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Study of Mitapivat for Patients with Sickle Cell Disease and Kidney Disease

Verified siteInvestigationalNo placebo
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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying the effects of a medication called Mitapivat on patients with Sickle Cell Disease and Nephropathy, which is a type of kidney disease. Sickle Cell Disease is a genetic condition that affects the shape of red blood cells, leading to various health complications. The purpose of the study is to evaluate how Mitapivat affects the albumin creatinine ratio, a measure used to assess kidney function, in these patients.

Participants in the study will receive Mitapivat in the form of a tablet. The study will be conducted over a period of time, during which participants will take the medication and attend regular check-ups to monitor their health and the effects of the treatment. Some participants may receive a placebo instead of the actual medication. The study aims to observe changes in kidney function and other health indicators over the course of the treatment.

The trial will also track other health outcomes, such as the frequency of emergency room visits and hospitalizations, as well as any side effects experienced by participants. The goal is to determine whether Mitapivat can provide a beneficial effect on kidney function and overall health in patients with Sickle Cell Disease and Nephropathy.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

  1. Step 1

    Joining the study

    Upon joining the study, the patient will begin participation in a clinical trial focused on evaluating the effects of mitapivat on kidney function in individuals with sickle cell disease and kidney disease.

  2. Step 2

    Medication administration

    The patient will receive mitapivat in the form of a tablet, taken orally. The specific dosage and frequency will be determined by the study protocol and communicated to the patient by the study team.

  3. Step 3

    Monitoring and assessments

    Throughout the study, the patient will undergo regular monitoring to assess the impact of mitapivat on kidney function. This includes measuring the albumin creatinine ratio (ACR) to evaluate changes from the baseline to six months.

    Additional assessments will include changes in estimated glomerular filtration rate (eGFR) and the frequency of emergency room visits and hospitalizations.

  4. Step 4

    Duration of participation

    The study is expected to continue until June 2028. The patient will be required to comply with all study procedures for the duration of their participation.

  5. Step 5

    Completion of the study

    Upon completion of the study, the patient will have contributed valuable data regarding the effects of mitapivat on kidney function in individuals with sickle cell disease.

Who can join the trial?

10 criteria

  • Must be at least 16 years old to participate. In France, participants must be at least 18 years old.
  • Females must have started menstruating.
  • Must have a confirmed diagnosis of Sickle Cell Disease (SCD), specifically types HbSS or HbS/β0-thalassemia.
  • Hemoglobin levels must be between 5.5 and 10.5 grams per deciliter (g/dL) during the screening period. If multiple tests are done, the average must be within this range.
  • If taking hydroxyurea (a medication for SCD), the dose must have been stable for at least 90 days before the study starts, with no planned changes during the study. If stopping hydroxyurea, a 90-day break is needed before joining the study.
  • Two urine tests for albumin creatinine ratio (ACR) must be done during the screening period, both showing results between 100 and 2000 mg/g. One test can be from a regular clinic visit, and the other must be from a morning urine sample on a different day.
  • One ACR test result must be over 100 mg/g within 24 weeks before joining the study.
  • If taking ACE inhibitors or ARB therapy (medications for blood pressure or kidney protection), the dose must have been stable for at least 90 days before joining the study, with no planned changes during the study.
  • Women who can become pregnant must either not engage in sexual activities that could lead to pregnancy or agree to use two forms of birth control, one of which must be highly effective, from the time of joining the study until 28 days after the last dose of the study drug. The second form can be a barrier method like a condom.
  • Must provide written consent to participate in the study. For those under 18 or not legally adults, parental permission and child assent are required. Participants must agree to follow all study procedures for the entire duration of the study.

Who cannot join the trial?

4 criteria

  • Patients who do not have Sickle Cell Disease (SCD) and Nephropathy cannot participate. Sickle Cell Disease is a blood disorder, and Nephropathy refers to kidney problems.
  • Patients who are not within the specified age ranges cannot participate. The age ranges are typically defined by the study.
  • Patients who do not meet the gender requirements cannot participate. This study includes both male and female participants.
  • Patients who are considered part of a vulnerable population may not be eligible. Vulnerable populations can include groups like children, pregnant women, or those with certain disabilities.
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Investigated drugs

Mitapivat is a medication being studied for its potential effects on sickle cell disease and nephropathy. It is being tested to see if it can improve kidney function by affecting the albumin creatinine ratio, which is a measure of kidney health.

What is already known about the treatment

Mitapivat – Mitapivat is administered orally in tablet form. It is currently being studied in clinical trials for its potential benefits in treating sickle cell disease and nephropathy. The main therapeutic indication is to improve kidney function in patients with these conditions by evaluating its effect on the albumin creatinine ratio. At the molecular level, mitapivat works by activating an enzyme called pyruvate kinase, which plays a role in energy production in red blood cells. It is classified as a pyruvate kinase activator in pharmacology.

Investigated diseases

  • Sickle Cell Disease

    Sickle Cell Disease is a genetic blood disorder characterized by the production of abnormal hemoglobin, leading to the distortion of red blood cells into a sickle shape. These sickle-shaped cells can block blood flow, causing pain and potential organ damage. The disease often results in anemia, as the sickle cells break down more quickly than normal red blood cells. Over time, individuals may experience episodes of pain, known as sickle cell crises, and are at risk for infections and other complications. The severity and frequency of symptoms can vary widely among individuals.

  • Nephropathy

    Nephropathy refers to kidney damage or disease, which can result from various causes, including diabetes, high blood pressure, or genetic conditions. In nephropathy, the kidneys' ability to filter waste and excess fluids from the blood is impaired. This can lead to the accumulation of waste products in the body, causing symptoms such as swelling, fatigue, and high blood pressure. Over time, nephropathy can progress to chronic kidney disease or even kidney failure if not managed properly. The progression of nephropathy can vary depending on the underlying cause and individual health factors.

Trial detailsLast updated 7 Oct 2026
Age18+ yearsPhasePhase IITrial ID2023-510289-28-00Protocol codeAG348-C-026Estimated enrolment47 patientsSponsorAgios Pharmaceuticals Inc.

sourced from the EU Clinical Trials Register and site verification

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