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

NCT03187756 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: 17-00042
• Study Type: interventional
• Target: 6
• Locations: 1 country
• Sites: 1

Brief 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. 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).

Conditions

Hematopoietic Malignancies

Keywords

Hematopoietic Malignancies; Nonmyeloablative Peripheral Blood Stem Cell Transplant; Posttransplantation

Outcome Measures

Primary Outcomes (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

  One Year

Secondary Outcomes (6)

• Number of Participants With Chronic GVHD and Grades I-IV GVHD

  1 year

• Number of Major Toxicities and Complications Associated With Transplantation Procedure

  1 year

• Cumulative Incidences of Systemic Steroid Initiation

  1 year

• Graft Failure Frequency

  1 year

• Time to Neutrophil Recovery

  1 year

Study Arms (1)

Cyclophosphamide

EXPERIMENTAL

Drug: Cyclophosphamide

Interventions

Cyclophosphamide DRUG

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

Cyclophosphamide

Eligibility Criteria

• Age: 18 Years - 90 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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

You may not qualify if:

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

Sponsors & Collaborators

• NYU Langone Health: lead

Study Sites (1)

New York University School of Medicine

New York, New York, 10016, United States

Location

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

Cyclophosphamide

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds

Results Point of Contact

• Title: David Kaminetzky, MD
• Organization: Perlmutter Cancer Center, NYU Langone Health

David.Kaminetzky@nyulangone.org

212-731-5855

Study Officials

• Samer Al-Homsi, MD

  NYU Langone Health

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 2, 2017
• First Posted: June 15, 2017
• Study Start: June 2, 2017
• Primary Completion: December 18, 2018
• Study Completion: December 18, 2018
• Last Updated: March 27, 2020
• Results First Posted: March 27, 2020

Record last verified: 2020-03

Locations

US (1)