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Sickle cell drug pociredir tested for Long-Term safety in 50 patients

NCT ID NCT07401823

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 This study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at the long-term safety of the drug pociredir in 50 adults with sickle cell disease who have already taken it and seen benefits in an earlier study. Participants will take pociredir once daily for up to 48 months. The main goal is to track side effects and other health changes.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pociredir (also called FTX-6058)
What this could lead to
If successful, this could show that pociredir is safe enough to use long-term for managing sickle cell disease, potentially reducing complications.
What could go wrong
This is a small, early-phase extension study with no comparison group, so results may not prove effectiveness. Long-term side effects are still unknown.

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

About 50 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Mar 2026

An estimate. Start dates often move.

Expected to finish

Jul 2030

An estimate. End dates often move.

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

18 years and older

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: * Participants aged ≥18 years and older must have previously participated in and successfully completed Study 6058-SCD-101. * Participant has signed and dated the informed consent form (ICF) before any study-specific procedures are performed and is willing and able to comply with the study procedures and restrictions. * Participants who meet all other inclusion and exclusion criteria for this study, and per Investigator's recommendation may continue standard of care as indicated with the exception of hydroxyurea (HU). Participants may continue crizanlizumab, and/or L-glutamine, but must be on a stable dose for at least 6 months. * Participants, who if female and of childbearing potential, agree to use 2 effective methods of contraception, 1 of which must be highly effective, or practice abstinence starting at the time of the ICF signing to 90 days after the last dose of study drug, and, who if male, should use condoms or practice abstinence from the time of ICF signing to 90 days after the last dose of study drug. * Documented HbF benefit, as judged by the Investigator, from prior study. * Participant must meet both of the following laboratory values during Screening: 1. Absolute neutrophil count ≥ 1.5 × 10\^9/liter, 2. Platelets ≥ 80 × 10\^9/liter * Absolute reticulocyte count during Screening \> 100 × 10\^9/liter. Exclusion Criteria: * Major surgery, serious illness (acute or chronic), infection (clinically significant bacterial, fungal, parasitic or viral infection which requires therapy), fever not resolved within 3 days of onset and requiring treatment, significant bleeding, cerebrovascular accident, or seizure within 14 days prior to signing the ICF and 14 days prior to starting study drug. * Sickle cell complication requiring more than 24-hour care from a medical provider in hospital or emergency care setting in the 14 days prior to starting study drug. * Use of medications that are moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or CYP2C8, inhibit P-glycoprotein, breast cancer resistance protein, or multidrug and toxin extrusion protein 2-K, or are substrates of CYP2B6 within 14 days prior to first dose of study drug or anticipated need for any of these medications during the study. * Participation in any other study with an investigational agent other than pociredir within the past 60 days prior to the first dose of study drug. * History of bone marrow transplant or hematopoietic stem cell transplant or gene therapies. * Vaccination in the previous 7 days prior to the first dose of study drug. Note: Other protocol specified criteria may apply

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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.

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    8 sites. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • Inova Schar Cancer Institute

    Fairfax, Virginia, 22031, United States

  • Jacobi Medical Center

    The Bronx, New York, 10461, United States

  • Our Lady of the Lake Hospital

    Baton Rouge, Louisiana, 70808, United States

  • Queens Hospital Cancer Center

    Jamaica, New York, 11432, United States

  • University of Arkansas for Medical Sciences

    Little Rock, Arkansas, 72205, United States

  • University of California, Los Angeles

    Los Angeles, California, 90095, United States

  • University of Texas Houston

    Houston, Texas, 77030, United States

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