BBI-001 in healthy volunteers and people with hereditary hemochromatosis
A Phase 1/2a, Two-Part, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability and Pharmacodynamics of Multiple Doses of Oral BBI-001 in Healthy Participants and in Participants With Hereditary Hemochromatosis
This project will try BBI-001 taken three times daily to see if it safely changes blood iron levels in people with hereditary hemochromatosis while also testing safety in healthy volunteers.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 34 (estimated) |
| Ages | 18 Years to 70 Years |
| Sex | All |
| Sponsor | Bond Biosciences Industry-sponsored |
| Locations | 1 site (Sydney, New South Wales) |
| Trial ID | NCT07371793 on ClinicalTrials.gov |
What this trial studies
The protocol runs in two parallel parts: a Phase 1 multiple-ascending-dose, randomized, double-blind, placebo-controlled portion that enrolls healthy volunteers in three sequential dose cohorts receiving BBI-001 or placebo three times daily for 14 days. The Phase 2a portion is a randomized, two-period, two-sequence crossover in patients with hereditary hemochromatosis who receive eight doses of BBI-001 or placebo TID in Period 1 and the reverse treatment in Period 2, with dosing periods separated by 12 days. Key outcomes include effects on blood iron parameters in patients and safety/tolerability assessments in both groups. Placebo control and crossover design in patients allow within-subject comparisons of the drug's short-term effects on iron measures.
Who should consider this trial
Good fit: Ideal participants are adults with hereditary hemochromatosis who are clinically stable on maintenance phlebotomy (and not receiving iron chelation) for at least six months, or healthy adult volunteers for the Phase 1 part.
Not a fit: People with significant organ damage from iron overload, those currently on iron chelation, active infections, uncontrolled medical or psychiatric conditions, or alcohol use disorder are unlikely to be eligible or to receive benefit from this protocol.
Why it matters
Potential benefit: If successful, BBI-001 could lower or normalize blood iron measures and reduce the need for frequent phlebotomy in people with hereditary hemochromatosis.
How similar studies have performed: This clinical approach appears relatively novel for hereditary hemochromatosis with limited prior human data, so efficacy has not yet been established.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Healthy volunteers (Part 1) or patients with hereditary hemochromatosis (Part 2) Exclusion Criteria: Serious or unstable medical or psychiatric conditions Significant medical history Current infections Alcohol use disorder Receiving iron chelation therapy or treatment other than stable maintenance phlebotomy for the prior 6 months (Part 2) Organ damage from iron overload in the view of the principal investigator would prevent successful completion of the protocol (Part 2)
Where this trial is running
Sydney, New South Wales
- Clinical Research Unit — Sydney, New South Wales, Australia (Recruiting)
Study contacts
- Study coordinator: Georgina Kilfoil
- Email: georgina@ashcroftlsc.com.au
- Phone: +61 (0)432 388 772
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.