Sickle Cell DiseasePhase 2NCT07401823
Open-Label Extension Study to Pioneer Study 6058-SCD-101
A Phase 2 study testing Pociredir in people with Sickle Cell Disease, run by Fulcrum Therapeutics at 8 sites in United States.
Trial summary
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder study 6058-SCD-101 (NCT05169580). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- Pociredir
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 8 sitesUnited States
- Study dates
- 30 Mar 2026to 5 Jul 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participants aged ≥18 years and older must have previously participated in and successfully completed Study 6058-SCD-101.
- Participant has signed and dated the informed consent form (ICF) before any study-specific procedures are performed and is willing and able to comply with the study procedures and restrictions.
- Participants who meet all other inclusion and exclusion criteria for this study, and per Investigator's recommendation may continue standard of care as indicated with the exception of hydroxyurea (HU). Participants may continue crizanlizumab, and/or L-glutamine, but must be on a stable dose for at least 6 months.
- Participants, who if female and of childbearing potential, agree to use 2 effective methods of contraception, 1 of 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, should use condoms or practice abstinence from the time of ICF signing to 90 days after the last dose of study drug.
- Documented HbF benefit, as judged by the Investigator, from prior study.
- Participant must meet both of the following laboratory values during Screening:
Show all 9Show fewer
- Absolute neutrophil count ≥ 1.5 × 10^9/liter,
- Platelets ≥ 80 × 10^9/liter
- Absolute reticulocyte count during Screening > 100 × 10^9/liter.
Who cannot join
- Major surgery, serious illness (acute or chronic), infection (clinically significant bacterial, fungal, parasitic or viral infection which requires therapy), fever not resolved within 3 days of onset and requiring treatment, significant bleeding, cerebrovascular accident, or seizure within 14 days prior to signing the ICF and 14 days prior to starting study drug.
- Sickle cell complication requiring more than 24-hour care from a medical provider in hospital or emergency care setting in the 14 days prior to starting study drug.
- Use of medications that are moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or CYP2C8, 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 other than pociredir within the past 60 days prior to the first dose of study drug.
- History of bone marrow transplant or hematopoietic stem cell transplant or gene therapies.
- Vaccination in the previous 7 days prior to the first dose of study drug.
- Note: Other protocol specified criteria may apply
Trial locations and contacts
8 sites in 1 country. Contact the study team or a site near you to ask about taking part.
8 sites in 1 country
United States8 sites
Our Lady of the Lake Hospital
Baton Rouge, Louisiana, 70808
Boston Medical Center
Boston, Massachusetts, 02118
Inova Schar Cancer Institute
Fairfax, Virginia, 22031
University of Texas Houston
Houston, Texas, 77030
Queens Hospital Cancer Center
Jamaica, New York, 11432
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
University of California, Los Angeles
Los Angeles, California, 90095
Jacobi Medical Center
The Bronx, New York, 10461
About this study
Official title: An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of Pociredir in Participants With Sickle Cell Disease (SCD) Who Have Participated in a Pociredir Study
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder study 6058-SCD-101 (NCT05169580). Participants in this study will receive once daily doses of pociredir for up to 48 months.
Read the detailed description
The first dose of study drug will be administered on Day 1 in the clinic and participants will continue at home dosing once daily (QD). Dosing will occur in the clinic on days where there are clinic visits. Treatment Period clinic visits are planned every other week through Week 12 (Weeks 2, 4, 6, 8, 10, and 12), monthly through Week 24 (Weeks 16, 20, and 24), and then every 12 weeks from Week 24 through Week 192. A final follow-up visit (Week 196) will occur 4 weeks after the final dose of study drug at Week 192.
Participants will receive pociredir at the dose level they received in Study 6058-SCD-101 through Week 192, unless data from that study indicates a change to a different optimized dose.
- Conditions
- Sickle Cell Disease
- Being tested
- Pociredir
- Keywords
- FTX-6058, Pociredir, Fetal hemoglobin, Sickle Cell Disease, Open-label, Anemia, Sickle Cell, Hematologic diseases
- Medical subject headings
- Anemia, Sickle Cell, beta-Thalassemia, Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PociredirDrug
Also called FTX-6058
Pociredir Oral Capsules will be administered
| Study group | Role | What they receive |
|---|---|---|
| Pociredir | Experimental | Participants will receive Pociredir orally once daily (QD) Receives: Drug: Pociredir |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
Measured: Up to Week 196
Number of participants with clinically significant changes in 12-lead Electrocardiogram (ECGs)
Measured: Up to Week 196
Number of participants with clinically significant changes in Vital signs
Measured: Up to Week 196
Number of participants with clinically significant changes in Clinical laboratory tests
Laboratory assessments including hematology, coagulation, serum chemistry and electrolytes, lipid panel, SCD characterization, serology, urinalysis and pregnancy tests will be performed.
Measured: Up to Week 196
Secondary outcomes (9)
Change from Baseline in percent Fetal hemoglobin (HbF)
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in percent Reticulocytes
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in Red cell distribution width
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in Unconjugated bilirubin
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in Lactate dehydrogenase (LDH)
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in Haptoglobin
Measured: Baseline (Day 1), and Up to Week 192
Change from Baseline in Reticulocyte count
Measured: Baseline (Day 1), and Up to Week 192
Number of participants reporting SCD-related complications
Measured: Up to Week 192
Annualized rate of Vaso-occlusive episode (VOE)
Measured: through end of month 48
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2026
- Study start30 Mar 2026 (estimated)
- Primary completion4 Feb 2030 (estimated)
- Study completion5 Jul 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Fulcrum Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07401823
- Sponsor's protocol ID
- 6058-SCD-102
- Status last verified
- Apr 2026
- First submitted
- 28 Jan 2026
- Last updated
- 14 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of Pociredir in Participants With Sickle Cell Disease (SCD) Who Have Participated in a Pociredir Study
- ClinicalTrials.gov ID
- NCT07401823
- Acronym / sponsor ID
- 6058-SCD-102
- Conditions
- Sickle Cell Disease
- Keywords
- FTX-6058, Pociredir, Fetal hemoglobin, Sickle Cell Disease, Open-label, Anemia, Sickle Cell, Hematologic diseases
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Fulcrum TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 30 Mar 2026 (estimated)
- Primary completion
- 4 Feb 2030 (estimated)
- Study completion
- 5 Jul 2030 (estimated)
- First posted
- 11 Feb 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an open-label study to evaluate the safety and tolerability of long-term treatment with pociredir without a comparator in participants with SCD who have previously been treated and shown benefit with pociredir in feeder study 6058-SCD-101 (NCT05169580). Participants in this study will receive once daily doses of pociredir for up to 48 months.
Detailed description
The first dose of study drug will be administered on Day 1 in the clinic and participants will continue at home dosing once daily (QD). Dosing will occur in the clinic on days where there are clinic visits. Treatment Period clinic visits are planned every other week through Week 12 (Weeks 2, 4, 6, 8, 10, and 12), monthly through Week 24 (Weeks 16, 20, and 24), and then every 12 weeks from Week 24 through Week 192. A final follow-up visit (Week 196) will occur 4 weeks after the final dose of study drug at Week 192.
Participants will receive pociredir at the dose level they received in Study 6058-SCD-101 through Week 192, unless data from that study indicates a change to a different optimized dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pociredir | Experimental | Drug: PociredirParticipants will receive Pociredir orally once daily (QD) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Participants aged ≥18 years and older must have previously participated in and successfully completed Study 6058-SCD-101.
- Participant has signed and dated the informed consent form (ICF) before any study-specific procedures are performed and is willing and able to comply with the study procedures and restrictions.
- Participants who meet all other inclusion and exclusion criteria for this study, and per Investigator's recommendation may continue standard of care as indicated with the exception of hydroxyurea (HU). Participants may continue crizanlizumab, and/or L-glutamine, but must be on a stable dose for at least 6 months.
- Participants, who if female and of childbearing potential, agree to use 2 effective methods of contraception, 1 of 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, should use condoms or practice abstinence from the time of ICF signing to 90 days after the last dose of study drug.
- Documented HbF benefit, as judged by the Investigator, from prior study.
Key exclusion criteria (7 listed)
- Major surgery, serious illness (acute or chronic), infection (clinically significant bacterial, fungal, parasitic or viral infection which requires therapy), fever not resolved within 3 days of onset and requiring treatment, significant bleeding, cerebrovascular accident, or seizure within 14 days prior to signing the ICF and 14 days prior to starting study drug.
- Sickle cell complication requiring more than 24-hour care from a medical provider in hospital or emergency care setting in the 14 days prior to starting study drug.
- Use of medications that are moderate or strong inducers or inhibitors of cytochrome P450 (CYP) 3A4 or CYP2C8, 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 other than pociredir within the past 60 days prior to the first dose of study drug.
- History of bone marrow transplant or hematopoietic stem cell transplant or gene therapies.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PociredirAlso: FTX-6058Pociredir Oral Capsules will be administered | Drug | Pociredir |
Primary outcome
Number of participants reporting Treatment Emergent Adverse Events (TEAEs)
Time frame: Up to Week 196
Number of participants with clinically significant changes in 12-lead Electrocardiogram (ECGs)
Time frame: Up to Week 196
Number of participants with clinically significant changes in Vital signs
Time frame: Up to Week 196
Number of participants with clinically significant changes in Clinical laboratory tests
Time frame: Up to Week 196
Laboratory assessments including hematology, coagulation, serum chemistry and electrolytes, lipid panel, SCD characterization, serology, urinalysis and pregnancy tests will be performed.
9 secondary outcomes
Change from Baseline in percent Fetal hemoglobin (HbF)
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in percent Reticulocytes
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Red cell distribution width
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Unconjugated bilirubin
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Lactate dehydrogenase (LDH)
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Haptoglobin
Time frame: Baseline (Day 1), and Up to Week 192
Change from Baseline in Reticulocyte count
Time frame: Baseline (Day 1), and Up to Week 192
Number of participants reporting SCD-related complications
Time frame: Up to Week 192
Annualized rate of Vaso-occlusive episode (VOE)
Time frame: through end of month 48
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
The record lists no contact. Use the official record to reach the sponsor.
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pociredir in people with Sickle Cell Disease, run by Fulcrum Therapeutics at 8 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: enrolling by invitation, as of the sponsor's last update on 14 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants reporting Treatment Emergent Adverse Events (TEAEs) (time frame: Up to Week 196). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Apr 2026.