NCT00483119

Brief Summary

The purpose of this study is to compare two standard treatments for pemphigus to determine which more effectively improves the clinical manifestations of the disease and decreases serum level of the autoantibodies which cause the disease.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
9

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Apr 2007

Typical duration for phase_2

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

April 1, 2007

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

June 5, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

June 6, 2007

Completed
2.7 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2010

Completed
1 year until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2011

Completed
5 years until next milestone

Results Posted

Study results publicly available

February 18, 2016

Completed
Last Updated

February 18, 2016

Status Verified

January 1, 2016

Enrollment Period

2.8 years

First QC Date

June 5, 2007

Results QC Date

October 5, 2015

Last Update Submit

January 21, 2016

Conditions

Pemphigus Vulgaris

Keywords

pemphigus vulgarisIVIgcyclophosphamidetreatmentpemphigus antibodies

Outcome Measures

Primary Outcomes (2)

  • Clinical Outcome: Extent and Severity of Disease

    6 - 10 weeks after initiation of therapy

  • Serum Levels of Pemphigus Antibodies

    6-10 weeks after initiation of therapy

Secondary Outcomes (2)

  • Toxicity of Treatment: Measured in Renal Toxicity, Myelosuppression or Hepatic Toxicity

    Throughout course of study

  • Ability to be Weaned Off Steroids

    Measured 6 and 10 weeks after initiation of IVIg treatment

Study Arms (2)

Group A

ACTIVE COMPARATOR

IVIg alone (intravenous immunoglobulin)

Drug: intravenous immunoglobulin

Group B

EXPERIMENTAL

IVIg with cyclophosphamide

Drug: intravenous immunoglobulinDrug: cyclophosphamide

Interventions

intravenous immunoglobulinDRUG

Gamunex 10% 500/mg/kg/day x four days per cycle total of four cycles

Also known as: IVIg, Gamunex
Group AGroup B
cyclophosphamideDRUG

cyclophosphamide dose of 2mg/kg/day divided into three-times daily oral administration

Also known as: CP
Group B

Eligibility Criteria

Age18 Years - 85 Years
Sexall
Healthy VolunteersYes
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Lesions consistent with pemphigus foliaceus or vulgaris
  • Diagnosis confirmed by histology and IIF ≥ 40 within past month
  • On ≥20mg/day of prednisone per day for two weeks or ≥ 80mg/day for one week
  • Women of childbearing potential negative HCG obtained two weeks prior to first IVIg
  • Agrees to two acceptable forms of contraception\* if randomized to cyclophosphamide group:
  • IUD (except progesterone T), Combination oral contraceptives, transdermal patch, vaginal ring, hormonal injectables or implantables, male latex condom, diaphragm, cervical cap, or vaginal sponge (contains spermicide)
  • Responds yes to at least one of the criteria below:
  • Persistence of clinical manifestations of disease despite steroid treatment
  • Flare in disease activity after an attempt at steroid tapering
  • Failure of established lesions to heal
  • Rapidly progressive disease.
  • Conventional therapy is relatively contraindicated i.e. side effects, co-morbid conditions
  • systemic infections, peptic ulcers, osteoporosis, hypertension, cataracts or others

You may not qualify if:

  • Use of IVIg within past 3 weeks or the use of a cytotoxic drug within the past 2 weeks
  • Participating in another clinical trial at the time of screening and enrollment
  • Medical condition that precludes use of IVIg or cyclophosphamide (i.e. pregnancy breastfeeding, underlying chronic infection, concurrent opportunistic infection, sepsis or volume depletion
  • Renal insufficiency ( GFR \<90, proteinuria (\>1+, x 2), creatinine \>1.8 or increased WBC or RBCs which cannot be explained by cystitis.)
  • Known hypersensitivity to study drugs, IVIg or cyclophosphamide

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

NYU Medical Center

New York, New York, 10016, United States

Location

Related Publications (4)

  • Green MG, Bystryn JC. Effect of intravenous immunoglobulin therapy on serum levels of IgG1 and IgG4 antidesmoglein 1 and antidesmoglein 3 antibodies in pemphigus vulgaris. Arch Dermatol. 2008 Dec;144(12):1621-4. doi: 10.1001/archdermatol.2008.503.

    PMID: 19075146BACKGROUND

  • Czernik A, Bystryn JC. Kinetics of response to conventional treatment in patients with pemphigus vulgaris. Arch Dermatol. 2008 May;144(5):682-3. doi: 10.1001/archderm.144.5.682. No abstract available.

    PMID: 18490602BACKGROUND

  • Czernik A, Beutner EH, Bystryn JC. Intravenous immunoglobulin selectively decreases circulating autoantibodies in pemphigus. J Am Acad Dermatol. 2008 May;58(5):796-801. doi: 10.1016/j.jaad.2008.01.007.

    PMID: 18423257BACKGROUND

  • Czernik A, Bystryn JC. Improvement of intravenous immunoglobulin therapy for bullous pemphigoid by adding immunosuppressive agents: marked improvement in depletion of circulating autoantibodies. Arch Dermatol. 2008 May;144(5):658-61. doi: 10.1001/archderm.144.5.658.

    PMID: 18490594BACKGROUND

MeSH Terms

Conditions

Pemphigus

Interventions

Immunoglobulins, IntravenousCyclophosphamide

Condition Hierarchy (Ancestors)

Skin Diseases, VesiculobullousSkin DiseasesSkin and Connective Tissue DiseasesAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin GImmunoglobulin IsotypesAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulinsPhosphoramide MustardsNitrogen Mustard CompoundsMustard CompoundsHydrocarbons, HalogenatedHydrocarbonsOrganic ChemicalsPhosphoramidesOrganophosphorus Compounds

Limitations and Caveats

Sadly, the trial PI, Dr. Jean-Claude Bystryn, died on August 19, 2010. As a result the study could not be completed and an analysis of the data collected was not performed.

Results Point of Contact

Title
Elise Kelman, Associate Director of Research Administration
Organization
NYU School of Medicine
elise.kelman@nyumc.org
2122639073

Study Officials

  • Jean-Claude Bystryn, M.D.

    NYU MEDICAL CENTER

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 5, 2007

First Posted

June 6, 2007

Study Start

April 1, 2007

Primary Completion

February 1, 2010

Study Completion

February 1, 2011

Last Updated

February 18, 2016

Results First Posted

February 18, 2016

Record last verified: 2016-01

Locations

US(1)