Patients with dermatomyositis and refractory skin disease on steroids and one steroid-sparing agent.

Dermatomyositis, Adult Type

Patients with refractory cutaneous dermatomyositis will be started on apremilast 30 mg twice a day in addition to a stable dose of their treatment regimen (including steroids and steroid sparing agents).

Apremilast 30mg

Tulane University

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

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

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 scale from 0-100. Higher scores represent worse outcome.

The proportion of participants experiencing adverse events and serious adverse events will be measured over 7 months period (6 months during the study and 1 month follow up).

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.

Complete delayed response is defined by a CDASI activity score of zero at 6 months. Partial delayed response is defined by a decrease of CDASI activity score of at least 4 points at 6 months compared to baseline. Calculation is performed as the CDASI activity score at 6 months minus the score at baseline. 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.

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.

Calculate the mean change in muscle enzymes value creatine kinase and aldolase at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: U/L, higher numbers represent worse outcomes

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.

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.

Data and research resources generated by this study will be made available to the research community and to the public at large. This strategy includes, but not limited to, presentations (poster or oral) at local, national, and international meetings; published abstracts, and journal articles (peer reviewed). Contact information will be provided in publications for direct inquiries of researchers. Data generated from sequencing and gene expression profiling will be deposited into the Gene Expression Omnibus (GEO), NCBI, to be accessible by general public. The study protocol will be shared with other researchers on the clinical trial.gov website. No identified personal patient informations will be shared.

We randomly allocated patients to treatment with IVIG or placebo. Children received either saline or intravenous immunoglobulin (IVIG) [ImmunoRel™ (batch 20081217)] at a dose of 400mg/kg/day for 5 days or an equivalent volume of 0.9% normal saline given intravenous at the rate of 0.01 to 0.02 ml/kg body weight/minute. All investigators, care providers and participants were blinded of the study drug. A second sealed envelope was kept with the patient's notes in case a physician urgently needed to know which drug a patient had received.

Japanese Encephalitis

IVIG group received 400mg/kg/day intravenous at the rate of 0.01 to 0.02 ml/kg body weight/minute for 5 days or appearance of side effect or adverse events.
Placebo group received 0.9% saline intravenous at similar rate.

Intravenous immunoglobulin [ImmunoRel™ (batch 20081217)]

Kanti Children's Hospital

BP Koirala Institute of Health Sciences

Safety and Efficacy Study of Intravenous Immunoglobulin to Treat Japanese Encephalitis

A Randomized Double Blind Placebo Controlled Trial to Assess the Safety and Efficacy of Intravenous Immunoglobulin (IVIG) in Children With Japanese Encephalitis in Nepal

Patients will be monitored for side effects such as infusion site reaction, diarrhea, rise in blood pressure (in mm Hg) and change in urinary output (in ml/Kg/hour) every 12 hours from first day of commencing treatment until until death or discharge. Patient on average are administered the study drug on the first day of admission. The study drug is administered daily for 5 days. Patients are discharged on an average on eighth day (192 hours) of hospital admission.

At the time of discharge(expected average of eighth day of admission) or death: Time to death, to recover from coma, to sit independently, to stand independently, to walk at least 5m independently, and to leave hospital.
At 6 months after discharge: history of further seizures, behavioral changes, evidence of recovery of neurological sequelae such as assessment of ability to sit independently, to stand independently, to walk at least 5 meters independently.

Pre-treatment was immediately prior to the first dose of study drug on the first day, mid-treatment was immediately prior to the 4th dose and post-treatment was 1hour after administering the fifth dose of the study drug.

Ajit Rayamajhi

Clinical Research Fellow (PhD Student)

University of Liverpool

B.P. Koirala Institute of Health Sciences

Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms.
ARM 1 has only Prednisone

MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.

Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms.
ARM 1 has only Prednisone
Prednisone: Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms

1Prednison

MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.
Methotrexate: MTX will be administered orally (in case of oral intolerance intramusculary (i.m.)), once weekly for 48 weeks. There will be a clinically oriented dose escalation starting from 10 up to 20-25 mg of MTX. Five to ten mg of folic acid will be given 24 hours after each methotrexate dose.

2Prednison MTX

Total

Sex: Female, Male

Region of Enrollment

The primary endpoint which has benn measured was the total dose of glucocorticoids administered between baseline and the end of treatment.

Prednison1

Prednison Methotrexate 2

The Primary Endpoint is the Total Dose of Glucocorticoids Administered Between Baseline and the End of Treatment.

Assessment of Disease Activity and Damage,Muscle Strength and Endurance, Enzyme Levels, Glucocorticoid Side-effects, Dose, HAQ,SF-36, Treatment Failures

Overall Study

Rheumatology Institute Prague

1 Prednison

2 Prednison Methotrexate

Polymyositis

Dermatomyositis

Prednisone will be administered orally, initially at 1.0 mg/kg/day dosage and then tapered gradually equally in the two arms

Prednisone

Methotrexate

Revmatologicky ustav

Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With PM and DM

A Prospective, Randomised, Assessor-blind, Multicenter Study of Efficacy and Safety of Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With Polymyositis and Dermatomyositis.

dermatomyositis/nàusea

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

Safety and Efficacy Study of Intravenous Immunoglobulin to Treat Japanese Encephalitis

Combined Treatment of Methotrexate + Glucocorticoids Versus Glucocorticoids Alone in Patients With PM and DM

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