Clinical Trials in Japan / 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 Japan tracker
Condition(s) studied
Pyruvate Kinase DeficiencyAnemia, HemolyticSickle Cell DiseaseThalassemia
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
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 | Manchester | United Kingdom |
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 Japan