Ongoing Clinical Trials for Red Blood Cell Abnormality
There is currently 1 ongoing clinical trial investigating new treatments for red blood cell abnormality. This trial is evaluating the safety and effectiveness of mitapivat, a medication designed to improve red blood cell function in adults with red blood cell membrane disorders. The trial is being conducted in Denmark and the Netherlands.
Clinical trial locations
- Denmark
- Netherlands
Study on the Safety of Mitapivat for Adults with Red Blood Cell Membrane Disorders
This trial is evaluating mitapivat, also known by its code name AG-348, for adults with red blood cell membrane disorders. The medication comes in the form of film-coated tablets available in 5 mg, 20 mg, and 50 mg doses. Mitapivat works by activating an enzyme called pyruvate kinase, which helps red blood cells produce energy more effectively.
Main inclusion criteria:
- Adults aged 18 years or older with a diagnosis of red blood cell membrane disorder or congenital dyserythropoietic anemia type II, confirmed by genetic testing
- Low hemoglobin levels: below 13.0 g/dL for men and below 11.0 g/dL for women
- If hemoglobin is above 10.0 g/dL, participants must also have an enlarged spleen measuring 12.5 cm or more, fatigue due to red blood cell breakdown, or active signs of red blood cell destruction on laboratory tests
- Adequate kidney and liver function based on specific laboratory values
- Willingness to take at least 0.8 mg of folic acid daily during the study
- Women of childbearing potential must have a negative pregnancy test and agree to use effective birth control methods
Main exclusion criteria:
- Individuals with red blood cell membrane disorders cannot participate in the trial
Focus and goals:
The primary goal of this study is to evaluate the safety of mitapivat in patients with red blood cell membrane disorders. The trial will monitor participants for any side effects or health changes that occur while taking the medication. The study involves a maximum treatment period of 56 days and includes regular check-ups with blood tests, physical examinations, ECGs, and DEXA scans.
Participants will go through several stages. After initial screening and assessment to confirm eligibility, they will begin taking mitapivat at assigned doses. The study includes two fixed dose periods where the dosage may be adjusted based on individual response. Throughout the trial, researchers will closely monitor hemoglobin levels and other health indicators to assess how well patients tolerate the medication and its potential benefits.
Some participants may receive a placebo, a substance with no active medication, to allow researchers to compare the effects. Regular monitoring helps determine whether mitapivat is a safe and potentially effective treatment option for people with these red blood cell membrane conditions.
Summary
Currently, there is one active clinical trial investigating treatments for red blood cell abnormality. This trial is being conducted in two European countries: Denmark and the Netherlands. The study focuses exclusively on mitapivat, a pyruvate kinase activator designed to improve red blood cell function by enhancing energy production within the cells.
The trial specifically targets adults with red blood cell membrane disorders and congenital dyserythropoietic anemia type II. The main emphasis is on evaluating the safety profile of mitapivat rather than primarily measuring effectiveness, though health indicators such as hemoglobin levels will be monitored throughout the study. This represents an important step in understanding whether mitapivat could become a viable treatment option for patients living with these challenging conditions.