Cystic FibrosisPhase 1NCT01093521
A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients
A Phase 1 study testing 100 mg/m2 dose and 200 mg/m2 dose in people with Cystic Fibrosis, run by University of Washington at 3 sites in United States.
Trial summary
The purpose of this research study is to test the pharmacokinetics, safety, and tolerability of an intravenous infusion of a drug called Ganite (gallium nitrate) in patients with cystic fibrosis. We want to see this drug is safe and tolerable and to see if high levels of the drug are found in the sputum. Read more about this study
- Who is it for?
- Ages 18 to 55 years
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- 100 mg/m2 dose and 200 mg/m2 dose
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 3 sitesUnited States
- Study dates
- Apr 2010to 31 Aug 2013
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 55 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Adult male or female, between 18 and 55 years of age
- Documented chronic colonization with Pseudomonas Aeruginosa (Pa)
- Confirmed diagnosis of CF:
- Documented history of > 60 mmol/L chloride concentration in pilocarpine sweat chloride test And/Or
- Genotype with two identifiable mutations consistent with CF, accompanied by one or more phenotypic features consistent with diagnosis of CF
- Forced expiratory volume in the first second (FEV1) ≥ 30% of predicted value
Show all 15Show fewer
- Able to expectorate sputum
- Serum liver function tests ≤ 2.5 x upper limit of normal
- Serum urea nitrogen (BUN) and creatinine ≤ 1.5 x upper limit of normal
- Serum creatinine ≤ 2.0 mg/dl
- Hemoglobin ≥ 9 g/dl, platelets ≥ 100,000/mm3, and white blood cells (WBC) ≥ 4,500/mm3 and ≤ 15,000/mm3
- Ionized calcium ≥ the lower limit of normal
- Able to understand and sign the informed consent document, communicate with the Investigator, and comply with the requirements of the protocol
- If female and of childbearing potential, must have a negative pregnancy test on Day 1 prior to receiving study drug
- If female and of childbearing potential, is willing to use adequate contraception, as determined by the investigator, for the duration of the study
Who cannot join
- Acute pulmonary exacerbation requiring antibiotic intervention within 2 weeks prior to screening
- Osteoporosis defined as the most recent dexa scan within the prior 5 years with a T-score ≤ -2.5
- Pregnant or lactating female
- Known sensitivity to gallium
- Use of biphosphonates
- Use of any investigational drug and/or participated in any clinical trial within 3 months prior to screening
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Johns Hopkins University
Baltimore, Maryland, 21201
University of Iowa
Iowa City, Iowa, 52242-1083
University of Washington
Seattle, Washington, 98195
About this study
The purpose of this research study is to test the pharmacokinetics, safety, and tolerability of an intravenous infusion of a drug called Ganite (gallium nitrate) in patients with cystic fibrosis. We want to see this drug is safe and tolerable and to see if high levels of the drug are found in the sputum.
Funding Source - Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD)
Read the detailed description
This is a two center pharmacokinetic and safety dosing study of IV gallium nitrate (Ganite®) in cystic fibrosis (CF) patients. Eighteen subjects are planned. Each subject will be administered a single 5-day infusion of study medication (one of 2 doses). No placebo is used. Each subject will receive 5 days of continuous infusion of the experimental treatment. There will be two dosing cohorts (cohort 1: 100 mg/m2/day and cohort 2: 200 mg/m2/day). Cohort 2 will begin enrollment only after Data Safety Monitoring Committee (DSMC) safety review and approval of cohort 1 data. Study visits occur at baseline (day 1), day 3 (visit 2), day 6 (visit 3), day 8 (visit 4), day 14 (visit 5), day 28 (visit 6), and day 56 (visit 7).
Screening data will be reviewed to determine subject eligibility. Subjects who meet all inclusion criteria and none of the exclusion criteria will be entered into the study.
The following treatment regimens will be used:
- Experimental treatment continuous infusion of gallium nitrate at the following doses cohort 1: 100 mg/m2/day and cohort 2: 200 mg/m2/day All subjects who receive at least one dose of study medication will be considered evaluable for safety and efficacy analyses. Incidence of adverse events will be monitored during the trial.
Primary endpoints will be assessment of pharmacokinetic and safety/tolerability data.
Secondary efficacy assessments will be based on changes in lung function and sputum P. aeruginosa density in sputum.
Total duration of subject participation will be five weeks. Total duration of the study is expected to be 20 months.
- Conditions
- Cystic Fibrosis
- Being tested
- 100 mg/m2 dose, 200 mg/m2 dose
- Keywords
- IV Gallium, Gallium, Ganite, Cystic Fibrosis, Gallium Infusion
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
100 mg/m2 doseDrug
Also called Ganite®, gallium nitrate
5 day infusion of gallium nitrate (IV Ganite®) at a dose of 100 mg/m2/day
200 mg/m2 doseDrug
Also called Ganite®, gallium nitrate
5 day infusion of gallium nitrate (IV Ganite®) at 200 mg/m2/day
| Study group | Role | What they receive |
|---|---|---|
| 100 mg/m2 dose | Experimental | Five day continuous IV Gallium Nitrate (Ganite®) infusion at 100 mg/m2 Receives: Drug: 100 mg/m2 dose |
| 200 mg/m2 dose | Experimental | Five day continuous IV Gallium Nitrate (Ganite®) infusion at 200 mg/m2 Receives: Drug: 200 mg/m2 dose |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic Assessment of a 5 Day Infusion of Gallium Nitrate (IV Ganite®)
To assess the summed area under the curves of a 5 day infusion of IV Ga from day 1 to day 28 at two doses: 100 mg/m2/day in adult subjects with CF; 200 mg/m2/day in adult subjects with CF.
To assess the safety of a 5 day infusion of IV Ga at two doses: 100 mg/m2/day in adult subjects with CF; 200 mg/m2/day in adult subjects with CF.
Safety and tolerability of 5 days of treatment with IV administered gallium nitrate (IV Ganite®) at a doses of 100 mg/m2/day and 200 mg/m2/day.
Measured: Day 1 at t=1, 2 and 6 hours, Day 3, Day 6 at t= 1, 2, 8, and 12, Day 14 and Day 28
Number of Serious Adverse Events
Safety as measured by serous adverse events
Measured: 56 days from starting dose
Number of Events When Study Drug Infusion Was Stopped Early
Tolerability as measured by adverse events of a 5 day continuous infusion of IV Gallium as assessed by stopping study drug infusion
Measured: 6 days from starting dose
Secondary outcomes (8)
Change in Spirometry From Baseline to Day 8
Change in spirometry as measured by FEV1 in liters from baseline to day 8
Measured: 8 days
Change in Lung Function From Baseline to Day 15
Change in FEV1 in liters from baseline to day 15
Measured: 15 days from starting dose
Change in Spirometry From Baseline to Day 28
Change in lung function as measured by FEV1 in liters from baseline to day 28
Measured: 28 days from starting dose
Change in Spirometry From Baseline to Day 56
Change in lung function as measured by FEV1 in liters from baseline to day 56
Measured: 56 days from starting dose
Change in Spirometry as Measured by FVC From Baseline to Day 8
Change from baseline in lung function assessed by FVC in liters after treatment with IV Ga at day 8
Measured: 8 days from starting dose
Change in P. Aeruginosa Density From Baseline to Day 8
Change in sputum microbiology (specifically P. aeruginosa density based on quantitative cultures) from baseline to day 8
Measured: 8 days from starting dose
Change in Sputum P. Aeruginosa Density From Baseline to Day 15
Change in sputum microbiology (specifically P. aeruginosa density based on quantitative cultures) from baseline to day 15
Measured: 15 days from starting dose
Change in P. Aeruginosa Density From Baseline to Day 56
Change in sputum microbiology (specifically P. aeruginosa density based on quantitative cultures) from baseline to day 56
Measured: 56 days from starting dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Mar 2010 (estimated)
- Study startApr 2010
- Primary completionApr 2012
- Study completion31 Aug 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by University of WashingtonThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Washington
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Responsible party
- Principal investigator, Chris Goss, University of Washington
- Investigators
- Christopher H Goss, MD, MSc, Principal investigator, University of Washington
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01093521
- Sponsor's protocol ID
- 35876
- Other IDs
- US FDA 5R01FD003704
- Status last verified
- Oct 2022
- First submitted
- 24 Mar 2010
- Last updated
- 29 Nov 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Pharmacokinetic and Safety Study of IV Gallium Nitrate (Ganite) in Cystic Fibrosis Patients
- ClinicalTrials.gov ID
- NCT01093521
- Acronym / sponsor ID
- 35876
- Conditions
- Cystic Fibrosis
- Keywords
- IV Gallium, Gallium, Ganite, Cystic Fibrosis, Gallium Infusion
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- University of WashingtonUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation
- Study start
- Apr 2010
- Primary completion
- Apr 2012 (actual)
- Study completion
- 31 Aug 2013 (actual)
- First posted
- 26 Mar 2010 (estimated)
- Results
- Posted 12 Jan 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this research study is to test the pharmacokinetics, safety, and tolerability of an intravenous infusion of a drug called Ganite (gallium nitrate) in patients with cystic fibrosis. We want to see this drug is safe and tolerable and to see if high levels of the drug are found in the sputum.
Funding Source - Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD)
Detailed description
This is a two center pharmacokinetic and safety dosing study of IV gallium nitrate (Ganite®) in cystic fibrosis (CF) patients. Eighteen subjects are planned. Each subject will be administered a single 5-day infusion of study medication (one of 2 doses). No placebo is used. Each subject will receive 5 days of continuous infusion of the experimental treatment. There will be two dosing cohorts (cohort 1: 100 mg/m2/day and cohort 2: 200 mg/m2/day). Cohort 2 will begin enrollment only after Data Safety Monitoring Committee (DSMC) safety review and approval of cohort 1 data. Study visits occur at baseline (day 1), day 3 (visit 2), day 6 (visit 3), day 8 (visit 4), day 14 (visit 5), day 28 (visit 6), and day 56 (visit 7).
Screening data will be reviewed to determine subject eligibility. Subjects who meet all inclusion criteria and none of the exclusion criteria will be entered into the study.
The following treatment regimens will be used:
- Experimental treatment continuous infusion of gallium nitrate at the following doses cohort 1: 100 mg/m2/day and cohort 2: 200 mg/m2/day All subjects who receive at least one dose of study medication will be considered evaluable for safety and efficacy analyses. Incidence of adverse events will be monitored during the trial.
Primary endpoints will be assessment of pharmacokinetic and safety/tolerability data.
Secondary efficacy assessments will be based on changes in lung function and sputum P. aeruginosa density in sputum.
Total duration of subject participation will be five weeks. Total duration of the study is expected to be 20 months.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 100 mg/m2 dose | Experimental | Drug: 100 mg/m2 doseFive day continuous IV Gallium Nitrate (Ganite®) infusion at 100 mg/m2 |
| 200 mg/m2 dose | Experimental | Drug: 200 mg/m2 doseFive day continuous IV Gallium Nitrate (Ganite®) infusion at 200 mg/m2 |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (15 listed)
- Adult male or female, between 18 and 55 years of age
- Documented chronic colonization with Pseudomonas Aeruginosa (Pa)
- Confirmed diagnosis of CF:
- Forced expiratory volume in the first second (FEV1) ≥ 30% of predicted value
- Able to expectorate sputum
Key exclusion criteria (6 listed)
- Acute pulmonary exacerbation requiring antibiotic intervention within 2 weeks prior to screening
- Osteoporosis defined as the most recent dexa scan within the prior 5 years with a T-score ≤ -2.5
- Pregnant or lactating female
- Known sensitivity to gallium
- Use of biphosphonates
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 100 mg/m2 doseAlso: Ganite®, gallium nitrate5 day infusion of gallium nitrate (IV Ganite®) at a dose of 100 mg/m2/day | Drug | 100 mg/m2 dose |
| 200 mg/m2 doseAlso: Ganite®, gallium nitrate5 day infusion of gallium nitrate (IV Ganite®) at 200 mg/m2/day | Drug | 200 mg/m2 dose |
Primary outcome
Pharmacokinetic Assessment of a 5 Day Infusion of Gallium Nitrate (IV Ganite®)
Time frame: Day 1 at t=1, 2 and 6 hours, Day 3, Day 6 at t= 1, 2, 8, and 12, Day 14 and Day 28
To assess the summed area under the curves of a 5 day infusion of IV Ga from day 1 to day 28 at two doses: 100 mg/m2/day in adult subjects with CF; 200 mg/m2/day in adult subjects with CF. To assess the safety of a 5 day infusion of IV Ga at two doses: 100 mg/m2/day in adult subjects with CF; 200 mg/m2/day in adult subjects with CF. Safety and tolerability of 5 days of treatment with IV administered gallium nitrate (IV Ganite®) at a doses of 100 mg/m2/day and 200 mg/m2/day.
Number of Serious Adverse Events
Time frame: 56 days from starting dose
Safety as measured by serous adverse events
Number of Events When Study Drug Infusion Was Stopped Early
Time frame: 6 days from starting dose
Tolerability as measured by adverse events of a 5 day continuous infusion of IV Gallium as assessed by stopping study drug infusion
8 secondary outcomes
Change in Spirometry From Baseline to Day 8
Time frame: 8 days
Change in Lung Function From Baseline to Day 15
Time frame: 15 days from starting dose
Change in Spirometry From Baseline to Day 28
Time frame: 28 days from starting dose
Change in Spirometry From Baseline to Day 56
Time frame: 56 days from starting dose
Change in Spirometry as Measured by FVC From Baseline to Day 8
Time frame: 8 days from starting dose
Change in P. Aeruginosa Density From Baseline to Day 8
Time frame: 8 days from starting dose
Change in Sputum P. Aeruginosa Density From Baseline to Day 15
Time frame: 15 days from starting dose
Change in P. Aeruginosa Density From Baseline to Day 56
Time frame: 56 days from starting dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher H Goss, MD, MSc principal investigator
University of Washington
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing 100 mg/m2 dose and 200 mg/m2 dose in people with Cystic Fibrosis, run by University of Washington at 3 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 15 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 12 Jan 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Pharmacokinetic Assessment of a 5 Day Infusion of Gallium Nitrate (IV Ganite®) (time frame: Day 1 at t=1, 2 and 6 hours, Day 3, Day 6 at t= 1, 2, 8, and 12, Day 14 and Day 28). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2022.