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Clinical Trials in the UK / NCT05777993
Enrolling by invitation Phase 4

A Study to Provide Access to Mitapivat for Participants Who Previously Participated in an Agios-Sponsored Mitapivat Study

NCT05777993 · tracked via the Priya Life Science UK tracker
Sponsor
Agios Pharmaceuticals, Inc.
Phase
Phase 4
Started
2023-07-06
Last updated
2026-08-12

Condition(s) studied

Pyruvate Kinase DeficiencyAnemia, HemolyticSickle Cell DiseaseThalassemia

Investigational drug(s) / intervention(s)

Mitapivat

Mitapivat: Tablets, granules

Study summary

The primary objective of the study is to provide continued access to participants who are deriving clinical benefit from ongoing mitapivat treatment in an Agios-sponsored mitapivat study, in the opinion of the Investigator.

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
Inclusion Criteria: * Participating in an Agios-sponsored study of mitapivat and deriving clinical benefit from ongoing mitapivat treatment, in the opinion of the Investigator. * For women of childbearing potential (WOCBP), must be abstinent of sexual activities that may result in pregnancy as part of their usual lifestyle or agree to use 2 forms of contraception, 1 of which must be considered highly effective, from the time of providing informed consent/assent, throughout the study, and for 28 days after the last dose of study drug. The second form of contraception can be an acceptable barrier method. * Written informed consent/assent from the participant (or their legally authorized representative, parent\[s\], or legal guardian) before any study-related procedures are conducted, and participants must be willing to comply with all study procedures for the duration of the study. * Agrees not to participate in other interventional clinical studies, including other studies of mitapivat, during participation in this study. Exclusion Criteria: * Known allergy or other contraindication to mitapivat or its inactive ingredients as defined in the current investigator brochure (IB). * Any medical, hematologic, psychological, or behavioral condition(s) or prior or current therapy that, in the opinion of the Investigator, may confer an unacceptable risk to participating in the study and/or could confound the interpretation of the study data. Also excluded are: * Participants who are institutionalized by regulatory or court order. * Participants with any condition(s) that could create undue influence (including but not limited to incarceration, involuntary psychiatric confinement, and financial or familial affiliation with the Investigator or Sponsor).

Primary outcome measure(s)

Trial sites (23)

FacilityCityRegionStatus
Hôpital Erasme Anderlecht Brussels Capital
Hospital Sao Lucas Da Pontificia Universidade Catolica Do Rio Grande Do Sul (PUCRS) Porto Alegre Rio Grande do Sul
Hospital de Clínicas da Unicamp Campinas São Paulo
HEMORIO Instituto Nacional de Hematologia Rio de Janeiro Brazil
Praxis Pesquisa Medica Santo André Brazil
GSH Banco de Sangue de São Paulo São Paulo Brazil
Hospital das Clínicas da Faculdade de Medicina da Universidad de São Paulo São Paulo Brazil
Hospital Das Clínicas da Faculdade de Medicina de Ribeirão Preto - USP São Paulo Brazil
McMaster Centre for Transfusion Medicine McMaster University Hamilton Ontario
University Health Network Toronto Ontario
Hopitaux de La Timone Marseille Bouches-du-Rhône
CHU de Bordeaux - Hôpital Pellegrin Bordeau France
CHU Hôpital Henri Mondor Créteil France
Hopital Edouard Herriot, CHU de Lyon Lyon France
AP-HP - Hôpital Européen Georges Pompidou Paris France
Hôpital Necker Enfants Malades Paris France
Rambam Medical Center Haifa Ḥeifā
Ziv Medical Center Safed Ḥeifā
Tohoku University Hospital Sendai Miyagi
Kyoto Katsura Hospital Kyoto Japan
Toho University - Omori Medical Center Ōta-ku Japan
Acibadem Adana Hospital Seyhan Adana
Manchester Royal Infirmary, Manchester University NHS Foundation Trust Manchester United Kingdom

More Agios Pharmaceuticals, Inc. trials in the UK

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05777993 on ClinicalTrials.gov ↗ ← All trials in the UK