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Evaluating Pociredir for Sickle Cell Disease

A Phase 1 Open-Label, Multiple-Dose Study to Evaluate Safety and Tolerability, Pharmacokinetics and Pharmacodynamics of FTX-6058 in Subjects With Sickle Cell Disease (SCD)

Phase 1 Interventional Fulcrum Therapeutics · NCT05169580

This study is testing a new drug called Pociredir to see if it is safe and effective for adults with sickle cell disease.

Quick facts

PhasePhase 1
Study typeInterventional
Enrollment70 (estimated)
Ages18 Years to 65 Years
SexAll
SponsorFulcrum Therapeutics Industry-sponsored
Drugs / interventionscrizanlizumab
Locations20 sites (Little Rock, Arkansas and 19 other locations)
Trial IDNCT05169580 on ClinicalTrials.gov

What this trial studies

This Phase 1 multicenter, international, open-label study assesses the safety, tolerability, pharmacokinetics, and pharmacodynamics of Pociredir in adults aged 18-65 with sickle cell disease. Participants will receive Pociredir orally for 12 weeks, followed by 4 weeks of follow-up, with multiple cohorts receiving varying doses. The study aims to determine the frequency of adverse events and the drug's pharmacokinetics, while also exploring its potential to induce fetal hemoglobin.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18-65 with documented sickle cell disease and a history of pain crises or related complications.

Not a fit: Patients with sickle cell disease who do not meet the inclusion criteria or have contraindications to Pociredir may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide a new treatment option that improves the management of sickle cell disease.

How similar studies have performed: While this approach is novel, previous studies have explored pharmacological interventions for sickle cell disease, indicating potential for success.

Eligibility criteria

Show full inclusion / exclusion criteria 
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.

Where this trial is running

Little Rock, Arkansas and 19 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Sickle Cell DiseaseSickle Cell AnemiaPharmacokineticsPharmacodynamicsPociredirOpen label
Last reviewed 2026-06-09 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.