Prove cliniche Nct

Summary
EudraCT Number:2021-003063-10
Sponsor's Protocol Code Number:ARGX-113-2010
National Competent Authority:Greece - EOF
Clinical Trial Type:EEA CTA
Trial Status:Ongoing
Date on which this record was first entered in the EudraCT database:2022-12-20
Trial 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
A. Protocol Information
A.1Member State ConcernedGreece - EOF
A.2EudraCT number2021-003063-10
A.3Full title of the trial
An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
A phase 3 study to evaluate the long-term safety, tolerability and efficacy of efgartigimod PH20 SC in adult participants with bullous pemphigoid
A.4.1Sponsor's protocol code numberARGX-113-2010
A.7Trial is part of a Paediatric Investigation Plan No
A.8EMA Decision number of Paediatric Investigation Plan
B. Sponsor Information
B.Sponsor: 1
B.1.1Name of Sponsorargenx BV
B.1.3.4CountryBelgium
B.3.1 and B.3.2Status of the sponsorCommercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1Name of organisation providing supportargenx BV
B.4.2CountryBelgium
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1Name of organisationargenx BV
B.5.2Functional name of contact pointRegulatory
B.5.3 Address:
B.5.3.1Street AddressIndustriepark Zwijnaarde 7
B.5.3.2Town/ cityZwijnaarde (Ghent)
B.5.3.3Post codeB-9052
B.5.3.4CountryBelgium
B.5.4Telephone number00329310 3400
B.5.6E-mailregulatory@argenx.com
D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3IMP RoleTest
D.2 Status of the IMP to be used in the clinical trial
D.2.1IMP to be used in the trial has a marketing authorisation No
D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
D.2.5.1Orphan drug designation number
D.3 Description of the IMP
D.3.1Product nameEfgartigimod PH20 SC
D.3.4Pharmaceutical form Solution for injection
D.3.4.1Specific paediatric formulation No
D.3.7Routes of administration for this IMPSubcutaneous use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8INN - Proposed INNEfgartigimod Alfa
D.3.9.1CAS number 1821402-21-4
D.3.9.2Current sponsor codeARGX-113
D.3.9.3Other descriptive nameEfgartigimod alfa
D.3.9.4EV Substance CodeSUB198780
D.3.10 Strength
D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
D.3.10.2Concentration typeequal
D.3.10.3Concentration number180
D.3.11 The IMP contains an:
D.3.11.1Active substance of chemical origin No
D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
The IMP is a:
D.3.11.3Advanced Therapy IMP (ATIMP) No
D.3.11.3.1Somatic cell therapy medicinal product No
D.3.11.3.2Gene therapy medical product No
D.3.11.3.3Tissue Engineered Product No
D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
D.3.11.5Radiopharmaceutical medicinal product No
D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
D.3.11.7Plasma derived medicinal product No
D.3.11.8Extractive medicinal product No
D.3.11.9Recombinant medicinal product Yes
D.3.11.10Medicinal product containing genetically modified organisms No
D.3.11.11Herbal medicinal product No
D.3.11.12Homeopathic medicinal product No
D.3.11.13Another type of medicinal product No
D.8 Information on Placebo
D.8 Placebo: 1
D.8.1Is a Placebo used in this Trial?Yes
D.8.3Pharmaceutical form of the placeboSolution for injection
D.8.4Route of administration of the placeboSubcutaneous use
E. General Information on the Trial
E.1 Medical condition or disease under investigation
E.1.1Medical condition(s) being investigated
Bullous Pemphigoid
E.1.1.1Medical condition in easily understood language
Autoimmune disease that causes painful or itchy blisters on the skin
E.1.1.2Therapeutic area Diseases [C] - Immune System Diseases [C20]
MedDRA Classification
E.1.2 Medical condition or disease under investigation
E.1.2Version 21.1
E.1.2Level LLT
E.1.2Classification code 10006567
E.1.2Term Bullous pemphigoid
E.1.2System Organ Class 10040785 - Skin and subcutaneous tissue disorders
E.1.3Condition being studied is a rare disease Yes
E.2 Objective of the trial
E.2.1Main objective of the trial
To assess the long-term safety and tolerability of treatment with efgartigimod PH20 SC in participants with BP
E.2.2Secondary objectives of the trial
To assess the long-term efficacy and durability of response with efgartigimod PH20 SC treatment in participants with BP
To evaluate the impact of efgartigimod PH20 SC treatment in reducing long-term glucocorticoid-associated toxicity in participants with BP
To evaluate the impact of efgartigimod PH20 SC treatment on QoL in participants with BP
To evaluate the PD and immunogenicity of efgartigimod PH20 SC in participants with BP
E.2.3Trial contains a sub-study Yes
E.2.3.1Full title, date and version of each sub-study and their related objectives
Substudy – Vaccination Response
Sites may collect additional blood samples for additional/optional/future vaccination research. Such research may include (but not limited to) the following:
1. Humoral (serum) responses to vaccinations received during the study
2. Cellular (peripheral blood mononuclear cells [PBMCs]) responses to vaccinations received during the study
Postvaccination samples will be collected at the visits identified in the Summary of Activities only from participants who took part in the vaccination substudy during ARGX-113-2009.
Data obtained from this substudy will not be included in the clinical database; the results may be described in a separate report.
E.3Principal inclusion criteria
1. Has completed the week 36 visit of ARGX-113-2009
2. Is capable of providing signed informed consent and complying with protocol requirements
3. Agrees to use contraceptive measures consistent with local regulations and the following:
a. Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP.
b. Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP
E.4Principal exclusion criteria
1. Clinically significant disease, recent major surgery (within 3 months of baseline), or intention to have surgery during the study; or any other medical condition that, in the investigator’s opinion would confound the results of the study or put the participant at undue risk
2. Known hypersensitivity to IMP or 1 of its excipients
3. Permanently discontinued IMP in ARGX-113-2009 due to an AE
considered related to IMP and for whom the benefit/risk balance is not
considered positive
E.5 End points
E.5.1Primary end point(s)
Incidence and severity of treatment-emergent adverse events (TEAEs), serious AEs (SAEs), and AEs of special interest (AESIs)
Rate of treatment discontinuation because of safety concerns
E.5.1.1Timepoint(s) of evaluation of this end point
Up to wk 56
E.5.2Secondary end point(s)
1. Proportions of participants achieving:
- complete remission (CR) while off oral corticosteroids (OCS) for ≥8 weeks
- CR or partial remission (PR) while off OCS for ≥8 weeks
- CR while on minimal OCS therapy for ≥8 weeks. (Minimal OCS therapy is defined as ≤0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS])
- CR while off both OCS and efgartigimod PH20 SC for ≥8 weeks
- CR or PR while off both OCS and efgartigimod PH20 SC for ≥8 weeks
2. Duration of sustained remission
3. Proportion of participants who relapse
4. Time to relapse
5. Incidence and severity of relapse
6. Bullous Pemphigoid Disease Area Index (BPDAI) activity scores, Investigator Global Assessment of Bullous Pemphigoid (IGA-BP) scores, and itch numerical rating scale (NRS) over time
7. Rate of treatment failure
8. GTI-related scores, including the GTI-AIS, GTI CWS, and GTI SL over time
9. EQ-5D-5L, DLQI, and ABQoL scores over time
10. Percent change from baseline over time for anti BP180 and anti BP230 antibody levels
11. Incidence and prevalence of ADA against efgartigimod (serum levels)
E.5.2.1Timepoint(s) of evaluation of this end point
1-5 & 7: Up to wk 56
6: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after & at wks 48, 52 & 56
8 & 9: At wks 0, 24 & 48 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 8, every 16 wks up to & after EFG stop, then at wk 48
10: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 2, 4, 8, then every 4 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56
11: At wks 0, 2, 4, 8, 16, 24, 32, 40, 48 & 56 when EFG is NA. When EFG continues/starts its evaluated at wks 0, 4, 8, then every 8 wks to EFG stop, every 8 wks after, then at wks 48, 52 & 56
E.6 and E.7 Scope of the trial
E.6Scope of the trial
E.6.1Diagnosis No
E.6.2Prophylaxis No
E.6.3Therapy No
E.6.4Safety Yes
E.6.5Efficacy Yes
E.6.6Pharmacokinetic No
E.6.7Pharmacodynamic No
E.6.8Bioequivalence No
E.6.9Dose response No
E.6.10Pharmacogenetic No
E.6.11Pharmacogenomic No
E.6.12Pharmacoeconomic No
E.6.13Others Yes
E.6.13.1Other scope of the trial description
Tolerability
E.7Trial type and phase
E.7.1Human pharmacology (Phase I) No
E.7.1.1First administration to humans No
E.7.1.2Bioequivalence study No
E.7.1.3Other No
E.7.1.3.1Other trial type description
E.7.2Therapeutic exploratory (Phase II) No
E.7.3Therapeutic confirmatory (Phase III) Yes
E.7.4Therapeutic use (Phase IV) No
E.8 Design of the trial
E.8.1Controlled No
E.8.1.1Randomised No
E.8.1.2Open Yes
E.8.1.3Single blind No
E.8.1.4Double blind No
E.8.1.5Parallel group No
E.8.1.6Cross over No
E.8.1.7Other No
E.8.2 Comparator of controlled trial
E.8.2.1Other medicinal product(s) No
E.8.2.2Placebo No
E.8.2.3Other No
E.8.2.4Number of treatment arms in the trial1
E.8.3 The trial involves single site in the Member State concerned No
E.8.4 The trial involves multiple sites in the Member State concerned Yes
E.8.4.1Number of sites anticipated in Member State concerned6
E.8.5The trial involves multiple Member States Yes
E.8.5.1Number of sites anticipated in the EEA70
E.8.6 Trial involving sites outside the EEA
E.8.6.1Trial being conducted both within and outside the EEA Yes
E.8.6.2Trial being conducted completely outside of the EEA No
E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
Australia
Canada
China
Israel
Japan
Russian Federation
Serbia
United Kingdom
United States
Bulgaria
Croatia
Czechia
France
Germany
Greece
Hungary
Italy
Latvia
Netherlands
Poland
Romania
Slovakia
Spain
E.8.7Trial has a data monitoring committee No
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
LVLS
E.8.9 Initial estimate of the duration of the trial
E.8.9.1In the Member State concerned years2
E.8.9.1In the Member State concerned months7
E.8.9.1In the Member State concerned days15
E.8.9.2In all countries concerned by the trial years2
E.8.9.2In all countries concerned by the trial months7
E.8.9.2In all countries concerned by the trial days15
F. Population of Trial Subjects
F.1 Age Range
F.1.1Trial has subjects under 18 No
F.1.1.1In Utero No
F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
F.1.1.3Newborns (0-27 days) No
F.1.1.4Infants and toddlers (28 days-23 months) No
F.1.1.5Children (2-11years) No
F.1.1.6Adolescents (12-17 years) No
F.1.2Adults (18-64 years) Yes
F.1.2.1Number of subjects for this age range: 40
F.1.3Elderly (>=65 years) Yes
F.1.3.1Number of subjects for this age range: 120
F.2 Gender
F.2.1Female Yes
F.2.2Male Yes
F.3 Group of trial subjects
F.3.1Healthy volunteers No
F.3.2Patients Yes
F.3.3Specific vulnerable populations Yes
F.3.3.1Women of childbearing potential not using contraception No
F.3.3.2Women of child-bearing potential using contraception Yes
F.3.3.3Pregnant women No
F.3.3.4Nursing women No
F.3.3.5Emergency situation No
F.3.3.6Subjects incapable of giving consent personally No
F.3.3.7Others No
F.4 Planned number of subjects to be included
F.4.1In the member state12
F.4.2 For a multinational trial
F.4.2.1In the EEA 97
F.4.2.2In the whole clinical trial 160
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)
Participants will receive standard of care as directed by their doctor
G. Investigator Networks to be involved in the Trial
N. Review by the Competent Authority or Ethics Committee in the country concerned
N.Competent Authority Decision Authorised
N.Date of Competent Authority Decision2023-02-01
N.Ethics Committee Opinion of the trial applicationFavourable
N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
N.Date of Ethics Committee Opinion2023-01-20
P. End of Trial
P.End of Trial StatusOngoing

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