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Could a pill protect the brains of kids with sickle cell disease?

NCT ID NCT05725902

Disease control Sponsor: Novo Nordisk A/S Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tested an experimental drug called etavopivat in 12 children and young adults (ages 12-21) with sickle cell disease. The goal was to see if the drug could improve blood flow and oxygen delivery to the brain and muscles. Participants took the drug and had scans to measure these effects. The study is complete, and results will help guide future research.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

12 people

The number who actually took part.

Started

Mar 2024

Finished

Mar 2026

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 21 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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 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

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Conditions

The condition(s) this trial relates to.

sickle cell disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Sickle cell disease

Contacts and locations

Locations

  • Emory University Children's Healthcare of Atlanta

    Atlanta, Georgia, 30342, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.