Skip to main content
UK clinical trials - updated daily from ClinicalTrials.gov
TrialConnect
← Back to Search
Looking for participantsPhase2

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

Sponsor: Forma Therapeutics, Inc.

NCT ID: NCT06198712

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
Etavopivat (drug)
How long the study runs
Study runs about 79 months (dates as stated)
About the drug or intervention
Etavopivat — drug: Participants will receive oral tablets or granules of etavopivat once daily.
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
6 Months to 18 Years
Who
All
Number of participants
95
Started
2023-01-12
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for sickle cell disease
  • • Phase2 - 95 participants
  • • The purpose of this study is to evaluate the pharmacokinetics and safety of etavopivat in paediatric participants with sickle cell disease (SCD)

Who can take part?

  • • Ages 6 Months to 18 Years
  • • Diagnosed with sickle cell disease

Where?

  • • London - Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
  • • London - King's College Hospital - Alex Mowat Research Hub
  • • Manchester - Manchester Royal Infirmary_1

This is a simplified summary. Always discuss with your doctor before making any decisions.

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 will undergo a 24-week primary treatment period followed by a 72-week extension treatment period to further evaluate long-term safety and pharmacokinetics of etavopivat. The total duration of the study will be approximately 96 weeks.

Sickle Cell Disease

How this trial compares with your answers

Answer 2 more questions to improve match

What we know so far

Condition· Matched your search
Age· Tell us your age for better matching
Gender· Tell us your sex for better matching

Still need:

  • • Tell us your age for better matching
  • • Tell us your sex for better matching

Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 6 Months - 18 Years
  • Who can join: All genders

Biomarkers mentioned

ER

What the study is looking for

  • ✓Type of Participant and Disease Characteristics
  • ✓Patient's parent, legal guardian, or legal representative has provided documented agreement to take part and patients have...
  • ✓Age greater than or equal to (≥) 6 months and lesser than (\<) 18 years of age at time of enrollment, according to...
  • ✓Cohort 1: age 12 to \< 18 years (adolescents)
  • ✓Cohort 2: age 6 to \< 12 years

Who cannot take part

  • ✗Medical Conditions
  • ✗Female who is breastfeeding or pregnant
  • ✗More than 15 VOCs within the 12 months prior to starting study treatment that required a hospital, emergency room...
  • ✗Hospitalized for sickle cell crisis or other vaso-occlusive event occurring in the 14 days prior to starting study...
  • ✗Abnormal TCD in the 12 months prior to starting study treatment
See the full 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 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)

Where Is This Study? (3 UK sites)

Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital

London SE1 7EH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Main Email: gstt.research.rbhh@nhs.net

gstt.research.rbhh@nhs.netn/a

King's College Hospital - Alex Mowat Research Hub

London SE5 9RS, United Kingdom

Recruiting
Hospital R&D contact (matched)

Jasmine Palmer

kch-tr.research@nhs.net0203 299 1980

Manchester Royal Infirmary_1

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

How to Get in Touch

Novo Nordisk

Sponsor contact

CONTACT

(+1) 866-867-7178 clinicaltrials@novonordisk.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09