Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial

Summary

ASSIST I was the first randomized trial in patients with scleroderma to not just slow disease progression but rather actually reverse it. It is the first treatment to have ever demonstrated reversal of lung disease in scleroderma with improvement in FVC, total lung capacity (TLC), high-resolution computed tomography (HRCT), and QOL. We now, therefore, purpose to compare the ASSIST I conditioning regimen of cyclophosphamide and rATG to a less intense regimen of rATG/cyclophosphamide/Fludarabine. In the new regimen the cyclophosphamide dose is decreased to 120mg/kg (60mg/kg/day x 2) compared to 200mg/kg (50mg/kg/day) in the standard regimen. The lower dose of cyclophosphamide will be less cardiotoxic. This study will determine if the less cardiotoxic regimen will be safer than the standard regimen and as effective as the standard regimen.

Conditions

• Scleroderma, Systemic

Interventions

BIOLOGICAL

Peripheral Blood Stem Cells

Mobilized leukapheresis product

DRUG

Cyclophosphamide

An alkylating agent which causes prevention of cell division by forming adducts with DNA

DRUG

Mesna

Medication used to decrease the risk of hemorrhagic cystitis prophylaxis

DRUG

rATG

A predominantly lymphocyte-specific immunosuppressive agent which contains antibodies specific to the antigens commonly found on the surface of T cells

DRUG

Methylprednisolone

Steroid

DRUG

Filgrastim

Granulocyte-colony stimulating factor (G-CSF); a glycoprotein that stimulates the bone marrow to produce granulocytes and stem cells and release them into the bloodstream

DRUG

Fludarabine

Purine analog which inhibits DNA synthesis or repair

Sponsors & Collaborators

• Northwestern University

  lead OTHER

Principal Investigators

• Richard Burt, MD · Northwestern University

Study Design

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Min Age

17 Years

Max Age

60 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2011-09-15

Primary Completion

2017-01-05

Completion

2019-10-10

FDA Drug

Yes

Countries

• United States

Study Locations