Sickle Cell Disease study: Mitapivat
Official title A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD)
ClinicalTrials.gov ID: NCT07656415
What this study is testing
What is Mitapivat?
an investigational oral tablet being studied as a potential treatment for sickle cell disease is designed to activate pyruvate kinase in red blood cells, which may improve red cell function
Also referred to as AG-348, Mitapivat sulfate.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to find out if taking mitapivat helps reduce how often participants need blood transfusions compared to a placebo.
- Who it's for
- People who have sickle cell disease.
- What's involved
- Participants will take either the study drug mitapivat or a placebo (a pill with no active medicine) and be monitored for how many transfusions they need.
- Time commitment
- The source text does not specify the duration or visit schedule for this study.
- Phase 3: a large, late-stage study
- You might receive a placebo (an inactive treatment) instead of the study drug.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Age ≥12 years.
- Documented diagnosis of SCD (hemoglobin SS (HbSS), combined heterozygosity for hemoglobins S and C (HbSC), sickle cell hemoglobin...
- 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...
You likely can't join if
- Pregnant, breastfeeding, or parturient.
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion)
- Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days prior to providing informed assent/consent or during the Screening Period.
- Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of...
- History of any malignancy except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.
- New York Heart Association Class III or IV heart failure or clinically significant dysrhythmia.
See the full eligibility criteria
Who can join
- Age ≥12 years.
- 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
- Hb \<8 g/dL, or
- Absolute reticulocyte count \> upper limit of normal (ULN), or
- Indirect bilirubin \>ULN, or
- LDH \>ULN
- If taking hydroxyurea, the hydroxyurea dose must be stable for at least 90 days prior to randomization. Discontinuation of hydroxyurea requires a 90-day washout prior to informed assent/consent.
- Women of childbearing potential (WOCBP) and pediatric female people who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle or agree to use a highly...
- Written informed assent/consent (for people under 18 years of age, or prior to the age at which a subject is considered legally an adult per local regulations, parental permission and child assent will be obtained) must...
What rules you out
- Pregnant, breastfeeding, or parturient.
- Receiving regularly scheduled RBC transfusion therapy (also termed chronic, prophylactic, or preventative transfusion); episodic transfusion in response to worsened anemia or vaso-occlusive crisis (VOC) is permitted.
- Hospitalized for an SCPC and/or other vaso-occlusive event within 14 days prior to providing informed assent/consent or during the Screening Period.
- Currently receiving treatment with a disease-modifying therapy for SCD (eg, voxelotor, crizanlizumab, L-glutamine), with the exception of hydroxyurea.
- History of any malignancy except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.
- 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 people and ≥450 milliseconds for male people, except for right or left bundle branch block.
- Severe pulmonary fibrosis as defined by severe hypoxia, evidence of right-sided heart failure, and radiographic pulmonary fibrosis \>50%.
- Severe pulmonary hypertension as defined by severe symptoms associated with hypoxia, right heart failure, and oxygen indicated.
- Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.
- Clinically symptomatic cholelithiasis or cholecystitis (people with prior cholecystectomy are eligible).
- History of drug-induced cholestatic hepatitis.
- Aspartate aminotransferase (AST) \>2.5× ULN (unless due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase (ALT) \>2.5× ULN (unless due to hepatic iron deposition).
- Renal dysfunction as defined by an estimated glomerular filtration rate \<30 milliliters per minute (mL/min)/1.73-meter square (m\^2) by the Chronic Kidney Disease Epidemiology Collaboration creatinine equation.
- Active uncontrolled infection requiring systemic antimicrobial therapy.
- Positive test for hepatitis C virus (HCV) antibody (Ab) with evidence of active HCV infection, or positive test for hepatitis B surface antigen (HBsAg).
- Positive test for human immunodeficiency virus (HIV)-1 antibody or HIV-2 antibody.
- History of major surgery (including splenectomy) ≤16 weeks before providing informed assent/consent and/or planning on undergoing a major surgical procedure during the study.
- Current enrollment or past participation (within 90 days before randomization or a time frame equivalent to 5 half-lives of the investigational study drug, whichever is longer) in any other clinical study involving an...
- Past enrollment in a clinical study involving mitapivat.
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation.
- Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered at least 90 days before randomization.
- Receiving products that are strong inhibitors of Cytochrome3A4/5 (CYP3A4/5) that have not been stopped for ≥5 days or a time frame equivalent to 5 half-lives (whichever is longer), or strong inducers of Cytochrome3A4...
- Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed
- Known allergy to mitapivat or tablet excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry Blue II film-coat [hypromellose, titanium...
- people unable to receive RBC transfusions (eg, due to presence of allo-antibodies, lack of blood availability).
- people deprived of liberty by court or administrative decision (eg, persons accommodated in an institution by order of an authority or court).
- people undergoing psychiatric care without their consent.
- people admitted to a health or social establishment for purposes other than research.
- Adult people subject to a legal protection measure (guardian, curatorship, legal protection).
- people unable to express their consent.
- Receiving herbal or dietary supplements that have not been stable in dose and preparation for ≥8 weeks prior to randomization.
The study team makes the final eligibility decision.
Where it is enrolling
- Indianapolis, Indiana, United States
- Grand Rapids, Michigan, United States
- Flowood, Mississippi, United States
- New York, New York, United States
- Houston, Texas, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Indianapolis, Indiana, United States; Grand Rapids, Michigan, United States; Flowood, Mississippi, United States; New York, New York, United States; Houston, Texas, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.