DermatomyositisNCT01140503
A Study to Evaluate the Safety and Efficacy of Apremilast in the Treatment of Skin Disease in Patients With Dermatomyositis
A study testing Apremilast in people with Dermatomyositis, run by Stanford University at 1 site in United States.
Trial summary
This study is designed to evaluate the safety and efficacy of an oral medicine (called apremilast) for treating skin involvement in patients with the disease dermatomyositis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dermatomyositis
- What is being tested?
- Apremilast
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2010to Sep 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Must understand and voluntarily sign an informed consent form
- Must be 18 years at time of signing informed consent form
- Must be able to adhere to the study visit schedule and other protocol requirements
- Patients must have a diagnosis of DM based upon the characteristic cutaneous findings proposed by Sontheimer1 and a skin biopsy consistent with DM
- Subjects must be a candidate for systemic therapy for their DM skin disease: a subject is considered a candidate, if, in the judgment of the investigator, they are not adequately responding to aggressive sun protection along with the use of potent (e.g. class I or II) topical corticosteroids and/or immunomodulators
- Must have cutaneous disease activity of at least "moderate" on a 5 point Likert scale (using the PGA)
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- Must have cutaneous disease activity score of at least 5 on the CDASI (activity) scale
- Concurrent therapy with topical corticosteroids and/or prednisone and/or antimalarials is permitted as defined in Exclusion Criteria.
- Concurrent therapy with methotrexate azathioprine, mycophenolate mofetil, or leflunomide is permitted as defined in Exclusion Criteria
- Must meet the following laboratory criteria:
- Hemoglobin \≥ 12 g/dL
- White blood cell (WBC) count \≥ 3000 /uL (\≥ 3.0 X 10^9/L) and < 14,000/uL (< 14 X 10^9/L)
- Platelets \≥ 100,000 /uL (\≥ 100 X 10^9/L)
- Serum creatinine \≤ 1.5 mg/dL (\≤ 132.6 \μmol/L)
- Total bilirubin \≤ 2.0 mg/dL
- Aspartate transaminase (AST [serum glutamic oxaloacetic transaminase, SGOT]) and alanine transaminase (ALT [serum glutamate pyruvic transaminase, SGPT]) ; 1.5x upper limit of normal (ULN) unless, in the opinion, of the investigator, the elevation is secondary to active muscle inflammation.
- Females of childbearing potential (FCBP) must have a negative urine pregnancy test at screening (Visit 1). In addition, sexually active FCBP must agree to use TWO of the following adequate forms of contraception while on study medication: oral, injectable, or implantable hormonal contraceptives; tubal ligation; intrauterine device; barrier contraceptive with spermicide; or vasectomized partner. A FCBP must agree to have pregnancy tests every 28 days while on study medication.
- Males (including those who have had a vasectomy) must agree to use barrier contraception (latex condoms) when engaging in sexual activity with FCBP while on study medication and for 84 days after taking the last dose of study medication
Who cannot join
- History of inadequate response of cutaneous DM disease to greater than 2 of the following agents: methotrexate, cyclosporine, azathioprine, mycophenolate mofetil, IVIG, leflunomide, cyclophosphamide.
- History of inadequate response to thalidomide for dermatomyositis skin disease.
- Receiving topical therapy within 14 days of Study Day 0 (including but not limited to topical corticosteroids, tacrolimus, pimecrolimus). Exceptions: low potency corticosteroids will be allowed as background therapy for treatment of the face and scalp as needed, but dose must be stable 14 days prior to Study Day 0 and throughout the study
- Concurrent therapy with prednisone (or equivalent dose of systemic corticosteroid) at greater than 10 mg daily.
- Concurrent therapy with more than one of the following agents: methotrexate, azathioprine, mycophenolate mofetil, leflunomide.
- Receiving the following dosages of medications during the study or within 28 days before Study Day 0:
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- Hydroxychloroquine at >600 mg/day
- Chloroquine at >400 mg/day
- Methotrexate at >25 mg/week
- Mycophenolate mofetil at >3 g/day
- Azathioprine at >3 mg/kg/day
- Leflunomide at >20mg/day
- Treatment with the following biologic agents:
- Adalimumab, etanercept, efalizumab, or infliximab within 12 weeks of Study Day 0 and for the study duration
- IVIG within 12 weeks of Study Day 0 and for the study duration
- Rituximab within 9 months of Study Day 0 and for the study duration
- Alefacept within 24 weeks of Study Day 0 and for the study duration
- Have received fluctuating doses of any of the following medications 28 days before Study Day 0: methotrexate, mycophenolate mofetil, azathioprine, leflunomide, dapsone
- Have received fluctuating doses of hydroxychloroquine 2 months before Study Day 0
- Have received fluctuating doses of chloroquine 3 months before Study Day 0
- Have received fluctuating doses of prednisone within 14 days prior to Study Day 0
- Received leflunomide >20 mg/day in the 6 months prior to Study Day 0
- Treatment with any investigational drug therapy within 28 days before Study Day 0 or biologic therapies within 30 days or 5 half-lives of the biologic agent, whichever is longer, before Study Day 0
- Currently receiving any of the following medications:
- Cyclophosphamide
- Intravenous immunoglobulin (IVIG)
- Any TNF inhibitor, including adalimumab, etanercept, infliximab, or certolizumab
- Rituximab
- Efalizumab
- Cyclosporine
- Oral FK506 (tacrolimus)
- Thalidomide
- History of any clinically significant (as determined by the investigator) cardiac, endocrinologic, pulmonary, neurologic, psychiatric, hepatic, renal, hematologic, immunologic, or other major uncontrolled disease
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Pregnant or breastfeeding
- Latent Mycobacterium tuberculosis infection as indicated by a positive Purified Protein Derivative (PPD) skin test.
- History of active Mycobacterium tuberculosis infection (any subspecies) within 3 years prior to the screening visit and without documentation of successful treatment
- Subjects who completed treatment at least 3 years prior to screening but lack documentation may not be enrolled in the study. Subjects who completed treatment at least 3 years prior to screening are allowed if successful treatment was completed at least 3 years prior to screening and is documented and available for verification
- History of incompletely treated latent Mycobacterium tuberculosis infection as indicated by:
- Subject's medical records documenting incomplete treatment for Mycobacterium tuberculosis
- Subject's self reported history of incomplete treatment for Mycobacterium tuberculosis
- Any clinically significant abnormality on 12-lead ECG at screening
- Presence of hepatitis B surface antigens (HBsAg) or Hepatitis B core antibody positive at screening
- Antibodies to hepatitis C virus at screening
- History of human immunodeficiency virus (HIV) infection
- Malignancy or history of malignancy except for treated (i.e. cured) basal-cell skin carcinomas
- Abnormal chest x-ray findings other than that consistent with dermatomyositis-associated interstitial lung disease. Chest x-rays performed within 3 months prior to start of study drug are acceptable
- Any condition, including the presence of laboratory abnormalities, that places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: An Open Label Study Evaluating the Safety and Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Dermatomyositis
This study is designed to evaluate the safety and efficacy of an oral medicine (called apremilast) for treating skin involvement in patients with the disease dermatomyositis.
- Conditions
- Dermatomyositis
- Being tested
- Apremilast
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ApremilastDrug
Apremilast 20mg PO BID
| Study group | Role | What they receive |
|---|---|---|
| apremilast | Experimental | apremilast 20mg bid Receives: Drug: Apremilast |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup.
Measured: 16 weeks
Secondary outcomes (2)
The Secondary Outcome Measure Will be Efficacy, as Measured by the Number of Participants Experiencing a 30% Decreased in the CDASI-a Score at 12 Weeks.
This was an intent to treat analysis--dropouts are considered treatment failures. Missing data at 12 weeks imputed by last observation carried forward.
Measured: Data collected at 12 weeks after baseline visit.
The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity at 12 Weeks
The CDASI (Cutaneous Dermatomyositis Activity and Severity Index) is a validated instrument to measure skin disease activity in dermatomyositis. A clinically meaningful change is a decrease of 4 points. All missing data are imputed using last observation carried forward. Calculation is performed as the score at 12 weeks minus the score at baseline.
Measured: Data collected at baseline at 12 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jun 2010 (estimated)
- Study startFeb 2010
- Primary completionAug 2011
- Study completionSep 2011
Why it stopped: Slow recruitment of participants
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for DermatomyositisStudies of the same condition, open and under way.
- More trials run by Stanford 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, David Fiorentino, Stanford University
- Investigators
- David Franklin Fiorentino, Principal investigator, Stanford UniversityKatharine Arefiev, Sub-investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01140503
- Sponsor's protocol ID
- SU-03302010-5522
- Other IDs
- 16975
- Status last verified
- Mar 2015
- First submitted
- 6 Apr 2010
- Last updated
- 4 Mar 2015
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 4 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study Evaluating the Safety and Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Dermatomyositis
- ClinicalTrials.gov ID
- NCT01140503
- Acronym / sponsor ID
- SU-03302010-5522
- Conditions
- Dermatomyositis
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Slow recruitment of participants
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Feb 2010
- Primary completion
- Aug 2011 (actual)
- Study completion
- Sep 2011 (actual)
- First posted
- 9 Jun 2010 (estimated)
- Results
- Posted 4 Mar 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to evaluate the safety and efficacy of an oral medicine (called apremilast) for treating skin involvement in patients with the disease dermatomyositis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| apremilast | Experimental | Drug: Apremilastapremilast 20mg bid |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Must understand and voluntarily sign an informed consent form
- Must be 18 years at time of signing informed consent form
- Must be able to adhere to the study visit schedule and other protocol requirements
- Patients must have a diagnosis of DM based upon the characteristic cutaneous findings proposed by Sontheimer1 and a skin biopsy consistent with DM
- Subjects must be a candidate for systemic therapy for their DM skin disease: a subject is considered a candidate, if, in the judgment of the investigator, they are not adequately responding to aggressive sun protection along with the use of potent (e.g. class I or II) topical corticosteroids and/or immunomodulators
Key exclusion criteria (48 listed)
- History of inadequate response of cutaneous DM disease to greater than 2 of the following agents: methotrexate, cyclosporine, azathioprine, mycophenolate mofetil, IVIG, leflunomide, cyclophosphamide.
- History of inadequate response to thalidomide for dermatomyositis skin disease.
- Receiving topical therapy within 14 days of Study Day 0 (including but not limited to topical corticosteroids, tacrolimus, pimecrolimus). Exceptions: low potency corticosteroids will be allowed as background therapy for treatment of the face and scalp as needed, but dose must be stable 14 days prior to Study Day 0 and throughout the study
- Concurrent therapy with prednisone (or equivalent dose of systemic corticosteroid) at greater than 10 mg daily.
- Concurrent therapy with more than one of the following agents: methotrexate, azathioprine, mycophenolate mofetil, leflunomide.
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 |
|---|---|---|
| ApremilastApremilast 20mg PO BID | Drug | apremilast |
Primary outcome
The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup.
Time frame: 16 weeks
2 secondary outcomes
The Secondary Outcome Measure Will be Efficacy, as Measured by the Number of Participants Experiencing a 30% Decreased in the CDASI-a Score at 12 Weeks.
Time frame: Data collected at 12 weeks after baseline visit.
The Secondary Outcome Measure Will be Efficacy as Measured by the Mean Change in CDASI-activity at 12 Weeks
Time frame: Data collected at baseline at 12 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Mar 2015.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
David Franklin Fiorentino principal investigator
Stanford University
Katharine Arefiev sub-investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Apremilast in people with Dermatomyositis, run by Stanford 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?
The sponsor lists 18 inclusion and 48 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 4 Mar 2015. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 4 Mar 2015). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Primary Endpoint Analysis Will be Safety, as Measured by the Number of Adverse Events and Serious Adverse Events Occuring During 12 Weeks of Therapy and 4 Weeks of Followup. (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 4 Mar 2015.