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)

Who is this study for? Patients with sickle-cell disease
What treatments are being studied? FTX-6058
Status: Recruiting
Location: See all (25) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 1
SUMMARY

This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 65
Healthy Volunteers: f
View:

• 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:

‣ ≥4 to 10 episodes of SCD pain crisis over 12 months, or ≥2 to 5 over 6 months prior to screening

⁃ ≥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

⁃ ≥2 of the following events over the previous 12 months:i. ACS ii. Hepatic or splenic sequestration iii. Priapism

⁃ 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;

⁃ SCD-related chronic kidney disease (CKD)

⁃ 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:

‣ Absolute neutrophil count ≥ 1.5 × 10\^9 per liter (/l)

⁃ Platelets ≥ 80 × 10\^9/l

⁃ Absolute reticulocyte count at screening ≥ 100 x 10\^9/l.

Locations
United States
Arkansas
University of Arkansas for Medical Sciences (UAMS)
RECRUITING
Little Rock
California
University of California, Los Angeles
RECRUITING
Los Angeles
Florida
Foundation for Sickle Cell Disease Research, LLC
WITHDRAWN
Hollywood
University of Miami Health System
WITHDRAWN
Miami
Georgia
Atlanta Center for Medical Research
WITHDRAWN
Atlanta
Sonar Research Center
RECRUITING
Atlanta
Visionaries Clinical Research
WITHDRAWN
Atlanta
Augusta University
WITHDRAWN
Augusta
Illinois
University of Illinois Chicago
RECRUITING
Chicago
Louisiana
Our Lady of the Lake Hospital
RECRUITING
Baton Rouge
Massachusetts
Boston Medical Center
RECRUITING
Boston
Maryland
Axon Clinical Research Institute
WITHDRAWN
Baltimore
Mississippi
Mississippi Center for Advanced Medicine
WITHDRAWN
Madison
North Carolina
University of North Carolina at Chapel Hill
RECRUITING
Chapel Hill
Eastern Carolina University
RECRUITING
Greenville
New York
Queens Hospital Cancer Center
RECRUITING
Jamaica
Jacobi Medical Center
RECRUITING
The Bronx
Oklahoma
Lynn Health Science Institute
RECRUITING
Oklahoma City
Texas
University of Texas Houston
RECRUITING
Houston
Virginia
Inova Schar Cancer Institute
RECRUITING
Fairfax
Virginia Commonwealth University
RECRUITING
Richmond
Other Locations
Nigeria
National Hospital, Abuja
RECRUITING
Abuja
University of Ibadan
RECRUITING
Ibadan
Barau Dikko Teaching Hospital
RECRUITING
Kaduna
South Africa
Charlotte Maxeke Johannesburg Academic Hospital
RECRUITING
Johannesburg
Contact Information
Primary
Call Center
clinicaltrials@fulcrumtx.com
617-651-8853
Time Frame
Start Date: 2021-12-13
Estimated Completion Date: 2026-01
Participants
Target number of participants: 70
Treatments
Experimental: 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.
Sponsors
Leads: Fulcrum Therapeutics

This content was sourced from clinicaltrials.gov