Haplo-identical SCT for High Risk (HR) Hematologic Malignancies w/Post-Transplant In-Vivo T-cell Depletion

NCT02053545 | Withdrawn Phase 1 DMC

• 1 other identifier: SCT 0813 Haplo
• Study Type: interventional
• Target: N/A
• Locations: 1 country
• Sites: 1

Brief Summary

Although a majority of children with leukemia and most hematological malignancies (Hodgkin's and Non-Hodgkin's lymphomas) can be cured with conventional chemotherapy, a subset of patients with resistant/recurrent high-risk disease are not cured with conventional treatment regimens. Investigators hypothesize that HSCT from a partially matched donor can be safe and effective for patients with very high risk hematologic malignancies when combined with post-transplant cyclophosphamide for prevention of graft-vs-host disease (GVHD).

Conditions

Leukemia; Lymphoma; MDS

Outcome Measures

Primary Outcomes (1)

• Cumulative non-relapse mortality at 100 days and 365 days post haplo-identical transplant

  Patients will be followed regularly for engraftment, complications, and disease control. Non-relapse mortality and overall survival will be recorded from date of HSCT.

  1 Year

Secondary Outcomes (1)

• Cumulative acute and chronic GVHD, cumulative relapse rates, and overall survival and event free survival

  1 Year

Other Outcomes (1)

• Engraftment Kinetics and Immune Reconstitution

  1 year

Study Arms (1)

Conditioning Regimen & GVHD Prophylaxis

EXPERIMENTAL

Stratum 1 (Refractory disease, relapse after previous transplant): Clofarabine, Melphalan,Thiotepa, Cyclophosphamide, Mesna, Tacrolimus and mycophenolate mofetil (MMF) Stratum 2 (Myeloid in remission): Busulfan, Fludarabine, Thiotepa, Cyclophosphamide, Mesna, Tacrolimus, MMF Stratum 3 (Lymphoid in remission): Fractionated total body irradiation (fTBI), Fludarabine, Thiotepa, Cyclophosphamide, Mesna, Tacrolimus, MMF

Drug: Conditioning Regimen & GVHD Prophylaxis

Interventions

Conditioning Regimen & GVHD Prophylaxis DRUG

Stratum 1 (Refractory disease, relapse after previous transplant): Radiation Therapy (if patient had prior Central Nervous System (CNS) disease), Clofarabine, Melphalan,Thiotepa, Stem Cell Transplant, Cyclophosphamide, Mesna, Tacrolimus and MMF Stratum 2 (Myeloid in remission): Radiation Therapy (if patient had prior CNS disease), Busulfan, Fludarabine, Thiotepa, Stem Cell Transplant, Cyclophosphamide, Mesna, Tacrolimus, MMF Stratum 3 (Lymphoid in remission): Radiation Therapy (if patient had prior CNS disease), TBI, Fludarabine, Thiotepa, Stem Cell Transplant, Cyclophosphamide, Mesna, Tacrolimus, MMF

Conditioning Regimen & GVHD Prophylaxis

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must have confirmed diagnosis of hematologic malignancy (leukemia, lymphoma or MDS) with the following:
• Resistant/refractory hematologic malignancies (disease exceeding 5% of marrow cells by morphology, which is a description of white blood cell types as assessed via light microscopy; or with measurable extramedullary disease, which is detection of leukemia at sites other than blood and marrow. This includes disease that infiltrates into tissues other than the spleen and marrow. i.e. nodal disease, which is leukemia and/or lymphoma involving a lymph node or chloroma, which is a collection of leukemic cells forming a mass/tumor mass) (Stratum 1).
• Or have relapsed following an initial allogeneic HSCT (Stratum 1).
• And/or lack an adequately matched unrelated donor (URD) or unrelated cord blood (URB) hematopoietic stem cell (HSC) source (see protocol section 2) (Strata 1, 2 \& 3).
• Adequate cardiac, pulmonary, renal, and hepatic functions
• Central vascular access providing a combined 3 access ports for all patients.
• Females of childbearing potential must have a negative pregnancy test prior to therapy. Pregnancy tests will only be done prior to therapy. Sexually active patients will be informed of the risk of not using adequate contraception.
• Recipient or legal guardian must be informed of the study, and have signed a consent form.
• Recipients must have a related haplo-identical donor.

You may not qualify if:

• High risk hematologic malignancies in remission (and no prior allogeneic HSCT), where allogeneic HSCT is indicated but an appropriately matched HSC source (sibling, unrelated adult or UCB) is available.
• Patients with systemic infections and/or organ dysfunction mandating a reduced intensity conditioning regimen are also excluded.

Sponsors & Collaborators

• Ann & Robert H Lurie Children's Hospital of Chicago: lead

Study Sites (1)

Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States

Location

MeSH Terms

Conditions

Leukemia; Lymphoma

Interventions

Transplantation Conditioning

Condition Hierarchy (Ancestors)

Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases; Lymphoproliferative Disorders; Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunosuppression Therapy; Immunotherapy; Immunomodulation; Biological Therapy; Therapeutics; Immunologic Techniques; Investigative Techniques

Study Officials

• Reggie Duerst, MD

  Ann & Robert H Lurie Children's Hospital of Chicago

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Primary Investigator

Study Record Dates

• First Submitted: December 31, 2013
• First Posted: February 3, 2014
• Study Start: December 1, 2013
• Primary Completion: May 11, 2017
• Study Completion: May 11, 2017
• Last Updated: August 30, 2019

Record last verified: 2019-08

Locations

US (1)