DermatomyositisPhase 3IIMNCT02728752
Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis (Idiopathic Inflammatory Myopathy)
A Phase 3 study testing Octagam 10% in people with Dermatomyositis, run by Octapharma at 37 sites in 10 countries.
Trial summary
Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study") Read more about this study
- Who is it for?
- Ages 18 to 79 years
- Conditions the study is for
- Dermatomyositis
- What is being tested?
- Octagam 10%
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 95 participants
- Where
- 37 sitesin 10 countries
- Study dates
- 27 Feb 2017to 5 Nov 2019
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 79 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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 overlap myositis (except for overlap with Sjögren's syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis, juvenile dermatomyositis or drug-induced myopathy.
- 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.
Show all 25Show fewer
- 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.
- History of hypersensitivity, anaphylaxis or severe systemic response to immuno-globulin, blood or plasma derived products or any component of Octagam 10%.
- Known blood hyperviscosity, or other hypercoagulable states.
- Subjects with a history of drug abuse within the past 5 years prior to study enrollment.
- Subjects unable or unwilling to understand or comply with the study protocol.
- Participating in another interventional clinical study with investigational treatment within 3 months prior to study enrollment.
- Women who are breast feeding, pregnant, or planning to become pregnant, or are unwilling to apply an effective birth control method (such as implants, injectables, combined oral contraceptives, some intrauterine devices [IUDs], sexual abstinence or vasectomized partner) up to four weeks after the last IMP infusion.
- Subjects who are accommodated in an institution or care facility based on an official directive or court order.
- Subjects who are in any way dependent on the Sponsor, Investigator or Study Site.
- Subjects who received forbidden medication within the washout period as defined in Section 4.2.2 (Table 3).
Trial locations and contacts
37 sites in 10 countries.
37 sites in 10 countries
United States17 sites
Octapharma Research Site
Ann Arbor, Michigan, 48109
Austin Neuromuscular Center
Austin, Texas, 78756
Octapharma Research Site
Great Neck, New York, 11021
Octapharma Research Site
Houston, Texas, 77034
Octapharma Research Site
Huntington Beach, California, 92647
Octapharma Research Site
Kansas City, Kansas, 66160
Octapharma Research Site
Los Angeles, California, 90095
Stones River Dermatology
Murfreesboro, Tennessee, 37130
University of California -Irvine
Orange, California, 92868
Octapharma Research Site
Orlando, Florida, 32819
NeuroMedical Research Center
Panama City, Florida, 32405
Octapharma Research Site
Pittsburgh, Pennsylvania, 15213
Octapharma Research Site
Plantation, Florida, 33324
Octapharma Research Site
Portland, Oregon, 97239
Octapharma Research Site
Rochester, Minnesota, 55905
University of South Florida
Tampa, Florida, 33612
Arthritis & Osteoporosis Clinic
Waco, Texas, 76710
Russia5 sites
Scientific Research Institute for Rheumatology (Moscow)
Moscow, 115522
I.M. Sechenov First Moscow State Medical University
Moscow, 119435
Orenburg State Medical University Based On Regional Clinical Hospital
Orenburg, 460018
3rd Rheumatology Department Of Clinical Rheumatology Hospital No. 25
Saint Petersburg, 190068
AVA-Peter clinic (Saint-Petersburg)
Saint Petersburg, 191028
Ukraine4 sites
Octapharma Research Site
Ivano-Frankivsk, 76018
Octapharma Research Site
Lviv, 79000
Octapharma Research Site
Sumy, 40022
Octapharma Research Site
Ternopil, 46002
Hungary3 sites
Semmelweis University Dermatology Clinic
Budapest, H-1085
University of Debrecen Dept of Internal Medicine
Debrecen, H-4032
University of Szeged Dermatology Clinic
Szeged, H-6720
Germany2 sites
Charité-Universitätsmedizin Berlin, Klinik für Dermatologie, Venerologie und Allergologie
Berlin, 10117
Uniklinikum Münster, Klinik für Hautkrankheiten
Münster, 48149
Poland2 sites
Centrum Medyczne Plejady
Krakow, 30-363
Narodowy Instytut Geriatrii, Reumatologii I Rehabilitacji - Warsaw
Warsaw, 02-637
Netherlands1 site
Academic Medical Centre University of Amsterdam
Amsterdam, 1105AZ
About this study
Official title: Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM Study")
Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")
- Conditions
- Dermatomyositis
- Being tested
- Octagam 10%, Placebo
- Keywords
- DM
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Octagam 10%Drug
Patients to be treated with Octagam 10%
PlaceboOther
Patients to be treated with a Placebo
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: Drug: Octagam 10% |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: At week 16
Secondary outcomes (19)
Proportion of TIS Responders by Improvement Category at Week 16
The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.
Primary: Total number of all patients who had at least minimal, moderate, or major response.
At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.
Measured: 16 weeks
Proportion of TIS Responders by Improvement Category at Week 40
The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.
At Least Minimal: Total number of all patients who had minimal, moderate, or major response.
At Least Moderate: Number of patients who had moderate or major response. At Least Major: Number of patients who had a major response.
Measured: 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)
The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed.
Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total activity score as well as for the total damage score the sub-scores will be added up. The score for total activity ranges from 0 to 100, for total damage from 0 to 32 points. Higher scores indicate greater disease activity or greater disease damage respectively.
Measured: 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)
The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed
Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total activity score as well as for the total damage score the sub-scores will be added up. The score for total activity ranges from 0 to 100, for total damage from 0 to 32 points. Higher scores indicate greater disease activity or greater disease damage respectively.
Measured: 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)
The modified CDASI has 3 activity measures (erythema, scale, and erosion/ulceration) and 2 damage measures (poikiloderma and calcinosis) which are assessed over 15 body areas. In addition, Gottron's papules on the hands are evaluated both for activity and damage. Lastly, the activity of periungual changes and alopecia is assessed.
Absence of skin lesions as described above is scored with "0" for each of the above described areas. Skin lesion will be scored with at least "1" (present), some lesions will be graded from "1" (less severe) to "2" (severe); others with "1", "2" or "3". For the total activity score as well as for the total damage score the sub-scores will be added up. The score for total activity ranges from 0 to 100, for total damage from 0 to 32 points. Higher scores indicate greater disease activity or greater disease damage respectively.
Measured: 40 weeks
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: SF-36v2 Health Survey
The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.
SF-36 scores ranging from 0 to 100. 0 represented the lowest possible score (worst health state) and 100 represented the highest possible score (best health state), with scores in between representing the percentages of the total possible score achieved by respondents on a given scale.
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Physician's Global Disease Activity
10 cm VAS assessing global disease activity from "No evidence of disease activity" to "Extremely active or severe disease activity"; Disease Activity being defined as potentially reversible pathology or physiology resulting from the myositis). Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Patient Global Disease Activity
Patient's Global Disease Activity (10cm VAS assessing the overall activity of the patient's disease today from "No evidence of disease activity" to "Extremely active or severe disease activity", Disease Activity being active inflammation in the patient's muscles, skin, joints, intestines, heart, lungs or other parts of the body, which can improve when treated with medicines). 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: MMT-8
Manual Muscle Testing - MMT-8; a set of 8 designated muscles tested bilaterally [potential score 0 - 150]. Higher score associated with better outcome.
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Health Assessment Questionnaire
Health Assessment Questionnaire (HAQ); a generic rather than a disease-specific instrument; comprised of 8 sections: dressing, arising, eating, walking, hygiene, reach, grip, and activities. There are 2 or 3 questions for each section. Scoring within each section is from 0 [without any difficulty] to 3 [unable to do]. For each section the score given to that section is the worst score within the section. The 8 scores of the 8 sections are summed and divided by 8). Assessment completed by patients. Lowest score 0 highest score 24. Higher score associated with worse outcome.
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Alanine Aminotransferase)
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aspartate Aminotransferase)
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Lactate Dehydrogenase)
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aldolase)
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Creatine Kinase)
Measured: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Extra-muscular Activity
10 cm VAS assessing extra-muscular activity from "No evidence of disease activity" to "Extremely active or severe disease activity". It encompasses an overall evaluation for the disease activity in all the extramuscular organ systems and excludes muscle disease activity. Assessment completed by physician. 0 is lowest score and 10 is highest score. Higher score associated with worse outcome.
Measured: From start of the trial until Week 40
Mean TIS From Baseline (Week 0) to End of First Period (Week 16) and From Baseline (Week 0) to End of Extension Period (Week 40)
The TIS (Total Improvement Score) is a scale from 0 to 100 that allows for the discrimination between minimal, moderate and major responders depending on their improvement in the combined 6 CSM: ≥20 to 39 points being minimal improvement, ≥40 to 59 points being moderate improvement, and ≥60 points being major improvement.
Measured: Up to 40 weeks
Time to Minimal, Moderate and Major Improvement in TIS
When interpreting these time to event evaluations, one has to keep in mind that eventually all patients were treated with octagam 10%, so that differences in time to response for the Overall Period reflect this delayed start of treatment rather than the true difference between treatment with octagam 10% and placebo.
Measured: Up to 40 weeks
Time to Confirmed Deterioration in the First Period and Overall
Confirmed deterioration is defined as disease worsening on 2 consecutive visits. Worsening criteria are defined as either Physician's Global Disease Activity worsening ≥2cm and Manual Muscle Testing worsening ≥20% or Extra-muscular Activity worsening ≥2cm or any 3 of the following scores worsening by ≥30%: Physician's Global Disease Activity, Manual Muscle Test, Extra-muscular Activity, Patient's Global Disease Activity or Health Assessment Questionnaire.
Measured: Up to 40 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2016 (estimated)
- Study start27 Feb 2017
- Primary completion5 Nov 2019
- Study completion5 Nov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for DermatomyositisStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 95 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wolfgang Frenzel, Study director, International Medical Director
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02728752
- Sponsor's protocol ID
- GAM10-08
- Acronym
- IIM
- Status last verified
- Nov 2021
- First submitted
- 11 Mar 2016
- Last updated
- 23 Dec 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
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.
PubMed 40616720Charles-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.
PubMed 39918968Aggarwal 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.
PubMed 38233885Werth 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.
PubMed 37799613Aggarwal 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.
PubMed 36198179Aggarwal 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.
PubMed 33429735
Study at a glance
From the registry record, last updated by the sponsor on 23 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM Study")
- ClinicalTrials.gov ID
- NCT02728752
- Acronym / sponsor ID
- IIM / GAM10-08
- Conditions
- Dermatomyositis
- Keywords
- DM
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 95 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 27 Feb 2017 (actual)
- Primary completion
- 5 Nov 2019 (actual)
- Study completion
- 5 Nov 2019 (actual)
- First posted
- 5 Apr 2016 (estimated)
- Results
- Posted 21 Apr 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Prospective, Double-blind, Randomized, Placebo-Controlled Phase III Study Evaluating Efficacy and Safety of Octagam 10% in Patients With Dermatomyositis ("ProDERM study")
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Other: PlaceboSubjects 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. |
| Octagam10% | Experimental | Drug: Octagam 10%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. |
Who may qualify
- Ages
- 18 to 79 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (25 listed)
- 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 overlap myositis (except for overlap with Sjögren's syndrome), connective tissue disease associated DM, inclusion body myositis, polymyositis, juvenile dermatomyositis or drug-induced myopathy.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Octagam 10%Patients to be treated with Octagam 10% | Drug | Octagam10% |
| PlaceboPatients to be treated with a Placebo | Other | Placebo |
Primary outcome
Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS)
Time frame: At week 16
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
19 secondary outcomes
Proportion of TIS Responders by Improvement Category at Week 16
Time frame: 16 weeks
Proportion of TIS Responders by Improvement Category at Week 40
Time frame: 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)
Time frame: 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)
Time frame: 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)
Time frame: 40 weeks
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: SF-36v2 Health Survey
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Physician's Global Disease Activity
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Patient Global Disease Activity
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: MMT-8
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Health Assessment Questionnaire
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Alanine Aminotransferase)
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aspartate Aminotransferase)
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Lactate Dehydrogenase)
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Aldolase)
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in Enzymes (Creatine Kinase)
Time frame: From start of the trial till Week 40
Mean Change From Baseline (Week 0) to End of First Period (Week 16) and Extension Period (Week 40) in: Extra-muscular Activity
Time frame: From start of the trial until Week 40
Mean TIS From Baseline (Week 0) to End of First Period (Week 16) and From Baseline (Week 0) to End of Extension Period (Week 40)
Time frame: Up to 40 weeks
Time to Minimal, Moderate and Major Improvement in TIS
Time frame: Up to 40 weeks
Time to Confirmed Deterioration in the First Period and Overall
Time frame: Up to 40 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Dec 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 40616720Aggarwal 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.
- Derived from this study: PubMed 39918968Charles-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.
- Derived from this study: PubMed 38233885Aggarwal 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.
- Derived from this study: PubMed 37799613Werth 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.
- Derived from this study: PubMed 36198179Aggarwal 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.
- Derived from this study: PubMed 33429735Aggarwal 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Wolfgang Frenzel study director
International Medical Director
37 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Octagam 10% in people with Dermatomyositis, run by Octapharma at 37 sites in 10 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 25 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 37 sites in United States, Russia, Ukraine, Hungary, Germany and 5 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 Apr 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measure the Number of Patients Who Had an Increase of ≥20 Points on the Total Improvement Score (TIS) (time frame: At week 16). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02728752, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Dec 2021.