NCT00684255

Brief Summary

The purpose of this study is to determine if a reduced intensity (RI) (non-myeloablative) chemoimmunotherapy followed by Allogeneic Stem Cell Transplantation AlloSCT (matched family donors and matched unrelated cord blood donors) will be well tolerated.

Trial Health

57
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
1

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Aug 2007

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 1, 2007

Completed
10 months until next milestone

First Submitted

Initial submission to the registry

May 22, 2008

Completed
4 days until next milestone

First Posted

Study publicly available on registry

May 26, 2008

Completed
1 month until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2008

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2008

Completed
1.4 years until next milestone

Results Posted

Study results publicly available

December 2, 2009

Completed
Last Updated

April 1, 2014

Status Verified

February 1, 2014

Enrollment Period

11 months

First QC Date

May 22, 2008

Results QC Date

December 22, 2008

Last Update Submit

February 6, 2014

Conditions

Systemic Lupus ErythematosusSystemic Sclerosis

Keywords

Autoimmune DiseaseReduced Intensity Transplant

Outcome Measures

Primary Outcomes (1)

  • Toxicity

    Toxicity associated with reduced intensity regimen of fludarabine/busulfan and Campath followed by allogeneic stem cell transplant in patients with medically refractory Systemic Lupus Erythematosus (SLE) or SSc is measured.

    1 year

Secondary Outcomes (3)

  • Chimerism

    1 year

  • Immune Reconstitution.

    1 year

  • Progression Free and Overall Survival.

    1 year

Study Arms (2)

Reduced Intensity Regimen for Refractory SLE

EXPERIMENTAL

RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with medically refractory Systemic Lupus Erythematosus (SLE).

Procedure: Reduced Intensity Allogeneic TransplantDrug: FludarabineDrug: BusulfanDrug: Campath

Reduced Intensity Regimen for SSc

EXPERIMENTAL

RI regimen of fludarabine/busulfan and Alemtuzumab (FBA) followed by AlloSCT in selected patients with Systemic Sclerosis (SSc).

Procedure: Reduced Intensity Allogeneic TransplantDrug: FludarabineDrug: BusulfanDrug: Campath

Interventions

Reduced Intensity Allogeneic TransplantPROCEDURE

Eeduced intensity allogeneic stem cell transplantation with a fludarabine/busulfan/alemtuzumab conditioning regimen is anticipated to result in mixed and/or complete donor chimerism and potentially alter the natural history and outcome of patients with medically refractory Systemic Lupus Erythematosus (SLE) or Systemic Sclerosis (SSc).

Reduced Intensity Regimen for Refractory SLEReduced Intensity Regimen for SSc
FludarabineDRUG

Fludarabine 30 mg/m2 Day -7, -6, -5, -4, -3, -2

Reduced Intensity Regimen for Refractory SLEReduced Intensity Regimen for SSc
BusulfanDRUG

Busulfan 3.2 mg/kg Days \_8, -7, -6, -5

Reduced Intensity Regimen for Refractory SLEReduced Intensity Regimen for SSc
CampathDRUG

Campath: 2 mg/m2 Day -5; 6 mg/m2 Day -4, -3; 20 mg/m2 Day -2

Also known as: Alemtuzumab
Reduced Intensity Regimen for Refractory SLEReduced Intensity Regimen for SSc

Eligibility Criteria

Age7 Years - 50 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Diffuse Systemic Sclerosis and variants as per ACR criteria
  • Medically refractory disease
  • Adequate Organ Function - Pulmonary function
  • Renal function, Cardiac function defined as:
  • SGOT (AST) or SGPT (ALT) \<5 x upper limit of normal
  • Diagnosis of SLE - Medically refractory disease

You may not qualify if:

  • Karnofsky/Lansky \<60%

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

Related Links

MeSH Terms

Conditions

Lupus Erythematosus, SystemicScleroderma, SystemicAutoimmune Diseases

Interventions

fludarabineBusulfanAlemtuzumab

Condition Hierarchy (Ancestors)

Connective Tissue DiseasesSkin and Connective Tissue DiseasesImmune System DiseasesSkin Diseases

Intervention Hierarchy (Ancestors)

Butylene GlycolsGlycolsAlcoholsOrganic ChemicalsMesylatesAlkanesulfonatesAlkanesulfonic AcidsAlkanesHydrocarbons, AcyclicHydrocarbonsSulfonic AcidsSulfur AcidsSulfur CompoundsAntibodies, Monoclonal, HumanizedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Limitations and Caveats

Study closed due to low accrual. Did not meet enrollment goals.

Results Point of Contact

Title
Dr. Mitchell S. Cairo
Organization
New York Medical College
mitchell_cairo@nymc.edu
914-594-3650

Study Officials

  • Mitchell Cairo, MD

    Columbia University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal Investigator

Study Record Dates

First Submitted

May 22, 2008

First Posted

May 26, 2008

Study Start

August 1, 2007

Primary Completion

July 1, 2008

Study Completion

July 1, 2008

Last Updated

April 1, 2014

Results First Posted

December 2, 2009

Record last verified: 2014-02

Locations

US(1)