Mesenchymal Stromal Cells for Haplo Hematopoietic Cell Transplantation for Sickle Cell Disease

Summary

This trial is being conducted as a step toward testing the long-term hypothesis that freshly cultured, autologous mesenchymal stromal cells (MSCs) grown in platelet lysate-containing medium will modulate recipient T-cell immune responses and promote engraftment in haploidentical hematopoietic cell transplant (HCT) recipients. As a phase I, dose escalation trial of autologous MSCs in patients with sickle cell disease (SCD) undergoing haploidentical HCT, the main aim is to evaluate the safety of this therapy with a secondary aim to evaluate its effects on engraftment and graft-versus-host disease (GVHD).

Conditions

• Sickle Cell Disease

Interventions

BIOLOGICAL

Autologous MSCs

Participants at dose level 1 receive four infusions of MSCs, given once per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10\^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.

BIOLOGICAL

Autologous MSCs

Participants at dose level 2 receive four infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10\^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.

BIOLOGICAL

Autologous MSCs

Participants at dose level 3 receive six infusions of MSCs, given twice per week. The first MSC infusion will be given no sooner than 4 hours after the bone marrow transplant. The total number of cells delivered to each patient will depend on their weight and assigned dose level. The maximal individual dose of MSCs any patient will receive is 2 x 10\^6 cells/kg, which will be delivered via intravenous infusion. The infusion can be administered to patients in the inpatient or outpatient setting.

Sponsors & Collaborators

• National Heart, Lung, and Blood Institute (NHLBI)

  collaborator NIH

• Emory University

  lead OTHER

Principal Investigators

• Elizabeth Stenger, MD · Emory University

• Lakshmanan Krishnamurti, MD · Emory University

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

SEQUENTIAL

Eligibility

Min Age

12 Years

Max Age

40 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2017-12-21

Primary Completion

2018-10-25

Completion

2018-10-25

FDA Drug

Yes