Anemia, Sickle CellPhase 1NCT03926819
A Study to Assess the Safety and Pharmacokinetics of HBI-002, an Oral Carbon Monoxide Therapeutic, in Healthy Volunteers
A Phase 1 study testing HBI-002 in people with Anemia, Sickle Cell, run by Hillhurst Biopharmaceuticals, Inc. at 1 site in United States.
Trial summary
This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days. Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Anemia, Sickle Cell
- What is being tested?
- HBI-002
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Jul 2022to 26 Apr 2023
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Signed informed consent.
- Healthy male or female 18-55 years of age inclusive.
- Negative HBsAg, aHCV, aHIV, and SARS-CoV-2 test.
- Non-smoker or vaper (no use of tobacco or marijuana products within 3 months of screening).
- Body weight between 60 kg and 110 kg (inclusive) and with BMI less than 30 kg/m2.
- Subjects must be healthy as defined by:
Show all 17Show fewer
- absence of clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic disease, as determined by the Investigator.
- liver function: alanine transaminase (ALT) and aspartate transaminase (AST) ≤2 times the upper limit of the normal range
- total bilirubin ≤1.5 times the upper limit of the normal range
- renal function: creatinine clearance within normal range as assessed by Cockcroft and Gault calculation
- carboxyhemoglobin level by venous blood gas ≤ 3.5% (any time prior to the first dose)
- venous lactate level <2.0 mmol/L at baseline.
- the absence of current clinically relevant abnormalities identified by a detailed medical history, full physical examination including blood pressure and pulse rate measurement, 12-lead ECG, and clinical laboratory tests (hematology and clinical chemistries), as determined by the Investigator.
- Negative pregnancy tests for females.
- Subjects must be willing to use a highly effective method of contraception for the duration of the study and for 30 days thereafter.
- Male subjects, without a vasectomy, must use a condom and be instructed that their female partner should use another form of contraception such as an IUD, diaphragm with spermicide, oral contraceptive, injectable progesterone, subdermal implant or a tubal ligation if the female partner could become pregnant
- Female subjects of childbearing potential (not surgically sterilized and less than one year post-menopausal) should use a form of contraception such as an IUD, diaphragm with spermicide, oral contraceptive, injectable progesterone, subdermal implant or a tubal ligation, and be instructed that their male partners should use a condom, if not vasectomized.
Who cannot join
- Subjects who meet any of the following criteria will be ineligible for participation in the study:
- Subjects with concurrent illness/disease as defined by:
- clinically significant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic or allergic disease, as determined by the Investigator.
- current clinically relevant abnormalities identified by a detailed medical history, full physical examination including blood pressure and pulse rate measurement, 12-lead ECG, and clinical laboratory tests (hematology, clinical chemistries and urinalysis), as determined by the Investigator.
- clinically significant illness and/or surgery within 4 weeks prior to dosing.
- Anemia of any cause.
Show all 29Show fewer
- Homozygous or heterozygous hemoglobinopathy.
- Blood transfusion within six weeks prior to the first administration of study drug.
- Carboxyhemoglobin ≥ 3.5% (any time prior to the first dose)
- Oxygen saturation by transcutaneous measurement consistently ≤ 95% (any time prior to the first dose)
- Exposure to any live vaccine within 28 days prior to study drug administration.
- History of febrile or infective illness within 14 days prior to dosing.
- Positive pregnancy test or breast feeding for females.
- Weight loss or gain of more than 5 kg within 3 months prior to dosing.
- History of alcohol abuse or dependence or regular use of alcohol within six months prior to dosing (defined as more than 14 units of alcohol per week; 1 Unit= 150 mL wine, 360 mL beer or 45 mL of 40% alcohol)
- Positive result on alcohol screen
- History of pulmonary infiltrate or pneumonia within 6 months prior to dosing or pulmonary/bronchial infection within 2 weeks prior to dosing.
- History of cancer, with the exception of adequately treated basal cell or squamous cell carcinoma of the skin more than 1 year prior.
- History of cardiac disease
- History of drug abuse or dependence.
- Positive results on drug screen (oxycodone, benzodiazepines, THC, cocaine, opiates, and methamphetamine).
- Use of prescription drugs within 7 days or 5 half-lives (whichever is longer) prior to dosing. Herbal and vitamin supplements must be discontinued 14 days prior to dosing.
- Unwilling or unable to comply with the requirements of the protocol.
- Treatment with an investigational drug within the longer of 30 days or five half-lives.
- Systolic blood pressure lower than 90 or above 140 mm Hg, diastolic blood pressure lower than 50 or above 90 mm Hg, heart rate less than 45 or above 100 bpm, or arrhythmia at screening and/or baseline. ECG abnormalities or other vital sign abnormalities that are clinically significant at screening and/or baseline, as determined by the Investigator.
- Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into the study.
- History of allergic reactions to any of the drug product excipients
- History of epilepsy or seizure
- History of suicide attempts or ideation (by medical history)
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase 1 Open Labeled Single Ascending Dose Followed by Multiple Dose Safety and Pharmacokinetic Study of HBI-002 Carbon Monoxide Oral Liquid Drug Product in Healthy Adult Volunteers.
This is a single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days.
- Conditions
- Anemia, Sickle Cell
- Being tested
- HBI-002
- Keywords
- Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Anemia, Hemolytic, Anemia, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Carbon Monoxide, CO, Heme Oxygenase, HO-1
- Medical subject headings
- Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Anemia, Hemolytic, Anemia, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HBI-002Drug
Oral liquid carbon monoxide drug product.
| Study group | Role | What they receive |
|---|---|---|
| Single Ascending Dose | Active comparator | Not described Receives: Drug: HBI-002 |
| Multiple Ascending Dose | Active comparator | Not described Receives: Drug: HBI-002 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Measured: Day 1 of dosing, up to 30 days post-dose for SAD cohorts. Day 1 of dosing to 30 days post last dose, up to 37 days for MAD cohort
Cmax
Maximum COHb Concentration (Cmax), measured by blood gas machine with co-oximetry
Measured: Blood samples will be drawn on Day 1 immediately before and after dosing at 30, 60, 90, 120 minutes and at 3, 4, 5, 6, 24, 48, and 72 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2019
- Study start25 Jul 2022
- Primary completion26 Apr 2023
- Study completion26 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Anemia, Sickle CellStudies 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
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hillhurst Biopharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03926819
- Sponsor's protocol ID
- HBI-002-001
- Other IDs
- 2R44HL131065
- Status last verified
- May 2025
- First submitted
- 22 Apr 2019
- Last updated
- 23 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- 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 23 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open Labeled Single Ascending Dose Followed by Multiple Dose Safety and Pharmacokinetic Study of HBI-002 Carbon Monoxide Oral Liquid Drug Product in Healthy Adult Volunteers.
- ClinicalTrials.gov ID
- NCT03926819
- Acronym / sponsor ID
- HBI-002-001
- Conditions
- Anemia, Sickle Cell
- Keywords
- Anemia, Sickle Cell, Anemia, Hemolytic, Congenital, Anemia, Hemolytic, Anemia, Hematologic Diseases, Hemoglobinopathies, Genetic Diseases, Inborn, Carbon Monoxide
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Hillhurst Biopharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- 25 Jul 2022 (actual)
- Primary completion
- 26 Apr 2023 (actual)
- Study completion
- 26 Apr 2023 (actual)
- First posted
- 25 Apr 2019 (actual)
- Results
- Posted 23 May 2025
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 single center, open label Phase 1 clinical trial in normal adult subjects to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with single ascending doses (SAD), followed by multiple dose with doses daily for 7 days.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Single Ascending Dose | Active comparator | Drug: HBI-002 |
| Multiple Ascending Dose | Active comparator | Drug: HBI-002 |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (17 listed)
- Signed informed consent.
- Healthy male or female 18-55 years of age inclusive.
- Negative HBsAg, aHCV, aHIV, and SARS-CoV-2 test.
- Non-smoker or vaper (no use of tobacco or marijuana products within 3 months of screening).
- Body weight between 60 kg and 110 kg (inclusive) and with BMI less than 30 kg/m2.
Key exclusion criteria (29 listed)
- Subjects who meet any of the following criteria will be ineligible for participation in the study:
- Subjects with concurrent illness/disease as defined by:
- Anemia of any cause.
- Homozygous or heterozygous hemoglobinopathy.
- Blood transfusion within six weeks prior to the first administration of study drug.
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 |
|---|---|---|
| HBI-002Oral liquid carbon monoxide drug product. | Drug | Multiple Ascending Dose, Single Ascending Dose |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Time frame: Day 1 of dosing, up to 30 days post-dose for SAD cohorts. Day 1 of dosing to 30 days post last dose, up to 37 days for MAD cohort
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs)
Cmax
Time frame: Blood samples will be drawn on Day 1 immediately before and after dosing at 30, 60, 90, 120 minutes and at 3, 4, 5, 6, 24, 48, and 72 hours
Maximum COHb Concentration (Cmax), measured by blood gas machine with co-oximetry
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Anemia, Sickle Cell
Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis
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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing HBI-002 in people with Anemia, Sickle Cell, run by Hillhurst Biopharmaceuticals, Inc. at 1 site 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 17 inclusion and 29 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 23 May 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 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?
Yes. Results are posted in the registry (first posted 23 May 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 With Treatment-Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) (time frame: Day 1 of dosing, up to 30 days post-dose for SAD cohorts. Day 1 of dosing to 30 days post last dose, up to 37 days for MAD cohort). 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?
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 23 May 2025; AzTrials last refreshed its copy on 8 Nov 2026.