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)
Not applicable as this protocol is to provide an option for continued access to mitapivat for participants who have previously participated in an Agios-sponsored mitapivat study — Not Applicable - Study Completion
Trial sites (23)
Facility
City
Region
Status
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
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.
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