Trial studying safety and effects for Non-Transfusion-dependent Alpha-Thalassemia
Official title A Study to Investigate the Efficacy, Pharmacokinetics, and Safety of Mitapivat in Pediatric Participants With α- or β-Non-Transfusion-Dependent Thalassemia
ClinicalTrials.gov ID: NCT07517133
What this study is testing
What is Mitapivat?
Mitapivat is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for non-transfusion-dependent alpha-thalassemia.
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
- The primary objective of this study is to compare the effect of mitapivat versus placebo on anemia in pediatric participants with alpha- or beta-non-transfusion-dependent thalassemia.
- 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 1 to 17
You may be able to join if
- Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained...
- Aged 1 to \<18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent.
- Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or...
- Hb concentration ≤10.0 grams per deciliter (g/dL) [100.0 grams per liter (g/L)], based on an average of at least 2 Hb concentration measurements...
- Non-transfusion dependent, defined as ≤5 transfusion episodes (also referred to as "transfusion events") during the 24-week period before...
You likely can't join if
- Pregnant or breastfeeding.
- Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.
- Any conditions other than thalassemia expected to affect sexual maturation.
- Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization.
- Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization.
See the full eligibility criteria
- Written informed consent/assent from the participant (or their legally authorized representative, parent(s), or legal guardian) must be obtained before any study-related procedures are conducted and participants must be...
- Aged 1 to \<18 years and weighing at least 7 kilograms (kg) at the time of providing informed consent/assent.
- Documented diagnosis of thalassemia (β-thalassemia with or without α-globin gene mutations, hemoglobin E (HbE)/β-thalassemia, or α-thalassemia/hemoglobin H (HbH) disease) based on Hemoglobin (Hb) electrophoresis, Hb...
- Hb concentration ≤10.0 grams per deciliter (g/dL) [100.0 grams per liter (g/L)], based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the Screening Period.
- Non-transfusion dependent, defined as ≤5 transfusion episodes (also referred to as "transfusion events") during the 24-week period before randomization and no red blood cells (RBC) transfusions ≤8 weeks before providing...
- If taking hydroxyurea, the hydroxyurea dose must be stable for ≥16 weeks before randomization.
- Female participants who have attained menarche must be abstinent of sexual activities that may induce pregnancy as part of their usual lifestyle, or agree to use 2 forms of contraception, 1 of which must be considered...
- Pregnant or breastfeeding.
- Documented history of homozygous or heterozygous hemoglobin S (HbS) or hemoglobin C (HbC).
- Prior exposure to gene therapy or prior bone marrow or stem cell transplantation, including any prior exposure to myeloablative chemotherapy.
- Any conditions other than thalassemia expected to affect sexual maturation.
- Currently receiving treatment with luspatercept; the last dose must have been administered ≥18 weeks before randomization.
- Currently receiving treatment with hematopoietic stimulating agents; the last dose must have been administered ≥18 weeks before randomization.
- History of malignancy (active or treated) ≤5 years before providing informed consent/assent, except for nonmelanomatous skin cancer in situ, cervical carcinoma in situ, or breast carcinoma in situ.
- History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent.
- Hepatobiliary disorders, including but not limited to:
- Liver disease with histopathological evidence or clinical diagnosis of cirrhosis or severe fibrosis.
- Clinically symptomatic cholelithiasis or cholecystitis (prior cholecystectomy is not exclusionary).
- History of drug-induced cholestatic hepatitis.
- Aspartate Aminotransferase (AST) \>2.5\ Upper Limit of Normal (ULN) (unless due to hemolysis and hepatic iron deposition) and Alanine Transaminase (ALT) \>2.5\ ULN (unless due to hepatic iron deposition).
- Renal dysfunction as defined by an estimated glomerular filtration rate \<60 milliliters per minute (mL/min)/1.73-meter square (m\^2).
- Nonfasting triglycerides \>215 milligrams per deciliter (mg/dL) [5 millimole per liter (mmol/L)].
- Active infection requiring systemic antimicrobial therapy at the time of providing informed consent/assent. If antimicrobial therapy is required during the Screening Period, screening procedures should not be performed...
- Participants with known active hepatitis B or hepatitis C virus infection.
- Participants with known human immunodeficiency virus (HIV) infection.
- History of major surgery (including splenectomy) ≤16 weeks before providing informed consent/assent and/or a major surgical procedure planned during the study.
- Current enrollment or past participation (within ≤12 weeks or a timeframe equivalent to 5 half-lives of the investigational study drug before administration of the first dose of study drug, whichever is longer) in any...
- Receiving strong Cytochrome3A4/5 (CYP3A4/5) inhibitors that have not been stopped for ≥5 days or a timeframe equivalent to 5 half-lives (whichever is longer), or strong CYP3A4/5 inducers that have not been stopped for...
- Receiving anabolic steroids that have not been stopped for at least 4 weeks before randomization. Testosterone replacement therapy to treat hypogonadism is allowed; the testosterone dose and preparation must be stable...
- Known allergy, or other contraindication, to mitapivat or its excipients (microcrystalline cellulose, croscarmellose sodium, sodium stearyl fumarate, mannitol, magnesium stearate, and the Opadry filmcoat [hypromellose...
- Any medical, hematological, 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...
- 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...
The study team makes the final eligibility decision.
Where it's taking place
- Palo Alto, California, United States
- New York, New York, United States
- Philadelphia, Pennsylvania, United States
- Dallas, Texas, United States
Compensation & support
This study's listing mentions compensation or reimbursement.
No amount is published here - ClinicalTrials.gov has no reliable field for payment or travel support. Ask the study team what's actually covered (payment for your time, travel, parking, or other study costs), and never join a trial for the compensation alone.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
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 - 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, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Palo Alto, California, United States; New York, New York, United States; Philadelphia, Pennsylvania, United States; Dallas, Texas, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.