Purpose

This clinical trial is a study to evaluate the pharmacokinetics of the tablet formulation Pociredir in fasted and fed state participants with Sickle Cell Disease (SCD).

Condition

Eligibility

Eligible Ages
Between 18 Years and 65 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Documented SCD at the time of screening, as confirmed through review of medical records or high-performance liquid chromatography (HPLC)/electrophoresis. - Participant, who if female and of childbearing potential, agrees to use 2 effective methods of contraception, one 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, agrees to use condoms or practice abstinence from the time of ICF signing to 90 days after the last dose of study drug. - Total Hb ≥ 5.5 grams/deciliter (g/dL) and ≤ 12 g/dL (males) or ≤ 10.6 g/dL (females) at screening - Participant must meet all of the following laboratory values at screening: 1. Absolute neutrophil count ≥ 1.5 × 10^9/L (cells/liter) 2. Platelets ≥ 80 × 10^9/L 3. Absolute reticulocyte count > 100 × 10^9/L - Participants who meet all other inclusion and

Exclusion Criteria

for this study, and per Investigator's recommendation may continue crizanlizumab, and/or L-glutamine, must be on a stable dose for at least 6 months Exclusion Criteria: - Participant has had any of the following in the 14 days prior to dosing: major surgery, serious illness, infection (clinically significant bacterial, fungal, parasitic or viral infection which requires therapy), fever not resolved within 3 days and requiring treatment, or sickle cell complication requiring care from a medical provider in a hospital or emergency care setting. - Participant has a serious medical condition other than SCD that, in the opinion of the Investigator, would preclude them from participating in the study, or which is unresolved or requiring ongoing treatment. - Elective surgery planned for the time period of the study. - Use of any medications that induce or inhibit cytochrome P450 (CYP) 3A4, 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 within the past 30 days or 5 half-lives, whichever is longer, prior to the first dose of study drug. - For Fed Cohort Only: Participant has special dietary restrictions or inability to consume standard meals as required in the study. Note: Other protocol specified criteria may apply.

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
N/A
Intervention Model
Sequential Assignment
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Fasted Cohort
Pociredir single dose tablet formulation under fasted conditions.
  • Drug: Pociredir
    Pociredir tablet formulation
Experimental
Fed Cohort
Pociredir single dose tablet formulation under fed conditions (after a high-fat breakfast).
  • Drug: Pociredir
    Pociredir tablet formulation

Recruiting Locations

Advanced Pharma - Miami
Miami 4164138, Florida 4155751 33147
Contact:
305-220-2727

Omega Research Group
Orlando 4167147, Florida 4155751 32808
Contact:
407-512-1054

Neuro-Behavioral Clinical Research
North Canton 5164706, Ohio 5165418 44720
Contact:
330-493-1118

Worldwide Clinical Trials
San Antonio 4726206, Texas 4736286 78217
Contact:
210-635-1515

More Details

Status
Recruiting
Sponsor
Fulcrum Therapeutics

Study Contact

Call Center
clinicaltrials@fulcrumtx.com

Detailed Description

The study is designed to assess the pharmacokinetics of the tablet formulation of pociredir under fasted conditions in up to approximately 12 study participants with SCD (Fasted Cohort). An additional cohort of up to approximately 12 study participants with SCD may be enrolled to evaluate the potential effect of food on the PK of the tablet formulation (Fed Cohort). The fasted cohort will be conducted first. The study will include screening period: Day -28 to Day -2; In-patient confinement period: Days -1 to 3; check-in on day -1; single dose of pociredir on day 1; discharge on Day 3; outpatient visit: Day 4; end of study (EOS) visit: Days 8-11 Eligible participants who meet all inclusion and none of the exclusion criteria will be admitted to the clinical site on Day -1, the day before dosing. Participants may be discharged on Day 3 following completion of 48-hour PK sampling or may remain in-clinic through Day 4 if needed. Fasted Cohort: Participants will fast for at least 10 hours prior to dosing and remain fasted for 4 hours post-dose. Water intake will be restricted for 1 hour before and after administration of the investigational medicinal product (IMP). Fed Cohort (if conducted): Participants will fast for at least 10 hours prior to breakfast and will receive a standard high-fat breakfast starting 30 minutes before dosing. Pharmacokinetic samples will be collected up to 72 hours post-dose. Participants are required to remain in-clinic for the first 48 hours post-dose and may return to the site for subsequent Outpatient and EOS visits.

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.