Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

  • STATUS
    Recruiting
  • End date
    Mar 28, 2022
  • participants needed
    56
  • sponsor
    AMO Pharma Limited
Updated on 28 August 2021
weakness
myotonic dystrophy

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).

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.

Details
Condition Muscular Dystrophy, Myotonic Dystrophy
Treatment Placebo, Tideglusib
Clinical Study IdentifierNCT03692312
SponsorAMO Pharma Limited
Last Modified on28 August 2021

Eligibility

Yes No Not Sure

Inclusion Criteria

Male or female children and adolescents aged 6 years and 16 years
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
Subject must be able to walk and complete the 10-meter walk-run test (orthotics/splints allowed, forearm crutches are not allowed)
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
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

Not able to walk; (full time wheel chair use)
Body mass index (BMI) less than 13.5 kg/m or greater than 40 kg/m
New or change in medications/therapies within 4 weeks prior to Screening
Use of strong CYP3A4 inhibitors (e.g clarithromycin, telithromycin, ketoconazole, itraconazole, posaconazole, nefazodone, idinavir and ritonavir) within 4 weeks prior to Baseline
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
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
Hypersensitivity to tideglusib and its excipients including allergy to strawberry
Clear my responses

How to participate?

Step 1 Connect with a site
What happens next?
  • You can expect the study team to contact you via email or phone in the next few days.
  • Sign up as volunteer to help accelerate the development of new treatments and to get notified about similar trials.

You are contacting

Investigator Avatar
Name

Primary Contact

site
Name

0/250
Preferred Language
Other Language
Please verify that you are not a bot.

Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

Learn more

If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

Learn more

Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

Learn more

Similar trials to consider

Loading...

Browse trials for

Not finding what you're looking for?

Every year hundreds of thousands of volunteers step forward to participate in research. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.

Sign up as volunteer

user name

Added by • 

 • 

Private

Reply by • Private
Loading...

Lorem ipsum dolor sit amet consectetur, adipisicing elit. Ipsa vel nobis alias. Quae eveniet velit voluptate quo doloribus maxime et dicta in sequi, corporis quod. Ea, dolor eius? Dolore, vel!

  The passcode will expire in None.
Loading...

No annotations made yet

Add a private note
  • abc Select a piece of text from the left.
  • Add notes visible only to you.
  • Send it to people through a passcode protected link.
Add a private note