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
221 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The study will test a new medicine, etavopivat, for sickle cell disease and see if it is safe and help-ful for participants with sickle cell disease who are at an increased risk of stroke. Participants will be divided into two cohorts depending on their transcranial doppler (TCD) ultrasound results and whether or not they receive hydroxyurea (medication that they may already be taking). In one cohort, participants with conditional transcranial doppler (TCD) or participants with abnormal TCD who are not able to receive hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. In another cohort, participants with conditional TCD or participants with abnormal TCD who are receiving a stable dose of hydroxyurea will be included. The study doctor will determine if the TCD result is conditional or abnormal. The participant will start a 52-week (1 year) treatment period. The participant will take 400 milligrams (mg) of etavopivat once a day for the 52 weeks. The dose of 400 mg will be taken as 2 tablets by mouth, each containing 200 mg of etavopivat. Etavopivat may be taken with or without food. Each dose should be taken with a glass of water. As part of the study, the participants will be asked to visit the clinic frequently. The participant will have the opportunity to participate in a 48-week optional extension treatment period. The optional extension treatment period will allow continued as-sessment of safety of etavopivat in paediatric patients. At the end of the study, if deemed appro-priate the participant, the caregiver, and the study doctor, the participant may be offered the op-portunity to participate in a separate study to continue receiving etavopivat. If/when this separate study becomes available, the participant may only transfer to the new study after completion of the 52-week primary treatment period and at any time during the 48-week optional extension treatment period.
CONDITIONS
Official 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...
- Patient's parent, legal guardian, or legal representative has given informed consent, and patient has provided age-appropriate assent
- Age between 12 and 16 years at screening
- Confirmed diagnosis of sickle cell disease of any genotype
- Blood flow velocity (TAMMV) of 170 cm/s or higher in internal carotid or middle cerebral artery confirmed twice, with no history of stroke, transient ischemic attack, or severe CNS vasculopathy
- Hemoglobin level between 6 and 9 grams per deciliter at screening
- If taking hydroxyurea, dose must be stable (no more than 20% change) for at least 90 days before study start with no expected dose changes except weight-based adjustments
- Female patients of childbearing potential must use acceptable contraception and agree not to donate ova during the study and for 90 days after last dose; males must agree to use contraception and not donate sperm during the same period
You will not qualify if you...
- Female who is pregnant or breastfeeding
- History of seizure disorder
- Prior overt stroke or significant neurological deficits suggesting stroke
- Severe CNS vasculopathy (Grade 4 or higher) based on local MRA read
- Significant low blood counts: neutrophils less than 1.5 x 10^3/µL, platelets less than 150,000/µL, or reticulocytes less than 80,000/µL
- Severe kidney dysfunction or on chronic dialysis
- Liver dysfunction with ALT more than 4 times upper limit of normal or direct bilirubin more than 3 times upper limit
- Active significant infections requiring systemic treatment or history of infections causing neurological impairment
- Known HIV infection
- Known hepatitis B infection (positive surface antigen and core antibody)
AI-Screening
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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
CONTACT
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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