Programa de Acesso Expandido para Tiratricol em Pacientes com Deficiência do Transportador de Monocarboxilato 8
Programa de Acesso Expandido para Tiratricol em Pacientes com Deficiência do Transportador de Monocarboxilato 8, Também Conhecida como Síndrome de Allan-Herndon-Dudley (AHDS)
The goal of this program is to provide expanded access (i.e., before marketing authorization) to tiratricol as treatment for patients with monocarboxylate transporter 8 deficiency (MCT8 deficiency, also known as Allan-Herndon-Dudley syndrome [AHDS]), who in their Treating Physician's opinion, could benefit from tiratricol and meet the eligibility criteria.
Visão geral do estudo
Status
Intervenção / Tratamento
Descrição detalhada
This will be an open-label, single-arm, multi-center EAP, designed to provide treatment access in the US to tiratricol for eligible patients with MCT8 deficiency, also known as AHDS.
Patients will undergo clinical and safety assessments before tiratricol treatment initiation, during the titration phase and then at approximately 3-6 monthly intervals or more frequently if clinically indicated, following the initiation of tiratricol treatment and until treatment is completed or discontinued.
Adverse events (AEs) and serious adverse events (SAEs) will be recorded and reported as per FDA regulations.
Tipo de estudo
Tipo de acesso expandido
- População de tamanho intermediário
- Tratamento IND/Protocolo
Contactos e Locais
Locais de estudo
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Alabama
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Birmingham, Alabama, Estados Unidos, 35249
- University of Alabama at Birmingham Hospital
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California
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Madera, California, Estados Unidos, 93636-8762
- Valley Children's Hospital
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San Diego, California, Estados Unidos, 92123
- Rady Children's Hospital
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Florida
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Jacksonville, Florida, Estados Unidos, 32207
- Nemour's Children Hospital
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Georgia
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Atlanta, Georgia, Estados Unidos, 30329
- Children's Healthcare of Atlanta
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Illinois
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Chicago, Illinois, Estados Unidos, 60611
- Ann & Robert H. Lurie Children's Hospital of Chicago
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Louisiana
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New Orleans, Louisiana, Estados Unidos, 70118
- Louisiana State University Health Sciences Center and Children's Hospital
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Massachusetts
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Springfield, Massachusetts, Estados Unidos, 01199
- Baystate Medical Center
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Minnesota
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Saint Paul, Minnesota, Estados Unidos, 55101
- Gillette's Children's Specialty Healthcare
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New York
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New York, New York, Estados Unidos, 10029
- Icahn School of Medicine at Mount Sinai
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New York, New York, Estados Unidos, 10032
- Columbia University Irving Medical Center
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Oregon
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Portland, Oregon, Estados Unidos, 97239
- Oregon Health and Science University
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Pennsylvania
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Philadelphia, Pennsylvania, Estados Unidos, 19104
- Children's Hospital of Philadelphia
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Tennessee
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Memphis, Tennessee, Estados Unidos, 38105
- Le Bonheur Children's Hospital Foundation
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Texas
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Fort Worth, Texas, Estados Unidos, 76106
- Cook Children's Health Care System
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Washington
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Tacoma, Washington, Estados Unidos, 98403
- MultiCare Mary Bridge Children's Hospital
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Wisconsin
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Milwaukee, Wisconsin, Estados Unidos, 53326
- Children's Wisconsin
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Critérios de participação
Critérios de elegibilidade
Idades elegíveis para estudo
- Filho
- Adulto
- Adulto mais velho
Aceita Voluntários Saudáveis
Gêneros Elegíveis para o Estudo
Descrição
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 of tiratricol 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 treatment with tiratricol outweigh the potential risks for the patient.
- Patient or legal representative provided signed and dated informed consent to be treated with tiratricol, through this EAP.
- Given the severity of the disease, sexual activity in these patients is deemed unlikely. However, where, at the discretion of the Treating Physician sexual activity is possible for the patient, patients must follow protocol-specified-contraception guidance.
- Patient is approved for enrolment by the sponsor RTT.
Exclusion Criteria:
Patients who meet any of the following criteria will be excluded from the EAP:
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Parents, legal representative or, if applicable, patients unwilling or unable to comply with the Treating Physician's treatment plan related to this EAP for any reason.
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Major illness or recent major surgery unrelated to MCT8 deficiency (in the Treating Physician's judgement), defined as:
- Conditions requiring repeated hospitalizations that are likely to confound ability to participate in the program.
- Major illness in the 3 months before the screening visit that is likely to confound the ability of the patient to participate fully within the program and/or confound the assessment of serum total T3 and/or safety.
- Major surgery within the 3 months before the screening visit, or planned to take place during the program, including but not limited to major abdominal/thoracic/neurosurgical procedures.
- Major/minor abdominal and/or maxillofacial surgery that may inhibit the administration and/or absorption of tiratricol.
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Patients with any contra-indication for treatment with tiratricol or any excipients in the program treatment.
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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 have stabilized after the cessation of these medications. For patients currently using these medications the switch to tiratricol should be made following the above and under the guidance of an endocrinologist with knowledge of MCT8 deficiency, if needed, after consultation with pharmacologist.
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Known hypersensitivity to tiratricol including any ingredient in the pharmaceutical formulation.
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Although very unlikely, as this is a severe X-linked disease: Women who are nursing or pregnant (or women who are planning to become pregnant during treatment with tiratricol).
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Patients eligible for clinical trials with tiratricol.
Plano de estudo
Como o estudo é projetado?
Colaboradores e Investigadores
Patrocinador
Publicações e links úteis
Publicações Gerais
- Groeneweg S, Peeters RP, Moran C, Stoupa A, Auriol F, Tonduti D, Dica A, Paone L, Rozenkova K, Malikova J, van der Walt A, de Coo IFM, McGowan A, Lyons G, Aarsen FK, Barca D, van Beynum IM, van der Knoop MM, Jansen J, Manshande M, Lunsing RJ, Nowak S, den Uil CA, Zillikens MC, Visser FE, Vrijmoeth P, de Wit MCY, Wolf NI, Zandstra A, Ambegaonkar G, Singh Y, de Rijke YB, Medici M, Bertini ES, Depoorter S, Lebl J, Cappa M, De Meirleir L, Krude H, Craiu D, Zibordi F, Oliver Petit I, Polak M, Chatterjee K, Visser TJ, Visser WE. Effectiveness and safety of the tri-iodothyronine analogue Triac in children and adults with MCT8 deficiency: an international, single-arm, open-label, phase 2 trial. Lancet Diabetes Endocrinol. 2019 Sep;7(9):695-706. doi: 10.1016/S2213-8587(19)30155-X. Epub 2019 Jul 31.
- van Geest FS, Groeneweg S, van den Akker ELT, Bacos I, Barca D, van den Berg SAA, Bertini E, Brunner D, Brunetti-Pierri N, Cappa M, Cappuccio G, Chatterjee K, Chesover AD, Christian P, Coutant R, Craiu D, Crock P, Dewey C, Dica A, Dimitri P, Dubey R, Enderli A, Fairchild J, Gallichan J, Garibaldi LR, George B, Hackenberg A, Heinrich B, Huynh T, Klosowska A, Lawson-Yuen A, Linder-Lucht M, Lyons G, Monti Lora F, Moran C, Muller KE, Paone L, Paul PG, Polak M, Porta F, Reinauer C, de Rijke YB, Seckold R, Menevse TS, Simm P, Simon A, Spada M, Stoupa A, Szeifert L, Tonduti D, van Toor H, Turan S, Vanderniet J, de Waart M, van der Wal R, van der Walt A, van Wermeskerken AM, Wierzba J, Zibordi F, Zung A, Peeters RP, Visser WE. Long-Term Efficacy of T3 Analogue Triac in Children and Adults With MCT8 Deficiency: A Real-Life Retrospective Cohort Study. J Clin Endocrinol Metab. 2022 Feb 17;107(3):e1136-e1147. doi: 10.1210/clinem/dgab750.
Datas de registro do estudo
Datas de inscrição no estudo
Enviado pela primeira vez
Enviado pela primeira vez que atendeu aos critérios de CQ
Primeira postagem (Real)
Atualizações de registro de estudo
Última Atualização Postada (Real)
Última atualização enviada que atendeu aos critérios de controle de qualidade
Última verificação
Mais Informações
Termos relacionados a este estudo
Palavras-chave
Termos MeSH relevantes adicionais
Outros números de identificação do estudo
- Tiratricol-EAP-Pro MCT8-2022-4
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