PemphigusPhase 1NCT00278642
Hematopoietic Stem Cell Support in Patients With Autoimmune Bullous Skin Disorders
A Phase 1 study testing Hematopoietic stem cell transplantation in people with Pemphigus, run by Richard Burt, MD at 1 site in United States.
Trial summary
Autoimmune Bullous Skin Disorders are believed to be due to immune cells, cells that normally protect the body and are now causing damage to the body. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) together with… Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Pemphigus
- What is being tested?
- Hematopoietic stem cell transplantation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 1 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2002to Sep 2011
- Phase
- Phase 1
- 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 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Less than chronologic age 60 years at the time of pre-transplant evaluation.
- An established diagnosis of an autoimmune skin disorder that includes any of the following:
- pemphigus vulgaris
- pemphigus foliaceus
- Diagnosis of bullous skin lesions will be based on history and physical, skin biopsy (light microscopy and indirect fluorescence), indirect immunofluorescence titer, BP 180 and 230 titers, Desmolglein-3 and Desmoglein-1 antibody, and photograph.
- Patients who failed corticosteroids (equivalent dosage of prednisone 0.5 mg/kg/day for more than 3 months), and at least two of the following: azathioprine, mycophenolate mofetil, gold, tetracycline (or minocycline), cyclosporin, methotrexate, gold, or plasmapheresis. Failure is defined as the inability to wean steroids to less than 0.5 mg/kg/day.
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- Potential candidates must have involvement of more than 10% of skin body surface area, involvement of one or more mucosal lesions, or recurrent infections requiring more than two hospitalizations in which the source of the infection was due to bullous skin disease.
- All candidates must be evaluated by two dermatologists, Dr. Joan Guitart and Dr. Joaquin Brieva, who must concur that the patient has refractory disease that may, in their clinical judgement, be associated with a 5-10% mortality if not controlled.
- A minimum of 2.0 x 106 CD34+ cells/kg after selection are necessary to proceed to transplant.
Who cannot join
- Individuals less than 18 years of age.
- Significant end organ damage such as:
- LVEF <40% or deterioration of LVEF during exercise test on MUGA or echocardiogram.
- Untreated life-threatening arrhythmia.
- Active ischemic heart disease or heart failure.
- DLCO <45% or FEV1/FEV < 50%.
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- Serum creatinine > 2.5 mg/dl.
- Liver cirrhosis, transaminases >3x of normal limits or bilirubin >2.0 unless due to Gilberts disease.
- HIV positive.
- Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
- Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
- Positive pregnancy test, inability or unable to pursue effective means of birth control, or failure to willingly accept or comprehend irreversible sterility as a side effect of therapy.
- Psychiatric illness or mental deficiency making compliance with treatment or informed consent impossible.
- Inability to give informed consent.
- Major hematological abnormalities such as platelet count less than 100,000/ul, or ANC less than 1000/ul.
- Presence of infected skin lesions. All skin lesions should be free of suppurative exudate.
Trial locations and contacts
1 site in 1 country.
United States1 site
Northwestern University, Feinberg School of Medicine
Chicago, Illinois, 60611
About this study
Official title: High Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Support in Patients With Autoimmune Bullous Skin Disorders: A Phase I Trial
Autoimmune Bullous Skin Disorders are believed to be due to immune cells, cells that normally protect the body and are now causing damage to the body. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) together with anti-thymocyte globulin (a protein that kills the immune cells that are thought to be causing your disease), followed by return of the previously collected special blood cells (stem cells) will result in improvement of this disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in the immune system which may be causing this skin disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and anti-thymocyte globulin.
- Conditions
- Pemphigus
- Being tested
- Hematopoietic stem cell transplantation
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hematopoietic stem cell transplantationBiological
Autologous hematopoietic stem cell transplantation
| Study group | Role | What they receive |
|---|---|---|
| stem cell transplantation | Experimental | Not described Receives: Biological: Hematopoietic stem cell transplantation |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percent surface area involved New skin or mucosal blister development Immune suppressive medication requirements Survival
Measured: 5 years after transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jan 2006 (estimated)
- Study startSep 2002
- Primary completionSep 2011
- Study completionSep 2011
Why it stopped: sponsor stopped the study because of lack of elligible participants
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.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 1 participants (actual)
Sponsor and investigators
- Lead sponsor
- Richard Burt, MD
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Richard Burt, MD, Northwestern University
- Investigators
- Richard Burt, MD, Principal investigator, Northwestern University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00278642
- Sponsor's protocol ID
- DIAD Derm.Auto2001
- Status last verified
- Apr 2013
- First submitted
- 16 Jan 2006
- Last updated
- 8 Apr 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- High Dose Cyclophosphamide & ATG With Hematopoietic Stem Cell Support in Patients With Autoimmune Bullous Skin Disorders: A Phase I Trial
- ClinicalTrials.gov ID
- NCT00278642
- Acronym / sponsor ID
- DIAD Derm.Auto2001
- Conditions
- Pemphigus
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: sponsor stopped the study because of lack of elligible participants
- Enrolment
- 1 participants (actual)
- Lead sponsor
- Richard Burt, MDUniversity, hospital or other
- Study start
- Sep 2002
- Primary completion
- Sep 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 18 Jan 2006 (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.
Autoimmune Bullous Skin Disorders are believed to be due to immune cells, cells that normally protect the body and are now causing damage to the body. This study is designed to examine whether treating patients with high dose cyclophosphamide (a drug which reduces the function of the immune system) together with anti-thymocyte globulin (a protein that kills the immune cells that are thought to be causing your disease), followed by return of the previously collected special blood cells (stem cells) will result in improvement of this disease. Stem cells are undeveloped cells that have the capacity to grow into mature blood cells, which normally circulate in the blood stream. The purpose of the intense chemotherapy is to destroy the cells in the immune system which may be causing this skin disease. The purpose of the stem cell infusion is to restore the body's blood production, which will be severely impaired by the high dose chemotherapy and anti-thymocyte globulin.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| stem cell transplantation | Experimental | Biological: Hematopoietic stem cell transplantation |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (9 listed)
- Less than chronologic age 60 years at the time of pre-transplant evaluation.
- An established diagnosis of an autoimmune skin disorder that includes any of the following:
- Diagnosis of bullous skin lesions will be based on history and physical, skin biopsy (light microscopy and indirect fluorescence), indirect immunofluorescence titer, BP 180 and 230 titers, Desmolglein-3 and Desmoglein-1 antibody, and photograph.
- Patients who failed corticosteroids (equivalent dosage of prednisone 0.5 mg/kg/day for more than 3 months), and at least two of the following: azathioprine, mycophenolate mofetil, gold, tetracycline (or minocycline), cyclosporin, methotrexate, gold, or plasmapheresis. Failure is defined as the inability to wean steroids to less than 0.5 mg/kg/day.
- Potential candidates must have involvement of more than 10% of skin body surface area, involvement of one or more mucosal lesions, or recurrent infections requiring more than two hospitalizations in which the source of the infection was due to bullous skin disease.
Key exclusion criteria (16 listed)
- Individuals less than 18 years of age.
- Significant end organ damage such as:
- HIV positive.
- Uncontrolled diabetes mellitus, or any other illness that in the opinion of the investigators would jeopardize the ability of the patient to tolerate aggressive treatment.
- Prior history of malignancy except localized basal cell or squamous skin cancer. Other malignancies for which the patient is judged to be cured by local surgical therapy, such as (but not limited to) head and neck cancer, or stage I or II breast cancer will be considered on an individual basis.
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 |
|---|---|---|
| Hematopoietic stem cell transplantationAutologous hematopoietic stem cell transplantation | Biological | stem cell transplantation |
Primary outcome
Percent surface area involved New skin or mucosal blister development Immune suppressive medication requirements Survival
Time frame: 5 years after transplant
Results and safety
The study was stopped early (reason given: sponsor stopped the study because of lack of elligible participants). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2013.
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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.
Richard Burt, MD principal investigator
Northwestern University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Hematopoietic stem cell transplantation in people with Pemphigus, run by Richard Burt, MD 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?
The sponsor lists 9 inclusion and 16 exclusion criteria. 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: stopped early, as of the sponsor's last update on 8 Apr 2013. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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 was stopped early (reason given: sponsor stopped the study because of lack of elligible participants). Results are not available in the source record.
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.
What does the primary outcome measure?
Percent surface area involved New skin or mucosal blister development Immune suppressive medication requirements Survival (time frame: 5 years after transplant). It is what the study was designed to measure, not a finding.
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 8 Apr 2013.