A Study to Evaluate BBI-001 in Hereditary Haemochromatosis (HH) Patients and Iron Deficient Volunteers

Study Overview
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Brief Summary
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This is a first in human, double-blind, randomized, placebo-controlled, two-arm crossover study evaluating the safety, tolerability and PD of ascending dose levels of BBI-001 after: a single administration in iron deficient male and female participants, and male and female HH patients (Part A), two administrations per day in HH patients (Part B). BBI-001 administrations will be accompanied with consumption of a meal enriched with stable iron isotope Fe57, while corresponding placebo dose administrations will be with a meal enriched with stable iron isotope Fe58.

Official Title
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A Phase Ia/Ib Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability and Pharmacodynamics of BBI-001 in Iron Deficient Volunteers and HH Patients

Conditions
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Hereditary Hemochromatosis

Intervention / Treatment
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* Drug: BBI-001
* Dietary Supplement: Original Fibre Metamucil


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Otherwise healthy iron deficient participants or patients with hereditary hemochromatosis Exclusion Criteria: Serious or unstable medical or psychiatric conditions Significant medical history Current infections Receiving iron chelation therapy or treatment other than stable maintenance phlebotomy for the prior 6 months Organ damage from iron overload in the view of the PI would prevent successful completion of the protocol

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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Accepts Healthy Volunteers

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BBI-001<br>BBI-001: Oral administration immediately prior to a meal enriched with stable iron isotope Fe57 | Drug: BBI-001<br>* Part A: 1 dose BBI-001 administered in a crossover fashion Part B: 2 doses BBI-001 administered in a crossover fashion<br>|
| Placebo Comparator: Placebo<br>Placebo: Oral administration immediately prior to a meal enriched with stable iron isotope Fe58 | Dietary Supplement: Original Fibre Metamucil<br>* Part A: 1 dose placebo administered in a crossover fashion Part B: 2 doses placebo administered in a crossover fashion<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Adverse Events (AE) | Incidence, type, and severity of AEs, to include clinically significant laboratory changes | up to 17 days | 
| Clinical Laboratory Testing - Iron Panel | Changes from baseline in iron panel | up to 17 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evaluation of Iron Isotope Blood Levels | Fe57 and Fe58 blood concentrations | up to 17 days |