Nonmyeloablative Conditioning and Transplantation for Patients With Refractory Systemic Lupus Erythematosus (SLE)

NCT02080195 | Terminated Phase 1 Results DMC

• 2 other identifiers: J13134NA_00082453
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

The main goal of the study is to determine if bone marrow transplant (BMT) from a less specific pool of donors in combination with high dose cyclophosphamide can induce remission of refractory systemic lupus erythematosus.

Conditions

Lupus Erythematosus; Graft-versus-host Disease

Outcome Measures

Primary Outcomes (1)

• The Feasibility of the Conditioning Regimen and Post Transplantation Cyclophosphamide in Refractory SLE Patients With Donors Having Various Degrees of Matching

  Number of participants who were alive at 1 year after transplant and who had not suffered graft rejection, acute or chronic GVHD, or Grade 3 or higher (CTCAE V4.0) adverse events.

  1 year

Secondary Outcomes (5)

• RIFLE Score

  1 year

• Survival

  1 year

• Graft Failure

  60 days

• Acute Graft Versus Host Disease (GVHD)

  Up to 2 years

• Chronic Graft Versus Host Disease (GVHD)

  Up to 2 years

Study Arms (1)

Nonmyeloablative Conditioning and BMT

EXPERIMENTAL

Nonmyeloablative conditioning with rabbit antithymocyte globulin, cyclophosphamide, fludarabine, and total body irradiation. Allogeneic bone marrow transplant on Day 0. Graft versus host disease (GVHD) prophylaxis with cyclophosphamide, mycophenolate mofetil, and tacrolimus.

Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Tacrolimus; Drug: Mycophenolate Mofetil; Drug: Rabbit antithymocyte globulin; Radiation: Total body irradiation; Biological: Allogeneic bone marrow transplant

Interventions

Cyclophosphamide DRUG

14.5 mg/kg/day on Days -6 and -5. 50 mg/kg/day on Days 3 and 4.

Also known as: Cytoxan, Cy

Nonmyeloablative Conditioning and BMT

Fludarabine DRUG

30 mg/m\^2/day on Days -6 through -2.

Also known as: Fludara

Nonmyeloablative Conditioning and BMT

Tacrolimus DRUG

Starting on Day 5. Dose will be adjusted according to blood levels.

Also known as: FK-506, Prograf

Nonmyeloablative Conditioning and BMT

Mycophenolate Mofetil DRUG

15 mg/kg three times per day from Day 5 to Day 35.

Also known as: Cellcept, MMF

Nonmyeloablative Conditioning and BMT

Rabbit antithymocyte globulin DRUG

0.5 mg/kg on Day -9. 2 mg/kg/day on Days -8 and -7.

Also known as: Thymoglobulin

Nonmyeloablative Conditioning and BMT

Total body irradiation RADIATION

200 centigray on Day -1.

Nonmyeloablative Conditioning and BMT

Allogeneic bone marrow transplant BIOLOGICAL

Infusion on Day 0.

Also known as: BMT

Nonmyeloablative Conditioning and BMT

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Four or more American College of Rheumatology (ACR) criteria for the classification of SLE or 4 or more of the SLICE criteria
• Involvement of one or more of the following organ systems: renal, neurologic, hematologic, cardiac, pulmonary, gastrointestinal
• A lack of response to corticosteroids in moderate-to-high doses, and to either an equivalent degree of immunosuppression with azathioprine, methotrexate, cyclosporin, tacrolimus, belimumab, rituximab, mycophenolate mofetil, and/or appropriate other treatment
• Patients should be eligible for transplantation according to the BMT Policy Manual

You may not qualify if:

• Age less than 18 years and over 75 years
• Any risk of pregnancy
• Patients who are preterminal or moribund

Sponsors & Collaborators

• Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins: lead
• Maryland Stem Cell Research Fund: collaborator

Study Sites (1)

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Baltimore, Maryland, 21205, United States

Location

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

Cyclophosphamide; fludarabine; fludarabine phosphate; Tacrolimus; Mycophenolic Acid; thymoglobulin; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

Immune System Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Macrolides; Lactones; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids; Radiotherapy; Therapeutics; Investigative Techniques

Results Point of Contact

• Title: Javier Bolaños Meade, MD
• Organization: The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

fbolano2@jhmi.edu

410-614-6398

Study Officials

• Javier Bolaños-Meade, MD

  The Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 28, 2014
• First Posted: March 6, 2014
• Study Start: September 13, 2016
• Primary Completion: March 28, 2017
• Study Completion: March 29, 2017
• Last Updated: October 2, 2019
• Results First Posted: October 2, 2018

Record last verified: 2019-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)