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Activator

Mitapivat for Pyruvate Kinase Deficiency (ACTIVATE-Kids Trial)

Phase 3
Recruiting
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
Hemoglobin concentration ≤10 grams per deciliter (g/dL) for participants 12 to <18 years of age or ≤9 g/dL for participants 1 to <12 years of age during the screening period. Hb concentration must be based on an average of at least 2 Hb concentration measurements (separated by ≥7 days) collected during the screening period
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 8: ≤30 minutes predose; week 12: ≤30 minutes predose
Awards & highlights

ACTIVATE-Kids Trial Summary

This trial will evaluate the efficacy and safety of a drug called mitapivat in children with a condition called pyruvate kinase deficiency. Children will be given either the drug or a placebo, and researchers will track how well they do.

Who is the study for?
This trial is for children and teenagers aged 1 to less than 18 with Pyruvate Kinase Deficiency (PKD) who aren't getting regular blood transfusions. They must have a certain level of hemoglobin, not had many blood transfusions recently, be on folic acid supplements, and agree to use contraception if applicable. Those with severe liver or heart issues, recent major surgery like splenectomy, or specific genetic mutations are excluded.Check my eligibility
What is being tested?
The study tests the oral drug Mitapivat against a placebo in young patients with PKD. Participants will randomly receive either the drug or placebo in a ratio of 2:1 and will be grouped by age for dosing. The trial includes an initial dose adjustment phase followed by a fixed-dose period before moving into a long-term extension phase lasting up to five years.See study design
What are the potential side effects?
While specific side effects for Mitapivat in this pediatric population are not detailed here, common side effects may include digestive discomforts such as nausea or diarrhea, headaches, tiredness, potential liver enzyme changes which would be monitored closely during the trial.

ACTIVATE-Kids Trial 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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My average hemoglobin level is low according to my age group's requirement.
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I have had 5 or fewer blood transfusions in the last year and none in the past 3 months.
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I am between 1 and 18 years old. If I'm under 2, I weigh at least 7 kg.

ACTIVATE-Kids 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 a Hemoglobin (Hb) Response
Secondary outcome measures
Average Change From Baseline in Hb Concentration at Weeks 12, 16, and 20
Average Change From Baseline in Indirect Bilirubin Concentration at Weeks 12, 16, and 20
Average Change From Baseline in Lactose Dehydrogenase (LDH) Concentration at Weeks 12, 16, and 20
+25 more

ACTIVATE-Kids 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 12 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 12 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
4,176 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
8,530 Total Patients Enrolled
5 Trials studying Pyruvate Kinase Deficiency
225 Patients Enrolled for Pyruvate Kinase Deficiency

Media Library

Mitapivat (Activator) Clinical Trial Eligibility Overview. Trial Name: NCT05175105 — Phase 3
Pyruvate Kinase Deficiency Research Study Groups: Mitapivat (OLE period), Placebo, Mitapivat
Pyruvate Kinase Deficiency Clinical Trial 2023: Mitapivat Highlights & Side Effects. Trial Name: NCT05175105 — Phase 3
Mitapivat (Activator) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05175105 — Phase 3

Frequently Asked Questions

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

Are there any negative side effects associated with taking Mitapivat?

"Mitapivat is considered safe and received a score of 3. This is because, as a Phase 3 trial drug, there are both data supporting its efficacy and multiple rounds of safety testing."

Answered by AI

Could I take part in this medical research if I qualify?

"Eligible patients for this clinical trial must lack pyruvate kinase and be between 1 and 17 years old. Right now, the study is looking to recruit 30 individuals in total."

Answered by AI

Are any patients who are twenty years or older being accepted into this research study?

"The age requirement to participate in this study is between 1 and 17 years old."

Answered by AI

Are there other instances where Mitapivat has been used in medical research?

"Since 2015, when mitapivat was first studied at Stanford University, 12 clinical trials have completed. There are currently 9 live trials being conducted; many of these studies taking place in Durham, North carolina."

Answered by AI

How many total subjects are being enrolled in this experiment?

"That is accurate. The clinical trial, which was created on June 6th of this year, is still looking for enrollees. So far, 30 people have signed up at 2 different locations."

Answered by AI

Are there any other similar studies that have been conducted in the past?

"Agios Pharmaceuticals, Inc. first sponsored a clinical trial for mitapivat in 2015 which completed that same year. With 52 patients enrolled, this was the study that pushed mitapivat into Phase 2 drug approval. Currently, there are 9 active studies being conducted in 54 cities and across 28 countries."

Answered by AI

Are investigators still looking for more participants for this trial?

"That is correct. The online clinical trials database has the most recent information, which says that this study began on 6/6/2022 and is still looking for 30 participants at 2 sites."

Answered by AI

Who else is applying?

What site did they apply to?
UT Southwestern Medical Center
What portion of applicants met pre-screening criteria?
Did not meet criteria

Why did patients apply to this trial?

I am interested in seeing if I can increase my hemoglobin level.
PatientReceived 2+ prior treatments
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 Not 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 Not Regularly Transfused, Followed by a 5-Year Extension Period". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05175105.
~6 spots leftby Oct 2024
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