Hematologic MalignancyPhase 1/2NCT03986086

MPH966 for Prevention of Graft-versus-host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

A Phase 1/2 study testing MPH966 in people with Hematologic Malignancy, run by Nelson Chao at 1 site in United States.

Withdrawn

Withdrawn. The study stopped before anyone enrolled.

Trial summary

The purpose of this study is evaluate the safety and tolerability of MPH966, a neutrophil elastase inhibitor, and its ability to prevent graft-versus-host disease after hematopoietic stem cell transplant. Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Hematologic Malignancy
What is being tested?: MPH966
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 0 participants
Where: 1 siteUnited States
Study dates: Sep 2021to Dec 2023
Phase: Phase 1/2
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 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Provision of written informed consent prior to any study specific procedures
Plan to undergo allogeneic HCT for any cancer or non-cancer illness with a planned cell dose of ≥2 x 106 CD34/kg using peripheral blood stem cells.
Plan to receive a myeloablative conditioning regimen (see 4.3.1).
Plan to receive GVHD prophylaxis with tacrolimus and methotrexate.
Having a donor who is a 10 of 10 HLA match;
Karnofsky Performance Scale KPS ≥60
Willing to abstain from sexual activity or use two methods of birth control while on study drug and for 5 half-lives (4 days) after last dose.

Who cannot join

If female, pregnant or nursing.
Life expectancy <6 months
Other malignancy or neoplastic disease (i.e. aside from the malignancy for which they are undergoing HCT) within the past 5 years with the exception of treated basal/squamous cell skin carcinoma or treated cervical cancer in situ
Clinically significant active infection within 1 week of starting study drug
Any of the following organ system function criteria:
Cardiac: Ejection fraction ≤40% or myocardial infarction within 6 months of transplant or QTc >450 msec for males and >470 msec for females or other EKG abnormality which in the opinion of the investigator may put the subject at risk or interfere with study assessments

Show all 19Show fewer

Renal: Creatinine clearance (CLcr) ≤ 60 mL/min as estimated by the Cockcroft-Gault equation
Pulmonary: FEV1, FVC, or corrected DLCO ≤40% predicted (forced expiratory volume in 1 second; forced vital capacity; and diffusing capacity of the lung for carbon monoxide, respectively)
Hepatic: Total bilirubin >1.5 x (in the absence of known inherited hyperbilirubinemia, e.g. Gilbert's) and/or aspartate transaminase (AST)/alanine transaminase (ALT) >3 x upper limit of institutional normal for age (grade 2 or higher) and/or INR >1.5 (unless on anticoagulant), or history or evidence of cirrhosis (e.g. esophageal varices, ascites, or hepatic encephalopathy) or other chronic liver disease (e.g. Wilson's disease, autoimmune liver disease, primary biliary cirrhosis, etc.). Abnormalities in platelet number or albumin will not be considered exclusion criteria given that these are often due to the hematologic malignancy for which the patient is undergoing HCT rather than actual liver dysfunction
Uncontrolled infection, including detection of hepatitis B virus (HBV) or hepatitis C virus (HCV) by serology or nucleic acid testing or HIV by polymerase chain reaction (PCR)
Treated HBV/HCV/HIV with documented clearance is ok f. Other significant organ dysfunction (cardiac, pulmonary, renal, metabolic or central nervous system) that is uncontrolled and may interfere with study completion
Any significant medial history of alcohol abuse within 3 months of starting study drug and/or unwillingness to abstain for the duration of the study and follow up periods
Prior (within 30 days) or concomitant use of another neutrophil elastase inhibitor (e.g. alpha-1 antitrypsin)
Plan for in vivo or ex vivo T cell depletion.
Participated in another clinical study involving an investigational drug or device within 30 days or 5 half-lives prior to planned start of MPH966/placebo, or scheduled to participate in another clinical study involving an investigational drug or device within Day 100 of transplant
If the patient develops GVHD within the first 100 days, they are allowed to enroll on trials of investigational drugs to treat GVHD provided they come off of this study.
Enrollment in biorepository or supportive care trials that do not involve investigational drugs or devices is allowed
Any clinically relevant abnormal findings in physical examination, vital signs, hematology, clinical chemistry, or urinalysis at visit, which in the opinion of the Investigator, may put the subject at risk because of his/her participation in the study, or may influence the results of the study, or the subject's ability to participate in the study
Low or intermediate risk acute leukemia in first complete remission, chronic myeloid leukemia in first chronic phase, and any benign (non-malignant) disorders (phase 1 dose-escalation portion only)

Trial locations and contacts

1 site in 1 country.

United States1 site

Duke University Adult Bone Marrow Transplant Clinic
Durham, North Carolina, 27705

Other trials in United States

About this study

Official title: A Phase 1/2 Study of MPH966, an Oral Neutrophil Elastase Inhibitor, for Prevention of Graft-versus-host Disease After Allogeneic Hematopoietic Stem Cell Transplantation

Read the detailed description

Phase 1 is a 3+3 dose escalation study to determine the safety and recommended phase 2 dose (RP2D) of MPH966 in patients undergoing allogeneic hematopoietic stem cell transplantation (HCT). We will evaluate up to 4 doses: 60 mg po bid, 120 mg po bid, 180 mg po bid, and 240 mg po bid. Safety, tolerability, and efficacy will be assessed in real time and pharmacokinetics and pharmacodynamics after each dose cohort before escalating to the next cohort.

Phase 2 is a randomized, double-blind, placebo-controlled study to determine the clinical efficacy of MPH966 vs. placebo in preventing acute graft-versus-host disease (GVHD) after HCT, using the RP2D as determined by the phase 1 trial.

Conditions: Hematologic Malignancy
Being tested: MPH966, Placebo
Medical subject headings: Hematologic Neoplasms

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

MPH966Drug
Also called alvelestat
RP2D tablet
PlaceboDrug
MPH966 placebo table

Study groups in NCT03986086
Study group	Role	What they receive
MPH966	Experimental	Participants receive MPH966 at RP2D tablet orally twice daily from the start of conditioning chemotherapy through 45 days post transplant. Receives: Drug: MPH966
Placebo	Placebo comparator	Participants receive MPH placebo tablet matching MPH966 orally twice daily from the start of conditioning chemotherapy through 45 days post transplant. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Incidence of grade 2-4 acute Graft vs Host Disease (GVHD) requiring systemic therapy
Measured: day 100

Secondary outcomes (74)

Incidence of grade 2-4 acute GVHD
Measured: day 100
Incidence of grade 3-4 acute GVHD
Measured: day 100
Incidence of grade 2-4 acute GVHD
Measured: month 6
Incidence of grade 3-4 acute GVHD
Measured: month 6
Kaplan-Meier analysis of time-to-event: percentage of participants who develop grade 2-4 acute GVHD by visit
Measured: day 0 and 100 days, 6 months
Kaplan-Meier analysis of time-to-event: percentage of participants who develop grade 3-4 acute GVHD by visit
Measured: Day 0 and day 100, month 6
Incidence of chronic GVHD
Measured: month 6
Incidence of chronic GVHD
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop chronic GVHD
Time to event distributions estimated by the Kaplan-Meier method
Measured: Day 0 and month 6, 12
Incidence of GVHD-free survival
GVHD-free survival is defined as freedom from GVHD requiring systemic steroids
Measured: month 6
Incidence of GVHD-free survival
GVHD-free survival is defined as freedom from GVHD requiring systemic steroids
Measured: month 12
Incidence of relapse-free survival
Relapse-free survival is defined as freedom from relapse
Measured: month 6
Incidence of relapse-free survival
Relapse-free survival is defined as freedom from relapse
Measured: month 12
Incidence of bacterial infections
Measured: Day 100
Incidence of fungal infections
Measured: Day 100
Incidence of viral infections
Measured: Day 100
Incidence of overall infections
Measured: Day 100
Incidence of bacterial infections
Measured: 6 months
Incidence of fungal infections
Measured: month 6
Incidence of viral infections
Measured: month 6
Incidence of overall infections
Measured: month 6
Incidence of bacterial infections
Measured: month 12
Incidence of fungal infections
Measured: month 12
Incidence of viral infections
Measured: month 12
Incidence of overall infections
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop bacterial infections
Measured: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop fungal infections
Measured: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop viral infections
Measured: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop overall infections
Measured: Day 0 and day 100, month 6,12
Incidence of relapse
Measured: month 6
Incidence of relapse
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who relapse
Measured: Day 0 and month 6,12
Incidence of non-relapse mortality
Non-relapse mortality is defined as death while in remission from the primary disease
Measured: month 6
Incidence of non-relapse mortality
Non-relapse mortality is defined as death while in remission from the primary disease
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who experience non-relapse mortality
Non-relapse mortality is defined as death while in remission from the primary disease
Measured: Day 0 and month 6,12
Incidence of hospital re-admission
Measured: Day 100
Incidence of hospital re-admission
Measured: month 6
Incidence of hospital re-admission
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who are re-admitted to the hospital
Measured: Day 0 and day 100, month 6,12
Length of hospital re-admission
Measured: Day 100
Length of hospital re-admission
Measured: month 6
Length of hospital re-admission
Measured: month 12
Incidence of intensive care unit (ICU) admission
Measured: Day 100
Incidence of intensive care unit (ICU) admission
Measured: month 6
Incidence of intensive care unit (ICU) admission
Measured: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who are admitted to ICU
Measured: Day 0 and month 6,12
Length of intensive care unit (ICU) admission
Measured: Day 100
Length of intensive care unit (ICU) admission
Measured: month 6
Length of intensive care unit (ICU) admission
Measured: month 12
Length of stay in days between transplant and discharge to home
To determine the length of stay between transplant (Day 0) and discharge to home for those alive to be discharged home
Measured: Day 0 until discharge from hospital, up to 100 days
Quality of life as measured by the FACT-BMT assessment
Measured: day 30
Quality of life as measured by the FACT-BMT assessment
Measured: day 100
Quality of life as measured by the EQ 5D-5L assessment
Measured: Day 30
Quality of life as measured by the EQ 5D-5L assessment
Measured: Day 100
Quality of life as measured by the Lorig Self-Efficacy assessment
Measured: Day 30
Quality of life as measured by the Lorig Self-Efficacy assessment
Measured: Day 100
Quality of life as measured by the PG-SGA (patient-generated subjective global assessment)
Measured: day 30
Quality of life as measured by the PG-SGA (patient-generated subjective global assessment)
Measured: day 100
Quality of life as measured by the PROMIS-Depression assessment
Measured: day 30
Quality of life as measured by the PROMIS-Depression assessment
Measured: day 100
Quality of life as measured by the PROMIS-Anxiety assessment
Measured: Day 30
Quality of life as measured by the PROMIS-Anxiety assessment
Measured: Day 100
Quality of life as measured by the PROMIS-Social Isolation assessment
Measured: Day 30
Quality of life as measured by the PROMIS-Social Isolation assessment
Measured: Day 100
Quality of life as measured by the PROMIS-Emotional Support assessment
Measured: day 30
Quality of life as measured by the PROMIS-Emotional Support assessment
Measured: Day 100
Quality of life as measured by the PROMIS-Cognitive Function assessment
Measured: Day 30
Quality of life as measured by the PROMIS-Cognitive Function assessment
Measured: Day 100
Quality of life as measured by the PROMIS-Physical Function assessment
Measured: Day 30
Quality of life as measured by the PROMIS-Physical Function assessment
Measured: Day 100
Rate of grade 2 or higher adverse events, causally related during treatment period
Measured: Day 75
Rate of grade 2 or higher adverse events, causally related during follow up period
Measured: Year 1
Rate of grade 2 or higher adverse events, non related during treatment period
Measured: Day 75
Rate of grade 2 or higher adverse events, non related during follow up period
Measured: Year 1

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Sep 2021Dec 2023

First posted14 Jun 2019
Study startSep 2021 (estimated)
Primary completionDec 2022 (estimated)
Study completionDec 2023 (estimated)

Why it stopped: Lack of Funding

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Hematologic MalignancyStudies 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 1/2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Care provider
Enrolment: 0 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03986086
Sponsor's protocol ID: Pro00102362
Status last verified: Mar 2020
First submitted: 12 Jun 2019
Last updated: 20 Mar 2020
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Hematologic Malignancy trials

Study at a glance

From the registry record, last updated by the sponsor on 20 Mar 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase 1/2 Study of MPH966, an Oral Neutrophil Elastase Inhibitor, for Prevention of Graft-versus-host Disease After Allogeneic Hematopoietic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT03986086
Acronym / sponsor ID: Pro00102362
Conditions: Hematologic Malignancy
Phase: Phase 1/2
Study type: Interventional
Recruitment status: WithdrawnReason stopped: Lack of Funding
Enrolment: 0 participants (actual)
Lead sponsor: Nelson ChaoUniversity, hospital or other
Collaborators: Mereo BioPharma, National Center for Advancing Translational Sciences (NCATS)
Study start: Sep 2021 (estimated)
Primary completion: Dec 2022 (estimated)
Study completion: Dec 2023 (estimated)
First posted: 14 Jun 2019 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, care provider)
Number of arms: 2

Arm	Type	Receives
MPH966	Experimental	Drug: MPH966Participants receive MPH966 at RP2D tablet orally twice daily from the start of conditioning chemotherapy through 45 days post transplant.
Placebo	Placebo comparator	Drug: PlaceboParticipants receive MPH placebo tablet matching MPH966 orally twice daily from the start of conditioning chemotherapy through 45 days post transplant.

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (7 listed)

Provision of written informed consent prior to any study specific procedures
Plan to undergo allogeneic HCT for any cancer or non-cancer illness with a planned cell dose of ≥2 x 106 CD34/kg using peripheral blood stem cells.
Plan to receive a myeloablative conditioning regimen (see 4.3.1).
Plan to receive GVHD prophylaxis with tacrolimus and methotrexate.
Having a donor who is a 10 of 10 HLA match;

Key exclusion criteria (19 listed)

If female, pregnant or nursing.
Life expectancy <6 months
Other malignancy or neoplastic disease (i.e. aside from the malignancy for which they are undergoing HCT) within the past 5 years with the exception of treated basal/squamous cell skin carcinoma or treated cervical cancer in situ
Clinically significant active infection within 1 week of starting study drug
Any of the following organ system function criteria:

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
MPH966Also: alvelestatRP2D tablet	Drug	MPH966
PlaceboMPH966 placebo table	Drug	Placebo

Primary outcome

Incidence of grade 2-4 acute Graft vs Host Disease (GVHD) requiring systemic therapy
Time frame: day 100

74 secondary outcomes

Incidence of grade 2-4 acute GVHD
Time frame: day 100
Incidence of grade 3-4 acute GVHD
Time frame: day 100
Incidence of grade 2-4 acute GVHD
Time frame: month 6
Incidence of grade 3-4 acute GVHD
Time frame: month 6
Kaplan-Meier analysis of time-to-event: percentage of participants who develop grade 2-4 acute GVHD by visit
Time frame: day 0 and 100 days, 6 months
Kaplan-Meier analysis of time-to-event: percentage of participants who develop grade 3-4 acute GVHD by visit
Time frame: Day 0 and day 100, month 6
Incidence of chronic GVHD
Time frame: month 6
Incidence of chronic GVHD
Time frame: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop chronic GVHD
Time frame: Day 0 and month 6, 12
Incidence of GVHD-free survival
Time frame: month 6
Incidence of GVHD-free survival
Time frame: month 12
Incidence of relapse-free survival
Time frame: month 6
Incidence of relapse-free survival
Time frame: month 12
Incidence of bacterial infections
Time frame: Day 100
Incidence of fungal infections
Time frame: Day 100
Incidence of viral infections
Time frame: Day 100
Incidence of overall infections
Time frame: Day 100
Incidence of bacterial infections
Time frame: 6 months
Incidence of fungal infections
Time frame: month 6
Incidence of viral infections
Time frame: month 6
Incidence of overall infections
Time frame: month 6
Incidence of bacterial infections
Time frame: month 12
Incidence of fungal infections
Time frame: month 12
Incidence of viral infections
Time frame: month 12
Incidence of overall infections
Time frame: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop bacterial infections
Time frame: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop fungal infections
Time frame: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop viral infections
Time frame: Day 0 and day 100, month 6,12
Kaplan-Meier analysis of time-to-event: percentage of participants who develop overall infections
Time frame: Day 0 and day 100, month 6,12
Incidence of relapse
Time frame: month 6
Incidence of relapse
Time frame: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who relapse
Time frame: Day 0 and month 6,12
Incidence of non-relapse mortality
Time frame: month 6
Incidence of non-relapse mortality
Time frame: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who experience non-relapse mortality
Time frame: Day 0 and month 6,12
Incidence of hospital re-admission
Time frame: Day 100
Incidence of hospital re-admission
Time frame: month 6
Incidence of hospital re-admission
Time frame: month 12
Kaplan-Meier analysis of time-to-event: percentage of participants who are re-admitted to the hospital
Time frame: Day 0 and day 100, month 6,12
Length of hospital re-admission
Time frame: Day 100

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 20 Mar 2020.
Individual participant dataSharing planned: No

Related trials for Hematologic Malignancy

RecruitingPhase 2
Tafasitamab With Acalabrutinib and Venetoclax for the Treatment of Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Chronic Lymphocytic Leukemia, Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma
Not yet recruitingPhase 2
Study of YKST02 in Adults With Relapsed or Refractory Multiple Myeloma
Relapsed or Refractory Multiple Myeloma (RRMM)
RecruitingPhase 2
Engaging T-cells to Eliminate MRD in Newly Diagnosed Myeloma Optimizing Response With Talquetmab and Teclistamab (ROTATE)
Multiple Myeloma
Active, not recruitingPhase 3
A Phase III Study of Eque-cel in Subjects With Len-refractory RRMM (FUMANBA-03)
Multiple Myeloma
Active, not recruitingPhase 1/2
A Window of Opportunity Trial to Learn if Linvoseltamab is Safe and Well Tolerated, and How Well it Works in Adult Participants With Recently Diagnosed Multiple Myeloma Who Have Not Already Received Treatment
Multiple Myeloma

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.

Anthony Sung, MD principal investigator

Duke University Health System (DUHS)

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing MPH966 in people with Hematologic Malignancy, run by Nelson Chao 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 7 inclusion and 19 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 20 Mar 2020. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

Incidence of grade 2-4 acute Graft vs Host Disease (GVHD) requiring systemic therapy (time frame: day 100). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03986086.

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 20 Mar 2020.