Suspended

Mitapivat for Long-term Safety in Stable Sickle Cell Disease

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Study Aim

This study aims to evaluate the long-term safety and tolerability of mitapivat in individuals with stable sickle cell disease over 48 weeks.

What is being tested

Mitapivat

Drug
Who is being recruted

Anemia+6

+ Anemia, Hemolytic

+ Anemia, Hemolytic, Congenital

From 18 to 70 Years
+41 Eligibility Criteria
See all eligibility criteria
How is the trial designed

Treatment Study

Phase 1 & 2
Interventional
Study Start: December 2020
See protocol details

Summary

Principal SponsorNational Heart, Lung, and Blood Institute (NHLBI)
Last updated: February 6, 2026
Sourced from a government-validated database.Claim as a partner

Study start date: December 9, 2020

Actual date on which the first participant was enrolled.

This clinical trial aims to evaluate the long-term safety and tolerability of the drug mitapivat in people with stable sickle cell disease (SCD). Participants in this study have previously completed an initial Phase I study of mitapivat and will now be taking a maintenance dose for 48 weeks. The study will focus on how safe and tolerable the drug is over this extended period. Researchers are also interested in understanding how the drug affects red blood cell metabolism and survival, which could potentially lead to clinical benefits for SCD patients. The goal is to improve the management of sickle cell disease by offering a new maintenance therapy option that can reduce symptoms and improve quality of life. Participants will receive mitapivat and undergo regular health evaluations to monitor safety, drug absorption, and drug effects. These evaluations include checking how the drug influences hemoglobin levels, markers of red blood cell breakdown, and overall health and quality of life. The study will also track how often participants experience pain crises, which are common in sickle cell disease, and will assess the usability of a digital health app designed for SCD patients. If participants benefit from the drug, they may continue the therapy for an additional five years, allowing for a comprehensive understanding of long-term effects.

Official TitleEvaluation of the Safety,Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
NCT04610866
Principal SponsorNational Heart, Lung, and Blood Institute (NHLBI)
Last updated: February 6, 2026
Sourced from a government-validated database.Claim as a partner

Protocol

This section provides details of the study plan, including how the study is designed and what the study is measuring.
Design Details

15 patients to be enrolled

Total number of participants that the clinical trial aims to recruit.

Treatment Study

These studies test new ways to treat a disease, condition, or health issue. The goal is to see if a new drug, therapy, or approach works better or has fewer side effects than existing options.

Eligibility

Researchers look for people who fit a certain description, called eligibility criteria: person's general health condition or prior treatments.
Conditions
Criteria

Any sex

Biological sex of participants that are eligible to enroll.

From 18 to 70 Years

Range of ages for which participants are eligible to join.

Healthy volunteers not allowed

If individuals who are healthy and do not have the condition being studied can participate.

Conditions

Pathology

AnemiaAnemia, HemolyticAnemia, Hemolytic, CongenitalAnemia, Sickle CellHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, Inborn

Criteria

16 inclusion criteria required to participate
c. Absolute neutrophil count >=1.0 x 10^9/L.
Subjects completing Study 19H0097 will first be screened for eligibility. Eligibility criteria are identical with the exception of criteria 5.1.4, 5.2.2, and 5.2.3.p. If any of the 15 subjects completing the 19H0097 study are unable to participate in or complete the current extension study (defined as completing 24 weeks of treatment with study drug to allow for assessment of the primary endpoint), then additional new subjects naive to mitapivat treatment may be enrolled to replace them.
Subjects will enroll onto the study and undergo screening. Subjects who do not meet any of the following criteria during screening will not receive the study intervention but will be counted toward study accrual. Screen failures may be rescreened at a later time.
d. Hemoglobin >= 7 g/dL
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25 exclusion criteria prevent from participating
6. History of drug-induced cholestatic hepatitis.
Documented pyruvate kinase deficiency
Screening hemoglobin level of >= 11 g/dL
Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound the interpretation of the study data. Such significant medical conditions include, but are not limited to the following:
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Study Plan

Find out more about all the medication administered in this study, their detailed description and what they involve.
Treatment Groups
Study Objectives

One single intervention group is designated in this study

This study does not include a placebo group 

Treatment Groups

Group I

Experimental
Subjects will be treated with a maintenance dose of mitapivat previously assessed for safety and tolerability in the Phase I study for an initial 48 weeks and undergo safety monitoring, evaluation of pharmacokinetics and pharmacodynamics, and assessment of secondary laboratory and clinical endpoints at pre-specified intervals during the study period.

Study Objectives

Primary Objectives

Secondary Objectives

Study Centers

These are the hospitals, clinics, or research facilities where the trial is being conducted. You can find the location closest to you and its status.

This study has 1 location

Suspended

National Institutes of Health Clinical Center

Bethesda, United StatesOpen National Institutes of Health Clinical Center in Google Maps
SuspendedOne Study Center
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