Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

Description

An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.

Conditions

Sickle Cell Disease

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Study Overview

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Study Details

Study overview

An open-label, single arm study in patients 12 to 21 years of age with SCD to evaluate the effects of etavopivat on cerebral and muscle hemodynamics.

A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease

Condition
Sickle Cell Disease
Intervention / Treatment

-

Contacts and Locations

Atlanta

Emory University Children's Healthcare of Atlanta, Atlanta, Georgia, United States, 30342

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • * Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/β0 thal)
  • * Hemoglobin (Hb): Hb ≤ 9.0 g/dL at baseline
  • * Concomitant hydroxyurea (HU) therapy is allowed if the dose has been stable for at least 3 months with no anticipated need for dose adjustments during the study and no sign of hematological toxicity
  • * Any one of the following requiring a medical facility visit within 14 days prior to signing the informed consent form:
  • * Vaso-occlusive crisis (VOC)
  • * Acute chest syndrome (ACS)
  • * Splenic sequestration
  • * Dactylitis
  • * Requires chronic transfusion therapy
  • * Abnormal TCD in the last 12 months
  • * RBC transfusion within 60 days of screening
  • * Severe renal dysfunction at the Screening Visit or on chronic dialysis
  • * Hepatic dysfunction
  • * Clinically relevant cardiac or pulmonary disease- e.g., congenital heart defect, uncompensated heart failure, or any unstable cardiac condition, arrhythmic heart condition, pulmonary fibrosis, pulmonary hypertension
  • * Major surgery involving the stomach or small intestine
  • * Chemotherapy or radiation within the past 2 years
  • * History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
  • * Clinically significant bacterial, fungal, parasitic, or viral infection currently receiving or that will require therapy
  • * Female who is breast feeding or pregnant

Ages Eligible for Study

12 Years to 21 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Forma Therapeutics, Inc.,

Clinical Transparency dept. 2834, STUDY_DIRECTOR, Novo Nordisk A/S

Amy Tang, MD, PRINCIPAL_INVESTIGATOR, Children's Healthcare of Atlanta

Study Record Dates

2026-02-02