Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)

NCT02728752 | Completed Phase 3 Results DMC FDA Drug

• 1 other identifier: GAM10-08
• Study Type: interventional
• Target: 95
• Locations: 10 countries
• Sites: 37

Brief Summary

Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")

Conditions

Dermatomyositis

Keywords

DM

Outcome Measures

Primary Outcomes (1)

• Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS)

  Proportion of responders in the 2.0 g/kg Octagam 10% and placebo arms at Week 16 relative to baseline (Week 0). A responder being defined as a patient with an increase of ≥20 points on the Total Improvement Score (TIS, a scale from 0 to 100; 20-39 points being minimal improvement, 40-59 points being moderate improvement, and ≥60 points being major improvement

  At week 16

Secondary Outcomes (19)

• Proportion of TIS Responders by Improvement Category at Week 16

  16 weeks

• Proportion of TIS Responders by Improvement Category at Week 40

  40 weeks

• Mean Change From Baseline (Week 0) to End of First Period (Week 16) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

  First 16 weeks

• Mean Change From End of First Period (Week 16) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

  From week 16 to Week 40

• Mean Change From Baseline (Week 0) to End of Extension Period (Week 40) in the Modified Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)

  40 weeks

Study Arms (2)

Placebo

PLACEBO COMPARATOR

Subjects randomized to placebo will receive 4 infusions of placebo every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to Octagam 10%. After response assessment at Week 16, all subjects with no confirmed deterioration and subjects switched to Octagam 10% due to confirmed deterioration but without further confirmed deterioration during the First Period will continue to receive 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to placebo and switched to Octagam 10% due to confirmed deterioration, who deteriorate also during Octagam 10% treatment at 2 consecutive visits will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Other: Placebo

Octagam10%

EXPERIMENTAL

Subjects randomized to Octagam will receive 4 infusions of 2.0 g/kg Octagam 10% every 4 weeks during the blinded First Period (16 weeks). If confirmed deterioration (deterioration at 2 consecutive visits) during the First Period, subjects will be switched to the alternate treatment. After response assessment at Week 16, all subjects with no confirmed deterioration during the First Period will continue receiving 2.0 g/kg of Octagam 10% every 4 weeks during the subsequent 6-months open-label Extension Period. At Week 28, subjects who are stable on 2.0 g/kg Octagam 10% can be switched to 1.0 g/kg Octagam 10%, at the discretion of the investigator. Subjects randomized to Octagam and switched to the alternate treatment due to confirmed deterioration will drop-out after response assessment at Week 16 and will not enter the Extension Period.

Drug: Octagam 10%

Interventions

Octagam 10% DRUG

Patients to be treated with Octagam 10%

Octagam10%

Placebo OTHER

Patients to be treated with a Placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 79 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects with diagnosis of definite or probable DM according to the Bohan and Peter criteria.
• Subjects under treatment with corticosteroids and/or maximally 2 immune-suppressants and being on stable therapy for at least 4 weeks (see Section 4.2.1) OR Subjects with previous failure of response or previous intolerance to corticosteroid and at least 1 additional immunosuppressive drug, and with steroid/immunosuppressive drugs washed out as per Section 4.2.1 (Table 2).
• Subjects with active disease, assessed and agreed upon by an independent adjudication committee.
• Manual Muscle Testing-8 (MMT-8) score \<142, with at least 2 other abnormal Core Set Measures (CSM) (Visual Analogue Scale \[VAS\] of patient global activity ≥2 cm, physician's global disease activity ≥2 cm, extra-muscular activity ≥2 cm; at least one muscle enzyme \>1.5 times upper limit of normal, Health Assessment Questionnaire ≥0.25).
• Males or females ≥ 18 to \< 80 years of age.
• Voluntarily given, fully informed written consent obtained from subject before any study-related procedures are conducted.
• Subject must be capable to understand and comply with the relevant aspects of the study protocol.

You may not qualify if:

• Cancer-associated myositis, defined as the diagnosis of myositis within 2 years of the diagnosis of cancer (except basal or squamous cell skin cancer or carcinoma in situ of the cervix that has been excised and cured and at least 1 or 5 years, respectively, have passed since excision).
• Evidence of active malignant disease or malignancies diagnosed within the previous 5 years (including hematological malignancies and solid tumors) or breast cancer diagnosed within the previous 10 years.
• Subjects with immune-mediated necrotizing myopathy with absence of typical DM rash.
• Subjects with generalized, severe musculoskeletal conditions other than DM that prevent a sufficient assessment of the subject by the physician.
• Subjects who have received IgG treatment within the last 6 months before enrolment.
• Subjects who received blood or plasma-derived products (other than IgG) or plasma exchange within the last 3 months before enrolment.
• Subjects starting or planning to start a physical therapy-directed exercise regimen during the trial.
• Cardiac insufficiency (New York Heart Association III/IV), cardiomyopathy, significant cardiac dysrhythmia requiring treatment, unstable or advanced ischemic heart disease.
• Severe liver disease, with signs of ascites and hepatic encephalopathy.
• Severe kidney disease (as defined by estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m2).
• Known hepatitis B, hepatitis C or HIV infection.
• Subjects with a history of TEE such as deep vein thrombosis, pulmonary embolism, myocardial infarction, ischemic stroke, transient ischemic attack, peripheral artery disease (Fontaine IV) ever.
• Body mass index ≥40 kg/m2.
• Medical conditions whose symptoms and effects could alter protein catabolism and/or IgG utilization (e.g. protein-losing enteropathies, nephrotic syndrome).
• Known IgA deficiency with antibodies to IgA.

Sponsors & Collaborators

• Octapharma: lead

Study Sites (37)

Octapharma Research Site

Huntington Beach, California, 92647, United States

Location

Octapharma Research Site

Los Angeles, California, 90095, United States

Location

University of California -Irvine

Orange, California, 92868, United States

Location

Octapharma Research Site

Orlando, Florida, 32819, United States

Location

NeuroMedical Research Center

Panama City, Florida, 32405, United States

Location

Octapharma Research Site

Plantation, Florida, 33324, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Octapharma Research Site

Kansas City, Kansas, 66160, United States

Location

Octapharma Research Site

Ann Arbor, Michigan, 48109, United States

Location

Octapharma Research Site

Rochester, Minnesota, 55905, United States

Location

Octapharma Research Site

Great Neck, New York, 11021, United States

Location

Octapharma Research Site

Portland, Oregon, 97239, United States

Location

Octapharma Research Site

Pittsburgh, Pennsylvania, 15213, United States

Location

Stones River Dermatology

Murfreesboro, Tennessee, 37130, United States

Location

Austin Neuromuscular Center

Austin, Texas, 78756, United States

Location

Octapharma Research Site

Houston, Texas, 77034, United States

Location

Arthritis & Osteoporosis Clinic

Waco, Texas, 76710, United States

Location

Octapharma Research Site

Montreal, Quebec, H3T 1E2, Canada

Location

Revmatologický ústav

Prague, 128 50, Czechia

Location

Charité-Universitätsmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie

Berlin, 10117, Germany

Location

Uniklinikum Münster, Klinik für Hautkrankheiten

Münster, 48149, Germany

Location

Semmelweis University Dermatology Clinic

Budapest, H-1085, Hungary

Location

University of Debrecen Dept of Internal Medicine

Debrecen, H-4032, Hungary

Location

University of Szeged Dermatology Clinic

Szeged, H-6720, Hungary

Location

Academic Medical Centre University of Amsterdam

Amsterdam, 1105AZ, Netherlands

Location

Centrum Medyczne Plejady

Krakow, 30-363, Poland

Location

Narodowy Instytut Geriatrii, Reumatologii I Rehabilitacji - Warsaw

Warsaw, 02-637, Poland

Location

Octapharma Research Site

Cluj-Napoca, 400006, Romania

Location

Scientific Research Institute for Rheumatology (Moscow)

Moscow, 115522, Russia

Location

I.M. Sechenov First Moscow State Medical University

Moscow, 119435, Russia

Location

Orenburg State Medical University Based On Regional Clinical Hospital

Orenburg, 460018, Russia

Location

3rd Rheumatology Department Of Clinical Rheumatology Hospital No. 25

Saint Petersburg, 190068, Russia

Location

AVA-Peter clinic (Saint-Petersburg)

Saint Petersburg, 191028, Russia

Location

Octapharma Research Site

Ivano-Frankivsk, 76018, Ukraine

Location

Octapharma Research Site

Lviv, 79000, Ukraine

Location

Octapharma Research Site

Sumy, 40022, Ukraine

Location

Octapharma Research Site

Ternopil, 46002, Ukraine

Location

Related Publications (6)

• Aggarwal R, Schessl J, Bata-Csorgo Z, Dimachkie MM, Griger Z, Moiseev S, Oddis CV, Schiopu E, Vencovsky J, Clodi E, Levine T, Charles-Schoeman C; ProDERM investigators. Efficacy of Intravenous Immunoglobulin for Systemic Manifestations of Dermatomyositis Beyond Muscular and Cutaneous: Sub-analysis of the ProDERM Study. Rheumatol Ther. 2025 Oct;12(5):855-871. doi: 10.1007/s40744-025-00775-5. Epub 2025 Jul 5.

  PMID: 40616720 DERIVED

• Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie MM, Griger Z, Moiseev S, Oddis CV, Schiopu E, Vencovsky J, Clodi E, Levine T, Aggarwal R. Predictors of response to intravenous immunoglobulin in patients with dermatomyositis: the ProDERM study. Rheumatology (Oxford). 2025 Jun 1;64(6):3767-3776. doi: 10.1093/rheumatology/keaf070.

  PMID: 39918968 DERIVED

• Aggarwal R, Schessl J, Charles-Schoeman C, Bata-Csorgo Z, Dimachkie MM, Griger Z, Moiseev S, Oddis CV, Schiopu E, Vencovsky J, Beckmann I, Clodi E, Levine T; ProDERM investigators. Safety and tolerability of intravenous immunoglobulin in patients with active dermatomyositis: results from the randomised, placebo-controlled ProDERM study. Arthritis Res Ther. 2024 Jan 17;26(1):27. doi: 10.1186/s13075-023-03232-2.

  PMID: 38233885 DERIVED

• Werth VP, Aggarwal R, Charles-Schoeman C, Schessl J, Levine T, Kopasz N, Worm M, Bata-Csorgo Z. Efficacy of intravenous immunoglobulins (IVIg) in improving skin symptoms in patients with dermatomyositis: a post-hoc analysis of the ProDERM study. EClinicalMedicine. 2023 Oct 2;64:102234. doi: 10.1016/j.eclinm.2023.102234. eCollection 2023 Oct.

  PMID: 37799613 DERIVED

• Aggarwal R, Charles-Schoeman C, Schessl J, Bata-Csorgo Z, Dimachkie MM, Griger Z, Moiseev S, Oddis C, Schiopu E, Vencovsky J, Beckmann I, Clodi E, Bugrova O, Danko K, Ernste F, Goyal NA, Heuer M, Hudson M, Hussain YM, Karam C, Magnolo N, Nelson R, Pozur N, Prystupa L, Sardy M, Valenzuela G, van der Kooi AJ, Vu T, Worm M, Levine T; ProDERM Trial Group. Trial of Intravenous Immune Globulin in Dermatomyositis. N Engl J Med. 2022 Oct 6;387(14):1264-1278. doi: 10.1056/NEJMoa2117912.

  PMID: 36198179 DERIVED

• Aggarwal R, Charles-Schoeman C, Schessl J, Dimachkie MM, Beckmann I, Levine T. Prospective, double-blind, randomized, placebo-controlled phase III study evaluating efficacy and safety of octagam 10% in patients with dermatomyositis ("ProDERM Study"). Medicine (Baltimore). 2021 Jan 8;100(1):e23677. doi: 10.1097/MD.0000000000023677.

  PMID: 33429735 DERIVED

MeSH Terms

Conditions

Dermatomyositis

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Results Point of Contact

• Title: Mikaela Raymond
• Organization: CRMG

ctgov@clinicalresearchmgt.com

866-337-1868

Study Officials

• Wolfgang Frenzel

  International Medical Director

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 11, 2016
• First Posted: April 5, 2016
• Study Start: February 27, 2017
• Primary Completion: November 5, 2019
• Study Completion: November 5, 2019
• Last Updated: December 23, 2021
• Results First Posted: April 21, 2021

Record last verified: 2021-11

Locations

HU (3); DE (2); CA (1); NL (1); RU (5); UA (4); CZ (1); US (17); RO (1); PL (2)