Active not recruitingPHASE3INTERVENTIONAL

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

This study, called ACTIVATE-Kids, is looking into a new oral medicine called mitapivat for children and teenagers aged 1 to 17 who have a blood disorder called Pyruvate Kinase Deficiency (PKD). These children are specifically those who do not regularly need blood transfusions. The main goals are to see how well mitapivat works and if it's safe. Participants will either receive the active medicine or a 'dummy' medicine (placebo) for a short period, and then those who complete this part may continue on mitapivat for up to five years. The study aims to make daily life better for children with PKD by finding new treatment options.

At a glance

Status

Active not recruiting

Phase

PHASE3

Sponsor

Agios Pharmaceuticals, Inc.

Enrolment target

Start

06 Jun 2022

Estimated completion

01 Jan 2030

Pediatric Pyruvate Kinase Deficiency Pediatric Hemolytic Anemia

What is this study about?

This clinical trial is designed for children and teenagers with a rare genetic blood condition called Pyruvate Kinase Deficiency (PKD). In PKD, red blood cells break down too quickly, which can lead to anaemia (low red blood cell count). The goal of this study is to test a new medicine called mitapivat. We want to see if it can help improve the condition and reduce its symptoms.

Children in the study will be randomly assigned to receive either the active medicine, mitapivat, or a 'dummy' medicine called a placebo. This helps us fairly compare how well mitapivat works. The study also aims to understand any side effects the medicine might have, making sure it's safe for children to use. This kind of research is really important because it helps doctors find better ways to treat conditions like PKD.

Finding new treatments is crucial for children living with PKD, as it can significantly improve their energy levels, reduce the need for medical interventions, and ultimately enhance their quality of life. By taking part, families can contribute to medical knowledge and potentially help future patients with this condition.

Key takeaways

This study is for children and teenagers (1-17 years old) with Pyruvate Kinase Deficiency (PKD).
It's testing a new oral medicine, mitapivat, against a dummy medicine (placebo).
Participants should not regularly need blood transfusions for their PKD.
The study involves an initial 20-week period, followed by an optional 5-year treatment extension.
Close medical monitoring is provided throughout the study.
Participation helps advance understanding and potential treatments for PKD.

Who may be eligible?

This study is looking for children and teenagers aged between 1 and 17 years old. For the very youngest children (12 to less than 24 months), they need to weigh at least 7 kilograms (about 1 stone).

To be considered, your child must have a confirmed diagnosis of Pyruvate Kinase Deficiency (PKD) from a genetic test. They also shouldn't have needed many blood transfusions in the past year (no more than 5) and none in the last 12 weeks before starting the study medicine. Their haemoglobin levels (a measure of red blood cells) also need to be below a certain level. All participants also need to be taking folic acid supplements.

For girls who have started puberty, they must either avoid activities that could lead to pregnancy or agree to use two reliable forms of contraception throughout the study and for a short period afterwards.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Is my child between 1 and 17 years old?
Does my child have a confirmed genetic diagnosis of Pyruvate Kinase Deficiency (PKD)?
Has my child received 5 or fewer blood transfusions in the last year, and none in the last 12 weeks?
Is my child currently taking folic acid supplements?
For girls who have started puberty: Are we able to meet the contraception requirements?
Does my child meet the haemoglobin level requirements for their age?

Answer every question to see your result.

What does participation involve?

Taking part in this study involves a few steps. First, there's a 'double-blind' period. This means neither you, your child, nor the study team will know if your child is getting the active medicine (mitapivat) or a dummy medicine (placebo). This period starts with 8 weeks where the dose might be slowly adjusted, followed by 12 weeks on a fixed dose. Regular visits to the clinic will be needed during this time for checks and blood tests.

If your child completes this initial period, they may then be able to join an 'open-label extension'. In this part, all participants will receive mitapivat, and everyone will know what medicine they are taking. This extension period can last for up to 5 years, with ongoing clinic visits to monitor your child’s health and the effectiveness of the medicine. The total duration of participation will depend on whether your child joins the extension period.

Potential risks and benefits

Taking part in a study like this offers potential benefits, such as access to a new medicine that might improve your child's condition before it's widely available. Your child's health will also be closely monitored by medical professionals throughout the study. However, there are also potential risks, including possible side effects from the study medicine, which will be carefully explained to you. There's also a chance your child might receive the placebo, meaning they wouldn't get the active treatment during the first phase. It's really important to remember that you have the right to withdraw your child from the study at any time, for any reason, without it affecting their regular medical care.

Locations (19)

Stanford Medicine
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Palo Alto, United States
Children's Hospital Colorado
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Aurora, United States
Children's Healthcare of Atlanta - Emory
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Atlanta, United States
UChicago Medicine
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Chicago, United States
Boston Children's Hospital
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Boston, United States
Children's Hospital of Michigan
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Detroit, United States
Duke University Medical Center
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Durham, United States
Children's Hospital of Philadelphia
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Philadelphia, United States
St Jude's Children's Research Hospital
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Memphis, United States
UT Southwestern Medical Center
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Dallas, United States
Texas Children's Hospital
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Houston, United States
Centre hospitalier Universitaire de Sainte-Justine
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Montreal, Canada

Common questions

What is Pyruvate Kinase Deficiency (PKD)?

PKD is an inherited blood condition where red blood cells don't work properly and break down too quickly, leading to anaemia and tiredness.

What does 'non-regularly transfused' mean?

It means your child doesn't usually need frequent blood transfusions to manage their PKD.

What is a 'placebo'?

A placebo is a dummy medicine that looks just like the real medicine but contains no active drug. It helps us fairly compare the effects of the actual medicine.

How long will my child be in the study?

The first part of the study lasts about 20 weeks. If your child completes this, they may be able to continue receiving the study medicine for up to 5 more years.

Will we know if my child is getting the actual medicine?

For the first 20 weeks, neither you nor the doctors will know. If your child continues into the extension phase, everyone will know they are receiving the active medicine, mitapivat.