RECRUITING

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir

Conditions

Sickle Cell Disease Sickle Cell Anemia Pharmacokinetics Pharmacodynamics Pociredir Open label

Quick Navigation

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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)

Quick Facts

Study Start:2021-12-13

Study Completion:2025-12

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT05169580

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.

Ages Eligible for Study:18 Years to 65 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* 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: 1. ≥4 episodes of SCD pain crisis over 12 months, or ≥2 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 * Participants who previously received voxelotor, crizanlizumab, and/ or L-glutamine and will be discontinuing before starting dosing with pociredir: 1. Must be off voxelotor and crizanlizumab for at least 60 days prior to initiating study drug; and 2. Must be off L-glutamine for at least 24-hours prior to initiating study drug. * 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.	* Sickle cell complication requiring care from a medical provider in the 14 days prior to starting study drug. * 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. * 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.

Inclusion Criteria

Exclusion Criteria

* 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:
1. ≥4 episodes of SCD pain crisis over 12 months, or ≥2 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
* Participants who previously received voxelotor, crizanlizumab, and/ or L-glutamine and will be discontinuing before starting dosing with pociredir:
1. Must be off voxelotor and crizanlizumab for at least 60 days prior to initiating study drug; and
2. Must be off L-glutamine for at least 24-hours prior to initiating study drug.
* 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.

* Sickle cell complication requiring care from a medical provider in the 14 days prior to starting study drug.
* 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.
* 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.

Contacts and Locations

Study Contact

Call Center

CONTACT

617-651-8853

clinicaltrials@fulcrumtx.com

Principal Investigator

Thomas Winkler, MD

STUDY_DIRECTOR

Fulcrum Therapeutics

Study Locations (Sites)

University of Arkansas for Medical Sciences (UAMS)

Little Rock, Arkansas, 72205

United States

University of California, Los Angeles

Los Angeles, California, 90095

United States

Foundation for Sickle Cell Disease Research, LLC

Hollywood, Florida, 33021

United States

University of Miami Health System

Miami, Florida, 33136

United States

Sonar Research Center

Atlanta, Georgia, 30315

United States

Visionaries Clinical Research

Atlanta, Georgia, 30329

United States

Atlanta Center for Medical Research

Atlanta, Georgia, 30331

United States

Augusta University

Augusta, Georgia, 30912

United States

Our Lady of the Lake Hospital

Baton Rouge, Louisiana, 70808

United States

Axon Clinical Research Institute

Baltimore, Maryland, 21237

United States

Boston Medical Center

Boston, Massachusetts, 02118

United States

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110

United States

Jacobi Medical Center

Bronx, New York, 10461

United States

Queens Hospital Cancer Center

Jamaica, New York, 11432

United States

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599

United States

Lynn Health Science Institute

Oklahoma City, Oklahoma, 73112

United States

University of Texas Houston

Houston, Texas, 77030

United States

Inova Schar Cancer Institute

Fairfax, Virginia, 22031

United States

Virginia Commonwealth University

Richmond, Virginia, 23298

United States

Collaborators and Investigators

Sponsor: Fulcrum Therapeutics

Thomas Winkler, MD, STUDY_DIRECTOR, 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 Registration Dates

Study Start Date2021-12-13

Study Completion Date2025-12

Study Record Updates

Study Start Date2021-12-13

Study Completion Date2025-12

Terms related to this study

Keywords Provided by Researchers

Sickle Cell Disease
Sickle Cell Anemia
Pharmacokinetics
Pharmacodynamics
Pociredir
Open label

Additional Relevant MeSH Terms

Sickle Cell Disease
Sickle Cell Anemia