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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03466073




Registration number
NCT03466073
Ethics application status
Date submitted
2/03/2018
Date registered
15/03/2018
Date last updated
27/01/2020

Titles & IDs
Public title
A Phase 1b/2a Study of the Safety and Pharmacokinetics of Rhu-plasma Gelsolin in Hospitalized Subjects With CAP
Scientific title
A Double-blind, Placebo-controlled, Dose-escalation Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Plasma Gelsolin Added to Standard of Care in Subjects Hospitalized for Acute Community-acquired Pneumonia
Secondary ID [1] 0 0
BTI-201
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Community-acquired Pneumonia 0 0
Condition category
Condition code
Respiratory 0 0 0 0
Other respiratory disorders / diseases
Infection 0 0 0 0
Other infectious diseases
Infection 0 0 0 0
Studies of infection and infectious agents

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Recombinant Human Plasma Gelsolin
Other interventions - Normal Saline Placebo

Experimental: Single Dose 6 mg/kg - Intravenous administration of recombinant human plasma gelsolin at 6 mg/kg v. placebo (NSS) in addition to standard of care

Experimental: Multiple Dose 6 mg/kg - Intravenous administration of recombinant human plasma gelsolin at 6 mg/kg once per day for 3 days v. placebo (NSS) in addition to standard of care

Experimental: Multiple Dose 12 mg/kg - Intravenous administration of recombinant human plasma gelsolin at 12 mg/kg once per day for 3 days v. placebo (NSS) in addition to standard of care

Experimental: Multiple Dose 24 mg/kg - Intravenous administration of recombinant human plasma gelsolin at 24 mg/kg once per day for 3 days v. placebo (NSS) in addition to standard of care


Treatment: Drugs: Recombinant Human Plasma Gelsolin
Recombinant human plasma gelsolin lyophilized for reconstitution, reconstituted in sterile water

Other interventions: Normal Saline Placebo
Normal saline in volume equivalent to drug
Intervention code [1] 0 0
Treatment: Drugs
Intervention code [2] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)
Timepoint [1] 0 0
0-28 days
Secondary outcome [1] 0 0
Pharmacokinetics (PK) (Area Under the Rhu-pGSN Concentration - Time Curve)
Timepoint [1] 0 0
On Days 0-3, specimens were obtained just prior and immediately post dose and 2, 8,12 and/or 16 , and 24 hours post completion of IV administration. In the single-dose arm, only 1 dose was given and thus no data from later days were obtained.
Secondary outcome [2] 0 0
Pharmacokinetics (PK) (Maximum Observed Rhu-pGSN Plasma Concentration (Cmax))
Timepoint [2] 0 0
On Days 0-3, specimens were obtained just prior and immediately post dose and 2, 8, 12 and/or 16, and 24 hours post completion of IV administration. In the single-dose arm, only 1 dose was given and thus no data from later days were obtained.

Eligibility
Key inclusion criteria
1. Informed consent obtained from subject
2. Domicile: home, assisted living, rehabilitation facility, or nursing home (as long as the prospective participant is capable of providing written informed consent)
3. Duration of infection precipitating hospitalization by history <14 days
4. Planned or actual admission to hospital with a primary diagnosis of CAP within 24 hours of presentation to the hospital
5. Primary admitting diagnosis of pneumonia supported by a compatible clinical presentation with a documented infiltrate consistent with pneumonia on chest radiograph or CT, as assessed by the admitting emergency-department (ED), clinic, or ward physician or equivalent caregiver

* Recommended (not mandatory) guidance/discretionary criteria defining patients with CAP:

* At least 2 symptoms: difficulty breathing, cough, production of purulent sputum, chest pain
* At least 2 vital sign abnormalities: fever, tachycardia, tachypnea
* At least one finding of other clinical signs and laboratory abnormalities: hypoxemia, clinical evidence of pulmonary consolidation, an elevated total white blood cell (WBC) count or leukopenia
* Chest imaging showing new (or presumed new or worsening) infiltrates
* Receipt of antibiotic treatment prior to presentation does not exclude the patient
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Pregnant or lactating women
2. Intubation, vasopressor support, or admission to the intensive care unit (ICU) directly from the ED/office (fluids for responsive hypotension is not a reason for exclusion)
3. Use of any investigational drug in the past 30 days
4. Hospitalization during the last 30 days
5. Residence within the last 30 days in long-term care facility where the patient remains persistently unable to participate in the routine activities of daily living
6. Active underlying cancer treated with systemic chemotherapy or radiation therapy during the last 30 days
7. Known or suspected immunosuppressive disease or therapy (including steroid use equivalent to prednisone =20 mg/day for >7 days or known advanced human immunodeficiency virus (HIV) infection with CD4 count =200/mm3; specific testing for HIV status or CD4 count is not required but can be done at the discretion of the caregivers)
8. Active congestive heart failure, myocardial infarction, or pulmonary embolism; cardiopulmonary arrest in last 30 days
9. Weight >100 kg
10. Otherwise unsuitable for study participation in the opinion of the investigator

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/08/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
2/04/2019
Sample size
Target
Accrual to date
Final
33
Recruitment in Australia
Recruitment state(s)
QLD,VIC
Recruitment hospital [1] 0 0
Cairns Hospital - Cairns
Recruitment hospital [2] 0 0
Box Hill Hospital - Box Hill
Recruitment hospital [3] 0 0
Footscray Hospital - Footscray
Recruitment postcode(s) [1] 0 0
4870 - Cairns
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
Recruitment postcode(s) [3] 0 0
3011 - Footscray
Recruitment outside Australia
Country [1] 0 0
Georgia
State/province [1] 0 0
Mtskheta
Country [2] 0 0
Georgia
State/province [2] 0 0
Rustavi
Country [3] 0 0
Georgia
State/province [3] 0 0
Tbilisi

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioAegis Therapeutics Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A Phase 1b/2a, Double-blind, Placebo-controlled, Dose-escalation Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Recombinant Human Plasma gelsolin (rhu-pGSN) Added to Standard of Care in Subjects Hospitalized for Acute Community-acquired Pneumonia (CAP)
Trial website
https://clinicaltrials.gov/study/NCT03466073
Trial related presentations / publications
Tannous A, Levinson SL, Bolognese J, Opal SM, DiNubile MJ. Safety and Pharmacokinetics of Recombinant Human Plasma Gelsolin in Patients Hospitalized for Nonsevere Community-Acquired Pneumonia. Antimicrob Agents Chemother. 2020 Sep 21;64(10):e00579-20. doi: 10.1128/AAC.00579-20. Print 2020 Sep 21.
Public notes

Contacts
Principal investigator
Name 0 0
Mark J DiNubile, MD
Address 0 0
BioAegis Therapeutics Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT03466073