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IV Gallium Study for Patients With Cystic Fibrosis Who Have NTM (ABATE Study) (ABATE)

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ClinicalTrials.gov Identifier: NCT04294043
Recruitment Status : Recruiting
First Posted : March 3, 2020
Last Update Posted : June 23, 2021
Sponsor:
Collaborator:
Cystic Fibrosis Foundation
Information provided by (Responsible Party):
Chris Goss, Seattle Children's Hospital

Brief Summary:

The purpose of this study is to assess the safety and tolerability of two 5-day infusion cycles of IV gallium in adult patients with CF who are infected with NTM.

Funding Source - FDA OOPD


Condition or disease Intervention/treatment Phase
Nontuberculous Mycobacterium Infection Drug: Gallium nitrate Phase 1

Detailed Description:

This is a prospective, multicenter open-label study in adults with CF who are colonized with M. avium complex and/or M. abscessus complex.

Subjects will receive two 5-day infusion cycles of IV gallium. The study will evaluate the safety and antimycobacterial effect of two 5-day infusions of IV gallium.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1b, Multi-center Study of Intravenous (IV) Gallium Nitrate in Patients With Cystic Fibrosis (CF) Who Are Colonized With Nontuberculous Mycobacteria (NTM) (The ABATE Study)
Actual Study Start Date : June 17, 2021
Estimated Primary Completion Date : September 30, 2023
Estimated Study Completion Date : September 30, 2024


Arm Intervention/treatment
Experimental: Infusion of IV Gallium
Gallium nitrate will be infused continuously over 5 days at 200 mg/m2/day. Study drug will be administered via a peripheral IV catheter, a peripherally inserted central catheter (PICC) line, midline catheter, or a chronic indwelling vascular access device using an ambulatory infusion pump infused over 24 hours for 5 sequential days for each cycle. There is a maximum of 2 cycles.
Drug: Gallium nitrate
Study subjects will receive an infusion of gallium nitrate.
Other Names:
  • Ga
  • GaN3O9
  • IV gallium
  • ganite




Primary Outcome Measures :
  1. Adverse Events of Special Interest [ Time Frame: Baseline to Day 57 ]
    Proportion of patients experiencing one or more Adverse Events of Special Interest (AESI). AESIs include the occurrence of either (1) a serious adverse event (SAE) of grade 3 or higher including hospitalizations or (2) study drug discontinuation because of an AE.


Secondary Outcome Measures :
  1. Clinically significant abnormal laboratory measures. (safety) [ Time Frame: Baseline to Day 57 ]
    Proportion of patients experiencing clinically significant abnormal laboratory measures. Clinically significant abnormal laboratory measures are identified by the site investigator.

  2. NTM clearance (efficacy) [ Time Frame: Day 6 to Day 111 ]
    Proportion of subjects who were NTM culture positive at baseline and have at least 2 sequential negative NTM cultures between visits 2 (Day 6) and 7 (Day 111). Those negative cultures must be at least 2 weeks apart.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Written informed consent obtained from subject or subject's legal representative
  2. Be willing and able to adhere to the study visit schedule and other protocol requirements
  3. Greater than or equal to 18 years of age at Visit 1
  4. Documentation of a CF diagnosis as evidenced by one or more clinical features consistent with the CF phenotype and one or more of the following criteria:

    • Sweat chloride ≥ 60 milliequivalent (mEq)/liter by quantitative pilocarpine iontophoresis test (QPIT)
    • Two well-characterized mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
    • Abnormal nasal potential difference (NPD) (change in NPD in response to a low chloride solution and isoproteronol of less than -5 mV)
  5. Documentation of NTM culture positive defined as follows:

    • The two most recent consecutive sputum (or bronchoalveolar lavage (BAL)) cultures were positive for M. avium complex and/or M. abscessus complex (cultures must be at least 4 weeks apart)
    • The two most recent consecutive cultures are of the same species or subspecies
    • The most recent positive culture result is within 4 months prior to screening AND the two most recent cultures are within 12 months prior to screening
  6. Current NTM species or subspecies has never been treated or previous treatment was associated with clearance of NTM and completed > 2 years prior to Day 1
  7. Forced expiratory volume in 1 second (FEV1) ≥ 25 % of predicted value at Screening
  8. Able to expectorate sputum
  9. Clinically stable with no significant changes in health status within 7 days prior to Day 1
  10. Enrolled in the CFF Cystic Fibrosis Foundation Patient Registry (CFFPR)
  11. Willing to discontinue chronic azithromycin use for the duration of the study

Exclusion Criteria:

  1. Any of the following abnormal lab values at screening:

    • Hemoglobin <10g/dL
    • Platelets <100,000/mm3
    • White blood cells (WBC) < 4,500/mm3
    • Aspartate transaminase (AST), alanine transaminase (ALT), gamma-glutamyl transferase (GGT), or alkaline phosphatase (ALP) ≥3 x upper limit of normal
    • Serum creatinine > 2.0 mg/dl and ≥1.5 x upper limit of normal
    • Ionized calcium ≤ lower limit of normal (only performed if total calcium is ≤ lower limit of normal)
  2. History of solid organ or hematological transplantation
  3. Use of bisphosphonates within 7 days prior to Day 1
  4. Known sensitivity to gallium
  5. Use of any investigational drug and/or participated in any interventional clinical trial within 28 days prior to Day 1
  6. In the opinion of the Investigator, features of active NTM disease are present (e.g., clinical worsening is likely due to NTM disease despite definitive treatment of co-pathogens and/or acute exacerbations)
  7. Undergoing treatment for NTM disease or anticipate beginning treatment within 3 months
  8. Current diagnosis of osteoporosis
  9. For people of childbearing potential:

    • Positive pregnancy test at Visit 1 or
    • Lactating or
    • Unwilling to practice a medically acceptable form of contraception (acceptable forms of contraception: abstinence, hormonal birth control, intrauterine device, or barrier method plus a spermicidal agent), unless surgically sterilized or postmenopausal during the study
  10. For people able to father a child: unwilling to use adequate contraception (as determined by the investigator) during the study
  11. Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives
  12. New initiation of chronic therapy (greater than 21 days) within 28 days prior to the Enrollment Visit

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04294043


Contacts
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Contact: Lauren Braam, MIH 206-884-1487 lauren.braam@seattlechildrens.org

Locations
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United States, Colorado
National Jewish Health Not yet recruiting
Denver, Colorado, United States, 80206
Contact: Alix Wilson       wilsona@njhealth.org   
United States, Washington
University of Washington Medical Center Recruiting
Seattle, Washington, United States, 98195
Contact: Teresa Gambol       TGambol@medicine.washington.edu   
Sponsors and Collaborators
Chris Goss
Cystic Fibrosis Foundation
Investigators
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Principal Investigator: Christopher H. Goss, MD, MSc University of Washington, Seattle Children's Hospital
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Responsible Party: Chris Goss, Professor of Medicine and Pediatrics, University of Washington, Seattle Children's Hospital
ClinicalTrials.gov Identifier: NCT04294043    
Other Study ID Numbers: ABATE-IP-18
R01FD006848 ( U.S. FDA Grant/Contract )
First Posted: March 3, 2020    Key Record Dates
Last Update Posted: June 23, 2021
Last Verified: June 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Chris Goss, Seattle Children's Hospital:
Cystic Fibrosis
Gallium Nitrate
IV Gallium
Nontuberculous mycobacterium
NTM
Mycobacterium abscessus
mycobacterium avium
Additional relevant MeSH terms:
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Mycobacterium Infections
Mycobacterium Infections, Nontuberculous
Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases
Actinomycetales Infections
Gram-Positive Bacterial Infections
Bacterial Infections
Gallium nitrate
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs
Antineoplastic Agents