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New sickle cell drug shows promise in early safety trial

NCT ID NCT05169580

Disease control Sponsor: Fulcrum Therapeutics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 early-stage study tested a new drug, Pociredir (FTX-6058), in 45 adults with sickle cell disease to see if it is safe and how the body processes it. The main goal was to check for side effects and measure drug levels in the blood. Researchers also looked at changes in fetal hemoglobin, which may help control the disease.

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 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

45 people

The number who actually took part.

Started

Dec 2021

Finished

Jan 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

18 to 65 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.

Key Inclusion Criteria: * Participant is 18 to 65 years of age, inclusive at the time informed consent is obtained. * Documented SCD at the time of screening (S/S, S/β0, S/β+, and S/C only) as confirmed through review of medical records or HPLC. * Participants who meet at least one the following criteria: 1. ≥4 to 10 episodes of SCD pain crisis over 12 months, or ≥2 to 5 over 6 months prior to screening 2. ≥2 episodes of SCD pain crisis plus at least one of the following over previous 12 months: i) Acute chest syndrome (ACS) ii. Hepatic or splenic sequestration iii. Priapism 3. ≥2 of the following events over the previous 12 months:i. ACS ii. Hepatic or splenic sequestration iii. Priapism 4. Tricuspid regurgitant jet velocity (TRV) ≥ 3.0 meter/second(m/s) OR TRV ≥ 2.5 m/s + N-terminal pro b-type natriuretic peptide (NT-proBNP) plasma level ≥ 160 picogram per milliliter; OR documented ongoing pulmonary hypertension diagnosed from previous echocardiogram or right-sided heart catheterization with mean pulmonary artery pressure \> 25 millimeter of mercury; 5. SCD-related chronic kidney disease (CKD) 6. Meet medical criteria to receive (e.g., post-cerebrovascular accident) but are contraindicated for chronic transfusions (e.g., alloimmunization, transfusion reactions) * Previous experience with Hydroxyurea (HU) but have shown to be unresponsive and/or intolerant or ineligible AND * Previous experience with a stable dose of voxelotor, crizanlizumab, and/ or L-glutamine but have shown to be unresponsive and/or intolerant or ineligible * Per Investigator's recommendation, participants may continue crizanlizumab and/or L-glutamine but must be on a stable dose for at least 6 months * HbF ≤ 20% of total Hb * Total Hb ≥ 5.5 g/dL and ≤ 12 g/dl (males) or ≤ 10.6 g/dl (females) at screening. * Participant must meet both of the following laboratory values at screening: 1. Absolute neutrophil count ≥ 1.5 × 10\^9 per liter (/l) 2. Platelets ≥ 80 × 10\^9/l 3. Absolute reticulocyte count at screening ≥ 100 x 10\^9/l. Key Exclusion Criteria: * Sickle cell complication requiring care from a medical provider in hospital or emergency care setting in the 14 days prior to starting study drug. * History of bone marrow transplant or human stem cell transplant or gene therapies. * • Participants with a history of severe renal disease defined as estimated glomerular filtration rate \< 30 mL/min/1.73m\^2. Participants on dialysis of any kind are excluded. * Participants receiving regularly scheduled transfusions or therapeutic phlebotomies, or any participant who has been transfused within 60 days prior to initiating study drug. * Participant with active malignancy, or history of cancer (except for squamous cell skin cancer, basal cell skin cancer, and stage 0 cervical carcinoma in situ, with no recurrence for the last 5 years), or has an immediate family member with known or suspected familial cancer syndrome. Known presence of a chromosomal abnormality or genetic mutation that may put the participant at an increased risk of myelodysplastic syndrome (MDS) or acute myeloid leukemia (AML). * Participant currently on HU, or have received HU, within 60 days prior to initiating study drug.

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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 anemia Sickle cell disease

Contacts and locations

Locations

  • Barau Dikko Teaching Hospital

    Kaduna, 800125, Nigeria

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • Charlotte Maxeke Johannesburg Academic Hospital

    Johannesburg, 2193, South Africa

  • Eastern Carolina University

    Greenville, North Carolina, 27834, United States

  • Inova Schar Cancer Institute

    Fairfax, Virginia, 22031, United States

  • Jacobi Medical Center

    The Bronx, New York, 10461, United States

  • Lynn Health Science Institute

    Oklahoma City, Oklahoma, 73112, United States

  • National Hospital, Abuja

    Abuja, 900247, Nigeria

  • Our Lady of the Lake Hospital

    Baton Rouge, Louisiana, 70808, United States

  • Queens Hospital Cancer Center

    Jamaica, New York, 11432, United States

  • Sonar Research Center

    Atlanta, Georgia, 30315, United States

  • University of Arkansas for Medical Sciences (UAMS)

    Little Rock, Arkansas, 72205, United States

  • University of California, Los Angeles

    Los Angeles, California, 90095, United States

  • University of Ibadan

    Ibadan, 200212, Nigeria

  • University of Illinois Chicago

    Chicago, Illinois, 60612, United States

  • University of North Carolina at Chapel Hill

    Chapel Hill, North Carolina, 27599, United States

  • University of Texas Houston

    Houston, Texas, 77030, United States

  • Virginia Commonwealth University

    Richmond, Virginia, 23298, United States

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