A Multiple Ascending Dose Study of PUL-042 in Stem Cell Transplant Recipients

An Open-Label, Multiple Ascending Dose Study to Evaluate the Safety and Tolerability of PUL-042 Inhalation Solution in Subjects With Hematologic Malignancies and Recipients of Stem Cell Transplantation

Subjects with hematologic malignancies or recipients of a first allogenic or autologus hematopoietic stem cell transplant, without any evidence or respiratory infection, will receive 4 doses of PUL-042 Inhalation Solution over a 2 week period. Subjects will be evaluated for tolerability of the drug. If tolerated, dose escalation may occur with up to 4 dose levels tested

Study Overview

• Status: Withdrawn
• Conditions: Hematologic Diseases; Stem Cell Transplants
• Intervention / Treatment: Drug: PUL-042

Detailed Description

This is an open-label multiple ascending dose study with a 3+3 study design to examine the safety and tolerability of PUL-042 Inhalation Solution in subjects with hematologic malignancies or recipients of a first allogenic or autologus hematopoietic stem cell transplant. Subjects will receive 4 doses over 2 weeks.

Cohort size will be 3 subjects, once 3 subjects have completed treatment and follow-up at a dose level, subject data will be evaluated by an independent data safety monitoring committee prior to dosing additional subjects. The Committee may recommend increasing the dose, maintaining the same dose, or decreasing the dose.

Once the maximum dose is determined, there may be up to 6 additional subjects dosed at that level.

• Study Type: Interventional
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Texas
    • Houston, Texas, United States, 77030
      • University of Texas MD Anderson MD Anderson Cancer Center

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

1. Subjects with hematologic malignancies or recipients of a first allogeneic or autologous hematopoietic stem cell transplantation and presently clinically stable
2. Pulse oximetry of hemoglobin saturation ≥92% on room air
3. Adult (≥18 years)
4. Spirometry (FEV1 and forced vital capacity [FVC]) ≥80% of predicted value
5. If female, must be either post-menopausal (one year or greater without menses), surgically sterile, or, for female subjects of child-bearing potential who are capable of conception must be: practicing two effective methods of birth control
6. If female, must not be pregnant, plan to become pregnant, or nurse a child during the study and through 30 days after completion of the study
7. If male, must be surgically sterile or willing to practice two effective methods of birth control
8. Ability to understand and give informed consent

Exclusion Criteria:

1. Subjects with any evidence of respiratory infection including any signs or symptoms of either a lower respiratory infection (LRI) or upper respiratory infection (URI)
2. Known history of chronic pulmonary disease
3. Subjects who are being treated for fungal, viral, or bacterial pneumonia
4. Exposure to any investigational agent (defined as any agent not approved by the Food and Drug Administration [FDA]) within 30 days prior to the Screening Visit
5. Patients with a relapsed and/or refractory underlying hematologic malignancy
6. HSCT recipients who underwent ex vivo T-cell depletion of the graft, or a mismatched, or cord or haplo identical blood transplantation
7. HSCT recipients with active and/or chronic graft versus host disease
8. Patients on systemic corticosteroids (oral or intravenous)
9. Absolute neutrophil count (ANC) < 1,000 cells/mL
10. Clinically significant bacteremia or fungemia
11. Current smokers or subjects with any history of smoking

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Prevention
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: PUL-042; PUL-042 Inhalation Solution	Drug: PUL-042; PUL-042 Inhalation Solution

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Forced Expiratory Volume in one Second (FEV1)	Evaluation of increasing doses of PUL-042 Inhalation Solution on FEV1	3 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Serum proteomics	Evalution of increasing doses of PUL-042 Inhalation Solution on serum proteomic profile	3 weeks

Collaborators and Investigators

• Sponsor: Pulmotect, Inc.
• Collaborators: National Heart, Lung, and Blood Institute (NHLBI); Cancer Prevention Research Institute of Texas

Study record dates

Study Major Dates

• Study Start (Anticipated): December 1, 2024
• Primary Completion (Anticipated): March 1, 2026
• Study Completion (Anticipated): May 1, 2026

Study Registration Dates

• First Submitted: March 27, 2017
• First Submitted That Met QC Criteria: March 27, 2017
• First Posted (Actual): March 31, 2017

Study Record Updates

• Last Update Posted (Actual): April 27, 2023
• Last Update Submitted That Met QC Criteria: April 25, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Respiratory System Agents; Pam2CSK4 acetate and ODN M362 combination
• Other Study ID Numbers: PUL-042-201A; R44HL127677 (U.S. NIH Grant/Contract)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No