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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 22 February 2021
Main ID:  NCT03529955
Date of registration: 24/04/2018
Prospective Registration: Yes
Primary sponsor: Tulane University
Public title: Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis
Scientific title: A Phase 2, Open Label Single Arm Study for Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis
Date of first enrolment: June 12, 2018
Target sample size: 10
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT03529955
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Carole Bitar, MD
Address: 
Telephone: 202-642-8122
Email: cbitar@tulane.edu
Affiliation: 
Name:     Carole Bitar, MD
Address: 
Telephone: 2026428122
Email: cbitar@tulane.edu
Affiliation: 
Name:     Carole Bitar, MD
Address: 
Telephone:
Email:
Affiliation:  Tulane University
Key inclusion & exclusion criteria

Inclusion Criteria:

- Must understand the risks and the benefits/purpose of the study and provide signed and
dated informed consent.

- Must be 18 years at time of signing the informed consent form.

- Willing to participate in all required evaluations and procedures in the study
including the ability to swallow pills without difficulty.

- Patients must have a diagnosis of DM based upon the characteristic cutaneous findings
proposed by Sontheimer[6] and/or a skin biopsy consistent with DM.

- Patients must be candidate for systemic therapy for their DM skin disease defined by
inadequate response to aggressive sun protection along with the use of potent topical
corticosteroids and/or immunomodulators.

- Patients with a diagnosis of dermatomyositis on steroid-sparing agent and/or systemic
steroids (maximum dose of prednisone 1mg/Kg) and still having cutaneous disease
activity of at least 5 on the CDASI scale.

- If on immunosuppressive treatments and/or steroids, patients must be on stable doses
for at least 4 weeks (28 days).

- Patients must undergo age appropriate cancer screening.

- Females of childbearing potential (FCBP) must have a negative pregnancy test at
screening (day 0 of the study and every month throughout the study). While on
investigational product and for at least 28 days after taking the last dose of
investigational product.

Exclusion Criteria:

- Increasing or changing dose of topical therapy within 14 days of study day 0
(including but not limited to topical corticosteroids, tacrolimus, pimecrolimus).

- Increasing or changing systemic steroids dosing within 28 days of study day 0.

- Increasing or changing dosing for concurrent therapy agents within 28 days or 5
half-lives of the biologic agent, whichever is longer, before study day 0:
methotrexate, azathioprine, mycophenolate mofetil, hydroxychloroquine, dapsone,
leflunomide, cyclosporine, biologic agents (anti-TNFs), IVIG, rituximab.

- History of any clinically significant (as determined by the investigators) 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
patient 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.

- Untreated Latent Mycobacterium tuberculosis infection or active tuberculosis infection
as indicated by a positive Purified Protein Derivative (PPD) skin test or T-spot.

- Any condition, including the presence of laboratory abnormalities that places the
patient at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study.

- Patients with acute dermatomyositis onset and rapid progression of muscle disease or
significant systemic involvement including pulmonary diseases associated with DM.

- Prior major surgery or major life-threatening medical illness within 2 weeks.

- Inflammatory bowel disease, malabsorption or any other gastrointestinal motility
disorders that limit the absorption of the study drug.

- Active hepatitis B or C infection with detectible viral nucleic acid in the blood or
known Human Immunodeficiency Virus (HIV) positivity.

- Prior history of suicide attempt at any time in the patient's lifetime prior to
screening or randomization, or major psychiatric illness requiring hospitalization
within the last 3 years.

- Active substance abuse or a history of substance abuse within 6 months prior to
screening.

- Use of any investigational drug within 4 weeks prior to randomization, or 5
pharmacokinetic/pharmacodynamic half-lives, if known (whichever is longer).

- Prior treatment with apremilast.

- Any severe systemic illness requiring IV antibiotics within the two weeks prior to
initiation of the study drug.

- Malignancy or history of malignancy within the past four years, except for:

- treated [ie, cured] basal cell or squamous cell in situ skin carcinomas;

- treated [ie, cured] cervical intraepithelial neoplasia (CIN) or carcinoma in situ
of cervix with no evidence of recurrence within the previous 4 years.



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All
Health Condition(s) or Problem(s) studied
Dermatomyositis, Adult Type
Intervention(s)
Drug: Apremilast 30mg
Primary Outcome(s)
The primary endpoint analysis would be overall response rate measured by the number of participants experiencing at least 4 points decrease in CDASI activity score at 3 months. [Time Frame: Data collected at 3 months after baseline visit]
Secondary Outcome(s)
2. The secondary endpoint analysis would be safety as measured by the number of participants experiencing adverse events and serious adverse events occurring during 6 months of therapy and 1 month follow up. [Time Frame: 7 months]
3. An additional secondary endpoint analysis would be durability of response measured by the number of participants with maintenance of their CDASI activity score or change in their CDASI activity score by at least 4 points compared to 3 months. [Time Frame: Data collected at 6 months compared to data collected at 3 months]
4. An additional secondary endpoint analysis would be delayed response measured by the number of participants experiencing at least 4 points decrease in CDASI activity score at 6 months compared to baseline. [Time Frame: Data collected at 6 months after baseline visit]
5. An additional secondary endpoint analysis would assess quality of life as measured by the number of participants experiencing at least 5 points decrease on DLQI score at 3 and 6 months. [Time Frame: Data collected at 3 and 6 months after baseline visit]
Secondary ID(s)
2017-978
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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