor ^ICMJE

AMO Pharma Limited

Collaborators ^ICMJE

Investigators ^ICMJE

Study Director: Joseph P Horrigan, MD AMO Pharma

PRS Account

Verification Date

September 2018

^ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP

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