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

NCT ID: NCT03097796

Last Updated: 2023-04-27

Basic Information

• Recruitment Status: WITHDRAWN
• Clinical Phase: PHASE1
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-12-31
• Study Completion Date: 2026-05-31

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

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.

Conditions

Hematologic Diseases; Stem Cell Transplants

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: PREVENTION
• Blinding Strategy: NONE

Study Groups

PUL-042

PUL-042 Inhalation Solution

Group Type: EXPERIMENTAL

PUL-042

Intervention Type: DRUG

PUL-042 Inhalation Solution

Interventions

PUL-042

PUL-042 Inhalation Solution

Intervention Type: DRUG

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role: collaborator

Cancer Prevention Research Institute of Texas

OTHER

Sponsor Role: collaborator

Pulmotect, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Roy F Chemaly, MD, MPH

Role: PRINCIPAL_INVESTIGATOR

M.D. Anderson Cancer Center

Locations

University of Texas MD Anderson MD Anderson Cancer Center

Houston, Texas, United States

Countries

United States

Other Identifiers

R44HL127677

Identifier Type: NIH

Identifier Source: secondary_id

View Link

PUL-042-201A

Identifier Type: -

Identifier Source: org_study_id