Single-dose Pociredir pharmacokinetics in people with sickle cell disease
A Phase 1, Open-Label Study to Assess Pharmacokinetics After Single Doses of Pociredir in Participants With Sickle Cell Disease
This will see if a single tablet of Pociredir is absorbed and processed differently in people with sickle cell disease when taken on an empty stomach versus after a high-fat meal.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Fulcrum Therapeutics Industry-sponsored |
| Drugs / interventions | crizanlizumab |
| Locations | 6 sites (Miami, Florida and 5 other locations) |
| Trial ID | NCT07431398 on ClinicalTrials.gov |
What this trial studies
This Phase 1, single-dose pharmacokinetic study will enroll up to ~12 participants in a fasted cohort and may enroll an additional ~12 in a fed cohort to compare drug exposure with and without food. Eligible participants are admitted the day before dosing, receive one oral dose on Day 1, remain in-clinic for at least 48 hours for intensive PK sampling, and have follow-up visits through Day 8–11 with PK samples collected up to 72 hours post-dose. Key eligibility includes confirmed sickle cell disease, specified hemoglobin and blood count ranges, and agreement to contraception where applicable. The fed cohort (if conducted) will receive a standard high-fat breakfast starting 30 minutes before dosing to evaluate the food effect on Pociredir absorption.
Who should consider this trial
Good fit: Adults with confirmed sickle cell disease who meet the study's hemoglobin and laboratory criteria and can agree to in-clinic confinement and required contraception are ideal candidates.
Not a fit: People seeking immediate therapeutic benefit, pregnant or breastfeeding individuals, or those with hemoglobin or laboratory values outside the study limits are unlikely to receive direct clinical benefit from this single-dose PK study.
Why it matters
Potential benefit: If successful, the results could guide safe dosing and provide clear instructions about taking Pociredir with or without food for people with sickle cell disease.
How similar studies have performed: Single-dose pharmacokinetic studies are a routine, well-established method and have successfully characterized absorption of other sickle cell drugs, while Pociredir's PK in this population represents an early-stage, product-specific evaluation.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Miami, Florida and 5 other locations
- Advanced Pharma - Miami — Miami, Florida, United States (Recruiting)
- Omega Research Group — Orlando, Florida, United States (Recruiting)
- Sonar Clinical Research — Riverdale, Georgia, United States (Recruiting)
- Neuro-Behavioral Clinical Research — North Canton, Ohio, United States (Recruiting)
- University of Texas Health Science Center Houston — Houston, Texas, United States (Not_yet_recruiting)
- Worldwide Clinical Trials — San Antonio, Texas, United States (Recruiting)
Study contacts
- Study coordinator: Call Center
- Email: clinicaltrials@fulcrumtx.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.