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New daily pill could ease sickle cell pain for kids

NCT ID NCT06198712

Disease control Sponsor: Forma Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Sep 18, 2026 · Updated 3 times

Summary

This study tests a once-daily pill called etavopivat in children (6 months to 18 years) with sickle cell disease. The main goals are to check safety and how the drug works in the body, and to see if it can raise hemoglobin levels and reduce painful crises. About 95 children will take the pill every day for 96 weeks, with follow-up visits to track results.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 95 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jan 2023

Expected to finish

Aug 2029

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 months to 18 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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 years of age at time of enrollment, according to the enrolling cohort: * Cohort 1: age 12 to \< 18 years (adolescents) * Cohort 2: age 6 to \< 12 years * Cohort 3: age 2 to \< 6 years * Cohort 4: age 6 months to \< 2 years 3. Patient has confirmed diagnosis of SCD • Documentation of SCD genotype (HbSS, HbSβ0-thalassemia or other sickle cell syndrome variants) based on prior history of laboratory testing. Molecular genotyping is not required. SCD genotype may be determined from the results of Hb electrophoresis, high-performance liquid chromatography (HPLC), or similar testing. Note that Hb electrophoresis is performed by the local laboratory at Screening. 4. Hemoglobin ≥ 5.5 and lesser than or equal to (≤) 10.5 grams per deciliter (g/dL) 5. Pediatric patients with severe SCD, as defined by at least 1 of the following: * 2-15 episodes of documented VOC within the 12 months prior to screening. Documentation must exist in the patient's medical record prior to screening. Events based solely on patient recall without supporting documentation should not be counted towards eligibility. * Hospitalization for any SCD-related complication in the last 12 months prior to starting study treatment * Proteinuria, defined as an albumin:creatinine ratio (ACR) \> 100 mg/g on 2 measures (separated by ≥ 1 month) as an indicator of early renal disease * History of a conditional TCD in the last 12 months prior to starting study treatment, but not currently being treated with chronic transfusion therapy (applicable to participants \> 2 years of age). Conditional TCD is defined as a TAMMV of 170-199 cm/s by TCD or 155-184 cm/s by imaging TCD (TCDi). 6. For participants taking hydroxyurea (HU), the dose of HU (mg/kg) must be stable (no more than a 20% change in dosing) for at least 90 days prior to start of study treatment with no anticipated need for dose adjustments during the study, in the opinion of the Investigator 7. Patients on crizanlizumab or L-glutamine treatment at the time of consent may be eligible if they: * Have been on a stable dose for ≥ 12 months at the time of consent (ie, no changes to the dose except for changes to weight or for safety reasons) * For patients on crizanlizumab, have been ≥ 80% compliant with the planned regimen during the 12 months prior to the time of consent 8. Female patients of childbearing potential who are using acceptable methods of contraception and agree not to donate ova from study start to 90 days after the last dose of study drug, and male patients who are willing to use acceptable methods of contraception and agree not to donate sperm, from study start to 90 days after the last dose of study drug. Exclusion Criteria: * Medical Conditions 1. Female who is breastfeeding or pregnant 2. More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room (ER), or clinic visit 3. Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study treatment 4. Abnormal TCD in the 12 months prior to starting study treatment Prior/Concomitant Therapy 5. Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventive transfusion) 6. Received any blood products within 30 days of starting study treatment 7. Receiving or use of concomitant medications that are strong inducers of cytochrome P450 (CYP) 3A4/5 within 2 weeks of starting study treatment 8. Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study 9. Receipt of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study 10. Receipt of prior cellular based therapy (eg, hematopoietic cell transplant, gene modification therapy)

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    15 sites in 5 countries. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • APHP - Centre de Référence des Syndromes

    NOT_YET_RECRUITING

    Paris, 75019, France

  • Acıbadem Adana Hastanesi

    COMPLETED

    Seyhan, 1130, Turkey (Türkiye)

  • Ahero Clinical Trials Unit

    RECRUITING

    Kisumu, 40101, Kenya

  • American University of Beirut Medical center

    RECRUITING

    Beirut, 1107 2020, Lebanon

  • Aminu Kano Teaching Hospital (AKTH)

    RECRUITING

    Tarauni, 700101, Nigeria

  • Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle

    NOT_YET_RECRUITING

    Roeun, 76031, France

  • Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital

    RECRUITING

    London, SE1 7EH, United Kingdom

  • HCL-HOPITAL LYON SUD_Institut d'Hématologie et d'Oncologie Pédiatrique (IHOPe)

    NOT_YET_RECRUITING

    Lyon, 69008, France

  • Hacettepe University pediatric hematology

    COMPLETED

    Ankara, 06100, Turkey (Türkiye)

  • Hospital Nini

    RECRUITING

    Tripoli, 113-6044, Lebanon

  • KEMRI CRDR Siaya Clinical Research Annex, Country Referral Hospital

    RECRUITING

    Siaya, Siaya County, 40600, Kenya

  • KEMRI-Walter-Reed Kericho

    RECRUITING

    Kericho, 20200, Kenya

  • King's College Hospital - Alex Mowat Research Hub

    RECRUITING

    London, SE5 9RS, United Kingdom

  • Kombewa Clinical Research Centre

    RECRUITING

    Kisumu, 40100, Kenya

  • Lagos University Teaching Hospital, Lagos

    RECRUITING

    Lagos, 102215, Nigeria

  • Manchester Royal Infirmary_1

    RECRUITING

    Manchester, M13 9WL, United Kingdom

  • The Hospital for Sick Children

    COMPLETED

    Toronto, Ontario, M5G 1X8, Canada

  • University of Nigeria Teaching Hospital (UNTH)

    RECRUITING

    Ituku-Ozalla, Enugu State, 400001, Nigeria

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