Clinical Trials Logo

Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT05725902
Other study ID # FT-4202-CBF
Secondary ID
Status Not yet recruiting
Phase Phase 2
First received
Last updated
Start date March 4, 2024
Est. completion date August 15, 2025

Study information

Verified date February 2024
Source Novo Nordisk A/S
Contact Amy Tang, MD
Phone +1-404-785-3518
Email amy.tang@choa.org
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


Description:

This study is a pilot, open-label, single-arm study to evaluate the effect of etavopivat on cerebral hemodynamics, as measured by frequency domain near-infrared spectroscopy/diffuse correlation spectroscopy (FDNIRS/DCS) in participants 12 to 21 years of age with sickle cell disease (SCD). Cerebral blood flow (CBF), oxygen ejection fraction (OEF), and cerebral metabolic rate of oxygen (CMRO2) will be assessed FDNIRS/DCS in participants prior to, periodically throughout, and after 24 weeks of treatment with etavopivat. Approximately 12 participants will be enrolled. The duration of study treatment will be 24 weeks. The study duration for individual participants may last up to 36 to 38 weeks and includes the Screening Period (up to 4 weeks before study treatment), the 24-week treatment period, a Safety Follow-up Visit at 4 weeks (+ 7 days) after the last dose of study drug, and an End of Study (EOS) visit approximately 8 weeks (± 7 days) after the last dose of study drug. A participant is considered to have completed the study if he or she has completed all phases of the study including the last visit or the last scheduled procedure shown in the Schedule of Events.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 12
Est. completion date August 15, 2025
Est. primary completion date June 15, 2025
Accepts healthy volunteers No
Gender All
Age group 12 Years to 21 Years
Eligibility Inclusion Criteria: - Homozygous hemoglobin SS (HbSS) or hemoglobin S/beta0 thalassemia (HbS/ß0 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 Exclusion Criteria: - 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

Study Design


Related Conditions & MeSH terms


Intervention

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

Locations

Country Name City State
United States Emory University Children's Healthcare of Atlanta Atlanta Georgia

Sponsors (2)

Lead Sponsor Collaborator
Forma Therapeutics, Inc. Emory University

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary 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
Primary 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
Primary 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 Relationship between CBF and change in Hb levels The change from baseline of CBF will be correlated to the corresponding post-baseline assessment for change in Hb. 24 weeks
Secondary Relationship between oxygen ejection fraction (OEF) and change in Hb levels The change from baseline of OEF will be correlated to the corresponding post-baseline assessment for change in Hb. 24 weeks
Secondary Relationship between cerebral metabolic rate of oxygen (CMRO2) and change in Hb levels The change from baseline of CMRO2 will be correlated to the corresponding post-baseline assessment for change in Hb. 24 weeks
Secondary Adverse events in participants with SCD Maximum intensity of treatment emergent adverse events (TEAEs) will be summarized by system organ class and preferred term. The tabulation of deaths, serious TEAEs, serious drug-related TEAEs and TEAEs leading to study drug discontinuation will also be provided 24 weeks
See also
  Status Clinical Trial Phase
Completed NCT02227472 - Working Memory and School Readiness in Preschool-Aged Children With Sickle Cell Disease
Recruiting NCT06301893 - Uganda Sickle Surveillance Study (US-3)
Recruiting NCT04398628 - ATHN Transcends: A Natural History Study of Non-Neoplastic Hematologic Disorders
Completed NCT02522104 - Evaluation of the Impact of Renal Function on the Pharmacokinetics of SIKLOS ® (DARH) Phase 4
Recruiting NCT04688411 - An mHealth Strategy to Improve Medication Adherence in Adolescents With Sickle Cell Disease N/A
Terminated NCT03615924 - Effect of Ticagrelor vs. Placebo in the Reduction of Vaso-occlusive Crises in Pediatric Patients With Sickle Cell Disease Phase 3
Not yet recruiting NCT06300723 - Clinical Study of BRL-101 in Severe SCD N/A
Recruiting NCT03937817 - Collection of Human Biospecimens for Basic and Clinical Research Into Globin Variants
Completed NCT04917783 - Health Literacy - Neurocognitive Screening in Pediatric SCD N/A
Completed NCT04134299 - To Assess Safety, Tolerability and Physiological Effects on Structure and Function of AXA4010 in Subjects With Sickle Cell Disease N/A
Completed NCT02580565 - Prevalence of Problematic Use of Equimolar Mixture of Oxygen and Nitrous Oxide and Analgesics in the Sickle-cell Disease
Recruiting NCT04754711 - Interest of Nutritional Care of Children With Sickle Cell Disease on Bone Mineral Density and Body Composition N/A
Completed NCT04388241 - Preliminary Feasibility and Efficacy of Behavioral Intervention to Reduce Pain-Related Disability in Pediatric SCD N/A
Recruiting NCT05431088 - A Phase 2/3 Study in Adult and Pediatric Participants With SCD Phase 2/Phase 3
Completed NCT01158794 - Genes Influencing Iron Overload State
Recruiting NCT03027258 - Point-of-Delivery Prenatal Test Results Through mHealth to Improve Birth Outcome N/A
Withdrawn NCT02960503 - Macrolide Therapy to Improve Forced Expiratory Volume in 1 Second in Adults With Sickle Cell Disease Phase 1/Phase 2
Completed NCT02567682 - Drug Interaction Study of GBT440 With Caffeine, S-warfarin, Omeprazole, and Midazolam in Healthy Subjects Phase 1
Completed NCT02620488 - A Brief Laboratory-Based Hypnosis Session for Pain in Sickle Cell Disease N/A
Withdrawn NCT02630394 - A Pilot Study of Azithromycin Prophylaxis for Acute Chest Syndrome in Sickle Cell Disease Phase 1