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

NCT05725902 | Completed Phase 2 FDA Drug

• 1 other identifier: FT-4202-CBF
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

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

Outcome Measures

Primary Outcomes (3)

• Effect of etavopivat on cerebral blood flow (CBF)

  Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.

  24 weeks

• Effect of etavopivat on oxygen ejection fraction (OEF)

  Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.

  24 weeks

• Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)

  Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.

  24 weeks

Secondary Outcomes (7)

• Relationship between CBF and change in Hb levels

  24 weeks

• Relationship between oxygen ejection fraction (OEF) and change in Hb levels

  24 weeks

• Relationship between cerebral metabolic rate of oxygen (CMRO2) and change in Hb levels

  24 weeks

• Adverse events in participants with SCD

  24 weeks

• Muscle hemodynamic effect of etavopivat on muscle blood flow

  24 weeks

Study Arms (1)

Etavopivat

EXPERIMENTAL

Single-arm, open-label

Drug: Etavopivat

Interventions

Etavopivat DRUG

The study intervention is etavopivat (400 mg), administered orally and once daily (QD)

Also known as: FT-4202

Etavopivat

Eligibility Criteria

• Age: 12 Years - 21 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (1)

Emory University Children's Healthcare of Atlanta

Atlanta, Georgia, 30342, United States

Location

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Transparency dept. 2834

  Novo Nordisk A/S

  STUDY DIRECTOR

• Amy Tang, MD

  Children's Healthcare of Atlanta

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 23, 2023
• First Posted: February 13, 2023
• Study Start: March 9, 2024
• Primary Completion: March 9, 2026
• Study Completion: March 9, 2026
• Last Updated: April 8, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)