Bronchiectasis Adult, Cystic Fibrosis, Non-Tuberculous Mycobacteria
Conditions
Keywords
BCG vaccine, Cystic fibrosis, Bronchiectasis, Non-tuberculosis mycobacteria
Brief summary
This is pilot study of the immunologic effects of intradermal Bacille Calmette-Guerin (BCG) vaccination of adults with cystic fibrosis (CF), non-CF bronchiectasis (NCFB), and healthy volunteers.
Detailed description
This single-site, open-label clinical trial to investigate the immune effects of BCG vaccination in adults with cystic fibrosis, non-cystic fibrosis bronchiectasis and healthy volunteers by measuring pre and post vaccination immune responses, BCG uptake and protection against infections, including nontuberculous mycobacteria (NTM). This study will also gather data on study feasibility, tolerability, and safety.
Interventions
BIOLOGICALBCG TICE Vaccine
Intradermal vaccination with BCG TICE
Sponsors
Cystic Fibrosis Foundation
Johns Hopkins University
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
Yes
Inclusion criteria
(CF and non-CF bronchiectasis): * Confirmed diagnosis of either CF or non-CF bronchiectasis * Forced expiratory volume over one second (FEV1) \> 40% * Willingness to participate in the study after all aspects of the protocol have been explained and written informed consent obtained. * Available for the study duration, including all planned follow-up visits Inclusion Criteria (Healthy Volunteers): * Negative HIV enzyme-linked immunosorbent assay (ELISA) and confirmatory test at screening. * Willingness to participate in the study after all aspects of the protocol have been explained and written informed obtained. * Available for the study duration, including all planned follow-up visits
Exclusion criteria
(All arms): * Current or prior history of active or latent tuberculosis (TB) (per report, not formally tested) or NTM infection * Prior BCG vaccination * Previous vaccine in the past 4 weeks * History of severe anaphylaxis to any vaccine or vaccine components * History of organ/bone marrow transplantation or other immunosuppressing condition, including HIV * Immunosuppressing drugs (including oral corticosteroids equivalent to \>10mg of prednisone for 5 days) in the 30 days prior to study enrollment * Cirrhosis or portal hypertension * Pregnant or breastfeeding * Receipt of another investigational product in the last 28 days or planned receipt during this study * Has any other condition that, in the opinion of the principal investigator, would preclude informed consent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving study objectives.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| BCG Uptake | 3 months | Change in concentration of interferon (IFN) gamma levels in blood after Bacille Calmette-Guérin (BCG) incubation relative to baseline as measured by SFUs. Spot forming units (SFU) is a measure of immune response of mononuclear cells in response to M. avium lysate. Higher values indicate a stronger immune response. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Cystic Fibrosis Participants with Cystic Fibrosis.
BCG TICE Vaccine: Intradermal vaccination with BCG TICE | 14 |
| Non Cystic Fibrosis Bronchiectasis Participants with Non Cystic Fibrosis Bronchiectasis.
BCG TICE Vaccine: Intradermal vaccination with BCG TICE | 2 |
| Healthy Volunteer Participants with no condition (healthy volunteers).
BCG TICE Vaccine: Intradermal vaccination with BCG TICE | 14 |
| Total | 30 |
Baseline characteristics
| Characteristic | Cystic Fibrosis | Non Cystic Fibrosis Bronchiectasis | Healthy Volunteer | Total |
|---|---|---|---|---|
| Age, Continuous | 32.4 years STANDARD_DEVIATION 13.5 | 44.4 years STANDARD_DEVIATION 22.3 | 35.8 years STANDARD_DEVIATION 10.2 | 34.8 years STANDARD_DEVIATION 12.5 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 4 Participants | 4 Participants |
| Race (NIH/OMB) More than one race | 1 Participants | 0 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 13 Participants | 2 Participants | 9 Participants | 24 Participants |
| Region of Enrollment United States | 14 Participants | 2 Participants | 14 Participants | 30 Participants |
| Sex: Female, Male Female | 6 Participants | 1 Participants | 6 Participants | 13 Participants |
| Sex: Female, Male Male | 8 Participants | 1 Participants | 8 Participants | 17 Participants |
| SFU count | 35.22 Spot forming units (SFU) STANDARD_DEVIATION 35.18 | 32.5 Spot forming units (SFU) STANDARD_DEVIATION 17.68 | 38.75 Spot forming units (SFU) STANDARD_DEVIATION 48.14 | 35.49 Spot forming units (SFU) STANDARD_DEVIATION 33.67 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 14 | 0 / 2 | 0 / 14 |
| other Total, other adverse events | 12 / 14 | 1 / 2 | 12 / 14 |
| serious Total, serious adverse events | 0 / 14 | 0 / 2 | 0 / 14 |
Outcome results
Primary
BCG Uptake
Change in concentration of interferon (IFN) gamma levels in blood after Bacille Calmette-Guérin (BCG) incubation relative to baseline as measured by SFUs. Spot forming units (SFU) is a measure of immune response of mononuclear cells in response to M. avium lysate. Higher values indicate a stronger immune response.
Time frame: 3 months
Population: Analyzed all participants enrolled
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Cystic Fibrosis | BCG Uptake | 48.06 spot forming units | Standard Error 23.52 |
| Non Cystic Fibrosis Bronchiectasis | BCG Uptake | 0.84 spot forming units | Standard Error 16.92 |
| Healthy Volunteer | BCG Uptake | 35.97 spot forming units | Standard Error 23.64 |