Sickle cell drug safety trial ends early
NCT ID NCT05632354
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study looked at the long-term safety of the drug osivelotor in people with sickle cell disease who had already taken it in an earlier trial. The goal was to track side effects and blood changes over time. However, the study was terminated early, so the full safety picture is not yet known.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- Osivelotor (a drug to help red blood cells carry oxygen better)
- What this could lead to
- If successful, this could provide a long-term treatment option to reduce painful crises in sickle cell disease.
- What could go wrong
- The trial was terminated early, so long-term safety and effectiveness data are limited. It is still an experimental drug with unknown risks.
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
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Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
47 people
The number who actually took part.
- Started
-
Jan 2023
- Finished
-
Feb 2025
- 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
-
6 months and older
- Sex
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Anyone
- Healthy volunteers
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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 Hide 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: 1. Male or female aged 6 months or older with SCD who participated and received study drug or placebo in a previous osivelotor clinical study and completed the end of treatment visit. Note: Participants who discontinued study drug in the originating study due to an TEAE, but who remained on study, may be eligible for treatment in this study provided the TEAE does not pose a risk for treatment with osivelotor. 2. Females of childbearing potential are required to have a negative urine pregnancy test prior to dosing on Day 1. Note: Females who become of childbearing potential during the study must be willing to have negative urine pregnancy tests to remain in the study. 3. If sexually active, females of childbearing potential must consistently use highly effective methods of contraception consistently throughout the study and for at least 120 days after the last dose of study drug. If sexually active, male participants must use barrier methods of contraception until 84 days after the last dose of study drug. Male participants are eligible to participate if they agree to the following requirements during the study intervention period and for 84 days after the last dose of study intervention: * Refrain from donating sperm PLUS either * Be abstinent from heterosexual intercourse as their preferred and usual lifestyle OR * Must agree to use a male condom when engaging in any activity that allows for passage of ejaculate to another person 4. Participant has provided written informed consent/assent. For underage participants, both the consent of the participant's legal representative or legal guardian and the participant's assent (where applicable) must be obtained based on local requirements. Exclusion Criteria: * Participant withdrew consent or was noncompliant from the originating osivelotor clinical study * Current or recent use of voxelotor. Recent use is defined as within 10 days prior to Day 1 * Current or recent use of crizanlizumab. Recent use is defined as within 90 days prior to Day 1 * Participant has any medical, psychological, safety, or behavioral conditions that, in the opinion of the Investigator, may confound safety interpretation, interfere with compliance, or preclude informed consent * Has received an investigational drug (including investigational vaccines) within 5 times the elimination half-life (if known) or within 30 days (if the elimination half-life- is unknown) prior to study drug administration or is concurrently enrolled in any research judged not to be scientifically or medically compatible with this study
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Aminu kano Teaching Hospital
Kano, 700233, Nigeria
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Inova Schar Cancer Institute
Fairfax, Virginia, 22031, United States
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Lagos University Teaching Hospital
Lagos, 100254, Nigeria
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Mississippi Center for Advanced Medicine
Madison, Mississippi, 39110, United States
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Our Lady of the Lake Hospital, Inc.
Baton Rouge, Louisiana, 70808, United States
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University College Hospital Ibadan
Ibadan, Oyo/ibadan North, 200212, Nigeria
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University Medical Center Inpatient Pharmacy
New Orleans, Louisiana, 70112, United States
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University Medical Center New Orleans
New Orleans, Louisiana, 70112, United States
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University of Texas Health Science Center
Houston, Texas, 77030, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can adding common pain drugs reduce morphine needs in sickle cell crises?
- Gene editing offers hope for a One-Time sickle cell cure
- Tiny biochip could reveal sickle cell severity
- Can a milder transplant cure sickle cell and thalassemia in adults?
- Can an antioxidant supplement calm sickle cell blood cells?
- Can a softer transplant cure sickle cell disease?