Sickle Cell DiseasePhase 1NCT05169580
Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Pociredir
A Phase 1 study testing Pociredir oral capsule(s) in people with Sickle Cell Disease and Sickle Cell Anemia, run by Fulcrum Therapeutics at 18 sites in 3 countries.
Trial summary
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Anemia
- What is being tested?
- Pociredir oral capsule(s)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 45 participants
- Where
- 18 sitesin 3 countries
- Study dates
- 13 Dec 2021to 20 Jan 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- ≥4 to 10 episodes of SCD pain crisis over 12 months, or ≥2 to 5 over 6 months prior to screening
- ≥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
- ≥2 of the following events over the previous 12 months:i. ACS ii. Hepatic or splenic sequestration iii. Priapism
Show all 18Show fewer
- 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;
- SCD-related chronic kidney disease (CKD)
- 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
- Previous experience with a stable dose of voxelotor, crizanlizumab, and/ or L-glutamine but have shown to be unresponsive and/or intolerant or ineligible
- Per Investigator's recommendation, participants may continue crizanlizumab and/or L-glutamine but must be on a stable dose for at least 6 months
- 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:
- Absolute neutrophil count ≥ 1.5 × 10^9 per liter (/l)
- Platelets ≥ 80 × 10^9/l
- Absolute reticulocyte count at screening ≥ 100 x 10^9/l.
Who cannot join
- Sickle cell complication requiring care from a medical provider in hospital or emergency care setting 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 within 60 days prior to initiating study drug.
- 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.
Trial locations and contacts
18 sites in 3 countries.
18 sites in 3 countries
United States14 sites
Sonar Research Center
Atlanta, Georgia, 30315
Our Lady of the Lake Hospital
Baton Rouge, Louisiana, 70808
Boston Medical Center
Boston, Massachusetts, 02118
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599
University of Illinois Chicago
Chicago, Illinois, 60612
Inova Schar Cancer Institute
Fairfax, Virginia, 22031
Eastern Carolina University
Greenville, North Carolina, 27834
University of Texas Houston
Houston, Texas, 77030
Queens Hospital Cancer Center
Jamaica, New York, 11432
University of Arkansas for Medical Sciences (UAMS)
Little Rock, Arkansas, 72205
University of California, Los Angeles
Los Angeles, California, 90095
Lynn Health Science Institute
Oklahoma City, Oklahoma, 73112
Virginia Commonwealth University
Richmond, Virginia, 23298
Jacobi Medical Center
The Bronx, New York, 10461
Nigeria3 sites
National Hospital, Abuja
Abuja, 900247
University of Ibadan
Ibadan, 200212
Barau Dikko Teaching Hospital
Kaduna, 800125
South Africa1 site
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, 2193
About this study
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)
This is a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
Read the detailed description
This is a Phase 1 multicenter, international, open-label study evaluating the safety, tolerability, pharmacokinetics (PK), fetal hemoglobin (HbF) induction and biological activity of Pociredir in participants 18-65 years of age, inclusive, with SCD.
Participants will receive 12 weeks of dosing with 4 weeks of follow-up. Approximately 10 participants will be enrolled in each cohort. A maximum of 3 participants with SCD HbSC+ genotype may be enrolled in each cohort.
Cohort 1 will receive 6 milligrams (mg) of Pociredir by mouth once daily. Doses for subsequent cohorts will be determined following review by the Data Monitoring Committee [DMC]. A total of seven cohorts may be included. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. Additional cohorts using alternative dosing schedules may be considered based on available data.
The primary endpoints of the study are to evaluate the safety and tolerability of Pociredir as measured by the frequency of adverse events and to evaluate single and multiple-dose pharmacokinetics of Pociredir in participants with sickle cell disease. Secondary endpoints include evaluating the effect of Pociredir on fetal hemoglobin induction in peripheral blood and evaluating the effects of Pociredir on hemolysis in participants with sickle cell disease.
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Being tested
- Pociredir oral capsule(s)
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Pharmacokinetics, Pharmacodynamics, Pociredir, Open label
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Pociredir oral capsule(s)Drug
Also called FTX-6058
Participants will receive Pociredir
| Study group | Role | What they receive |
|---|---|---|
| Pociredir oral capsule(s) in Sickle Cell participants | Experimental | Cohort 1 will receive 6 mg of Pociredir by mouth once daily. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. A total of seven cohorts may be included. Following the first cohort, doses for all subsequent cohorts will be determined following DMC review of the safety and pharmacokinetic data observed in participants from the prior and ongoing cohorts. Alternate dosing schedules may be evaluated in some of the cohorts. Receives: Drug: Pociredir oral capsule(s) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-Emergent Adverse Events
To evaluate the safety and tolerability of Pociredir in adult participants with sickle cell disease based on the frequency of adverse events (AEs) and changes in clinically significant laboratory test results, vital signs and electrocardiograms (ECGs) parameters.
Measured: Up to approximately 16 weeks of monitoring
Plasma Concentrations of Pociredir
Blood samples will be collected to measure the plasma concentration of Pociredir at specified timepoints.
Measured: Days 1, 14, 28, 42, 56, 70, 84 and 91
Secondary outcomes (5)
Change from Baseline in percentage fetal hemoglobin (%HbF) biomarkers in peripheral blood
The percentage of HbF will be measured in peripheral whole blood.
Measured: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in % Reticulocytes
The percentage of reticulocytes will be measured in peripheral whole blood.
Measured: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in Absolute Reticulocyte Count
The absolute reticulocyte count will be measured in peripheral whole blood.
Measured: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in Red cell distribution width
Blood samples will be collected for the analysis of hematology parameter: red cell distribution width
Measured: Baseline and at Days 1, 14, 28, 42, 56, 70, 84 and 91
Change from Baseline in unconjugated bilirubin
Blood samples will be collected for the analysis of clinical chemistry parameter: unconjugated bilirubin
Measured: Baseline and at Days 1, 14, 28, 42, 56, 70, 84 and 91
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Dec 2021
- Study start13 Dec 2021
- Primary completion20 Jan 2026
- Study completion20 Jan 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Model description
- Open-label, multiple-dose study.
- Masking
- None (open label)
- Enrolment
- 45 participants (actual)
Sponsor and investigators
- Lead sponsor
- Fulcrum Therapeutics
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Adeyemi Adenola, MD, Study director, Fulcrum Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05169580
- Sponsor's protocol ID
- 6058-SCD-101
- Status last verified
- Feb 2026
- First submitted
- 14 Dec 2021
- Last updated
- 12 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Ngo DA, Aygun B, Akinsheye I, Hankins JS, Bhan I, Luo HY, Steinberg MH, Chui DH. Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin. Br J Haematol. 2012 Jan;156(2):259-64. doi: 10.1111/j.1365-2141.2011.08916.x. Epub 2011 Oct 24.
PubMed 22017641Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
PubMed 28423290Sankaran VG, Orkin SH. The switch from fetal to adult hemoglobin. Cold Spring Harb Perspect Med. 2013 Jan 1;3(1):a011643. doi: 10.1101/cshperspect.a011643.
PubMed 23209159Saraf SL, Molokie RE, Nouraie M, Sable CA, Luchtman-Jones L, Ensing GJ, Campbell AD, Rana SR, Niu XM, Machado RF, Gladwin MT, Gordeuk VR. Differences in the clinical and genotypic presentation of sickle cell disease around the world. Paediatr Respir Rev. 2014 Mar;15(1):4-12. doi: 10.1016/j.prrv.2013.11.003. Epub 2013 Nov 15.
PubMed 24361300Steinberg MH, Chui DH, Dover GJ, Sebastiani P, Alsultan A. Fetal hemoglobin in sickle cell anemia: a glass half full? Blood. 2014 Jan 23;123(4):481-5. doi: 10.1182/blood-2013-09-528067. Epub 2013 Nov 12.
PubMed 24222332Abbasi M, Srivastava A, Saraf SL. Management of Kidney Disease with Sickle Cell Disease. J Am Soc Nephrol. 2025 Oct 1;36(10):2041-2054. doi: 10.1681/ASN.0000000804. Epub 2025 Jun 26.
PubMed 40569673
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT05169580
- Acronym / sponsor ID
- 6058-SCD-101
- Conditions
- Sickle Cell Disease, Sickle Cell Anemia
- Keywords
- Sickle Cell Disease, Sickle Cell Anemia, Pharmacokinetics, Pharmacodynamics, Pociredir, Open label
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 45 participants (actual)
- Lead sponsor
- Fulcrum TherapeuticsIndustry (pharma, biotech, devices)
- Study start
- 13 Dec 2021 (actual)
- Primary completion
- 20 Jan 2026 (actual)
- Study completion
- 20 Jan 2026 (actual)
- First posted
- 27 Dec 2021 (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 a study to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of Pociredir in participants with sickle cell disease.
Detailed description
This is a Phase 1 multicenter, international, open-label study evaluating the safety, tolerability, pharmacokinetics (PK), fetal hemoglobin (HbF) induction and biological activity of Pociredir in participants 18-65 years of age, inclusive, with SCD.
Participants will receive 12 weeks of dosing with 4 weeks of follow-up. Approximately 10 participants will be enrolled in each cohort. A maximum of 3 participants with SCD HbSC+ genotype may be enrolled in each cohort.
Cohort 1 will receive 6 milligrams (mg) of Pociredir by mouth once daily. Doses for subsequent cohorts will be determined following review by the Data Monitoring Committee [DMC]. A total of seven cohorts may be included. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. Additional cohorts using alternative dosing schedules may be considered based on available data.
The primary endpoints of the study are to evaluate the safety and tolerability of Pociredir as measured by the frequency of adverse events and to evaluate single and multiple-dose pharmacokinetics of Pociredir in participants with sickle cell disease. Secondary endpoints include evaluating the effect of Pociredir on fetal hemoglobin induction in peripheral blood and evaluating the effects of Pociredir on hemolysis in participants with sickle cell disease.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Open-label, multiple-dose study.
| Arm | Type | Receives |
|---|---|---|
| Pociredir oral capsule(s) in Sickle Cell participants | Experimental | Drug: Pociredir oral capsule(s)Cohort 1 will receive 6 mg of Pociredir by mouth once daily. Cohort 2 will be dosed at 2 mg once daily by mouth, and cohort 3 will be dosed at 12 mg once daily by mouth. The Sponsor will reinitiate enrolment in the 3rd cohort (12 mg cohort) with the updated inclusion and exclusion criteria. Based on review of available safety and biomarker data and with the recommendation of the DMC, a subsequent 4th cohort of 20 mg and potentially a 5th cohort of 30 mg may be initiated. A total of seven cohorts may be included. Following the first cohort, doses for all subsequent cohorts will be determined following DMC review of the safety and pharmacokinetic data observed in participants from the prior and ongoing cohorts. Alternate dosing schedules may be evaluated in some of the cohorts. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 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:
- Previous experience with Hydroxyurea (HU) but have shown to be unresponsive and/or intolerant or ineligible AND
- Previous experience with a stable dose of voxelotor, crizanlizumab, and/ or L-glutamine but have shown to be unresponsive and/or intolerant or ineligible
Key exclusion criteria (6 listed)
- Sickle cell complication requiring care from a medical provider in hospital or emergency care setting 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 within 60 days prior to initiating study drug.
- 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).
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 |
|---|---|---|
| Pociredir oral capsule(s)Also: FTX-6058Participants will receive Pociredir | Drug | Pociredir oral capsule(s) in Sickle Cell participants |
Primary outcome
Treatment-Emergent Adverse Events
Time frame: Up to approximately 16 weeks of monitoring
To evaluate the safety and tolerability of Pociredir in adult participants with sickle cell disease based on the frequency of adverse events (AEs) and changes in clinically significant laboratory test results, vital signs and electrocardiograms (ECGs) parameters.
Plasma Concentrations of Pociredir
Time frame: Days 1, 14, 28, 42, 56, 70, 84 and 91
Blood samples will be collected to measure the plasma concentration of Pociredir at specified timepoints.
5 secondary outcomes
Change from Baseline in percentage fetal hemoglobin (%HbF) biomarkers in peripheral blood
Time frame: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in % Reticulocytes
Time frame: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in Absolute Reticulocyte Count
Time frame: Baseline and at Days 1, 14, 28, 42, 56, 70, 84, 91, and 112
Change from Baseline in Red cell distribution width
Time frame: Baseline and at Days 1, 14, 28, 42, 56, 70, 84 and 91
Change from Baseline in unconjugated bilirubin
Time frame: Baseline and at Days 1, 14, 28, 42, 56, 70, 84 and 91
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2026.
- Background: PubMed 22017641Ngo DA, Aygun B, Akinsheye I, Hankins JS, Bhan I, Luo HY, Steinberg MH, Chui DH. Fetal haemoglobin levels and haematological characteristics of compound heterozygotes for haemoglobin S and deletional hereditary persistence of fetal haemoglobin. Br J Haematol. 2012 Jan;156(2):259-64. doi: 10.1111/j.1365-2141.2011.08916.x. Epub 2011 Oct 24.
- Background: PubMed 28423290Piel FB, Steinberg MH, Rees DC. Sickle Cell Disease. N Engl J Med. 2017 Apr 20;376(16):1561-1573. doi: 10.1056/NEJMra1510865. No abstract available.
- Background: PubMed 23209159Sankaran VG, Orkin SH. The switch from fetal to adult hemoglobin. Cold Spring Harb Perspect Med. 2013 Jan 1;3(1):a011643. doi: 10.1101/cshperspect.a011643.
- Background: PubMed 24361300Saraf SL, Molokie RE, Nouraie M, Sable CA, Luchtman-Jones L, Ensing GJ, Campbell AD, Rana SR, Niu XM, Machado RF, Gladwin MT, Gordeuk VR. Differences in the clinical and genotypic presentation of sickle cell disease around the world. Paediatr Respir Rev. 2014 Mar;15(1):4-12. doi: 10.1016/j.prrv.2013.11.003. Epub 2013 Nov 15.
- Background: PubMed 24222332Steinberg MH, Chui DH, Dover GJ, Sebastiani P, Alsultan A. Fetal hemoglobin in sickle cell anemia: a glass half full? Blood. 2014 Jan 23;123(4):481-5. doi: 10.1182/blood-2013-09-528067. Epub 2013 Nov 12.
- Derived from this study: PubMed 40569673Abbasi M, Srivastava A, Saraf SL. Management of Kidney Disease with Sickle Cell Disease. J Am Soc Nephrol. 2025 Oct 1;36(10):2041-2054. doi: 10.1681/ASN.0000000804. Epub 2025 Jun 26.
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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. This study is not recruiting at the moment.
Adeyemi Adenola, MD study director
Fulcrum Therapeutics
18 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Pociredir oral capsule(s) in people with Sickle Cell Disease and Sickle Cell Anemia, run by Fulcrum Therapeutics at 18 sites in 3 countries.
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 18 inclusion and 6 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: finished, as of the sponsor's last update on 12 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in United States, Nigeria, South Africa. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Treatment-Emergent Adverse Events (time frame: Up to approximately 16 weeks of monitoring). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05169580, which also lists 6 linked publications.
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 12 Feb 2026.