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Hematopoietic MalignanciesPhase 2NCT03187756

Study of Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Hematopoietic Malignancies Including Those That Are Challenging to Engraft

A Phase 2 study testing Cyclophosphamide in people with Hematopoietic Malignancies, run by NYU Langone Health at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

This is an open label phase II single arm study of peripheral blood stem cell transplantation and posttransplantation cyclophosphamide, using HLA full match or haploidentical related donors, in hematological malignancies including those difficult to engraft. Read more about this study

Who is it for?
Ages 18 to 90 years
Conditions the study is for
Hematopoietic Malignancies
What is being tested?
Cyclophosphamide
Could I receive a placebo?
No, everyone receives an active treatment
How many people
6 participants
Where
1 siteUnited States
Study dates
2 Jun 2017to 18 Dec 2018
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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 90 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • The following are eligibility for study entry and transplantation.
  • Presence of a suitable related, HLA-haploidentical or HLA-matched stem cell donor
  • The donor and recipient must be identical at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, HLA-DRB1, and HLA-DQB1. A minimum match of 5/10 is therefore required for related donors, and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.
  • Eligible diagnoses:
  • Myelodysplastic syndrome (MDS) including chronic myelomonocytic leukemia [CMML] with at least one poor risk factor
  • No active extramedullary leukemia or known active CNS involvement by malignancy. Such disease treated into remission is permitted.
Show all 31Show fewer
  • Any previous autologous HSCT must have occurred at least 3 months prior to start of conditioning
  • No previous allogeneic HSCT
  • Adequate end-organ function Note: Infection is permitted if there is evidence of response to medication. Eligibility of HIV infected patients will be determined on a case-by-case basis.
  • ECOG performance status < 2 or Karnofsky or Lansky score > 60.
  • Age > 18 years and older.
  • Not pregnant or breast-feeding.
  • No uncontrolled infection.
  • Eligible diagnoses:
  • Myelodysplastic syndrome (MDS) including chronic myelomonocytic leukemia [CMML] with at least one of the following poor-risk features
  • SLL or CLL with 17p deletion, or with progression < 6 months after second or greater treatment regimen. Must have the following to be an acceptable candidate as well:
  • < 20% of bone marrow cellularity involved by SLL/CLL (to lower risk of graft rejection)
  • No lymph nodes > 5 cm in any dimension
  • No massive splenomegaly, defined as > 6 cm below the left costal margin
  • T-cell PLL in PR or better prior to transplantation. Must also have < 20% of bone marrow cellularity involved by PLL (to lower risk of graft rejection).
  • Interferon- or tyrosine kinase-refractory CML in first chronic phase, TKI-intolerant CML in first chronic phase, or CML in second or subsequent chronic phase
  • Philadelphia chromosome negative myeloproliferative disease (including myelofibrosis)
  • o Intermediate-2 or High risk score by DIPSS Plus is required for a diagnosis of myelofibrosis
  • Multiple myeloma or plasma cell leukemia with a PR or better to the last treatment regimen, based on the International Myeloma Working Group (IMWG) criteria.49
  • Hematologic malignancy in complete remission with minimal residual disease (MRD) non detectable OR detectable by conventional cytogenetics, FISH, flow cytometry, or molecular testing or hematologic malignancies in partial remission
  • Donor eligibility
  • Donors must be either:
  • HLA-haploidentical or HLA-identical relatives of the patient based on allele or allele group level typing as defined in Section 4.1.
  • Medically fit to and willing to donate
  • Lack of recipient anti-donor HLA antibody
  • Has not donated blood products to patient

Who cannot join

  • Any individual that does not meet the eligibility criteria for transplantation or donor eligibility will not be a part of this trial.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • New York University School of Medicine

    New York, New York, 10016

Other trials in United States

About this study

Official title: Phase II Study of Nonmyeloablative Peripheral Blood Stem Cell Transplant With High-dose Posttransplantation Cyclophosphamide in Hematopoietic Malignancies Including Those That Are Challenging to Engraft

This is an open label phase II single arm study of peripheral blood stem cell transplantation and posttransplantation cyclophosphamide, using HLA full match or haploidentical related donors, in hematological malignancies including those difficult to engraft. The objective of this study is to evaluate the safety and feasibility in nonmyeloablative, partially HLA-mismatched or HLA-matched PBSC transplant from haploidentical donors or fully matched donors with post-grafting immunosuppression that includes high-dose cyclophosphamide, tacrolimus, and Mycophenolate mofetil (MMF).

Read the detailed description

Primary Objective Estimate event free survival (EFS) (relapse, progression, or death) rate one year after transplant.

Secondary Objectives:

  1. Estimate the cumulative incidences of severe acute grade III or higher GVHD, chronic GVHD (overall and by extent)
  2. Estimate the cumulative incidence of systemic steroid initiation,
  3. Summarize the graft failure frequency,
  4. Summarize the kinetics of neutrophil and platelet recovery, and kinetics of donor chimerism in unsorted and CD3+ sorted peripheral blood.
  5. Summarize major toxicities and complications associated with the transplantation procedure selected toxicities.

Exploratory Objectives:

Explore the association between the amount of donor T cell chimerism at ~ Day 28 and patient/graft characteristics (e.g., prior therapies, graft cell dose) and transplantation outcomes (sustained engraftment, relapse or progression, GVHD).

Being tested
Cyclophosphamide
Keywords
Hematopoietic Malignancies, Nonmyeloablative Peripheral Blood Stem Cell Transplant, Posttransplantation
Medical subject headings
Hematologic Neoplasms

What participants receive

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

  • CyclophosphamideDrug

    Shortened duration immunosuppression following nonmyeloablative peripheral blood stem cell transplant with high dose post transplantation cyclophosphamide in malignancies to engraft.

Study groups in NCT03187756
Study groupRoleWhat they receive
CyclophosphamideExperimentalNot described
Receives: Drug: Cyclophosphamide

What is being measured

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

  1. Event Free Survival (EFS)

    Estimate the one year after transplantation event free survival (EFS) rate using a Kaplan-Meier curve with a 90% confidence interval. An event for EFS is defined as the first of any of the following failures: relapse or disease progression or death from any cause

    Measured: One Year

Secondary outcomes (6)

  1. Number of Participants With Chronic GVHD and Grades I-IV GVHD

    Acute GVHD is graded by standard criteria, and all suspected cases of acute GVHD will be confirmed histologically by biopsy of an affected organ. The severity of acute GVHD is determined by an assessment of the degree of involvement of the skin, liver, and gastrointestinal tract. Grade I is characterized as mild disease (skin involvement alone), Grade II as moderate, Grade III as severe (involvement of any organ system), and Grade IV as life-threatening. The diagnosis of a chronic GVHD per NIH criteria requires a) at least 1 diagnostic manifestation or b) 1 distinctive manifestation confirmed by biopsy or testing of the same or other involved organ.

    Measured: 1 year

  2. Number of Major Toxicities and Complications Associated With Transplantation Procedure

    Summarize major toxicities and complications associated with the transplantation procedure

    Measured: 1 year

  3. Cumulative Incidences of Systemic Steroid Initiation

    Estimate the cumulative incidence of systemic steroid initiation, by 1 year after HSCT. This is will be reported as number of participants who started steroids over the course of the study.

    Measured: 1 year

  4. Graft Failure Frequency

    Graft failure and death, or graft failure, death and treatment of relapse/progressions. This will be reported as the number of participants with graft failures.

    Measured: 1 year

  5. Time to Neutrophil Recovery

    Neutrophil recovery is defined as post-nadir ANC greater than or equal to 500/mm3 for three consecutive measurements on different days. The first of the three days will be designated as the day of neutrophil recovery.

    Measured: 1 year

  6. Time to Platelet Recovery

    Platelet recovery is defined as sustained platelet count greater than or equal to 20,000/mm3 or greater than or equal to 50,000/mm3 with no platelet transfusions in the preceding seven days. The first of three consecutive measurements on different days will be designated as the day of initial platelet recovery.

    Measured: 1 year

Timeline

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

  1. First posted15 Jun 2017
  2. Study start2 Jun 2017
  3. Primary completion18 Dec 2018
  4. Study completion18 Dec 2018

Why it stopped: Procedure has become standard of care - protocol is no longer necessary

Resources

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
6 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT03187756
Sponsor's protocol ID
17-00042
Status last verified
Mar 2020
First submitted
2 Jun 2017
Last updated
27 Mar 2020
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Cyclophosphamide in people with Hematopoietic Malignancies, run by NYU Langone Health 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 31 inclusion and 1 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: stopped early, as of the sponsor's last update on 27 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. 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 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?

Yes. Results are posted in the registry (first posted 27 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Event Free Survival (EFS) (time frame: One Year). 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 NCT03187756.

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 Mar 2020.

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