NCT01445821

Autologous Stem Cell Systemic Sclerosis Immune Suppression Trial

Randomized Study of Different Non-myeloablative Conditioning Regimens With Hematopoietic Stem Cell Support in Patients With Scleroderma (Autologous Systemic Sclerosis Immune Suppression Trial - II ASSIST-IIb)

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 44 (actual)
• Sponsor: Northwestern University · Academic / Other
• Sex: All
• Age: 17 Years – 60 Years
• Healthy volunteers: Not accepted

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.

Detailed description

Mobilization. For patients in both arms undergoing hematopoietic stem cell transplantation (HSCT), peripheral blood stem cells (PBSC) will be mobilized with cyclophosphamide (2 g/m2) followed by 5-10 mcg/kg subcutaneous filgrastrim daily from day 5 until completion of apheresis. Mobilized hematopoietic stem cells (HSC) will be collected by apheresis on day 10 and cryopreserved without selection or manipulation. There will be an interval of at least 17 days between mobilization of PBSC and start of conditioning regimen.

Conditions

• Scleroderma, Systemic

Interventions

Type	Name	Description
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

Timeline

• Start date: 2011-09-15
• Primary completion: 2017-01-05
• Completion: 2019-10-10
• First posted: 2011-10-04
• Last updated: 2020-07-23
• Results posted: 2020-07-23

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study