Actively Recruiting
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
Led by Hillhurst Biopharmaceuticals, Inc. · Updated on 2026-01-08
9
Participants Needed
2
Research Sites
71 weeks
Total Duration
On this page
Sponsors
H
Hillhurst Biopharmaceuticals, Inc.
Lead Sponsor
N
National Heart, Lung, and Blood Institute (NHLBI)
Collaborating Sponsor
AI-Summary
What this Trial Is About
This is a multi-center, open label Phase 2a clinical trial in subjects with sickle cell disease to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of HBI-002, an orally administered liquid containing carbon monoxide (CO), with doses daily for 14 days.
CONDITIONS
Official Title
A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent.
- Male or female aged 14 to 55 years inclusive.
- Negative tests for Hepatitis B surface antigen, anti-Hepatitis C, anti-Human Immunodeficiency Virus, and SARS-CoV-2.
- Non-smoker and non-vaper (no tobacco or marijuana use within 3 months before screening).
- Body weight between 45 and 110 kg with BMI under 30 kg/m2 for adults; over 35 kg with BMI under 30 kg/m2 for ages 14 to 18.
- Confirmed Hb-SS or Sβ0 sickle cell disease with 10 or fewer vaso-occlusive crises per year over the past two years and history of acute chest syndrome, stroke, priapism, bone avascular necrosis, or splenic sequestration.
- Stable use or non-use of hydroxyurea for at least one month prior to study.
- Normal heart function based on clinical, ECG, and lab results.
- Carboxyhemoglobin level of 3.5% or less before first dose.
- No significant medical abnormalities based on medical history, physical exam, ECG, and lab tests.
- Hemoglobin greater than 6 g/dL, white blood cell count over 7,000/uL, neutrophil count over 3,500/uL, platelet count over 80,000/uL, and stable coagulation labs.
- Negative pregnancy test for females.
- Willingness to use effective contraception during the study and for 30 days after, with specific methods required based on gender and reproductive status.
You will not qualify if you...
- Hemoglobinopathies other than sickle cell disease or Sβ0 thalassemia.
- Evidence of heart dysfunction based on clinical, ECG, or lab findings.
- Acute chest syndrome or vaso-occlusive crisis within 21 days before first dosing or signs of impending crisis.
- Current smoker.
- Significant illness or surgery unrelated to sickle cell disease within 3 months before dosing.
- Blood transfusion within 6 weeks before first dose.
- Live vaccine exposure within 28 days before dosing.
- Febrile or infectious illness within 14 days before dosing.
- Positive pregnancy test or breastfeeding.
- Weight change over 5 kg within 3 months before dosing.
- History of alcohol abuse or regular alcohol use exceeding 14 units per week within 6 months.
- Renal dysfunction with glomerular filtration rate below 30 mL/min/1.73m2.
- Pulmonary infiltrate or pneumonia within 6 months or bronchial infection within 2 weeks before dosing.
- Use of domiciliary oxygen.
- History of cancer except treated basal or squamous cell skin carcinoma over 1 year ago.
- History of cardiac disease.
- History of drug abuse except opioid use for pain control.
- Use of certain prescription drugs (voxelotor, crizanlizumab) or investigational drugs within specified time frames before dosing.
- Unwillingness or inability to comply with study protocol.
- Clinically significant ECG or vital sign abnormalities at screening or baseline.
- Any other severe medical or psychiatric condition or laboratory abnormality increasing risk or interfering with study participation.
- History of allergic reactions to study drug components.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Cevaxin - The Panama Clinic
Panama City, Panama
Actively Recruiting
2
Hospital Pacífica Salud
Panama City, Panama
Actively Recruiting
Research Team
A
Andrew Gomperts
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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