Publication31 December 2021

ProDERM Trial

Efficacy, Tolerability and Safety of IVIg (octagam® 10%) in Adult Patients with Active Dermatomyositis - successfully meeting the primary endpoint

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Dermatomyositis (DM) is a rare chronic systemic autoimmune disease with characteristic skin rash and progressive proximal muscle weakness.

Current therapies encompass corticosteroids, other immunosuppressants as well as intravenous immunoglobulins (IVIg). However, none of these therapies are proven by randomized controlled phase 3 studies.1

The ProDERM (Progress in DERMatomyositis) study was a prospective, double-blind, randomised, parallel-group, placebo-controlled, multicentre, phase 3 study of octagam® (intravenous human normal immunoglobulin) in adults with active dermatomyositis.1,2

Watch Prof. Rohit Aggarwal, Medical Director of the Arthritis and Autoimmunity Center at the University of Pittsburgh School of Medicine and a member of the ProDERM study Steering Committee, providing insights on the study

Study Design

The ProDERM study consisted of a Screening phase, First Period, and Extension Period. Patients were screened and, if eligible, were randomised 1:1 to receive either 2 g/kg of octagam® or placebo, administered every 4 weeks until Week 16 (First Period). During the First Period, patients were switched to the alternate treatment if they showed confirmed deterioration (defined as deterioration at 2 consecutive visits) at or after Week 8. After response assessment at Week 16, all patients on placebo and those without deterioration on octagam® entered the open-label Extension Period, receiving 2 g/kg octagam® every 4 weeks for 24 weeks.1,2

Study Endpoints

Primary Efficacy Endpoint1:

Proportion of responders at Week 16 (First Period). A responder was defined as a patient with a total improvement score (TIS) ≥20 (at least minimal improvement) at Week 16 and no confirmed deterioration up to and including Week 16.

Key Secondary Endpoints1:

•Proportion of responders at Week 16 (First Period) and Week 40 (Extension Period) by improvement category (minimal/moderate/major)

•Time to at least minimal improvement

•TIS change from baseline, individual CSMs, QoL, total activity and damage scores in the modified CDASI

•Safety

CDASI: cutaneous dermatomyositis disease area and severity index; CSM: core set measures; QoL: quality of life.

Patient Population

Patients aged ≥ 18 and < 80 years with definite or probable dermatomyositis and active disease (Bohan and Peter criteria) were eligible for study. Inclusion and exclusion criteria for the ProDERM study have been published. A total of 95 patients were included in the study, of whom 48 were randomised to placebo and 47 to octagam®.2

Baseline Characteristics

Efficacy Data

The primary endpoint was met, with significantly more responders in the octagam® group than in the placebo group (79% vs 44%; p = 0.0008) at the end of the First Period (Week 16). In addition, significantly more patients in the octagam® group achieved a moderate response or a major response at Week 16 than in the placebo group.3

Minimal responder was defined as a patient with an improvement of at least 20 points, moderate responder was defined as a patient with an improvement of at least 40 points and major responder was defined as a patient with an improvement of at least 60 points on the TIS at Week 16 compared to baseline and who did not meet ‘Deterioration’ criteria at 2 consecutive visits up to and including Week 16.

Safety and Tolerability

octagam® was generally well tolerated throughout the study. The majority of treatment-emergent adverse events (TEAEs) were mild in intensity. The most commonly reported related TEAEs were headache (42%), nausea (16%) and pyrexia (19%).

The incidence of serious TEAEs was low and similar in the two treatment groups. No deaths occurred during the study.

Patients with dermatomyositis are at an increased risk of thromboembolic events (TEEs)2,3. A total of 8 TEEs were reported in 6 patients treated with octagam®. The maximum allowable infusion rate was reduced after 6 TEEs had occurred, leading to a decrease in the exposure-adjusted incidence rate for TEEs.2

Download the Safety Poster here

CONCLUSIONS

The ProDERM study met the primary and secondary endpoints and was the first large trial proving the efficacy and safety of IVIg in dermatomyositis. Results from this study have led to the approval of octagam® as a therapy for the treatment of adults with dermatomyositis in Europe as well as in the USA. octagam® was efficacious and generally well tolerated, and may allow sparing of corticosteroids in adults with dermatomyositis.²

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