A Safety, Efficacy, and Pharmacokinetic (PK) Study of HBI-002, an Oral Carbon Monoxide (CO) Therapeutic, in Subjects With Sickle Cell Disease (SCD)

NCT06144749 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: HBI-002-1212R44HL131065-05
• Study Type: interventional
• Target: 9
• Locations: 1 country
• Sites: 2

Brief Summary

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

Anemia, Sickle Cell

Keywords

Anemia, Sickle Cell; Anemia, Hemolytic; Congenital Anemia; Hemolytic Anemia; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn; Carbon Monoxide; CO; Heme Oxygenase; HO-1

Outcome Measures

Primary Outcomes (1)

• Frequency and severity of Treatment Emergent Adverse Events, including adverse events of special interest

  Day 1 to 44

Secondary Outcomes (5)

• Maximum hemoglobin concentration (Cmax) as measured by carboxyhemoglobin (COHb) saturation

  Days 1 and 4

• Elimination half-life (t1/2) as measured by COHb saturation

  Days 1 and 4

• Change from baseline in complete blood count (CBC) test parameters, including hemoglobin, hematocrit, red blood cell (RBC) count, white blood cell (WBC) count, and neutrophil count

  Day 0 to 15

• Change from baseline in inflammation and oxidation, as assessed by levels of protein and/or mRNA indicators, particularly those associated with heme oxygenase-1 (HO-1)

  Day 0 to 15

• Change from baseline in patient pain levels as measured by an emoji faces pain scale with pain assessment from 0 to 10 and with higher scores indicating more severe pain

  Days 1 to 44

Study Arms (1)

Ascending Multiple Dose

EXPERIMENTAL

Drug: HBI-002 (oral liquid carbon monoxide drug product)

Drug: HBI-002

Interventions

HBI-002 DRUG

Oral liquid carbon monoxide drug product.

Ascending Multiple Dose

Eligibility Criteria

• Age: 18 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Signed informed consent.
• Male or female 14-55 years of age inclusive.
• Negative Hepatitis B surface antigen (HBsAg), anti-Hepatitis C (aHCV), anti-Human Immunodeficiency Virus (aHIV), and SARS-CoV-2 test. Subjects that test SARS-CoV-2 positive at screen or baseline can be re-screened at least four weeks after the positive test so long as they are asymptomatic and test negative.
• Non-smoker or vaper (no use of tobacco or marijuana products within 3 months of screening).
• Body weight. For 18 years of age and above, between 45 kg and 110 kg (inclusive) and with body mass index (BMI) less than 30 kg/m2. For 14 to 18 years of age, above 35 kg and with BMI less than 30 kg/m2.
• Established Hb-SS or Sβ0 SCD with:
• ≤10 vaso-occlusive crises (VOC) per year over the prior two years; and
• a history of one or more episodes of one of the following: acute chest syndrome, stroke, priapism, bone avascular necrosis, or splenic sequestration.
• Normal cardiac function as evidenced by clinical, ECG and laboratory findings.
• Carboxyhemoglobin level by co-oximetry ≤ 3.5% (prior to first dose).
• 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 chemistries including liver and kidney function, as determined by the Investigator.
• Hematology including hemoglobin \>6g/dL, baseline white blood cell count \>7,000/uL and absolute neutrophil count (ANC) \>3,500/uL, platelet count \>80,000/uL and including coagulation labs (PT, PTT), with all levels documented as stable or not clinically significant changes over the period between screening and baseline.
• Negative pregnancy test 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, if applicable.
• 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 if the female partner could become pregnant.

You may not qualify if:

• Hemoglobinopathy other than SCD or S/beta 0 thalassemia.
• Clinical, ECG or laboratory evidence of cardiac dysfunction as determined by the PI.
• Acute chest syndrome or VOC within 21 days or signs or symptoms of an impending vaso-occlusive crisis as determined by both the subject and PI immediately prior to first dosing with HBI-002/placebo.
• Current smoker.
• Clinically significant illness or surgery other than that associated with SCD within 3 months prior to dosing.
• Blood transfusion within six weeks prior to the first administration of study drug.
• 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)
• History of renal dysfunction with glomerular filtration rate \<30 mL/min/1.73m2.
• History of pulmonary infiltrate or pneumonia within 6 months prior to dosing or pulmonary/bronchial infection within 2 weeks prior to dosing.
• Subject on domiciliary oxygen
• History of cancer, with the exception of adequately treated basal cell or squamous cell carcinoma of the skin more than 1 year prior.

Sponsors & Collaborators

• Hillhurst Biopharmaceuticals, Inc.: lead
• National Heart, Lung, and Blood Institute (NHLBI): collaborator

Study Sites (2)

Cevaxin - The Panama Clinic

Panama City, Panama

RECRUITING

Hospital Pacífica Salud

Panama City, Panama

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell; Anemia, Hemolytic; Hematologic Diseases; Hemoglobinopathies; Genetic Diseases, Inborn

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, Congenital; Anemia; Hemic and Lymphatic Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Andrew Gomperts

CONTACT

858 232 9495; hillhurstinfo@hillhurstbio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 10, 2023
• First Posted: November 22, 2023
• Study Start: February 15, 2025
• Primary Completion: March 31, 2026
• Study Completion (Estimated): June 30, 2026
• Last Updated: January 8, 2026

Record last verified: 2025-05

Locations

PA (2)