Etavopivat for Sickle Cell Disease in Children

A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease

Recruiting Phase 2 Interventional Study
Sickle Cell Disease
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
6 Months – 18
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
95 (estimated)
Sponsor
Forma Therapeutics, Inc. · Industry
Who this trial is looking for

This trial is looking for children and adolescents with sickle cell disease. Participants will take a drug called etavopivat for about 24 weeks, followed by a 72-week extended treatment period to check how safe it is and how the body processes it.

Are You a Good Fit for This Trial?

You may be able to join if

  • I am between 6 months and 18 years old.
  • I have been diagnosed with sickle cell disease.
  • My hemoglobin level is between 5.5 and 10.5 grams per deciliter.
  • I have had severe complications from sickle cell disease.
  • I have consent from my parent or guardian.
  • I can give age-appropriate assent to participate.
  • I have been stable on hydroxyurea for the past 90 days, if applicable.
  • I agree to use contraception if I could become pregnant or father a child.

You may not be able to join if

  • I am pregnant or breastfeeding.
  • I have had more than 15 vaso-occlusive events in the past 12 months.
  • I was hospitalized for a sickle cell crisis in the last 14 days.
  • I have had an abnormal transcranial Doppler test in the past 12 months.
  • I receive regular blood transfusions.
  • I received any blood products in the last 30 days.
  • I am taking medications that speed up certain liver enzymes within the last 2 weeks.
  • I have had a cell-based therapy in the past.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial
The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD). Participants will receive etavopivat and will be enrolled in a staggered manner, starting with the oldest age group and followed sequentially by younger cohorts after review of pharmacokinetic and safety data from the preceding cohort. All participants w…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Type of Participant and Disease Characteristics 1. Patient's parent, legal guardian, or legal representative has provided documented informed consent and patients have provided age-appropriate assent 2. Age greater than or equal to (≥) 6 months and lesser than (\<) 18 yea…
Contacts

Novo Nordisk

(+1) 866-867-7178

clinicaltrials@novonordisk.com

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