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Study identifier: NCT05953584 Synced from ClinicalTrials.gov · July 29, 2026
● 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

Condition: Sickle Cell Disease  ·  Sponsor: Forma Therapeutics, Inc.

PhasePhase 2
Planned participants27
Who can joinAll sexes, 12 Years to 16 Years
Healthy volunteersNo

About this study

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 paed…

This description comes directly from the study's public registry record.

Talk to the study team

Novo Nordisk  ·  (+1) 866-867-7178  ·  clinicaltrials@novonordisk.com

Always discuss trial participation with your own doctor first.

Locations (9)

All India Institute of Medical Sciences (AIIMS), RaipurRaipur, Chhattisgarh, IndiaRecruiting
All India Institute of Medical Sciences-DelhiDelhi, IndiaRecruiting
Nirmal Hospital Pvt. Ltd.Gujarat, IndiaRecruiting
Suretech Hospital and Research Centre Ltd.Maharashtra, IndiaRecruiting
Indira Gandhi Government Medical College & HospitalNagpur, IndiaNot Yet Recruiting
University of Ibadan, University College HospitalIbadan, NigeriaRecruiting
Lagos University Teaching Hospital, LagosLagos, NigeriaRecruiting
Aminu Kano Teaching Hospital (AKTH)Tarauni, NigeriaRecruiting
Sultan Qaboos University HospitalMuscat, Sultanet of Oman/Muscat/Al Khoud, OmanRecruiting

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Source record: clinicaltrials.gov/study/NCT05953584