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

• Sponsor: Pulmotect, Inc. (Industry)
• Overall Status: Suspended
• CT.gov ID: NCT03097796
• Collaborator: National Heart, Lung, and Blood Institute (NHLBI) (NIH), Cancer Prevention Research Institute of Texas (Other)
• Enrollment: 18
• Location: 1
• Arm: 1
• Anticipated Duration (Months): 17
• Patients Per Site Per Month: 1.1

Study Details

Study Description

Brief Summary

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

Condition or Disease	Intervention/Treatment	Phase
Hematologic Diseases; Stem Cell Transplants	Drug: PUL-042	Phase 1

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 Design

• Study Type: Interventional
• Anticipated Enrollment: 18 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Prevention
• Official Title: 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
• Anticipated Study Start Date: Dec 1, 2023
• Anticipated Primary Completion Date: Mar 1, 2025
• Anticipated Study Completion Date: May 1, 2025

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures

1. Forced Expiratory Volume in one Second (FEV1) [3 weeks]

   Evaluation of increasing doses of PUL-042 Inhalation Solution on FEV1

Secondary Outcome Measures

1. Serum proteomics [3 weeks]

   Evalution of increasing doses of PUL-042 Inhalation Solution on serum proteomic profile

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	University of Texas MD Anderson MD Anderson Cancer Center	Houston	Texas	United States	77030

Sponsors and Collaborators

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

Investigators

• Principal Investigator: Roy F Chemaly, MD, MPH, M.D. Anderson Cancer Center

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Pulmotect, Inc.
• ClinicalTrials.gov Identifier: NCT03097796
• Other Study ID Numbers: PUL-042-201A; R44HL127677
• First Posted: Mar 31, 2017
• Last Update Posted: Apr 20, 2022
• Last Verified: Apr 1, 2022

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Hematologic Diseases; Pam2CSK4 acetate and ODN M362 combination

Study Results

No Results Posted as of Apr 20, 2022