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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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 8: ≤30 minutes predose; week 12: ≤30 minutes predose
Awards & highlights

ACTIVATE-KidsT Trial Summary

This trial is designed to study the efficacy and safety of a drug called mitapivat in children with pyruvate kinase deficiency who regularly receive blood transfusions. Children will be given either mitapivat or a placebo, and the effects will be monitored over a period of 32 weeks. Those who complete the trial will be eligible to receive the drug for up to 5 years in an extension period.

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.Check my eligibility
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.See study design
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.

ACTIVATE-KidsT Trial Eligibility Criteria

Inclusion Criteria

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

ACTIVATE-KidsT Trial Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~week 8: ≤30 minutes predose; week 12: ≤30 minutes predose
This trial's timeline: 3 weeks for screening, Varies for treatment, and week 8: ≤30 minutes predose; week 12: ≤30 minutes predose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Percentage of Participants Achieving Transfusion Reduction Response (TRR)
Secondary outcome measures
Change From Baseline in Body Mass Index (BMI)-for-age Z-score
Change From Baseline in Bone Mineral Density (BMD) Z-score
Change From Baseline in Estradiol Concentration
+23 more

ACTIVATE-KidsT 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

Find a Location

Who is running the clinical trial?

Agios Pharmaceuticals, Inc.Lead Sponsor
52 Previous Clinical Trials
4,157 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
8,511 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

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the projected or estimated enrollment for this research project?

"Yes, that is correct. The listing for this trial on clinicaltrials.gov says that it is recruiting patients. This particular trial was first posted on 6/8/2022 and was last updated on 10/19/2022. They are looking for 45 patients total from 3 different medical centers."

Answered by AI

Who would be an ideal candidate to help with this medical research?

"This clinical trial requires 45 participants that have anemia and hemolytic. These individuals must also be between 1 and 17 years of age."

Answered by AI

Does age restrict participation in this experiment?

"This particular study is only recruiting pediatric patients that are between 1 and 17 years old."

Answered by AI

Are there any open positions in this research project?

"The listed information on clinicaltrials.gov suggests that this study is looking for participants. The trial was first announced on June 8th, 2022 and updated on October 19th of the same year."

Answered by AI

Could you please explain what prior research has been done on Mitapivat?

"The first study involving mitapivat was conducted in 2015 at Stanford University. So far, 12 studies have been completed while 9 are ongoing. Many of these active trials are based in Las Vegas, Nevada."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period
2022. "A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05144256.
~0 spots leftby May 2024
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