Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir
NCT ID: NCT05169580
Last Updated: 2025-10-27
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
RECRUITING
PHASE1
70 participants
INTERVENTIONAL
2021-12-13
2026-01-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Safety, Tolerability and Pharmacokinetics of FTX-6058
NCT04586985
A Study of Nicotinamide With Oral Tetrahydrouridine and Decitabine to Treat High Risk Sickle Cell Disease
NCT04055818
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
NCT05904093
A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease
NCT06546670
Interest of Famotidine in Children With Sickle Cell Disease
NCT05084521
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
Participants will receive 12 weeks of dosing with 4 weeks of follow-up. Approximately 10 participants will be enrolled in each cohort. A maximum of 3 participants with SCD HbSC+ genotype may be enrolled in each cohort.
Cohort 1 will receive 6 milligrams (mg) of Pociredir by mouth once daily. Doses for subsequent cohorts will be determined following review by the Data Monitoring Committee \[DMC\]. A total of seven cohorts may be included. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. Additional cohorts using alternative dosing schedules may be considered based on available data.
The primary endpoints of the study are to evaluate the safety and tolerability of Pociredir as measured by the frequency of adverse events and to evaluate single and multiple-dose pharmacokinetics of Pociredir in participants with sickle cell disease. Secondary endpoints include evaluating the effect of Pociredir on fetal hemoglobin induction in peripheral blood and evaluating the effects of Pociredir on hemolysis in participants with sickle cell disease.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SEQUENTIAL
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
Pociredir oral capsule(s) in Sickle Cell participants
Cohort 1 will receive 6 mg of Pociredir by mouth once daily. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. A total of seven cohorts may be included. Following the first cohort, doses for all subsequent cohorts will be determined following DMC review of the safety and pharmacokinetic data observed in participants from the prior and ongoing cohorts. Alternate dosing schedules may be evaluated in some of the cohorts.
Pociredir oral capsule(s)
Participants will receive Pociredir
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
Pociredir oral capsule(s)
Participants will receive Pociredir
Other Intervention Names
Discover alternative or legacy names that may be used to describe the listed interventions across different sources.
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
18 Years
65 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Fulcrum Therapeutics
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Adeyemi Adenola, MD
Role: STUDY_DIRECTOR
Fulcrum Therapeutics
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
University of Arkansas for Medical Sciences (UAMS)
Little Rock, Arkansas, United States
University of California, Los Angeles
Los Angeles, California, United States
Foundation for Sickle Cell Disease Research, LLC
Hollywood, Florida, United States
University of Miami Health System
Miami, Florida, United States
Sonar Research Center
Atlanta, Georgia, United States
Visionaries Clinical Research
Atlanta, Georgia, United States
Atlanta Center for Medical Research
Atlanta, Georgia, United States
Augusta University
Augusta, Georgia, United States
University of Illinois Chicago
Chicago, Illinois, United States
Our Lady of the Lake Hospital
Baton Rouge, Louisiana, United States
Axon Clinical Research Institute
Baltimore, Maryland, United States
Boston Medical Center
Boston, Massachusetts, United States
Mississippi Center for Advanced Medicine
Madison, Mississippi, United States
Queens Hospital Cancer Center
Jamaica, New York, United States
Jacobi Medical Center
The Bronx, New York, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Eastern Carolina University
Greenville, North Carolina, United States
Lynn Health Science Institute
Oklahoma City, Oklahoma, United States
University of Texas Houston
Houston, Texas, United States
Inova Schar Cancer Institute
Fairfax, Virginia, United States
Virginia Commonwealth University
Richmond, Virginia, United States
National Hospital, Abuja
Abuja, , Nigeria
University of Ibadan
Ibadan, , Nigeria
Barau Dikko Teaching Hospital
Kaduna, , Nigeria
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, , South Africa
Countries
Review the countries where the study has at least one active or historical site.
Central Contacts
Reach out to these primary contacts for questions about participation or study logistics.
References
Explore related publications, articles, or registry entries linked to this study.
Steinberg MH, Chui DH, Dover GJ, Sebastiani P, Alsultan A. Fetal hemoglobin in sickle cell anemia: a glass half full? Blood. 2014 Jan 23;123(4):481-5. doi: 10.1182/blood-2013-09-528067. Epub 2013 Nov 12.
Abbasi M, Srivastava A, Saraf SL. Management of Kidney Disease with Sickle Cell Disease. J Am Soc Nephrol. 2025 Oct 1;36(10):2041-2054. doi: 10.1681/ASN.0000000804. Epub 2025 Jun 26.
Ngo DA, Aygun B, Akinsheye I, Hankins JS, Bhan I, Luo HY, Steinberg MH, Chui DH. Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin. Br J Haematol. 2012 Jan;156(2):259-64. doi: 10.1111/j.1365-2141.2011.08916.x. Epub 2011 Oct 24.
Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
Sankaran VG, Orkin SH. The switch from fetal to adult hemoglobin. Cold Spring Harb Perspect Med. 2013 Jan 1;3(1):a011643. doi: 10.1101/cshperspect.a011643.
Saraf SL, Molokie RE, Nouraie M, Sable CA, Luchtman-Jones L, Ensing GJ, Campbell AD, Rana SR, Niu XM, Machado RF, Gladwin MT, Gordeuk VR. Differences in the clinical and genotypic presentation of sickle cell disease around the world. Paediatr Respir Rev. 2014 Mar;15(1):4-12. doi: 10.1016/j.prrv.2013.11.003. Epub 2013 Nov 15.
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
6058-SCD-101
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.