Adults 18 to 75, any sex, with Dermatomyositis, Adult Type. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
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.Primary· Data collected at 3 months after baseline visit
Cutaneous dermatomyositis disease area and severity index (CDASI) activity score is a validated tool to measure skin disease activity in dermatomyositis. The overall response rate (ORR) includes partial and complete responses. Complete response is defined by a CDASI activity score of zero. Partial response is defined by a decrease of CDASI activity score of at least 4 points. Calculation is performed as the CDASI activity score at 3 month(s) minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a sc
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
7
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.Secondary· 7 months
The proportion of participants experiencing adverse events and serious adverse events was measured over 7 months period (6 months during the study and 1 month follow up) using Common Terminology Criteria for Adverse Events (CTCAE) v5.0.
Grade refers to severity of the AE. The CTCAE displays Grades 1 to 5 with unique clinical descriptions of severity for each AE:
Grade 1 Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated Grade 2 Moderate; minimal, local or noninvasive intervention indicated; limiting age- appropriate instrumental Activity
Headache Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
7
Nausea Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
5
Diarrhea Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
4
Herpes Zoster Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
2
Influenza Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
1
Pneumonia Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
1
Acute sinusitis Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
1
Hypertension Grade 1-2
Group
Value
95% CI
Dermatomyositis Patients With Refractory Cutaneous Disease
1
An Additional Secondary Endpoint Analysis Would be Durability of Response Measured Participants CDASI Activity Score or Change in Their CDASI Activity Score at 6 Months Compared to 3 Months.Secondary· Data collected at 6 months compared to data collected at 3 months
The durability of response will be measured using the CDASI activity score at 6 months minus CDASI activity score at 3 months. Complete response durability is defined as zero or minus difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Partial response durability is defined as \>4 points difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.
Group
Value
95% CI
CDASI Score at 3 Months
16.9
± 8.3
CDASI Score at 6 Months
14
± 6.4
An Additional Secondary Endpoint Analysis Would Assess Quality of Life as Measured at 3 Months Compared to Quality of Life Measured at 6 MonthsSecondary· Data collected at 3 and 6 months after baseline visit
Dermatology Life Quality Index (DLQI) is a validated tool to measure quality of life in patients with skin disease. Complete response is defined by a DLQI of zero at 3, and 6 months. Partial response is defined by a decrease of DLQI of at least 5 points at 3, and 6 months compared to baseline. Calculation is performed as the DLQI at 3, and 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
Units : Units on a scale from 0-30, higher scores represent worse outcome.
Group
Value
95% CI
DLQI Score at 3 Months
6.3
± 4.6
DLQI Score at 6 Months
4.2
± 2.1
An Additional Endpoint Analysis Would Assess the MMT-8 Score in Patients With Muscle Disease as Measured at 3 and 6 Months Compared to Baseline.Secondary· Data collected at 3 and 6 months after baseline visit
MMT-8 (Manual Muscle Testing-8) score is a validated tool to assess muscle strength. Calculate the mean change in MMT-8 score at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: Units on a scale. Scale goes from 0-150. 150 is perfect strength.
Group
Value
95% CI
MMT-8 Score at 3 Months
143.3
± 10.9
MMT-8 Score at 6 Months
144.5
± 8.0
An Additional Endpoint is to Assess the Gene Expression Profiling and Immunohistochemistry Analysis Change on Skin Biopsies at 3 Months Compared to Baseline.Secondary· Data collected at 3 months after baseline visit
Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in gene expression profiling and immunohistochemistry stain. Gene expression profiling will be analyzed using inferential statistics with a False Discovery Rate (FDR) of \< 0.05.
Down regulated genes
Group
Value
95% CI
Skin Biopsy at Baseline for Gene Expression Profiling
0
Skin Biopsy at 3 Months Into Apremilast Therapy for Gene Expression Profiling
123
Up regulated genes
Group
Value
95% CI
Skin Biopsy at Baseline for Gene Expression Profiling
0
Skin Biopsy at 3 Months Into Apremilast Therapy for Gene Expression Profiling
72
An Additional Endpoint is to Assess the Immunohistochemistry Analysis Change on Skin Biopsies at 3 Months Compared to Baseline.Secondary· Data collected at 3 months after baseline visit
Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in immunohistochemistry stain.
STAT1
Group
Value
95% CI
Skin Biopsy at Baseline for IHC
96.2
± 48.6
Skin Biopsy at 3 Months Into Apremilast Therapy for IHC
50.1
± 28.6
STAT3
Group
Value
95% CI
Skin Biopsy at Baseline for IHC
44.3
± 25.3
Skin Biopsy at 3 Months Into Apremilast Therapy for IHC
17.4
± 7.7
Adverse events — posted to ClinicalTrials.gov
Time frame: Over 6 months period..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Dermatomyositis Patients With Refractory Cutaneous Disease
With limited treatment options available for dermatomyositis, the investigators hypothesize that apremilast, a phosphodiesterase-4 (PDE-4) inhibitor, is a safe and efficacious add-on treatment in patients with refractory cutaneous dermatomyositis.
The study will investigate the efficacy, safety and toxicity of apremilast given at 30 mg twice daily to patients with refractory cutaneous dermatomyositis. Clinical response will be assessed at 1 and 3 months. Patients will also be evaluated for durability of their response for up to 6 months.
Treatment will be monitored with frequent clinical visits (0, 1, 3 and 6 months) and blood tests (CBC, CMP, creatine kinase, aldolase). Treatment will be discontinued at disease progression or unacceptable adverse events. Disease progression is defined as 4 points increase in the cutaneous dermatomyositis disease area and severity index (CDASI) score, worsening of muscle disease by manual muscle testing (MMT-8) score and 5 points increase in dermatomyositis life quality index (DLQI).
5 mm skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment for gene expression profiling and confirmatory immunohistochemical stains.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Tulane University
Last refreshed: 3 March 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03529955.