Single-dose Pharmacokinetics of Pociredir in Participants With Sickle Cell Disease

June 16, 2026 updated by: Fulcrum Therapeutics

A Phase 1, Open-Label Study to Assess Pharmacokinetics After Single Doses of Pociredir in Participants With Sickle Cell Disease

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
13

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Florida
      • Miami, Florida, United States, 33147
        • Advanced Pharma - Miami
      • Orlando, Florida, United States, 32808
        • OMEGA Research Group
    • Georgia
      • Riverdale, Georgia, United States, 30274
        • Sonar Clinical Research
    • Ohio
      • North Canton, Ohio, United States, 44720
        • Neuro-Behavioral Clinical Research
    • Texas
      • Houston, Texas, United States, 77030
        • University of Texas Health Science Center Houston
      • San Antonio, Texas, United States, 78217
        • Worldwide Clinical Trials

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Plasma concentration of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Maximum plasma concentration (Cmax) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Area under the plasma concentration-time curve from time 0 to 24 hours (AUC(0-24)) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Area under the plasma concentration-time curve from time 0 to the last measurable concentration (AUC(0-tlast)) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Area under the plasma concentration-time curve from time 0, extrapolated to infinity (AUC(0-inf)), of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Time to maximum plasma concentration (Tmax) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Terminal disposition rate constant (λz) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Terminal half-life (t1/2) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Apparent volume of distribution during the terminal phase (Vz/F) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Apparent clearance (CL/F) of pociredir under fasted and fed conditions
Time Frame: Day 1 through Day 4
Day 1 through Day 4

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Number of participants with treatment emergent adverse events (TEAEs) under fasted and fed conditions
Time Frame: Up to Day 11
Up to Day 11
Number of participants with clinically significant changes in safety assessments under fasted and fed conditions
Time Frame: Up to Day 11
Up to Day 11
Number of participants with clinically significant findings in clinical laboratory evaluations, vital signs, electrocardiogram (ECGs) and physical examination
Time Frame: Up to Day 11
Up to Day 11
Plasma concentration of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Cmax of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
AUC(0-24) of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
AUC(0-tlast) of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
AUC(0-inf), under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Tmax of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
λz of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
t1/2 of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
Vz/F of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4
CL/F of pociredir under fasted and fed conditions compared to healthy participants
Time Frame: Day 1 through Day 4
Day 1 through Day 4

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Fulcrum Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 13, 2025

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2026

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 1, 2026

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
February 18, 2026

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
February 18, 2026

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
February 24, 2026

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 18, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 16, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
June 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 6058-CLP-104

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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