Back to results

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Primary Purpose

Myotonic Dystrophy 1

Status

Completed

Phase

Phase 2

Locations

United Kingdom

Study Type

Interventional

Intervention

Tideglusib

About this trial

This is an interventional treatment trial for Myotonic Dystrophy 1

Eligibility Criteria

12 Years - 45 Years (Child, Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Adolescents or adults with diagnosis of congenital or juvenile-onset type 1 myotonic dystrophy (DM-1)
Diagnosis must be genetically confirmed
Subjects must be male or female aged 12 years to 45 years
Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2)
Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints allowed)
Subject's legally authorized representative (LAR) must provide written informed consent and there must be written consent or assent (as age applicable and developmentally appropriate) by the subject before any study-related procedures are conducted

Exclusion Criteria:

Non-ambulatory (full time) wheel chair user
Receiving stimulant medication
Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run-in (V2)
Medical illness or other concern which would cause investigator to conclude subjects will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment.
Current enrolment in a clinical trial of an investigational drug or enrolment in a clinical trial of an investigational drug in the last 6 months
Women of child bearing potential who are pregnant, lactating or not willing to use a protocol defined acceptable contraception method if sexually active and not surgically sterile.
Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results
Current clinically significant (as determined by the investigator) cardiovascular, renal, hepatic, endocrine or respiratory disease
Clinically significant heart disease (in the opinion of the investigator) or second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia
A history of chronic liver disease with current out of range values for Alanine transaminase (ALT), clinically relevant hepatic steatosis or other clinical manifestations of ongoing liver disease
A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis)
A history of alcohol or substance use disorders

Sites / Locations

Newcastle-upon-Tyne Hospitals NHS Trust

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Arm Label

Cohort 1 - Tideglusib

Cohort 2 - Tideglusib

Arm Description

1000 mg tideglusib, orally, once daily

400 mg tideglusib, orally, once daily

Outcomes

Primary Outcome Measures

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.

Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.

Secondary Outcome Measures

Full Information

NCT ID

NCT02858908

First Posted

August 4, 2016

Last Updated

December 20, 2018

Sponsor

AMO Pharma Limited

1. Study Identification

Unique Protocol Identification Number

NCT02858908

Brief Title

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Official Title

A Single-Blind, Phase 2 Study To Evaluate The Safety And Efficacy Of Tideglusib 400mg Or 1000mg For The Treatment Of Adolescent And Adult Congenital And Juvenile-Onset Myotonic Dystrophy

Study Type

Interventional

2. Study Status

Record Verification Date

December 2018

Overall Recruitment Status

Completed

Study Start Date

July 20, 2016 (Actual)

Primary Completion Date

January 2018 (Actual)

Study Completion Date

January 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

AMO Pharma Limited

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

5. Study Description

Brief Summary

The purpose of this study is to determine whether Tideglusib is safe and efficacious in the treatment of adolescents and adults with congenital and juvenile-onset Myotonic Dystrophy. The pharmacokinetics of tideglusib and its primary metabolite will also be investigated.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myotonic Dystrophy 1

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Sequential Assignment

Masking

Participant

Allocation

Non-Randomized

Enrollment

16 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Cohort 1 - Tideglusib

Arm Type

Experimental

Arm Description

1000 mg tideglusib, orally, once daily

Arm Title

Cohort 2 - Tideglusib

Arm Type

Experimental

Arm Description

400 mg tideglusib, orally, once daily

Intervention Type

Drug

Intervention Name(s)

Tideglusib

Intervention Description

Tideglusib for oral suspension,

Primary Outcome Measure Information:

Title

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study.

Description

Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.

Time Frame

14 weeks (baseline through end of study)

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

45 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Adolescents or adults with diagnosis of congenital or juvenile-onset type 1 myotonic dystrophy (DM-1) Diagnosis must be genetically confirmed Subjects must be male or female aged 12 years to 45 years Subjects must have a Clinical Global Impression - Severity (CGI-S) score of 4 or greater at Screening and Run-in (V2) Subjects must be ambulatory and able to complete the 10 metre walk/run test (splints allowed) Subject's legally authorized representative (LAR) must provide written informed consent and there must be written consent or assent (as age applicable and developmentally appropriate) by the subject before any study-related procedures are conducted Exclusion Criteria: Non-ambulatory (full time) wheel chair user Receiving stimulant medication Receiving other medications/therapies not stable (changed) within 4 weeks prior to Run-in (V2) Medical illness or other concern which would cause investigator to conclude subjects will not be able to perform the study procedures or assessments or would confound interpretation of data obtained during assessment. Current enrolment in a clinical trial of an investigational drug or enrolment in a clinical trial of an investigational drug in the last 6 months Women of child bearing potential who are pregnant, lactating or not willing to use a protocol defined acceptable contraception method if sexually active and not surgically sterile. Gastrointestinal disease which may interfere with the absorption, distribution, metabolism or excretion of the study medication and impact the interpretability of the study results Current clinically significant (as determined by the investigator) cardiovascular, renal, hepatic, endocrine or respiratory disease Clinically significant heart disease (in the opinion of the investigator) or second or third degree heart block, atrial flutter, atrial fibrillation, ventricular arrhythmias, or is receiving medication for treatment of a cardiac arrhythmia A history of chronic liver disease with current out of range values for Alanine transaminase (ALT), clinically relevant hepatic steatosis or other clinical manifestations of ongoing liver disease A history of significant drug allergy (such as Steven-Johnson syndrome, anaphylaxis) A history of alcohol or substance use disorders

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP

Organizational Affiliation

Institute of Neuroscience, Newcastle University.

Official's Role

Principal Investigator

Facility Information:

Facility Name

Newcastle-upon-Tyne Hospitals NHS Trust

City

Newcastle Upon Tyne

State/Province

Tyne And Wear

ZIP/Postal Code

NE1 4LP

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

32942085

Citation

Horrigan J, Gomes TB, Snape M, Nikolenko N, McMorn A, Evans S, Yaroshinsky A, Della Pasqua O, Oosterholt S, Lochmuller H. A Phase 2 Study of AMO-02 (Tideglusib) in Congenital and Childhood-Onset Myotonic Dystrophy Type 1 (DM1). Pediatr Neurol. 2020 Nov;112:84-93. doi: 10.1016/j.pediatrneurol.2020.08.001. Epub 2020 Aug 5.

Results Reference

derived

Learn more about this trial

Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

We'll reach out to this number within 24 hrs