RecruitingPhase 3interventionalRules rewrite

Phase 3: testing Mitapivat Matched Placebo for Sickle Cell Disease

The primary objective of this study is to determine the effect of mitapivat versus dummy treatment (placebo) on the need for transfusions in subjects with SCD.

Official registry: https://clinicaltrials.gov/study/NCT07656415 · Agios Pharmaceuticals, Inc. · enrollment listed as 159

What this phase means

Phase 3: this treatment already looked useful in smaller studies. Now it is tested in a large group, often against standard care or a dummy treatment, to see if it should be approved.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are Mitapivat Matched Placebo, or Mitapivat. The registry lists allocation as randomized. The primary objective of this study is to determine the effect of mitapivat versus dummy treatment (placebo) on the need for transfusions in subjects with SCD.

They plan to enroll about 159 people at 5 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease

Interventions: Mitapivat Matched Placebo, Mitapivat

Primary outcome (registry wording): Percentage of Subjects who are Transfusion Free From Week 4 Through Week 52

Who it is for (from inclusion criteria)

Ages 12 Years to any
Documented diagnosis of SCD (hemoglobin SS (HbSS), combined heterozygosity for hemoglobins S and C (HbSC), sickle cell hemoglobin (HbS)/β0-thalassemia, HbS/β+-thalassemia, or other sickle cell syndrome variants)
No more than 10 SCPCs in the 12 months before providing informed assent/consent
At least 1 transfusion of packed RBCs in the 12 months before informed assent/consent
Hb ≥5.5 and ≤10.5 g/dL. Hb concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period
Additional signs or symptoms of hemolysis, as evidenced by any laboratory assessment during the Screening Period with
Absolute reticulocyte count \> upper limit of normal (ULN), or
Indirect bilirubin \>ULN, or

Who the registry excludes

Pregnant, breastfeeding, or parturient
Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea. The last dose of voxelotor, crizanlizumab, and L-glutamine must have been administered at least 90 days before randomization
History of any malignancy except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ. Subjects must not have active disease or received anticancer treatment ≤5 years before providing informed assent/consent
History of active and uncontrolled cardiac or pulmonary disease within 6 months before randomization, including but not limited to:
New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia
Myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; deep venous thrombosis; or pulmonary or arterial embolism
Heart rate-corrected QT interval using Fridericia's method of ≥470 milliseconds for female subjects and ≥450 milliseconds for male subjects, except for right or left bundle branch block

Public study contact (from the registry)

Agios Medical Affairs

08332288474

medinfo@agios.com

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

5 listed

Indiana Hemophilia & Thrombosis Center — Indianapolis, Indiana (recruiting)
The Cancer and Hematology Centers (CHC) — Grand Rapids, Michigan (recruiting)
Southern Specialty Clinic — Flowood, Mississippi (recruiting)
Icahn School of Medicine at Mount Sinai — New York, New York (recruiting)
University of Texas Health Science Center at Houston — Houston, Texas (recruiting)

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Natural History of Sickle Cell Disease

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.

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Background: Some people with the same disorder on a genetic level have more complications than others. Researchers want to look for a link between the PKLR gene and sickle cell disease (SCD) symptoms.

NCT03685721 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

FAQ about this NCT record

Is NCT07656415 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT07656415?

Phase 3. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT07656415?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary