A Phase 2 Multiple Dose Study to Evaluate the Efficacy and Safety of PUL-042 Inhalation Solution in Reducing Lower Respiratory Tract Complications in Patients with Hematologic Malignancies and Recipients of Hematopoietic Stem Cell Transplantation (HSCT) with Documented Viral Infections with Parainfluenza Virus (PIV), Human Metapneumovirus (hMPV) or Respiratory Syncytial Virus (RSV)

Cancer Categories

Hematologic (Blood Cancers)

Karmanos Trial ID

2024-076

NCT ID

NCT06665100

Age Group

Adult

Scope

National

Phase

Phase II

Principal Investigator

Joseph
Uberti, M.D., Ph.D.
Oncology - Hematology, Oncology - Medical View Profile

Objective:

Primary Objective:

To determine the efficacy of PUL-042 Inhalation Solution on lower respiratory tract complications (LRTC) using the peak post-treatment radiologic severity index (RSI) score 18 , 19 in subjects with hematologic malignancies (HM [lymphoma, multiple myeloma and leukemia]) and hematopoietic stem cell transplant (HSCT) recipients with documented parainfluenza virus (PIV), human metapneumovirus (hMPV) or respiratory syncytial virus (RSV) infection. The primary endpoint is the difference in the peak RSI score observed during the period following randomized treatment between Day 1 and Day 29.

Secondary Objectives

To determine the treatment difference in the change from pre-treatment to peak post-treatment RSI score through Day 29.
To determine the effect of PUL-042 through Day 29 on:
- Incidence of pneumonia
- Respiratory symptom scores
- Incidence of hospitalization
- Duration of hospitalization
- Incidence of ICU admission
- Duration of ICU care
- Oxygenation requirements
- Mortality
- Post-treatment viral RNA titers
- Change in viral RNA shedding relative to baseline
- Proportion of subjects positive for each virus at each sampling timepoint
To evaluate the dose/response of two dose levels of PUL-042 on the primary and secondary endpoints.
To evaluate the tolerability of PUL-042 in this population.

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Eligibility

Inclusion Criteria:

Subjects will be eligible for entry into the study if a nasopharyngeal swab is positive for PIV, RSV, or hMPV (as a single pathogen or a mixed infection with rhinovirus) by molecular assay by a local laboratory AND subjects must fulfill the following inclusion criteria to be eligible for participation in the study:

Subjects with hematologic malignancies (i.e., leukemia, lymphoma, or multiple myeloma) or recipients of an allogeneic or autologous hematopoietic stem cell transplantation for one of the following diagnoses: leukemia, lymphoma, Hodgkin's lymphoma, non-Hodgkin's lymphoma, multiple myeloma, and myelodysplastic and myeloproliferative disorder.
Subjects who have undergone active cytotoxic chemotherapy within 6 months or subjects who are on an immunosuppressive therapy (e.g., alemtuzumab, ibrutinib, mycophenolate mofetil, corticosteroids ≥1mg/kg prednisone equivalent).
Subjects who are recipients of an allogeneic hematopoietic stem cell transplant (HSCT) must be deemed high risk with an Immunodeficiency Scoring Index (ISI) , of greater or equal to 4.
Subjects who are recipients of an autologous HSCT must be within 3 months of the transplant procedure.
Subjects must be symptomatic with upper or lower respiratory tract symptoms such as rhinorrhea, sore throat or cough and must be dosed within 6 days from the onset of symptoms.
Chest X-ray with a Radiologic Severity Index (RSI) score of 6 or lower.
Subjects must have pulse oximetry of hemoglobin saturation ≥ 93% on room air.
Spirometry (forced expiratory volume in one second [FEV1] and forced vital capacity [FVC]) ≥70% of predicted value.
Adult (≥ 18 years of age).
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 (acceptable methods include intrauterine device, spermicide, barrier, male partner surgical sterilization, and hormonal contraception) during the study and through 30 days after completion of the study. Abstinence is not classified as an effective method of birth control.
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. A pregnancy test must be negative at the Screening Visit, prior to dosing on Day 1.
If male, must be surgically sterile or willing to practice two effective methods of birth control (acceptable methods include barrier, spermicide, or female partner surgical sterilization) during the study and through 30 days after completion of the study. Abstinence is not classified as an effective method of birth control.
Ability to understand and give informed consent.

Exclusion Criteria:

Subjects will be excluded if they fulfill any of the following exclusion criteria:

Patients with a pulse oximetry of hemoglobin saturation less than 93% on room air.
Known history of chronic pulmonary disease (e.g., asthma [including atopic asthma, exercise-induced asthma, or asthma triggered by respiratory infection], chronic pulmonary disease, pulmonary fibrosis, COPD), pulmonary hypertension, or heart failure.
Subjects treated for fungal, viral, or bacterial pneumonia in the previous 30 days.
Exposure to any investigational agent (defined as any non-FDA-approved agent) within 30 days, or 5 half-lives of the investigational agent, whichever is longer, prior to the Screening Visit.
Allogeneic HSCT recipients with an ISI of 3 or less.
Autologous HSCT recipients more than 3 months after the transplant procedure.
Patients with a relapsed and/or refractory underlying hematologic malignancy with a life expectancy of less than 2 months.
HSCT recipients in the pre-engraftment period.
Chest X-ray with an RSI of &gt;6.
Patients documented to be positive for other respiratory viruses (limited to influenza, SARS-CoV-2, adenovirus, or coronavirus) within 7 days prior to the Screening Visit, as determined by local testing (additional screening testing is not required).
Clinically significant bacteremia or fungemia within 7 days prior to the Screening Visit that has not been adequately treated, as determined by the Principal Investigator.
Any condition which, in the opinion of the Principal Investigator, would prevent full participation in this trial or would interfere with the evaluation of the trial endpoints.

Locations

Karmanos Cancer Institute - Detroit Headquarters

4100 John R
Detroit, MI 48201
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Phone: 1-800-527-6266

Applicable Disease Site

Therapies, Drugs, Devices