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Tests treatment safety and results for Anemia
Official title To Assess Safety of Mitapivat and Provide Proof of Concept of the Efficacy of the Drug in Patients With RBC Membranopathies or CDAII.
ClinicalTrials.gov ID: NCT07055243
What this study is testing
What is Mitapivat?
Mitapivat is an investigational medicine, being studied as a potential treatment for anemia.
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 is a prospective exploratory phase 2 study designed to evaluate the safety and efficacy of mitapivat in RBC membranopathies and CDAII, a rare sub type of anemia. Nine patients from Princess Margaret who are diagnosed with CDAII will be enrolled to the study.
- Phase 2: a mid-size study of how well it works
- 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 18 and older
You may be able to join if
- Male or female with RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII). Diagnosis must be supported genetically by a ACMG class...
- Age ≥18 years.
- Average hemoglobin (Hb) concentration (average of at least 2 Hb measurements separated by a minimum of 7 days the during screening period) must be...
- Splenomegaly (length ≥12.5 cm)
- Fatigue attributed to hemolysis
You likely can't join if
- Known history of pyruvate kinase deficiency (decreased PK activity or two pathogenic PKLR alleles). PK activity and PKLR testing is not required.
- Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic or preventive transfusion), defined as more than 5...
- Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound interpretation of...
- Poorly controlled hypertension (defined as systolic blood pressure \>150 mm Hg or diastolic blood pressure \>90 mm Hg) refractory to medical...
- Any history of congestive heart failure; myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; or recent (\<...
- Cardiac dysrhythmias judged as clinically significant by the Investigator.
See the full eligibility criteria
- Male or female with RBC membranopathy or congenital dyserythropoietic anemia type II (CDAII). Diagnosis must be supported genetically by a ACMG class 3 (VUS), 4 or 5 variant.
- Age ≥18 years.
- Average hemoglobin (Hb) concentration (average of at least 2 Hb measurements separated by a minimum of 7 days the during screening period) must be less than 13.0 g/dL for males and 11.0 g/dL for females. Patients with...
- Splenomegaly (length ≥12.5 cm)
- Fatigue attributed to hemolysis
- Active hemolysis as evaluated by one or more of the following: haptoglobin, bilirubin, LDH, reticulocytes
- people must start or continue taking at least the equivalent of daily 0.8 mg oral folic acid for the duration of the study.
- Have adequate organ function, as defined by:
- Serum aspartate aminotransferase (AST) ≤2.5 × ULN (unless the increased AST is assessed by the Investigator as due to hemolysis and/or hepatic iron deposition) and alanine aminotransferase (ALT) ≤2.5 × ULN.
- Normal or elevated levels of serum bilirubin. In people with serum bilirubin \> ULN, the elevation must be attributed to hemolysis with or without Gilbert's syndrome and must not be associated with choledocholithiasis...
- Estimated glomerular filtration rate ≥45 mL/min/1.73 m2 by Chronic Kidney Disease Epidemiology Collaboration creatinine
- Be willing and able to give written informed consent and to comply to all study procedures for the duration of the study.
- For women of reproductive potential, have a negative urine or serum pregnancy test during the Screening Period (Day -50 to Day -1). Women of reproductive potential are defined as sexually mature women who have not...
- For women of reproductive potential, be abstinent as part of their usual lifestyle, or agree to use a highly effective method of contraception. A highly effective form of contraception is defined as combined (estrogen...
- Known history of pyruvate kinase deficiency (decreased PK activity or two pathogenic PKLR alleles). PK activity and PKLR testing is not required.
- Receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic or preventive transfusion), defined as more than 5 transfusion episodes in the 12-month period up to the first day of...
- Have a significant medical condition that confers an unacceptable risk to participating in the study, and/or that could confound interpretation of the study data. Such significant medical conditions include, but are not...
- Poorly controlled hypertension (defined as systolic blood pressure \>150 mm Hg or diastolic blood pressure \>90 mm Hg) refractory to medical management.
- Any history of congestive heart failure; myocardial infarction or unstable angina pectoris; hemorrhagic, embolic, or thrombotic stroke; or recent (\< 6 months prior to signing informed consent) deep venous thrombosis...
- Cardiac dysrhythmias judged as clinically significant by the Investigator.
- Clinically symptomatic cholelithiasis or cholecystitis. Prior cholecystectomy is not exclusionary. people with symptomatic cholelithiasis or cholecystitis may be rescreened once the disorder has been treated and...
- History of drug-induced cholestatic hepatitis.
- Severe iron overload as evaluated by the Investigator. This includes cardiac (eg, clinically significant impaired left ventricular ejection fraction) or hepatic (eg, fibrosis, cirrhosis) dysfunction.
- Have a diagnosis of any other congenital or acquired blood disorder or any other hemolytic process, except mild allo-immunization, as a consequence of transfusion therapy. g. Positive test for HBsAg or HCVAb with signs...
The study team makes the final eligibility decision.
Where it's taking place
- Toronto, Ontario, Canada
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 - 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, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Toronto, Ontario, Canada. 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.