A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 in Adults With Dermatomyositis (DM)

Phase 3

Active, not recruiting

• Conditions: Dermatomyositis
• Interventions: Drug: Placebo; Drug: human immunoglobulin G

• Registration Number: NCT04044690
• Lead Sponsor: CSL Behring

Brief Summary

This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of IgPro20 (subcutaneous Ig) treatment in adult subjects with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of IgPro20 subcutaneous (SC) doses in comparison to placebo in adult subjects with DM, as measured by responder status based on Total Improvement Score (TIS) assessments.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2019-08-01
• Study First Submitted QC: 2019-08-01
• Study First Posted: 2019-08-05
• Study Start: 2019-10-21
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-03
• Last Update Posted: 2024-07-05
• Primary Completion: 2028-05-22
• Study Completion: 2028-05-22

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 126

Inclusion Criteria

• Male or female subjects ≥ 18 years of age
• Diagnosis of at least probable idiopathic inflammatory myopathies (IIM) per European League Against Rheumatism/American College of Rheumatology (EULAR/ACR) Classification Criteria which includes confirmation of dermatomyositis (DM) rash/manifestation, disease activity defined by presence of DM rash / manifestation or an objective disease activity measure
• Disease severity defined by Physician global activity visual analog scale (VAS) with a minimum value of 2.0 cm on a 10 cm scale and MMT-8 ≤ 142 or CDASI total activity score ≥ 14.
• Corticosteroid daily dose less than that or equal to 20 mg prednisolone equivalent

Read More

Exclusion Criteria

• Cancer-associated myositis
• Evidence of active malignant disease or malignancies diagnosed within the previous 5 years
• Physician Global Damage score ≥ 3, or clinically relevant improvement between Screening Visit and Baseline

Read More

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	human albumin solution administered subcutaneously
IgPro20	human immunoglobulin G	human immunoglobulin G administered subcutaneously

Primary Outcome Measures

Name	Time	Method
Responder Rate	Weeks 17, 21, and 25	A responder is defined as a subject with a total improvement score (TIS) ≥ 20 points at Week 25 and at least 1 of the previous scheduled visits (Week 17 or Week 21), who completes 24 weeks of randomized investigational medicinal product (IMP) treatment without the use of rescue corticosteroid treatment. The TIS is a sum response criterion which incorporates 6 weighted IMACS core set measures (CSMs). Thresholds for minimal, moderate, and major improvement were ≥ 20, ≥ 40, and ≥ 60 points on the TIS.

Secondary Outcome Measures

Name	Time	Method
Mean Total Improvement Score (TIS)	Up to Week 25	The TIS is a sum response criterion which incorporates 6 weighted IMACS core set measures (CSMs). A total improvement score (range 0 to 100), determined by summing scores for each CSM, was based on improvement in and relative weight of each CSM. Thresholds for minimal, moderate, and major improvement were ≥ 20, ≥ 40, and ≥ 60 points on the TIS.
Mean change difference (IgPro20 minus placebo) in MMT-8	Up to week 25	MMT-8 is a set of 8 designated muscles which will be tested bilaterally (potential score 0 to 150): 7 biaxial muscles with potential score 0 to140 and 1 axial (neck flexors) with potential score 0 to10. Improvement is documented with an increase in score.
Mean change difference (IgPro20 minus placebo) in CDASI	Up to week 25	The CDASI in its modified version (v2) is a validated tool of skin disease activity (3 items) and damage (3 items) assessment. Scores range from 0-100 for activity and from 0-32 for damage. Improvement is documented with a decrease in score.
Time to first achieving TIS ≥ 20, ≥ 40, and ≥ 60 points on the TIS	Week 5 up to Week 53
Percentage of subjects achieving TIS ≥ 20 points at the end of study period 2	Up to week 53
Percentage of subjects meeting DOW at least once, twice, or > twice	Baseline up to Week 53
Number of subjects meeting DOW and receiving rescue steroid treatment	Baseline up to Week 53
Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%	Up to Week 25
Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%	Up to Week 25
Number of subjects who start oral corticosteroid dose taper	Baseline up to Week 53
Mean difference (IgPro20 minus placebo) in TIS	Up to week 25	The TIS is a sum response criterion which incorporates 6 weighted IMACS core set measures (CSMs). A total improvement score (range 0 to 100), determined by summing scores for each CSM, was based on improvement in and relative weight of each CSM. Thresholds for minimal, moderate, and major improvement were ≥ 20, ≥ 40, and ≥ 60 points on the TIS.
Mean changes from Baseline in Manual Muscle Testing (MMT-8)	Up to week 25	MMT-8 is a set of 8 designated muscles which will be tested bilaterally (potential score 0 to 150): 7 biaxial muscles with potential score 0 to140 and 1 axial (neck flexors) with potential score 0 to10. Improvement is documented with an increase in score.
Mean changes from Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) total activity score	Up to week 25	The CDASI in its modified version (v2) is a validated tool of skin disease activity (3 items) and damage (3 items) assessment. Scores range from 0-100 for activity and from 0-32 for damage. Improvement is documented with a decrease in score.
Mean TIS	Week 5 up to Week 53	The TIS is a sum response criterion which incorporates 6 weighted IMACS core set measures (CSMs). A total improvement score (range 0 to 100), determined by summing scores for each CSM, was based on improvement in and relative weight of each CSM. Thresholds for minimal, moderate, and major improvement were ≥ 20, ≥ 40, and ≥ 60 points on the TIS.
Percentage of subjects achieving TIS ≥ 20, ≥ 40, and ≥ 60 points	Week 5 up to Week 53
Number of subjects meeting Definition of Worsening (DOW) at least once, twice, or > twice	Baseline up to Week 53	The DOW consists of meeting 1 of the following criteria on 2 consecutive visits at least 2 weeks apart: * Physician Global Activity Assessment Visual Analog Scale (VAS) worsening ≥ 2 cm\* and Manual Muscle Test (MMT)-8 worsening ≥ absolute 10%, or; * Extramuscular Global Assessment worsening ≥ 2 cm on the Myositis Disease Activity Assessment Tool (MDAAT) VAS, or; * Any 3 of 6 CSM worsening by ≥ absolute 20%
Time to meeting DOW for the first time	Baseline up to Week 53
Number of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EuroQoL 5-Dimension Questionnaire (EQ-5D-5L)	Baseline up to Week 53	The subject will select which best describes his own health state at the study visit in the following dimensions: Mobility, Self-care, Usual Activities. The subject will also select a point on a scale drawn like a thermometer to indicate how good or bad the health state is, with best state as "100" and worst state as "0."
Mean changes from baseline in individual CSMs (except muscle enzymes) and CDASI	Between Week 5 and Week 25
Mean changes in individual CSMs (except muscle enzymes) and CDASI	From week 29 to week 53
Percentage of subjects meeting DOW and receiving rescue steroid treatment	Baseline up to Week 53
Percentage of subjects receiving rescue steroid treatment	Baseline up to Week 25
Percentage of subjects whose rescue steroid treatment is tapered	Baseline up to Week 25
Percentage of subjects with Treatment Emergent Adverse Events (TEAEs)	Up to 8 years
Percentage of subjects with related TEAEs	Up to 8 years
Rate of TEAEs per days with infusion, by severity	Up to 8 years
Rate of related TEAEs per days with infusion	Up to 8 years
Rate of serious TEAEs per days with infusion	Up to 8 years
The odds ratio (IgPro20:Placebo) of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%	Up to Week 25
Percentage of subjects who start oral corticosteroid dose taper	Baseline up to Week 53
Number of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%	Baseline up to Week 53
Percentage of subjects who are able to reduce the oral corticosteroid dose by ≥ 25%, ≥ 50%, ≥ 75%	Baseline up to Week 25
Percentage of subjects having at least 1 level, 2 levels, and more than 2 levels of improvement from Baseline in mobility, self-care, and usual activities domains of EQ-5D-5L	Baseline up to Week 53
Rate of TEAEs per days with infusion	Up to 8 years
Number of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L from Week 25	From Week 25 up to week 53
Percentage of subjects having no reduction in levels, at least 1 level, 2 levels, and more than 2 levels of improvement in mobility, self-care, and usual activities domains of EQ-5D-5L from Week 25	From Week 25 up to week 53
Percentage of subjects with serious TEAEs	Up to 8 years
Time to first intake of rescue corticosteroid treatment	Baseline up to Week 25

Trial Locations

Locations (76)

8401210 - University of Pennsylvania; 🇺🇸 Philadelphia, Pennsylvania, United States

3920125 - Tokyo Medical And Dental University Medical Hospital; 🇯🇵 Bunkyo, Tokyo, Japan

4840081 - Centro Integral en Reumatologia, SA de CV; 🇲🇽 Guadalajara, Jalisco, Mexico

3920091 - Nippon Medical School Hospital; 🇯🇵 Bunkyō-Ku, Tokyo, Japan

7560033 - University Hospital Bern Inselspital; 🇨🇭 Bern, Switzerland

8401151 - Biomedical Science Tower; 🇺🇸 Pittsburgh, Pennsylvania, United States

0560049 - CHU de Liège - Sart Tilman; 🇧🇪 Liège, Belgium

8401160 - Center For Rheumatology; 🇺🇸 Fort Lauderdale, Florida, United States

0560056 - Universitair Ziekenhuis Leuven; 🇧🇪 Leuven, Belgium

2760203 - Charité; 🇩🇪 Berlin, Germany

2760212 - University Hospital Of Tuebingen; 🇩🇪 Tuebingen, Germany

8401473 - RecioMed Clinical Research Network, Inc.; 🇺🇸 Boynton Beach, Florida, United States

0320083 - Hospital Italiano de Buenos Aires; 🇦🇷 Ciudad Autónoma Buenos Aires, Buenos Aires, Argentina

2500146 - CHU De Dijon Hopital Du Bocage; 🇫🇷 Dijon, France

2760210 - University of Münster; 🇩🇪 Münster, Germany

0560048 - Universitair Ziekenhuis (UZ) Leuven; 🇧🇪 Leuven, Belgium

8401487 - Ohio State University; 🇺🇸 Columbus, Ohio, United States

0320077 - Centro Medico Privado de Reumatolgia; 🇦🇷 Tucumán, Argentina

2500135 - CHU Nice-Hopital Archet I; 🇫🇷 Nice, France

0560050 - Ghent Universit Hospital (UZ Gent); 🇧🇪 Gent, Belgium

8401476 - DS Research; 🇺🇸 Louisville, Kentucky, United States

2500132 - Hopital Pitie-Salpetriere; 🇫🇷 Paris, France

0320081 - Fundacion Respirar; 🇦🇷 Buenos Aires, Argentina

8401129 - UCLA - Rheumatology Los Angeles; 🇺🇸 Los Angeles, California, United States

8401115 - The University of Texas Medical School at Houston; 🇺🇸 Houston, Texas, United States

8401117 - Arizona Arthritis & Rheumatology Research; 🇺🇸 Glendale, Arizona, United States

2500188 - Centre Hospitalier Regional Universitaire de Lille; 🇫🇷 Lille Cedex, France

8401474 - West Tennessee Research Institute, LLC; 🇺🇸 Jackson, Tennessee, United States

2500144 - Hopitaux Universitaire de Strasbourg; 🇫🇷 Strasbourg, France

3800133 - Universita degli Studi Di Brescia - Azienda Ospedaliera Spedali Civili di Brescia; 🇮🇹 Brescia, Italy

3920096 - Chukyo Hospital; 🇯🇵 Nagoya, Aichi, Japan

4840084 - Centro de Alta Especialidad en Reumatologia e Investigacion del Potosí, S.C.; 🇲🇽 San Luis Potosí, Mexico

7560028 - Kantonsspital St. Gallen; 🇨🇭 Saint Gallen, Switzerland

8401199 - Neuromuscular Research Center; 🇺🇸 Phoenix, Arizona, United States

8401486 - Oregon Health and Science University; 🇺🇸 Portland, Oregon, United States

3920035 - Yamaguchi University Hospital; 🇯🇵 Yamaguchi, Japan

3920090 - University Of Fukui Hospital; 🇯🇵 Yoshida-Gun, Fukui, Japan

0320084 - DIM Clinica Privada; 🇦🇷 Buenos Aires, Argentina

2500133 - CHU - Hospital de la Timone; 🇫🇷 Marseille, France

2760036 - University Medicine Göttingen; 🇩🇪 Göttingen, Germany

2760271 - Universitatsklinikum Carl Gustav Carus TU Dresden; 🇩🇪 Dresden, Germany

2760273 - Hautklinik des Uni-Klinikums Erlangen; 🇩🇪 Erlangen, Germany

2760201 - Medizinische Hochschule Hannover (MHH); 🇩🇪 Hannover, Germany

2760199 - University Hospital Köln; 🇩🇪 Köln, Germany

2760268 - University of Ulm; 🇩🇪 Ulm, Germany

3480048 - University of Debrecen; 🇭🇺 Debrecen, Hungary

3800132 - Universitaria Vittorio Emanuele; 🇮🇹 Catania, Italy

3800139 - Universita degli Studi Firenze; 🇮🇹 Firenze, Italy

3800134 - Azienda Ospedaliero Universitaria Pisana; 🇮🇹 Pisa, Italy

3920088 - Gunma University Hospital; 🇯🇵 Maebashi City, Gunma, Japan

3920086 - St. Marianna University Hospital; 🇯🇵 Kawasaki, Kanagawa, Japan

3920087 - Wakayama Medical University Hospital; 🇯🇵 Wakayama, Japan

3920097 - Tokyo Women's Medical University Hospital; 🇯🇵 Shinjuku-Ku, Tokyo, Japan

6160104 - Zespol Poradni Specjalistycznych REUMED, ONYKSOWA Filia nr 2; 🇵🇱 Lublin, Poland

6430122 - City Clinical Hospital No. 5; 🇷🇺 Nizhniy Novgorod, Russian Federation

6430125 - Medical Centre-Healthy Family; 🇷🇺 Novosibirsk, Russian Federation

6160117 - MedicalConcierge Centrum Medyczne; 🇵🇱 Warszawa, Poland

6430124 - ORIS Firm Limited Liability Company; 🇷🇺 Moscow, Russian Federation

6430120 - St. Petersburg City Rheumatological Hospital 25; 🇷🇺 Saint Petersburg, Russian Federation

7240086 - Complejo Hospitalario Universitario A Coruña; 🇪🇸 A Coruña, Spain

7240112 - Hospital Clinic Barcelona; 🇪🇸 Barcelona, Spain

7240011 - Hospital Universitario Valle de Hebron; 🇪🇸 Barcelona, Spain

8040057 - Khmelnitskiy Regional Hospital; 🇺🇦 Khmelnytskyi, Ukraine

8040053 - Cherkassy Regional Hospital; 🇺🇦 Cherkasy, Ukraine

8040058 - State Institution National Scientific Center Strazhesko; 🇺🇦 Kiev, Ukraine

8040055 - Mechnikov Institute of Microbiology and Immunology; 🇺🇦 Kharkiv, Ukraine

6430123 - Yaroslavl Oblast Clinical Hospital; 🇷🇺 Yaroslavl, Russian Federation

8040051 - Kyiv Railway Clinical Hospital No.2; 🇺🇦 Kyiv, Ukraine

8040054 - Modern Clinic LLC; 🇺🇦 Zaporizhia, Ukraine

3920089 - Hokkaido University Hospital; 🇯🇵 Sapporo, Hokkaido, Japan

8401107 - Morsani Center for Advanced Health Care (CAHC); 🇺🇸 Tampa, Florida, United States

2760211 - Charité - Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany

4840082 - CINTRE, Centro de Investigacion y Tratamiento Reumatologico S.C.; 🇲🇽 Ciudad de México, Distrito Federal, Mexico

8040052 - Institute of Rheumatology; 🇺🇦 Kyiv, Ukraine

8401132 - Omega Research Maitland; 🇺🇸 Orlando, Florida, United States

8401152 - The University of Kansas Medical Center; 🇺🇸 Fairway, Kansas, United States