Actively Recruiting
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
Led by Forma Therapeutics, Inc. · Updated on 2025-09-18
27
Participants Needed
9
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying etavopivat, a new medicine, in children aged 12 to 16 years with sickle cell disease who have an increased risk of stroke. The trial focuses on patients with conditional or abnormal transcranial doppler (TCD) ultrasound results, assessing whether etavopivat is safe and helpful for these participants. The study is a Phase 2 open-label trial sponsored by Forma Therapeutics, Inc., aiming to understand the effect of etavopivat on blood flow velocities in brain arteries. Participants will be divided into two groups based on their TCD results and whether they are already taking the medication hydroxyurea. One group includes participants with abnormal or conditional TCD who are not on hydroxyurea, while the other group includes those with similar TCD results who are on a stable dose of hydroxyurea. All participants will take 400 mg of etavopivat orally once daily for 52 weeks, with the option to continue in a 48-week extension period to further monitor safety. Etavopivat is taken as two 200 mg tablets and may be taken with or without food. During the study, participants will visit the clinic frequently for monitoring. Assessments include measuring blood flow velocities in brain arteries using TCD at various time points, tracking changes in velocity categories, and monitoring safety. The study also includes evaluating blood counts and liver and kidney function. At the end of the treatment and extension periods, participants may be offered the chance to join another study to continue receiving etavopivat. Total participation can last up to about two years depending on extension and further studies.
CONDITIONS
Brief Title
A Phase 2 Open-label Study to Evaluate the Activity of Etavopivat on Transcranial Doppler Velocities in Pediatric Patients With Sickle Cell Disease Who Are at Increased Risk for Primary Stroke
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Parent or legal guardian has provided informed consent and patient has given age-appropriate assent
- Aged 12 to 16 years inclusive at screening
- Confirmed diagnosis of sickle cell disease with documented genotype
- Transcranial doppler velocity greater than or equal to 170 cm/s in key brain arteries confirmed twice
- No history of primary ischemic or hemorrhagic stroke, transient ischemic attack, or severe CNS vasculopathy
- Hemoglobin level between 6 and 9 grams per deciliter at screening
- Stable hydroxyurea dose for at least 90 days before treatment for those already taking it
- Females of childbearing potential must use contraception and agree not to donate ova during and 90 days after treatment; males must use contraception and agree not to donate sperm during and 90 days after treatment
You will not qualify if you...
- Female who is pregnant or breastfeeding
- History of seizure disorder
- Prior overt stroke or significant neurological deficits related to stroke
- Severe central nervous system vasculopathy (Grade 4 or higher)
- Significant low blood counts (neutrophils, platelets, reticulocytes below specified levels)
- Severe kidney dysfunction or on chronic dialysis
- Liver dysfunction with specific elevated enzyme or bilirubin levels
- Active or recent serious infections requiring systemic treatment
- Known HIV infection
- Known hepatitis B infection
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 52 weeks
Participants receive Etavopivat 400 mg orally once daily, either alone or in combination with a stable dose of hydroxyurea.
Visits at Baseline, Weeks 2, 4, 12, 24, and 52
Trial Site Locations
Total: 9 locations
1
All India Institute of Medical Sciences (AIIMS), Raipur
Raipur, Chhattisgarh, India, 492099
Actively Recruiting
2
All India Institute of Medical Sciences-Delhi
Delhi, India, 110029
Actively Recruiting
3
Nirmal Hospital Pvt. Ltd.
Gujarat, India, 395002
Actively Recruiting
4
Suretech Hospital and Research Centre Ltd.
Maharashtra, India, 440012
Actively Recruiting
5
Indira Gandhi Government Medical College & Hospital
Nagpur, India, 440018
Not Yet Recruiting
6
University of Ibadan, University College Hospital
Ibadan, Nigeria, 200285
Actively Recruiting
7
Lagos University Teaching Hospital, Lagos
Lagos, Nigeria, 102215
Actively Recruiting
8
Aminu Kano Teaching Hospital (AKTH)
Tarauni, Nigeria, 700101
Actively Recruiting
9
Sultan Qaboos University Hospital
Muscat, Sultanet of Oman/Muscat/Al Khoud, Oman, 123
Actively Recruiting
Research Team
N
Novo Nordisk
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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