Anti-GBM disease (Goodpasture’s disease) with Adverse Renal Prognosis MedDRA version: 20.1 Level: LLT Classification code 10063039 Term: Anti-GBM antibody System Organ Class: 100000004848
Conditions
Interventions
Product Name: HMED-IdeS
Product Code: HMED-IdeS
Pharmaceutical Form: Powder for concentrate for solution for infusion
INN or Proposed INN: Imlifidase
CAS Number: 1947415-68-0
Other descriptive name: I
Sponsors
Linköping University
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Anti-GBM antibodies detected by Enzyme-Linked Immunosorbent Assay (ELISA) above a level that is considered toxic by the investigator. Patients double-positive for anti-GBM and ANCA may be entered in the trial, but only if their level of anti-GBM antibodies fulfil the criteria above. 2. eGFR 15 ml/min/1.73 m2 after start of treatment. 3. Haematuria on dipstick and/or urinary sediment. 4. Male or female patients aged at least 18 years; Female patients of childbearing potential may participate if highly effective contraception is used during the study. 5. Willing and able to give written Informed Consent and to comply with the requirements of the study protocol; and 6. Judged to be otherwise healthy by the Investigator, based on medical history, physical examination, and clinical laboratory assessments. Patients with clinical laboratory values that are outside of normal limits (other than those specified in the Exclusion Criteria) and/or with other abnormal clinical findings that are judged by the Investigator not to be of clinical significance, may be entered into the study. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 8 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 7
Exclusion criteria
Exclusion criteria: 1. Anuria for more than 48 hours (less than 200 ml) 2. Dialysis dependency for more than 5 days (maximum 3 sessions before signing informed consent) 3. Moderate to severe pulmonary haemorrhage as defined in the protocol 4. Pregnant 5. Symptomatic congestive heart failure (NYHA class 2-4) requiring prescription medication or clinically evident peripheral edema of cardiac origin 6. Myocardial infarction, unstable angina or stroke within 3 months prior to screening 7. Ongoing bacterial infection requiring antibiotic therapy or viral infection with Hepatitis B, C or HIV; or active tuberculosis as indicated by chest x-ray. 8. Patients should not have received investigational drugs within 30 days prior to screening or within 4 half-lives (whichever is longer); and 9. History or presence of any medical condition or disease which, in the opinion of the Investigator, may place the subject at unacceptable risk for study participation.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The primary efficacy objective is to evaluate the efficacy of an IdeS based regimen to salvage independent renal function defined as no need for dialysis at 6 months and after IdeS treatment. ;Secondary Objective: The secondary objectives of this study include assessment of: 1. Renal function 3 months 2. Change in eGFR during the study period; 3. Number of days with anti-GBM antibodies above a toxic level (i.e. >30 ELISA units) 4. Disappearance of hematuria, days from start of treatment; 5. Change in proteinuria during the study measured as u-albumin/creatinine ratio in morning void; 6. Number of PLEX needed; 7. Changes in renal histology (optional) 8. Anti-IdeS antibodies (ADA) 9. Pharmacokinetics, Pharmacodynamics (IgG degradation);Primary end point(s): The primary efficacy objective is to evaluate the efficacy of an IdeS based regimen to salvage independent renal function defined as no need for dialysis at 6 months after IdeS treatment. ;Timepoint(s) of evaluation of this end point: 6 months after IdeS treatment | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary objectives of this study include assessment of: 1. Renal function at 3 and 6 months expressed as eGFR; 2. Number of days with anti-GBM antibodies above a toxic level (i.e. >30 ELISA units) 3. Disappearance of hematuria, days from start of treatment; 4. Change in proteinuria during the study measured as u-albumin/creatinine ratio in morning void; 5. Number of PLEX needed; 6. Renal histology taken (optional) if clinically warranted 7. Anti-IdeS antibodies (ADA) 8. Pharmacokinetics, Pharmacodynamics (IgG degradation);Timepoint(s) of evaluation of this end point: Days after IdeS treatment 1, 3, 7, 10, 15, 22, 29, 50, 93, 135, 180 (end of study) | — |
Countries
Austria, Denmark, France, Sweden
Contacts
Public ContactMårten Segelmark
Linköping University
Outcome results
None listed