Clinical Trials Register

Summary
EudraCT Number:	2016-004082-39
Sponsor's Protocol Code Number:	GOOD-IDES-01
National Competent Authority:	Sweden - MPA
Clinical Trial Type:	EEA CTA
Trial Status:	Completed
Date on which this record was first entered in the EudraCT database:	2016-11-07
Trial results	View results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Sweden - MPA

A.2

EudraCT number

2016-004082-39

A.3

Full title of the trial

An Open-Label Phase II Study in anti-GBM disease (Goodpasture’s disease) with Adverse Renal Prognosis to Evaluate the Efficacy and Safety of IdeS --GOOD-IDES

En öppen fas II-studie för att utvärdera effekt och säkerhet av IdeS --GOOD-IDES vid anti-GBM sjukdom (Goodpastures sjukdom) med dålig prognos för bibehållen njurfunktion

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

An Phase II study of IdeS in anti-GBM disease (Goodpasture’s disease) with severe renal failure--GOOD-IDES

En fas II studie av IdeS vid Goodpastures sjukdom med svår njursvikt – GOOD-IDES

A.3.2

Name or abbreviated title of the trial where available

GOOD-IDES

A.4.1

Sponsor's protocol code number

GOOD-IDES-01

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	Linköping University
B.1.3.4	Country	Sweden
B.3.1 and B.3.2	Status of the sponsor	Non-Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	Linköping University
B.4.2	Country	Sweden
B.4.1	Name of organisation providing support	Hansa Biopharma AB
B.4.2	Country	Sweden
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	Linköping University
B.5.2	Functional name of contact point	Mårten Segelmark
B.5.3	Address:
B.5.3.1	Street Address	University Hospital
B.5.3.2	Town/ city	Linköping
B.5.3.3	Post code	581 83
B.5.3.4	Country	Sweden
B.5.4	Telephone number	+4610103 2297
B.5.5	Fax number	+4610103 4514
B.5.6	E-mail	marten.segelmark@liu.se

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	HMED-IdeS
D.3.2	Product code	HMED-IdeS
D.3.4	Pharmaceutical form	Powder for concentrate for solution for infusion
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Intravenous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	Imlifidase
D.3.9.1	CAS number	1947415-68-0
D.3.9.3	Other descriptive name	Imlifidase
D.3.9.4	EV Substance Code	SUB191871
D.3.10	Strength
D.3.10.1	Concentration unit	mg/ml milligram(s)/millilitre
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	No
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	Yes
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	Yes
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

Anti-GBM disease (Goodpasture’s disease) with Adverse Renal Prognosis

E.1.1.1

Medical condition in easily understood language

A pathogenic process is driven by autoantibodies (IgG) directed against part of the blood vessel wall in the kidneys (the glomerular basement membrane).

E.1.1.2

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10063039
E.1.2	Term	Anti-GBM antibody
E.1.2	System Organ Class	100000004848

E.1.3

Condition being studied is a rare disease

Yes

E.2 Objective of the trial

E.2.1

Main objective of the trial

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.

E.2.2

Secondary objectives of the trial

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)

E.2.3

Trial contains a sub-study

E.3

Principal 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 (by MDRD equation) or if the patient is non-responsive to standard treatment, and has lost >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.

E.4

Principal 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.

E.5 End points

E.5.1

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.

E.5.1.1

Timepoint(s) of evaluation of this end point

6 months after IdeS treatment

E.5.2

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)

E.5.2.1

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)

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

Yes

E.7.3

Therapeutic confirmatory (Phase III)

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

E.8.1.1

Randomised

E.8.1.2

Open

Yes

E.8.1.3

Single blind

E.8.1.4

Double blind

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

E.8.2.3

Other

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.7

Trial has a data monitoring committee

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

Last visit last subject

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

F.4.2.2

In the whole clinical trial

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

Follow up according to clinical standard treatment for anti-GBM nephritis with adverse renal prognosis

G. Investigator Networks to be involved in the Trial

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2016-12-29

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2017-01-23

P. End of Trial
P.	End of Trial Status	Completed
P.	Date of the global end of the trial	2020-07-24

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