Phase
Condition
Muscular Dystrophy
Martin-bell Syndrome
Myasthenia Gravis (Chronic Weakness)
Treatment
Tiratricol
Clinical Study ID
All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Each patient must meet all of the following criteria to be eligible:
Diagnosis of MCT8 deficiency confirmed with a genetic test.
Either tiratricol treatment naïve, or patients who may be on a stable dose oftiratricol having transferred from the Phase 3 MCT8-2021-3 (ReTRIACt) study (NCT05579327) or prior individual investigational new drug (IND).
In the Treating Physician's medical opinion, the potential benefits of treatmentwith tiratricol outweigh the potential risks for the patient.
Patient or legal representative provided signed and dated informed consent to betreated with tiratricol, through this EAP.
Given the severity of the disease, sexual activity in these patients is deemedunlikely. However, where, at the discretion of the Treating Physician sexualactivity is possible for the patient, patients must followprotocol-specified-contraception guidance.
Patient is approved for enrolment by the sponsor RTT.
Exclusion
Exclusion Criteria:
Patients who meet any of the following criteria will be excluded from the EAP:
Parents, legal representative or, if applicable, patients unwilling or unable tocomply with the Treating Physician's treatment plan related to this EAP for anyreason.
Major illness or recent major surgery unrelated to MCT8 deficiency (in the TreatingPhysician's judgement), defined as:
Conditions requiring repeated hospitalizations that are likely to confoundability to participate in the program.
Major illness in the 3 months before the screening visit that is likely toconfound the ability of the patient to participate fully within the programand/or confound the assessment of serum total T3 and/or safety.
Major surgery within the 3 months before the screening visit, or planned totake place during the program, including but not limited to majorabdominal/thoracic/neurosurgical procedures.
Major/minor abdominal and/or maxillofacial surgery that may inhibit theadministration and/or absorption of tiratricol.
Patients with any contra-indication for treatment with tiratricol or any excipientsin the program treatment.
Patients using thyroid hormone analogues- such as levothyroxine -or thionamides,such as propylthiouracil. Prior use of these drugs is not an exclusion criterion,provided the use of the medication has subsided and the thyroid hormone levels havestabilized after the cessation of these medications. For patients currently usingthese medications the switch to tiratricol should be made following the above andunder the guidance of an endocrinologist with knowledge of MCT8 deficiency, ifneeded, after consultation with pharmacologist.
Known hypersensitivity to tiratricol including any ingredient in the pharmaceuticalformulation.
Although very unlikely, as this is a severe X-linked disease: Women who are nursingor pregnant (or women who are planning to become pregnant during treatment withtiratricol).
Patients eligible for clinical trials with tiratricol.
Study Design
Study Description
Connect with a study center
University of Alabama at Birmingham Hospital
Birmingham, Alabama 35249
United StatesPlanned
Childrens Valley Hospital in
Madera, California 93636-8762
United StatesPlanned
Valley Children's Hospital
Madera, California 93636-8762
United StatesPlanned
Rady Children's Hospital
San Diego, California 92123
United StatesPlanned
Nemour's Children Hospital
Jacksonville, Florida 32207
United StatesPlanned
Children's Healthcare of Atlanta
Atlanta, Georgia 30329
United StatesPlanned
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois 60611
United StatesPlanned
Louisiana State University Health Sciences Center and Children's Hospital
New Orleans, Louisiana 70118
United StatesPlanned
Baystate Medical Center
Springfield, Massachusetts 01199
United StatesPlanned
Gillette's Children's Specialty Healthcare
Saint Paul, Minnesota 55101
United StatesPlanned
Oregon Health and Science University
Portland, Oregon 97239
United StatesPlanned
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United StatesPlanned
Le Bonheur Children's Hospital Foundation
Memphis, Tennessee 38105
United StatesPlanned
Cook Children's Health Care System
Fort Worth, Texas 76106
United StatesPlanned
MultiCare Mary Bridge Children's Hospital
Tacoma, Washington 98403
United StatesPlanned
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