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Allosteric Activator

Mitapivat for Pyruvate Kinase Deficiency (ACTIVATE-KidsT Trial)

Phase 3
Waitlist Available
Research Sponsored by Agios Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical laboratory confirmation of pyruvate kinase deficiency (PKD), defined as documented presence of at least 2 mutant alleles in the pyruvate kinase L/R (PKLR) gene, of which at least 1 is a missense mutation, as determined per the genotyping performed by the study central genotyping laboratory
Aged 1 to <18 years. Participants between 12 and 24 months of age must weigh a minimum of 7 kilograms (kg)
Must not have
History of active and/or uncontrolled cardiac or pulmonary disease or clinically relevant QT prolongation within 6 months before providing informed consent/assent
Homozygous for the R479H mutation or have 2 nonmissense mutations, without the presence of another missense mutation, in the PKLR gene as determined per the genotyping performed by the study central genotyping laboratory
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 9 to week 32
Awards & highlights

Summary

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.

Who is the study for?
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.
What is being tested?
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.
What are the potential side effects?
Possible side effects of Mitapivat include but are not limited to allergic reactions to the drug's ingredients, potential liver enzyme elevations due to interaction with other medications, and any general risks associated with taking new medication under clinical trial conditions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have a confirmed genetic condition called pyruvate kinase deficiency.
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I am between 1 and 18 years old. If I am 1-2 years old, I weigh at least 7 kg.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I haven't had serious heart or lung problems or abnormal heart rhythms in the last 6 months.
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My genetic test shows I have specific mutations in the PKLR gene.
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I do not have severe liver issues, uncontrolled infections, recent major surgery, or HIV/Hepatitis B/C.
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I have had cancer before.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 9 to week 32
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 9 to week 32 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Percentage of Participants Achieving Transfusion Reduction Response (TRR)
Secondary study objectives
Change From Baseline in Sexual Maturity Rating with Tanner Stage
Change from Baseline in Pediatric Quality of Life (PedsQL) Multidimensional Fatigue Scale
Change from Baseline in PedsQL Generic Core Scale (GCS)
+4 more

Trial Design

3Treatment groups
Experimental Treatment
Placebo Group
Group I: Mitapivat (OLE period)Experimental Treatment2 Interventions
Participants who have completed the double-blind period will be eligible to receive mitapivat for up to 5 years in the OLE period. Participants entering the OLE period will first receive blinded mitapivat and placebo for 8 weeks to maintain the double-blind treatment assignment before being transitioned to only receive active, open-label drug (mitapivat).
Group II: MitapivatExperimental Treatment1 Intervention
Double-Blind Period: Participants will receive mitapivat orally, at doses based on age and weight, for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.
Group III: PlaceboPlacebo Group1 Intervention
Double-Blind Period: Participants will receive mitapivat-matching placebo orally for 8 weeks in the dose titration period and for 24 weeks in the fixed-dose period.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Mitapivat
2023
Completed Phase 1
~20

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatment for Pyruvate Kinase Deficiency (PKD) involves the activation of the pyruvate kinase enzyme, which is crucial for the glycolytic pathway in red blood cells. Mitapivat, a pyruvate kinase activator, works by increasing the activity of the defective enzyme, thereby improving ATP production and reducing the accumulation of upstream glycolytic intermediates. This is significant for PKD patients as it helps to alleviate hemolytic anemia, decrease the need for regular blood transfusions, and improve overall quality of life by stabilizing red blood cell function and lifespan.

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
52 Previous Clinical Trials
4,123 Total Patients Enrolled
10 Trials studying Pyruvate Kinase Deficiency
1,137 Patients Enrolled for Pyruvate Kinase Deficiency
Medical AffairsStudy ChairAgios Pharmaceuticals, Inc.
39 Previous Clinical Trials
8,532 Total Patients Enrolled
5 Trials studying Pyruvate Kinase Deficiency
225 Patients Enrolled for Pyruvate Kinase Deficiency

Media Library

Mitapivat (Allosteric Activator) Clinical Trial Eligibility Overview. Trial Name: NCT05144256 — Phase 3
Pyruvate Kinase Deficiency Research Study Groups: Mitapivat, Placebo, Mitapivat (OLE period)
Pyruvate Kinase Deficiency Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT05144256 — Phase 3
Mitapivat (Allosteric Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05144256 — Phase 3
~15 spots leftby Oct 2025