Pemphigus VulgarisPhase 2NCT00606749
Use of KC706 for the Treatment of Pemphigus Vulgaris
A Phase 2 study testing KC706 in people with Pemphigus Vulgaris, run by Kemia, Inc at 1 site in United States.
Trial summary
The purpose of this study is to determine whether KC706 is effective in the prevention and healing of blisters in patients with pemphigus vulgaris, while the patient remains on stable doses of corticosteroids and/or immunosuppressants. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Pemphigus Vulgaris
- What is being tested?
- KC706
- How many people
- 20 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2007to Jun 2008
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- At least 18 years of age;
- Diagnosis of pemphigus vulgaris
- Patients must be taking and require either corticosteroid therapy or immunosuppressive therapy or both;
- Immunosuppressive therapy, if any,should have been administered at a stable dose for at least 60 days prior to the Baseline Visit and be well-tolerated, without the expectation that there will be a need to increase that dose during the next 30 days;
- Corticosteroids, if any, should have been administered at a stable dose for at least 30 days prior to the Baseline Visit without expectation that there will be a need to increase that dose during the next 30 days;
- Patients should have active PV skin, scalp or mucosal lesions that meet at least one of the following criteria at the Baseline Visit:
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- > 3 new lesions/week every week in the previous 3 weeks (skin, scalp, and/or mucosal), with healing occurring at a rate to match the appearance of new lesions; or
- At least 3 active, established lesions with a Pemphigus Lesion Score of at least 2; skin or scalp lesions must be ≥ 5mm in diameter to qualify; there is no size requirement for mucosal lesions; or,
- 1 large active established skin, scalp, or mucosal lesion > 10 mm;
- Accessibility to veins suitable for venipuncture;
- Patients must be cooperative, able to read, understand and give informed consent, and able to adhere to the study visit schedule and protocol requirements; and,
- Patients must be willing to follow adequate contraceptive measures during the study (both sexes).
Who cannot join
- Any history of opportunistic infections within 3 months prior to receiving study drug;
- Infection with HIV;
- Past or present diagnosis of hepatitis confirmed by serology or elevated hepatic enzymes;
- History of alcoholic liver disease or cirrhosis;
- Clinically significant concurrent medical disease or laboratory abnormalities evidenced by one or more of the following:
- Hepatobiliary AST or ALT ≥ 1.5 × upper limit of normal (ULN);alkaline phosphatase ≥ 1.5 × ULN; or, total bilirubin > 90% of the ULN;
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- Renal serum creatinine > 1.5 mg/dL; or, significant proteinuria > 2+ on urinary dip test;
- Hematologic hemoglobin < 11 mg/dL; leukocytes < 3.5 × 109/L; neutrophils < 1.5 × 109/L; or, platelets < 100 × 109/L;
- Presence or history of malignancy;
- Uncontrolled diabetes (defined as diabetes requiring hospitalization or emergency care in the 3 months prior to first dose of study drug);
- History or suspicion of Gilbert's syndrome;
- Significant blood loss (> 500 mL) within 28 days prior to receipt of study drug;
- Use of an investigational drug within 30 days of screening, or longer if that drug is expected to have long-acting effects (e.g., modulation of B-cell activity);
- Use of Rituximab within the past 6 months;
- Use of intravenous IgG within the past 3 months,
- Current or recent history (within 12 months of screening) of drug or substance abuse, including alcohol;
- Known or suspected pregnancy; nursing mothers;
- Clinically significant abnormality on the screening physical examination performed at the Baseline Visit, laboratory testing, vital signs or electrocardiogram suggestive of significant unstable medical condition other than the disease under study;
- Condition which, in the opinion of the Investigator, could interfere with participation in the study or would put the patient at unacceptable risk;
- History of noncompliance with medical regimens.
Trial locations and contacts
1 site in 1 country.
United States1 site
Victoria Werth, MD
Philadelphia, Pennsylvania, 19104
About this study
Official title: A Phase 2 Open-Label Uncontrolled Pilot Study of KC706 in Patients With Stable, Active Pemphigus Vulgaris
The purpose of this study is to determine whether KC706 is effective in the prevention and healing of blisters in patients with pemphigus vulgaris, while the patient remains on stable doses of corticosteroids and/or immunosuppressants.
Read the detailed description
The present study is designed to follow-up on pre-clinical observations that administration of KC706 is associated with prevention of the development of lesions in a mouse model of PV. Patients participating in this study will be those with active disease in spite of ongoing treatment with corticosteroids and/or immunosuppressive agents. The dose chosen for this clinical study is 300 mg once daily. The primary assessment of interest will be pemphigus lesion status during dosing with KC706.
- Conditions
- Pemphigus Vulgaris
- Being tested
- KC706
- Medical subject headings
- Pemphigus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KC706Drug
300 mg once daily (QD) for 12 weeks.
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the ability of KC706 to prevent the appearance of new lesions and heal existing lesions, while maintaining stable doses of corticosteroids and/or immunosuppressants in patients with pemphigus vulgaris.
Measured: 16 weeks
Secondary outcomes (1)
Evaluate the safety of KC706 in patients with PV and to assess plasma levels of KC706 with once daily dosing.
Measured: 16 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Feb 2008 (estimated)
- Study startNov 2007
- Primary completionJun 2008
- Study completionJun 2008
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Pemphigus VulgarisStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Kemia, Inc
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Victoria Werth, MD, Principal investigator, University of PennsylvaniaBruce Strober, MD, Principal investigator, NYU MEDICAL CENTERFrancisco Kerdel, MD, Principal investigator, Florida Academic Dermatology CentersMichael Kolodney, MD, Principal investigator, University of California, Los AngelesNeil Korman, MD, Principal investigator, University Hospitals Cleveland Medical CenterAmit Pandya, MD, Principal investigator, University of Texas Southwestern Medical CenterDavid Rubenstein, MD, PhD, Principal investigator, University of North Carolina, Chapel Hill
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00606749
- Sponsor's protocol ID
- KC706-C08
- Status last verified
- Jun 2008
- First submitted
- 22 Jan 2008
- Last updated
- 19 Jun 2008
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jun 2008. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2 Open-Label Uncontrolled Pilot Study of KC706 in Patients With Stable, Active Pemphigus Vulgaris
- ClinicalTrials.gov ID
- NCT00606749
- Acronym / sponsor ID
- KC706-C08
- Conditions
- Pemphigus Vulgaris
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Kemia, IncIndustry (pharma, biotech, devices)
- Study start
- Nov 2007
- Primary completion
- Jun 2008 (actual)
- Study completion
- Jun 2008 (actual)
- First posted
- 5 Feb 2008 (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.
The purpose of this study is to determine whether KC706 is effective in the prevention and healing of blisters in patients with pemphigus vulgaris, while the patient remains on stable doses of corticosteroids and/or immunosuppressants.
Detailed description
The present study is designed to follow-up on pre-clinical observations that administration of KC706 is associated with prevention of the development of lesions in a mouse model of PV. Patients participating in this study will be those with active disease in spite of ongoing treatment with corticosteroids and/or immunosuppressive agents. The dose chosen for this clinical study is 300 mg once daily. The primary assessment of interest will be pemphigus lesion status during dosing with KC706.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- At least 18 years of age;
- Diagnosis of pemphigus vulgaris
- Patients must be taking and require either corticosteroid therapy or immunosuppressive therapy or both;
- Immunosuppressive therapy, if any,should have been administered at a stable dose for at least 60 days prior to the Baseline Visit and be well-tolerated, without the expectation that there will be a need to increase that dose during the next 30 days;
- Corticosteroids, if any, should have been administered at a stable dose for at least 30 days prior to the Baseline Visit without expectation that there will be a need to increase that dose during the next 30 days;
Key exclusion criteria (20 listed)
- Any history of opportunistic infections within 3 months prior to receiving study drug;
- Infection with HIV;
- Past or present diagnosis of hepatitis confirmed by serology or elevated hepatic enzymes;
- History of alcoholic liver disease or cirrhosis;
- Clinically significant concurrent medical disease or laboratory abnormalities evidenced by one or more of the following:
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 |
|---|---|---|
| KC706300 mg once daily (QD) for 12 weeks. | Drug | – |
Primary outcome
To evaluate the ability of KC706 to prevent the appearance of new lesions and heal existing lesions, while maintaining stable doses of corticosteroids and/or immunosuppressants in patients with pemphigus vulgaris.
Time frame: 16 weeks
1 secondary outcome
Evaluate the safety of KC706 in patients with PV and to assess plasma levels of KC706 with once daily dosing.
Time frame: 16 weeks
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 19 Jun 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.
Victoria Werth, MD principal investigator
University of Pennsylvania
Bruce Strober, MD principal investigator
NYU MEDICAL CENTER
Francisco Kerdel, MD principal investigator
Florida Academic Dermatology Centers
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 KC706 in people with Pemphigus Vulgaris, run by Kemia, Inc 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 12 inclusion and 20 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: finished, as of the sponsor's last update on 19 Jun 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.
What does the primary outcome measure?
To evaluate the ability of KC706 to prevent the appearance of new lesions and heal existing lesions, while maintaining stable doses of corticosteroids and/or immunosuppressants in patients with pemphigus vulgaris. (time frame: 16 weeks). 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 19 Jun 2008.