PemphigusPhase 2NCT00010413
Phase II Study of High-Dose Cyclophosphamide in Patients With Refractory Pemphigus
A Phase 2 study testing cyclophosphamide and filgrastim in people with Pemphigus, run by Johns Hopkins University at 1 site in United States.
Trial summary
OBJECTIVES: I. Determine the response rate and 1-year event-free survival of patients with refractory pemphigus treated with high-dose cyclophosphamide. Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Pemphigus
- What is being tested?
- cyclophosphamide and filgrastim
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 1999to Aug 2007
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
PROTOCOL ENTRY CRITERIA:
--Disease Characteristics--
Definitive diagnosis of pemphigus, including all of the following: Typical clinical lesions Histologically proven by skin or mucous membrane biopsy Pemphigus IgG autoantibodies in skin or mucous membrane by direct immunofluorescence (IF) Circulating pemphigus IgG autoantibodies in serum by indirect IF
Dependence on high-dose corticosteroids
Persistent disease activity despite treatment with mycophenolate mofetil or azathioprine, or inability to tolerate these drugs (for paraneoplastic pemphigus, persistent disease activity despite treatment with cyclosporine)
--Prior/Concurrent Therapy--
No concurrent cytotoxic therapy
--Patient Characteristics--
Performance status: Karnofsky 20-100%
Renal: Creatinine no greater than 2.5 mg/dL
Cardiovascular: LVEF at least 40%
Pulmonary: FVC, FEV1, or DLCO at least 50% predicted
Other: Not pregnant Fertile patients must use effective contraception during and for 9 months after study Not preterminal or moribund
Trial locations and contacts
1 site in 1 country.
United States1 site
Johns Hopkins University School of Medicine
Baltimore, Maryland, 21205
About this study
OBJECTIVES:
- Determine the response rate and 1-year event-free survival of patients with refractory pemphigus treated with high-dose cyclophosphamide.
Read the detailed description
PROTOCOL OUTLINE: Patients receive cyclophosphamide IV on days 1-4 and filgrastim (G-CSF) beginning on day 10 and continuing until blood counts recover.
Patients are followed monthly for 6 months, every 2 months for 6 months, every 4 months for a year, and then annually thereafter.
- Conditions
- Pemphigus
- Being tested
- cyclophosphamide, filgrastim
- Keywords
- immunologic disorders and infectious disorders, pemphigus, rare disease
- Medical subject headings
- Pemphigus, Immune System Diseases, Communicable Diseases, Rare Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cyclophosphamideDrug
filgrastimDrug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2001 (estimated)
- Study startApr 1999
- Primary completionAug 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for PemphigusStudies of the same condition, open and under way.
- More trials run by Johns Hopkins UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Enrolment
- 35 participants
Sponsor and investigators
- Lead sponsor
- Johns Hopkins University
- Sponsor type
- University, hospital or other
- Investigators
- Grant J. Anhalt, Study chair, Johns Hopkins University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00010413
- Sponsor's protocol ID
- 199/15674
- Other IDs
- JHOC-J9912, JHOC-99022610
- Status last verified
- Oct 2008
- First submitted
- 2 Feb 2001
- Last updated
- 2 Oct 2008
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Oct 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase II Study of High-Dose Cyclophosphamide in Patients With Refractory Pemphigus
- ClinicalTrials.gov ID
- NCT00010413
- Acronym / sponsor ID
- 199/15674
- Conditions
- Pemphigus
- Keywords
- immunologic disorders and infectious disorders, pemphigus, rare disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants
- Lead sponsor
- Johns Hopkins UniversityUniversity, hospital or other
- Study start
- Apr 1999
- Primary completion
- Aug 2007 (actual)
- First posted
- 2 Feb 2001 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
OBJECTIVES:
- Determine the response rate and 1-year event-free survival of patients with refractory pemphigus treated with high-dose cyclophosphamide.
Detailed description
PROTOCOL OUTLINE: Patients receive cyclophosphamide IV on days 1-4 and filgrastim (G-CSF) beginning on day 10 and continuing until blood counts recover.
Patients are followed monthly for 6 months, every 2 months for 6 months, every 4 months for a year, and then annually thereafter.
Study design
- Primary purpose
- Treatment
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| cyclophosphamide | Drug | – |
| filgrastim | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Oct 2008.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Grant J. Anhalt study chair
Johns Hopkins University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing cyclophosphamide and filgrastim in people with Pemphigus, run by Johns Hopkins University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 2 Oct 2008. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2008.