ACTIVE_NOT_RECRUITING

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

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Conditions

Sickle Cell Disease

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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.

Official Title

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

Quick Facts

Study Start:2024-03-25
Study Completion:2026-02-26
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:ACTIVE_NOT_RECRUITING

Study ID

NCT05725902

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.

Ages Eligible for Study:12 Years to 21 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:CHILD, ADULT
Inclusion CriteriaExclusion Criteria
  1. * Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/β0 thal)
  2. * Hemoglobin (Hb): Hb ≤ 9.0 g/dL at baseline
  3. * 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
  1. * Any one of the following requiring a medical facility visit within 14 days prior to signing the informed consent form:
  2. * Vaso-occlusive crisis (VOC)
  3. * Acute chest syndrome (ACS)
  4. * Splenic sequestration
  5. * Dactylitis
  6. * Requires chronic transfusion therapy
  7. * Abnormal TCD in the last 12 months
  8. * RBC transfusion within 60 days of screening
  9. * Severe renal dysfunction at the Screening Visit or on chronic dialysis
  10. * Hepatic dysfunction
  11. * 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
  12. * Major surgery involving the stomach or small intestine
  13. * Chemotherapy or radiation within the past 2 years
  14. * History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
  15. * Clinically significant bacterial, fungal, parasitic, or viral infection currently receiving or that will require therapy
  16. * Female who is breast feeding or pregnant

Contacts and Locations

Principal Investigator

Clinical Transparency dept. 2834
STUDY_DIRECTOR
Novo Nordisk A/S
Amy Tang, MD
PRINCIPAL_INVESTIGATOR
Children's Healthcare of Atlanta

Study Locations (Sites)

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

Collaborators and Investigators

Sponsor: 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

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-25
Study Completion Date2026-02-26

Study Record Updates

Study Start Date2024-03-25
Study Completion Date2026-02-26

Terms related to this study

Additional Relevant MeSH Terms

  • Sickle Cell Disease