Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy

Sponsor

AMO Pharma Limited (Industry)

Overall Status

Completed

CT.gov ID

NCT02858908

Collaborator

(none)

Enrollment

Location

Arms

17.4

Actual Duration (Months)

0.9

Patients Per Site Per Month

Study Details

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.

Condition or Disease	Intervention/Treatment	Phase
Myotonic Dystrophy 1	Drug: Tideglusib	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

16 participants

Allocation:

Non-Randomized

Intervention Model:

Sequential Assignment

Masking:

Single (Participant)

Primary Purpose:

Treatment

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

Actual Study Start Date :

Jul 20, 2016

Actual Primary Completion Date :

Jan 1, 2018

Actual Study Completion Date :

Jan 1, 2018

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Cohort 1 - Tideglusib 1000 mg tideglusib, orally, once daily	Drug: Tideglusib Tideglusib for oral suspension,
Experimental: Cohort 2 - Tideglusib 400 mg tideglusib, orally, once daily	Drug: Tideglusib Tideglusib for oral suspension,

Arm

Intervention/Treatment

Experimental: Cohort 1 - Tideglusib

1000 mg tideglusib, orally, once daily

Drug: Tideglusib

Tideglusib for oral suspension,

Experimental: Cohort 2 - Tideglusib

400 mg tideglusib, orally, once daily

Drug: Tideglusib

Tideglusib for oral suspension,

Outcome Measures

Primary Outcome Measures

Incidence of Adverse events (AEs), including serious adverse events (SAEs), between baseline to end of study. [14 weeks (baseline through end of study)]
Adverse events may be volunteered spontaneously by the subject, or discovered as a result of general, non-leading questioning by physician.

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 45 Years

Sexes Eligible for Study:

All

Accepts 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Newcastle-upon-Tyne Hospitals NHS Trust	Newcastle Upon Tyne	Tyne And Wear	United Kingdom	NE1 4LP

Sponsors and Collaborators

AMO Pharma Limited

Investigators

Principal Investigator: Grainne Gorman, MB BCh BAO LRCP&SI MRCP FRCP, Institute of Neuroscience, Newcastle University.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

AMO Pharma Limited

ClinicalTrials.gov Identifier:

NCT02858908

Other Study ID Numbers:

AMO-02-MD-2-001
2016-000067-16

First Posted:

Aug 8, 2016

Last Update Posted:

Dec 27, 2018

Last Verified:

Dec 1, 2018

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Myotonic Dystrophy

Study Results

No Results Posted as of Dec 27, 2018