# A Phase 1b, Multi-center Study of IV Gallium Nitrate in Patients with Cystic Fibrosis who are colonized with Nontuberculosis Mycobacterium (The ABATE Study).

> **NIH FDA R01** · SEATTLE CHILDREN'S HOSPITAL · 2020 · $853,982

## Abstract

Project Summary:
Nontuberculous Mycobacteria (NTM) infection in cystic fibrosis (CF) is common, particularly
Mycobacterium avium complex (MAC) and M. abscessus complex and appears to be increasing
in prevalence. New approaches are needed as current drug regimens involve prolonged
administration of drugs with significant toxicity and rates of treatment failure are high. Despite
prolonged courses of multidrug therapy, effective cure for MAC lung disease is just under 60%
in subjects without immune suppression or CF. In regard to M. abscessus complex, treatment
response has been variable. M. abscessus ssp. massiliense demonstrated a better treatment
response with more sustained negative cultures compared to M. abscessus ssp. abscessus (88
vs 25%) in non-CF disease. Thus, novel treatments are needed to improve clinical outcomes.
While still controversial, recent evidence supporting patient to patient spread of M. abscessus
complex in CF centers raises the urgency of development of better treatments in CF. We
propose to exploit the iron vulnerability of both MAC and M. abscessus complex by using the
metal gallium as a "Trojan horse" to disrupt iron metabolism. Gallium has a nearly identical ionic
radius as Fe, and some bacterial uptake systems are unable to distinguish gallium from iron.
Gallium disrupts iron-dependent processes because Ga3+ cannot be reduced in physiological
conditions, and iron's biological functions involves redox cycling. Thus, gallium incorporation
into iron-containing proteins disrupts their functioning. Previous work by our group and others
found that gallium compounds had antibacterial activity against a number of human pathogens
including Pseudomonas aeruginosa, Fransella tulerensis, Acinetobacter baumanai, Klebsiella
pneumoniae, and other important pathogens including MAC and M. abscessus complex. Our
prior trials (phase 1b and 2b) in CF patients infected with P. aeruginosa demonstrated that a
single course of IV gallium was safe and showed evidence of efficacy. Here we propose a
phase 1b open label phase 1b bridging study to assess the safety and pharmacokinetics of two
sequential 5-day infusions of gallium nitrate in adults with CF who are infected with either MAC
and M. abscessus complex at a dose of 200 mg/m2/day. We will also assess for preliminary
microbiologic effect using the study population and a contemporary control population enrolled
in a parent observational PREDICT trial (PRospective Evaluation of NTM Disease In CysTic
Fibrosis; NCT02073409), a 10 center observational study of NTM in CF funded by the US CF
Foundation.

## Key facts

- **NIH application ID:** 9962047
- **Project number:** 1R01FD006848-01
- **Recipient organization:** SEATTLE CHILDREN'S HOSPITAL
- **Principal Investigator:** Christopher Hooper Goss
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $853,982
- **Award type:** 1
- **Project period:** 2020-08-15 → 2026-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9962047

## Citation

> US National Institutes of Health, RePORTER application 9962047, A Phase 1b, Multi-center Study of IV Gallium Nitrate in Patients with Cystic Fibrosis who are colonized with Nontuberculosis Mycobacterium (The ABATE Study). (1R01FD006848-01). Retrieved via AI Analytics 2026-05-27 from https://api.ai-analytics.org/grant/nih/9962047. Licensed CC0.

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