Sickle Cell DiseasePhase 2NCT05725902
Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
A Phase 2 study testing Etavopivat in people with Sickle Cell Disease, run by Novo Nordisk A/S at 1 site in United States.
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. Read more about this study
- Who is it for?
- Ages 12 to 21 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Etavopivat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Mar 2024to 9 Mar 2026
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Show all 16Show fewer
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Emory University Children's Healthcare of Atlanta
Atlanta, Georgia, 30342
About this study
Official title: A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
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.
Read the detailed 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.
- Conditions
- Sickle Cell Disease
- Being tested
- Etavopivat
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EtavopivatDrug
Also called FT-4202
The study intervention is etavopivat (400 mg), administered orally and once daily (QD)
| Study group | Role | What they receive |
|---|---|---|
| Etavopivat | Experimental | Single-arm, open-label Receives: Drug: Etavopivat |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: 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.
Measured: 24 weeks
Secondary outcomes (7)
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.
Measured: 24 weeks
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.
Measured: 24 weeks
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.
Measured: 24 weeks
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
Measured: 24 weeks
Muscle hemodynamic effect of etavopivat on muscle blood flow
Change in muscle blood flow assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Measured: 24 weeks
Muscle hemodynamic effect of etavopivat on oxygen ejection fraction (OEF)
Change in OEF from baseline will be summarized with descriptive statistics by nominal study visit.
Measured: 24 weeks
Muscle hemodynamic effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Change in CMRO2 from baseline will be summarized with descriptive statistics by nominal study visit.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Feb 2023
- Study start9 Mar 2024
- Primary completion9 Mar 2026
- Study completion9 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Transparency dept. 2834, Study director, Novo Nordisk A/SAmy Tang, MD, Principal investigator, Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05725902
- Sponsor's protocol ID
- FT-4202-CBF
- Status last verified
- Apr 2026
- First submitted
- 23 Jan 2023
- Last updated
- 8 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pilot Study of the Effect of Etavopivat on Cerebral Hemodynamic Response in Children With Sickle Cell Disease
- ClinicalTrials.gov ID
- NCT05725902
- Acronym / sponsor ID
- FT-4202-CBF
- Conditions
- Sickle Cell Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 9 Mar 2024 (actual)
- Primary completion
- 9 Mar 2026 (actual)
- Study completion
- 9 Mar 2026 (actual)
- First posted
- 13 Feb 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Etavopivat | Experimental | Drug: EtavopivatSingle-arm, open-label |
Who may qualify
- Ages
- 12 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- 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
Key exclusion criteria (16 listed)
- Any one of the following requiring a medical facility visit within 14 days prior to signing the informed consent form:
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| EtavopivatAlso: FT-4202The study intervention is etavopivat (400 mg), administered orally and once daily (QD) | Drug | Etavopivat |
Primary outcome
Effect of etavopivat on cerebral blood flow (CBF)
Time frame: 24 weeks
Change in cerebral blood flow (CBF) assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Effect of etavopivat on oxygen ejection fraction (OEF)
Time frame: 24 weeks
Change in OEF assessments from baseline will be summarized with descriptive statistics by nominal study visit.
Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Time frame: 24 weeks
Change in CMRO2 assessments from baseline will be summarized with descriptive statistics by nominal study visit.
7 secondary outcomes
Relationship between CBF and change in Hb levels
Time frame: 24 weeks
Relationship between oxygen ejection fraction (OEF) and change in Hb levels
Time frame: 24 weeks
Relationship between cerebral metabolic rate of oxygen (CMRO2) and change in Hb levels
Time frame: 24 weeks
Adverse events in participants with SCD
Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on muscle blood flow
Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on oxygen ejection fraction (OEF)
Time frame: 24 weeks
Muscle hemodynamic effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Time frame: 24 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Transparency dept. 2834 study director
Novo Nordisk A/S
Amy Tang, MD principal investigator
Children's Healthcare of Atlanta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Etavopivat in people with Sickle Cell Disease, run by Novo Nordisk A/S at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effect of etavopivat on cerebral blood flow (CBF) (time frame: 24 weeks). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2026.