Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy

Part of paid clinical trials in Little Rock, Arkansas.

Sponsor
AMO Pharma Limited
Study ID
NCT05004129
Phase
PHASE2/PHASE3
Status
Recruiting
Apply to this trial

Conditions

  • Congenital Myotonic Dystrophy

Eligibility Criteria

Sex
ALL
Age
6 Years - 45 Years
Healthy Volunteers
Not accepted

Interventions

  • Tideglusib — DRUG
    Tideglusib dosing will be weight-adjusted at 400 mg, 600 mg, or 1000 mg dose levels, or weight banded fixed doses of 400 mg, 600 mg, 800 mg or 1000 mg, with each subject starting at a weight-adjusted 400 mg dose level for 2 weeks, then up titrating to a weight-adjusted 600 mg dose level for the next 2 weeks.

Study Details

This is an open-label phase 2/3 study for individuals with Congenital Myotonic Dystrophy (Congenital DM1) who participated in the preceding AMO-02-MD-2-003 study or individuals with either Congenital or Childhood Onset DM1 who are treatment naïve.

Key Dates

Start date
Aug 23, 2021
Status verified
May 2025
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
76 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Tideglusib
    Weight adjusted or weight banded tideglusib, orally, once daily

Primary Outcome Measure

Safety (Adverse Events) [ Time Frame: 52 Weeks ]

Locations (10)

FacilityCityStateZIPSite coordinators
Arkansas Children's HospitalLittle RockArkansas72202
Annette Guy
[email protected]
501-364-3380
Caitlyn Daley
[email protected]
Aravindhan Veerapandiyan, MD (PRINCIPAL_INVESTIGATOR)
University of California, Los Angeles (UCLA)Los AngelesCalifornia90095-
Stanford UniversityPalo AltoCalifornia94304-
Lurie's Children's HospitalChicagoIllinois60611
Joseph Alberts
[email protected]
312-227-4449
Caitlyn Daley
[email protected]
Nancy Kuntz, MD (PRINCIPAL_INVESTIGATOR)
University of Iowa Hospitals and ClinicsIowa CityIowa52242
Laura Knosp
[email protected]
319-384-6277
Caitlyn Daley
[email protected]
Katherine Mathews, MD (PRINCIPAL_INVESTIGATOR)
University of Rochester - Medical CenterRochesterNew York14642
James Hilbert
[email protected]
585-273-5590
Caitlyn Daley
[email protected]
Bo Hoon Lee, MD (PRINCIPAL_INVESTIGATOR)
University of Pittsburgh Medical CenterPittsburghPennsylvania15213
Heather DiCostanzo
[email protected]
412-692-7660
Caitlyn Daley
[email protected]
Hoda Abdel-Hamid, MD (PRINCIPAL_INVESTIGATOR)
University of Utah Clinical Neurosciences CenterSalt Lake CityUtah84132
Domink May
[email protected]
801-587-4864
Caitlyn Daley
[email protected]
Stephanie Manberg, DO (PRINCIPAL_INVESTIGATOR)
Children's Hospital of The King's DaughtersNorfolkVirginia23507-
Virginia Commonwealth University-Department of Neurology - Muscular Dystrophy Translational Research ProgramRichmondVirginia23219-

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By research site
Arkansas Children's Hospital· Little Rock, ARUniversity of California, Los Angeles (UCLA)· Los Angeles, CAStanford University· Palo Alto, CALurie's Children's Hospital· Chicago, ILUniversity of Iowa Hospitals and Clinics· Iowa City, IAUniversity of Rochester - Medical Center· Rochester, NY

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