Набор по приглашению NCT05777993

A Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Фаза IV С лечением Pyruvate Kinase Deficiency Anemia, Hemolytic

Ориентир для пациента и семьи

Простыми словами

Автоматическая сводка по структурированным данным реестра. Она помогает сориентироваться, но не заменяет официальный протокол или оценку врача.

Что изучают: В протоколе указаны: Mitapivat.
Кому может быть актуально: Состояния в реестре: Pyruvate Kinase Deficiency, Anemia, Hemolytic. Базовые параметры: от 18 лет · Все.
Что важно проверить: Возраст, диагноз и пол — только базовые ориентиры. Предыдущее лечение, анализы и другие обязательные условия указаны ниже в критериях участия.
Где проводится: Канада, Япония
Следующий шаг: Сохраните исследование, покажите его лечащему врачу и уточните актуальный статус у исследовательского центра. Расходы, документы и поездка →

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Официальное название

A Rollover Study to Provide Continued Access to Mitapivat for Participants Who Previously Completed an Agios-Sponsored Mitapivat Study

Обзор

The purpose of this study is to provide continued access to mitapivat for participants who completed an Agios-sponsored mitapivat study (antecedent) and do not have commercial access to mitapivat.

Подробное описание

Participants will continue the mitapivat dose regimen that they were receiving at the final visit of their antecedent study.

Вмешательства

Препарат Mitapivat
Tablets

Первичные конечные точки

Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) [Срок оценки: Up to approximately 6.2 years]
Percentage of Participants With AEs and SAEs, Graded by Severity [Срок оценки: Up to approximately 6.2 years]
Percentage of Participants With AEs Considered by the Investigator to be Related to Study Drug [Срок оценки: Up to approximately 6.2 years]
Percentage of Participants With SAEs Considered by the Investigator to be Related to Study Drug [Срок оценки: Up to approximately 6.2 years]

Критерии участия

Критерии включения

Has completed an Agios-sponsored adult pyruvate kinase (PK) deficiency study of mitapivat within 1 day of enrollment in this rollover study;
Is unable to obtain mitapivat treatment due to country regulations or lack of commercial access;
Was deriving clinical benefit from mitapivat treatment at the completion of the antecedent study, in the opinion of the Investigator;
For women of childbearing potential, 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, 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 from the participant before any study-related procedures are conducted and 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.

Критерии исключения

Has discontinued treatment with mitapivat before completing the antecedent study;
Known allergy or other contraindication to mitapivat or its inactive ingredients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, Opadry II Blue \[hypromellose, titanium dioxide, lactose monohydrate, triacetin, and FD\&C Blue #2\]);
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 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).

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет