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Etavopivat for Sickle Cell Disease

Phase 2
Waitlist Available
Led By Amy Tang, MD
Research Sponsored by Novo Nordisk A/S
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 24 weeks
Awards & highlights

Study Summary

This trial will study a new drug to treat sickle cell disease & inherited hemoglobinopathies. The aim is to reduce red blood cell sickling & hemolysis to improve blood flow & reduce anemia. #SickleCell #Hemoglobinopathies #Etavopivat

Who is the study for?
This trial is for children aged 12-21 with Sickle Cell Disease, specifically those with hemoglobin SS or S/beta0 thalassemia and a hemoglobin level ≤ 9.0 g/dL. Stable hydroxyurea therapy is okay, but not if they've had recent severe complications like organ dysfunction, transfusions, surgery on stomach/small intestine, or unstable heart conditions.Check my eligibility
What is being tested?
The study tests Etavopivat's effects on brain and muscle blood flow in young patients with Sickle Cell Disease. It's an open-label trial where all participants receive the drug to see how it influences their cerebral hemodynamics.See study design
What are the potential side effects?
While specific side effects of Etavopivat are not listed here, common risks may include potential reactions at the infusion site, fatigue, headache or dizziness due to changes in blood flow; however individual experiences can vary.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~24 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Effect of etavopivat on cerebral blood flow (CBF)
Effect of etavopivat on cerebral metabolic rate of oxygen (CMRO2)
Effect of etavopivat on oxygen ejection fraction (OEF)
Secondary outcome measures
Adverse events in participants with SCD
Relationship between CBF and change in Hb levels
Relationship between cerebral metabolic rate of oxygen (CMRO2) and change in Hb levels
+1 more

Trial Design

1Treatment groups
Experimental Treatment
Group I: EtavopivatExperimental Treatment1 Intervention
Single-arm, open-label

Find a Location

Who is running the clinical trial?

Novo Nordisk A/SLead Sponsor
1,514 Previous Clinical Trials
2,415,056 Total Patients Enrolled
Forma Therapeutics, Inc.Lead Sponsor
12 Previous Clinical Trials
1,237 Total Patients Enrolled
Emory UniversityOTHER
1,640 Previous Clinical Trials
2,560,684 Total Patients Enrolled

Media Library

Etavopivat (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05725902 — Phase 2
Sickle Cell Disease Research Study Groups: Etavopivat
Sickle Cell Disease Clinical Trial 2023: Etavopivat Highlights & Side Effects. Trial Name: NCT05725902 — Phase 2
Etavopivat (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05725902 — Phase 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment accessible to adults over the age of eighteen?

"The minimum age for eligibility in this medical trial is 12, whereas the upper limit of participation has been set at 21."

Answered by AI

Has Etavopivat been given the greenlight by the FDA?

"The safety of etavopivat was assessed by our team at Power and scored a 2. As this is only in its second phase, there is some research that supports the drug's security but not yet any evidence to verify efficacy."

Answered by AI

Are there still opportunities for people to join this experiment?

"Data hosted on clinicaltrials.gov reveals that the recruitment period for this investigation has expired, last edited on February 10th 2023. Despite this trial not being open to new participants, there are an abundance of other trials with 188 studies accepting applicants currently."

Answered by AI

Who should consider participating in this clinical investigation?

"In order to be considered for this trial, a potential participant must have been diagnosed with sickle cell disease and lie in the 12-21 age range. In total, around a dozen patients are being admitted into the experiment."

Answered by AI

What is the ambition of this clinical exploration?

"Forma Therapeutics, Inc., the clinical trial sponsor, has reported that their primary outcome will be measuring effect of etavopivat on oxygen ejection fraction (OEF) over a 24-week time frame. Secondary outcomes include: examining correlation between OEF and change in Hb levels; exploring connection between cerebral metabolic rate of oxygen (CMRO2) and alteration in Hb levels; as well as evaluation of adverse events experienced by participants with SCD. Finally, they aim to furnish tabulated information regarding deaths attributed to TEAEs or drug discontinuation caused by these events."

Answered by AI
~8 spots leftby Jun 2025