Clinical trial NCT03097796: A Multiple Ascending Dose Study of PUL-042 in Stem Cell Transplant Recipients
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
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
Conditions: Hematologic Diseases
Stem Cell Transplants
Lead sponsor: Pulmotect, Inc.
Sponsor class: INDUSTRY
Collaborators: name: National Heart, Lung, and Blood Institute (NHLBI)
name: Cancer Prevention Research Institute of Texas
First posted: 2017-03-31
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: PREVENTION

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.

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

Primary outcomes:
measure: Forced Expiratory Volume in one Second (FEV1)
timeFrame: 3 weeks
Secondary outcomes:
measure: Serum proteomics
timeFrame: 3 weeks
Other outcomes:
Locations:
city: Houston
country: United States
facility: University of Texas MD Anderson MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Roy F Chemaly, MD, MPH
role: PRINCIPAL_INVESTIGATOR
References: