49 Participants Needed

Mitapivat for Pyruvate Kinase Deficiency

(ACTIVATE-KidsT Trial)

Recruiting at 22 trial locations
AM
Overseen ByAgios Medical Affairs
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Agios Pharmaceuticals, Inc.
Must be taking: Folic acid
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing mitapivat, a medication that may help children with a blood disorder called pyruvate kinase deficiency. These children often need frequent blood transfusions. Mitapivat aims to improve the function of their red blood cells, potentially reducing the need for these transfusions.

Will I have to stop taking my current medications?

The trial requires that you stop taking certain medications before participating. Specifically, you must stop taking strong inhibitors or inducers of CYP3A4, anabolic steroids, and any pyruvate kinase activators for a specified period before joining the study. You must also continue taking folic acid supplements during the study.

Is Mitapivat safe for humans?

Mitapivat has been generally well tolerated in clinical trials for pyruvate kinase deficiency, with most side effects being mild to moderate. It is approved for use in adults with this condition in the USA and Europe, indicating a favorable safety profile.12345

What makes the drug Mitapivat unique for treating pyruvate kinase deficiency?

Mitapivat is unique because it is the first approved drug that directly targets the underlying cause of pyruvate kinase deficiency by activating the pyruvate kinase enzyme in red blood cells, improving hemoglobin levels and reducing the need for blood transfusions.12345

What data supports the effectiveness of the drug Mitapivat for treating pyruvate kinase deficiency?

Mitapivat has been shown to improve hemoglobin levels and reduce the need for blood transfusions in adults with pyruvate kinase deficiency. It also enhances quality of life and reduces symptoms of hemolytic anemia, making it a valuable treatment option for this condition.12456

Who Is on the Research Team?

MA

Medical Affairs

Principal Investigator

Agios Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for kids with Pyruvate Kinase Deficiency who need regular blood transfusions. They must be between 1-18 years old, not pregnant or breastfeeding, and without serious heart, liver, or kidney issues. Participants should have a history of receiving multiple transfusions and agree to use contraception if applicable.

Inclusion Criteria

Have complete records of transfusion history for the 52 weeks before providing informed consent/assent, defined as having all the following available: (1) all the transfusion dates, (2) the RBC transfusion volume (milliliters and/or number of units) for all the transfusions, and (3) hemoglobin concentrations within 1 week before transfusion for at least 80% of the transfusions
I have been taking folic acid for at least 21 days before starting the study drug and will continue during the study.
I have a confirmed genetic condition called pyruvate kinase deficiency.
See 3 more

Exclusion Criteria

I haven't had serious heart or lung problems or abnormal heart rhythms in the last 6 months.
Pregnant or breastfeeding
My genetic test shows I have specific mutations in the PKLR gene.
See 2 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Dose Titration

Participants undergo an 8-week dose titration period to optimize dosing based on age and weight

8 weeks

Fixed-dose Treatment

Participants receive a fixed dose of mitapivat or placebo for 24 weeks

24 weeks

Open-label Extension

Participants who complete the double-blind period may continue receiving mitapivat in an open-label extension period

Up to 262 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • Mitapivat
  • Mitapivat-matching placebo
Trial Overview The study tests Mitapivat against a placebo in children with PKD who get regular transfusions. It's randomized (2:1 ratio), considering age and splenectomy status. After an initial period of dose adjustments followed by a fixed-dose period, participants may continue treatment for up to five years.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: MitapivatExperimental Treatment2 Interventions
Group II: PlaceboPlacebo Group2 Interventions

Find a Clinic Near You

Who Is Running the Clinical Trial?

Agios Pharmaceuticals, Inc.

Lead Sponsor

Trials
55
Recruited
4,200+

Citations

Mitapivat in adult patients with pyruvate kinase deficiency receiving regular transfusions (ACTIVATE-T): a multicentre, open-label, single-arm, phase 3 trial. [2022]
Safety and Efficacy of Mitapivat in Pyruvate Kinase Deficiency. [2019]
Mitapivat versus Placebo for Pyruvate Kinase Deficiency. [2022]
Mitapivat: A Review in Pyruvate Kinase Deficiency in Adults. [2023]
Pyruvate kinase activators for treatment of pyruvate kinase deficiency. [2023]
6.United Arab Emiratespubmed.ncbi.nlm.nih.gov
Mitapivat: A Novel Treatment of Hemolytic Anemia in Adults with Pyruvate Kinase Deficiency. [2023]
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