Stage 1/2a Arm 2 for Cystic Fibrosis

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
University of Minnesota Medical Center, Fairview - Infectious Diseases and International Medicine, Minneapolis, MN
Cystic Fibrosis+4 More
WRAIR_PAM-CF1 - Biological
Eligibility
18+
All Sexes
What conditions do you have?
Select

Study Summary

This is a phase 1b/2 study of a single dose of intravenous (IV) bacteriophage in males and non-pregnant females, at least 18 years old, diagnosed with Cystic Fibrosis (CF). This clinical trial is designed to assess the safety and microbiological activity of bacteriophage product WRAIR-PAM-CF1, directed at Pseudomonas aeruginosa in clinically stable CF individuals chronically colonized with P. aeruginosa. WRAIR-PAM-CF1 is a 4 component anti-pseudomonal bacteriophage mixture containing between 4 x 10^7 and 4 x 10^9 Plaque Forming Units (PFU) of bacteriophage. Enrollment will occur at up to 20 clinical sites in the United States. In stage 1, two eligible subjects will be assigned to each of the three dosing arms receiving a single dosage of the IV bacteriophage therapy (4 x 10^7 PFU, 4 x 10^8 PFU, and 4 x 10^9 PFU; total of 6 sentinel subjects), followed by 30 ± 7 days observation period. If no SAEs (related to the study product) are identified during the 96 hours after bacteriophage administration for all Sentinel Subjects in Stage 1, the study will proceed to Stage 2. In Stage 2a, 32 subjects will be enrolled into one of 4 arms (placebo IV, 4 x 10^7 PFU, 4 x 10^8 PFU, and 4 x 10^9 PFU) in a 1:1:1:1 allocation. An interim analysis will be performed after all subjects have completed follow up visit 7 on Day 30 to select the IV bacteriophage dose with the most favorable safety and microbiological activity profile. During Stage 2b, subjects will be randomized into the bacteriophage (dose selected based on Interim Analysis following Stage 2a) or placebo arm. The final sample size is expected to be up to 72 subjects total with up to 25 subjects in the placebo arm and up to 25 subjects in the Stage 2b bacteriophage dose.

Eligible Conditions

  • Cystic Fibrosis
  • SLC25A16 protein, human

Treatment Effectiveness

Effectiveness Progress

1 of 3

Other trials for Cystic Fibrosis

Study Objectives

3 Primary · 0 Secondary · Reporting Duration: Day 1 through Day 30 ± 7

Day 30
Change from baseline in log10 P. aeruginosa total colony counts in quantitative sputum cultures
Day 30
Number of grade 2 or higher treatment-emergent Adverse Events
Day 8
Desirability of Outcome Ranking (DOOR)

Trial Safety

Safety Progress

1 of 3

Other trials for Cystic Fibrosis

Trial Design

6 Treatment Groups

Stage 1/2a Arm 2
1 of 6
Stage 1/2a Arm 3
1 of 6
Stage 2b Arm 2
1 of 6
Stage 1/2a Arm 4
1 of 6
Stage 2b Arm 1
1 of 6
Stage 2a Arm 1
1 of 6
Active Control
Non-Treatment Group

72 Total Participants · 6 Treatment Groups

Primary Treatment: Stage 1/2a Arm 2 · Has Placebo Group · Phase 1 & 2

Stage 1/2a Arm 2
Biological
ActiveComparator Group · 1 Intervention: WRAIR_PAM-CF1 · Intervention Types: Biological
Stage 2b Arm 1
Other
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Other
Stage 1/2a Arm 3
Biological
ActiveComparator Group · 1 Intervention: WRAIR_PAM-CF1 · Intervention Types: Biological
Stage 2a Arm 1
Other
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Other
Stage 2b Arm 2
Biological
ActiveComparator Group · 1 Intervention: WRAIR_PAM-CF1 · Intervention Types: Biological
Stage 1/2a Arm 4
Biological
ActiveComparator Group · 1 Intervention: WRAIR_PAM-CF1 · Intervention Types: Biological

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: day 1 through day 30 ± 7
Closest Location: University of Minnesota Medical Center, Fairview - Infectious Diseases and International Medicine · Minneapolis, MN
Photo of Minneapolis 1Photo of Minneapolis 2Photo of Minneapolis 3
2011First Recorded Clinical Trial
1 TrialsResearching Cystic Fibrosis
5 CompletedClinical Trials

Who is running the clinical trial?

National Institute of Allergy and Infectious Diseases (NIAID)Lead Sponsor
3,108 Previous Clinical Trials
4,672,372 Total Patients Enrolled

Eligibility Criteria

Age 18+ · All Participants · 9 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You are capable and willing to complete all study visits and perform all procedures required by the protocol.
Can be obtained from documentation in medical records; actual test results not necessary.
You are able to produce at least 2 mL of sputum following a hypertonic saline treatment or other approach to increase sputum production.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.