Scleroderma Treatment With Autologous Transplant (STAT) Study

Summary

This phase II trial studies how well giving cyclophosphamide and anti-thymocyte globulin together followed by peripheral blood stem cell transplant (PBSCT) and mycophenolate mofetil works in treating patients with systemic scleroderma (SSc). Stem cells are collected from the patient's blood and stored prior to treatment. To store the stem cells patients are given colony-stimulating factors, such as filgrastim (G-CSF) or chemotherapy (cyclophosphamide) to help stem cells move from the bone marrow to the blood so they can be collected and stored. After storage, patients are then given high-dose chemotherapy, cyclophosphamide, and immunosuppression with anti-thymocyte globulin to suppress the immune system to prepare for the transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy and immunosuppression. After the stem cells have "engrafted" and have matured enough to support the immune system at approximately 2-3 months, patients are given a medication called mycophenolate mofetil (MMF) or Myfortic. This medication is given to prevent worsening or reactivation of SSc and is referred to as maintenance therapy.

Conditions

• Systemic Scleroderma

Interventions

BIOLOGICAL

Anti-Thymocyte Globulin

Given IV

PROCEDURE

Autologous Hematopoietic Stem Cell Transplantation

Undergo autologous PBSCT

DRUG

Cyclophosphamide

Given IV

BIOLOGICAL

Filgrastim

Given SC

OTHER

Laboratory Biomarker Analysis

Correlative studies

DRUG

Mycophenolate Mofetil

Given PO

PROCEDURE

Peripheral Blood Stem Cell Transplantation

Undergo autologous PBSCT

DRUG

Plerixafor

Given SC

OTHER

Quality-of-Life Assessment

Ancillary studies

OTHER

Questionnaire Administration

Ancillary studies

Sponsors & Collaborators

• National Cancer Institute (NCI)

  collaborator NIH

• Fred Hutchinson Cancer Center

  lead OTHER

Principal Investigators

• Leona Holmberg · Fred Hutch/University of Washington Cancer Consortium

Study Design

Allocation

NA

Purpose

TREATMENT

Masking

NONE

Model

SINGLE_GROUP

Eligibility

Max Age

70 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-09-15

Primary Completion

2023-09-15

Completion

2024-09-11

Countries

• United States

Study Locations