Hematologic DiseasesPhase 1NCT03097796
A Multiple Ascending Dose Study of PUL-042 in Stem Cell Transplant Recipients
A Phase 1 study testing PUL-042 in people with Hematologic Diseases and Stem Cell Transplants, run by Pulmotect, Inc. at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hematologic Diseases, Stem Cell Transplants
- What is being tested?
- PUL-042
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Dec 2024to May 2026
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subjects with hematologic malignancies or recipients of a first allogeneic or autologous hematopoietic stem cell transplantation and presently clinically stable
- Pulse oximetry of hemoglobin saturation ≥92% on room air
- Adult (≥18 years)
- Spirometry (FEV1 and forced vital capacity [FVC]) ≥80% of predicted value
- 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
- 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
- If male, must be surgically sterile or willing to practice two effective methods of birth control
- Ability to understand and give informed consent
Who cannot join
- Subjects with any evidence of respiratory infection including any signs or symptoms of either a lower respiratory infection (LRI) or upper respiratory infection (URI)
- Known history of chronic pulmonary disease
- Subjects who are being treated for fungal, viral, or bacterial pneumonia
- 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
- Patients with a relapsed and/or refractory underlying hematologic malignancy
- HSCT recipients who underwent ex vivo T-cell depletion of the graft, or a mismatched, or cord or haplo identical blood transplantation
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- HSCT recipients with active and/or chronic graft versus host disease
- Patients on systemic corticosteroids (oral or intravenous)
- Absolute neutrophil count (ANC) < 1,000 cells/mL
- Clinically significant bacteremia or fungemia
- Current smokers or subjects with any history of smoking
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Texas MD Anderson MD Anderson Cancer Center
Houston, Texas, 77030
About this study
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
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
Read the 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
- Being tested
- PUL-042
- Medical subject headings
- Hematologic Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PUL-042Drug
PUL-042 Inhalation Solution
| Study group | Role | What they receive |
|---|---|---|
| PUL-042 | Experimental | PUL-042 Inhalation Solution Receives: Drug: PUL-042 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Forced Expiratory Volume in one Second (FEV1)
Evaluation of increasing doses of PUL-042 Inhalation Solution on FEV1
Measured: 3 weeks
Secondary outcomes (1)
Serum proteomics
Evalution of increasing doses of PUL-042 Inhalation Solution on serum proteomic profile
Measured: 3 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 Mar 2017
- Study startDec 2024 (estimated)
- Primary completionMar 2026 (estimated)
- Study completionMay 2026 (estimated)
Why it stopped: Development plan revised
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematologic DiseasesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Pulmotect, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Cancer Prevention Research Institute of Texas
- Responsible party
- Sponsor
- Investigators
- Roy F Chemaly, MD, MPH, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03097796
- Sponsor's protocol ID
- PUL-042-201A
- Other IDs
- R44HL127677
- Status last verified
- Apr 2023
- First submitted
- 27 Mar 2017
- Last updated
- 27 Apr 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Apr 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03097796
- Acronym / sponsor ID
- PUL-042-201A
- Conditions
- Hematologic Diseases, Stem Cell Transplants
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Development plan revised
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Pulmotect, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI), Cancer Prevention Research Institute of Texas
- Study start
- Dec 2024 (estimated)
- Primary completion
- Mar 2026 (estimated)
- Study completion
- May 2026 (estimated)
- First posted
- 31 Mar 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Prevention
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| PUL-042 | Experimental | Drug: PUL-042PUL-042 Inhalation Solution |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (8 listed)
- Subjects with hematologic malignancies or recipients of a first allogeneic or autologous hematopoietic stem cell transplantation and presently clinically stable
- Pulse oximetry of hemoglobin saturation ≥92% on room air
- Adult (≥18 years)
- Spirometry (FEV1 and forced vital capacity [FVC]) ≥80% of predicted value
- 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
Key exclusion criteria (11 listed)
- Subjects with any evidence of respiratory infection including any signs or symptoms of either a lower respiratory infection (LRI) or upper respiratory infection (URI)
- Known history of chronic pulmonary disease
- Subjects who are being treated for fungal, viral, or bacterial pneumonia
- 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
- Patients with a relapsed and/or refractory underlying hematologic malignancy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PUL-042PUL-042 Inhalation Solution | Drug | PUL-042 |
Primary outcome
Forced Expiratory Volume in one Second (FEV1)
Time frame: 3 weeks
Evaluation of increasing doses of PUL-042 Inhalation Solution on FEV1
1 secondary outcome
Serum proteomics
Time frame: 3 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Apr 2023.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Roy F Chemaly, MD, MPH principal investigator
M.D. Anderson Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing PUL-042 in people with Hematologic Diseases and Stem Cell Transplants, run by Pulmotect, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 27 Apr 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Forced Expiratory Volume in one Second (FEV1) (time frame: 3 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Apr 2023.