Haploidentical Hematopoietic Cell Transplantation for Children With Hematologic Malignancies and Myelodysplasia

NCT03431090 | Active Not Recruiting Phase 1 DMC FDA Device

• 1 other identifier: IRB17-00516
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase I/II study designed to evaluate the kinetics of hematopoietic reconstitution and the incidence of acute chronic GVHD after partially matched related donor hematopoietic cell transplantation using an αβTCR/CD19+ cell depleted graft.

Conditions

Myelodysplasia; Hematologic Malignancy

Outcome Measures

Primary Outcomes (5)

• Measure rates of neutrophil and platelet engraftment

  Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have an improved rate of engraftment.

  4 years

• Measure incidence of acute GVHD

  Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a lower incidence of acute and chronic GVHD.

  4 years

• Measure rates of immune reconstitution

  Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a improved rate of immune reconstitution.

  4 years

• Measure rates of platelet engraftment

  Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have an improved rate of engraftment.

  4 years

• Measure incidence of chronic GVHD

  Patients undergoing partially matched related donor hematopoietic cell transplantation with an αβT cell / CD19+ B cell depleted graft should have a lower incidence of acute GVHD.

  4 years

Secondary Outcomes (4)

• Measure overall survival

  4 years

• Define nonhematopoietic regimen related toxicities

  4 years

• Measure relapse rate

  4 years

• Measure disease free survival

  4 years

Study Arms (1)

CliniMACS Isolation

EXPERIMENTAL

The mobilized peripheral blood cell collection (apheresis product) will be processed using a Miltenyi CliniMACS device according to the manufacturing instructions. The processing will deplete the αβTCR+ cells and CD19+ cells from the apheresis product to formulate the graft.

Device: CliniMACS® TCRαβ/CD19 Combined Depletion System

Interventions

CliniMACS® TCRαβ/CD19 Combined Depletion System DEVICE

The mobilized peripheral blood cell collection (apheresis product) will be processed using a Miltenyi CliniMACS device according to the manufacturing instructions. The processing will deplete the αβTCR+ cells and CD19+ cells from the apheresis product to formulate the graft.

CliniMACS Isolation

Eligibility Criteria

• Age: Up to 30 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Patient lacks an HLA matched sibling donor.
• Meets criteria nonhematopoietic organ function according to NCH BMT SOP09.
• If subjects have received a first HCT, they must be eligible for a second HCT if their disease has recurred.
• High resolution HLA and KIR typing
• The subject cannot have an active untreated infection. Viremia by PCR analysis is not considered an active infection but may require immediate viral prophylaxis. Patients with possible fungal infections must have had at least 2 weeks of appropriate anti- fungal therapy and be asymptomatic.
• Negative pregnancy test for females ≥11 years of age or post- menarche.
• Sexually active males and females of childbearing potential must agree to use a form of contraception considered effective and medically acceptable by the Investigator. (Non-childbearing potential defined as pre-menarche, greater than one year post-menopausal or surgically sterilized).
• Subjects must be ≤30 years at the time of consent.
• Signed consent by parent/guardian and assent if appropriate for subjects \< 18 years of age. Signed consent by patient/subject if ≥18 years of age.

You may not qualify if:

• Patient does not have a suitable donor who is willing and able (meets donor criteria).
• Patient has donor-specific anti-HLA antibodies at the time of enrollment
• Patient reports a history of allergic reactions to murine protein
• Donor Eligibility:
• The donor must be ≥18 years of age at the time of the informed consent conference.
• The donor must be a related donor
• The donor will be evaluated according to the current NCH BMT SOP 04 and must meet all criteria.
• The donor must be able and willing to undergo G-CSF mobilization and stem cell apheresis.
• The patient does not have donor specific anti-HLA antibodies

Sponsors & Collaborators

• Nationwide Children's Hospital: lead

Study Sites (1)

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

Location

MeSH Terms

Conditions

Anemia, Refractory, with Excess of Blasts; Hematologic Neoplasms

Condition Hierarchy (Ancestors)

Anemia, Refractory; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases; Neoplasms by Site; Neoplasms

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The mobilized peripheral blood cell collection (apheresis product) will be processed using a Miltenyi CliniMACS device according to the manufacturing instructions. The processing will deplete the αβTCR+ cells and CD19+ cells from the apheresis product to formulate the graft.

Study Record Dates

• First Submitted: December 27, 2017
• First Posted: February 13, 2018
• Study Start: March 2, 2018
• Primary Completion: December 1, 2025
• Study Completion: January 1, 2026
• Last Updated: May 13, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)