Actively Recruiting
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Led by Forma Therapeutics, Inc. · Updated on 2026-04-24
95
Participants Needed
18
Research Sites
342 weeks
Total Duration
On this page
Sponsors
F
Forma Therapeutics, Inc.
Lead Sponsor
N
Novo Nordisk A/S
Collaborating Sponsor
AI-Summary
What this Trial Is About
This study is being done to learn about etavopivat, a once a day medicine taken by mouth in adolescents with sickle cell disease. The main goals are to study safety and how long etavopivat stays in the bloodstream, while also studying if there are benefits from taking etavopivat. Eligible participants who enter the study will start a 96-week treatment period. At the end of the 96 weeks, participants will have an end of study visit that occurs 4 weeks later. The participants will receive etavopivat every day throughout the treatment period.
CONDITIONS
Official Title
A Study to Evaluate the Pharmacokinetics and Safety of Etavopivat in Pediatric Patients With Sickle Cell Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent or legal guardian has provided informed consent and patients have given age-appropriate assent
- Age between 6 months and under 18 years, grouped into four age cohorts
- Confirmed diagnosis of sickle cell disease with documented genotype
- Hemoglobin level between 5.5 and 10.5 grams per deciliter
- Severe sickle cell disease defined by 2-15 vaso-occlusive episodes in prior 12 months, or hospitalization for sickle cell complications, or proteinuria, or history of conditional transcranial Doppler (TCD) within 12 months
- Stable hydroxyurea dose for at least 90 days if taking
- Stable treatment with crizanlizumab or L-glutamine for at least 12 months if applicable
- Use of acceptable contraception for females of childbearing potential and males during study and 90 days after last dose
You will not qualify if you...
- Female who is pregnant or breastfeeding
- More than 15 vaso-occlusive episodes requiring medical visits in the last 12 months
- Hospitalization for sickle cell crisis within 14 days before starting treatment
- Abnormal transcranial Doppler (TCD) within 12 months before treatment
- Regularly scheduled blood transfusion therapy
- Blood transfusion within 30 days before treatment
- Use of strong CYP3A4/5 inducers within 2 weeks before treatment
- Use of voxelotor within 28 days before treatment or planned during study
- Use of erythropoietin or other hematopoietic growth factors within 28 days before treatment or planned during study
- Prior cellular-based therapy such as bone marrow transplant or gene therapy
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 18 locations
1
The Hospital for Sick Children
Toronto, Ontario, Canada, M5G 1X8
Withdrawn
2
APHP - Centre de Référence des Syndromes
Paris, France, 75019
Not Yet Recruiting
3
Hospices Civils de Lyon-Hopital Lyon Sud
Pierre-Bénite, France, 69310
Not Yet Recruiting
4
Centre Hospitalier Universitaire de Rouen-Hopital Charles Nicolle
Roeun, France, 76031
Not Yet Recruiting
5
KEMRI-Walter-Reed Kericho
Kericho, Kenya, 20200
Not Yet Recruiting
6
Kombewa Clinical Research Centre
Kisumu, Kenya, 40100
Not Yet Recruiting
7
Ahero Clinical Trials Unit
Kisumu, Kenya, 40101
Actively Recruiting
8
Kenya Medical Research Institute-Centre for Respiratory Disease Research, Siaya Clinical Research Annexe
Siaya, Kenya, 40100
Not Yet Recruiting
9
American University of Beirut Medical center
Beirut, Lebanon, 1107 2020
Actively Recruiting
10
Hospital Nini
Tripoli, Lebanon, 113-6044
Actively Recruiting
11
University of Nigeria Teaching Hospital (UNTH)
Ituku-Ozalla, Enugu State, Nigeria, 400001
Actively Recruiting
12
Lagos University Teaching Hospital, Lagos
Lagos, Nigeria, 102215
Actively Recruiting
13
Aminu Kano Teaching Hospital (AKTH)
Tarauni, Nigeria, 700101
Actively Recruiting
14
Hacettepe University pediatric hematology
Ankara, Turkey (Türkiye), 06100
Withdrawn
15
Acıbadem Adana Hastanesi
Seyhan, Turkey (Türkiye), 1130
Withdrawn
16
Guys and St Thomas NHS Foundation Trust / Evelina Childrens Hospital
London, United Kingdom, SE1 7EH
Actively Recruiting
17
King's College Hospital - Alex Mowat Research Hub
London, United Kingdom, SE5 9RS
Actively Recruiting
18
Manchester Royal Infirmary_1
Manchester, United Kingdom, M13 9WL
Actively Recruiting
Research Team
N
Novo Nordisk
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here