Clinical trial • Phase II • Immunology | Rare Disease

frexalimab for Focal segmental glomerulosclerosis | Minimal change disease

Phase II trial of frexalimab for Focal segmental glomerulosclerosis | Minimal change disease.

Overview

Trial Therapeutic Area
Immunology | Rare Disease
Trial Disease
Focal segmental glomerulosclerosis | Minimal change disease
Trial Stage
Phase II
Drug Modality
Monoclonal antibody | Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
19-08-2024
First CTIS Authorization Date
09-12-2024

Trial design

Randomised, placebo (matching placebo arms for the investigational product arms). no dose or dosing schedule details are specified in the ctis summary.-controlled Phase II trial in Czechia, France, Germany and others.

Randomised
Yes
Comparator
Placebo (matching placebo arms for the investigational product arms). No dose or dosing schedule details are specified in the CTIS summary.
Target Sample Size
67

Eligibility

Recruits 67 paediatric patients.

Vulnerable Population
The trial includes participants aged 16 to 75 years (minors aged 16–17 are therefore included). isVulnerablePopulationSelected is true; national requirements for inclusion of minors/legal guardian consent apply (Poland decision text flagged that persons under 18 require additional legal/ethical protections and appropriate consent/guardian documentation). Subject information and ICFs are provided in multiple country/language versions.

Inclusion criteria

  • {"criterion_text":"- Biopsy report indicative of primary FSGS or primary MCD, with supportive clinical presentation per Investigator's judgement."}
  • {"criterion_text":"- UPCR ≥3 g/g at screening, or ≥ 1.5 g/g in those with eGFR ≥ 60."}
  • {"criterion_text":"- eGFR ≥45 mL/min/1.73 m^2 at screening."}
  • {"criterion_text":"- Documented history of UPCR (or 24-hour urine protein) reduction by ≥40% in response to corticosteroid or other immunosuppressive therapy when pre-treatment UPCR was ≥3.5 g/g (or pre-treatment 24-hr urine protein was ≥3.5 g/day if 24-hour urine protein is used)."}
  • {"criterion_text":"- ≤10 mg/day prednisone or equivalent and stable starting at least 1 week prior to randomization."}
  • {"criterion_text":"- For those on a RAAS inhibitor prior to screening, the dose must be stable ≥4 weeks prior to screening; starting RAAS inhibitors or changing the dose will not be allowed during the double-blind or OLE treatment period."}
  • {"criterion_text":"- For those on an SGLT2 inhibitor prior to screening, the dose must be stable ≥4 weeks prior to screening; starting SGLT2 inhibitor treatment or changing the dose will not be allowed during the double-blind or OLE treatment periods."}
  • {"criterion_text":"- Body weight within 45 to 120 kg (inclusive) at screening."}

Exclusion criteria

  • {"criterion_text":"- Genetic or secondary FSGS or MCD. Those with APOL1 risk alleles are eligible."}
  • {"criterion_text":"- Collapsing variant of FSGS."}
  • {"criterion_text":"- ESKD requiring dialysis or transplantation."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Percent reduction in urine protein to creatinine ratio (UPCR)","definition_or_measurement_approach":"Percent reduction in urine protein-to-creatinine ratio (UPCR) measured as percent change in UPCR from baseline."}

Secondary endpoints

  • {"endpoint_text":"- Percentage of participants achieving FSGS partial remission endpoint","definition_or_measurement_approach":"Proportion of participants meeting the protocol-defined FSGS partial remission criteria (as recorded in study assessments)."}
  • {"endpoint_text":"- Percentage of participants achieving CR","definition_or_measurement_approach":"Proportion of participants achieving complete remission (CR) per protocol-defined criteria."}
  • {"endpoint_text":"- Incidence of treatment-emergent adverse events, treatment-emergent serious adverse events (SAEs), treatment-emergent adverse events of special interest (AESIs) and IMP discontinuation due to TEAEs during the study","definition_or_measurement_approach":"Incidence rates of TEAEs, SAEs, AESIs and IMP discontinuations collected throughout the study duration."}
  • {"endpoint_text":"- Plasma concentrations of frexalimab and rilzabrutinib and serum concentrations of SAR442970","definition_or_measurement_approach":"Pharmacokinetic measurements: plasma (for frexalimab and rilzabrutinib) and serum (for SAR442970) concentrations at scheduled PK timepoints."}
  • {"endpoint_text":"- Occurrence of anti-drug antibodies (ADAs) against frexalimab and SAR442970","definition_or_measurement_approach":"Assessment of immunogenicity by measuring anti-drug antibodies (ADAs) to frexalimab and SAR442970 at defined timepoints."}

Recruitment

Planned Sample Size
67
Recruitment Window Months
37
Consent Approach
Informed consent documents (L1/I C F) and other subject information materials are provided in multiple language versions (English, French, German, Spanish, Italian, Portuguese, Polish, Hungarian, Dutch, Slovak, Czech, Greek etc., as listed in CTIS documents). The trial population includes participants aged 16–75; participation of minors (16–17) requires legal guardian consent/assent according to national legislation (Poland review specifically noted guardian/legal consent requirements). Specific age-stratified consent/assent forms are not detailed in the CTIS summary but country-specific ICFs and patient-facing materials are provided.

Methods

  • Study brochures, flyers and posters (country-specific materials documented: brochure/flyer/poster in EN, FR, DE, ES, IT, PT, PL, HU, NL, SK, CS, EL, etc.)
  • Dear-colleague / doctor-to-doctor letters to clinicians (documented for several countries)
  • Site-based recruitment via participating nephrology clinics and hospitals (site lists provided per country)
  • Use of third-party recruitment services (e.g., Jumo Health USA Inc. listed with role 'Recruitment Services')
  • Patient-facing information sheets and informed consent materials in multiple languages

Geography

Total Number Of Sites
37
Total Number Of Participants
40

Czechia

Earliest CTIS Part Ii Submission Date
22-11-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
503
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Vseobecna Fakultni Nemocnice V Praze
Department Name
Klinika nefrologie VFN a 1. LF UK
Contact Person Name
Vladimir Tesar
Contact Person Email
vladimir.tesar@vfn.cz
Site Name
University Hospital Olomouc
Department Name
Internal clinic - nephrology, rheumatology and endocrinology
Contact Person Name
Karel Krejci
Contact Person Email
karel.krejci@fnol.cz

France

Earliest CTIS Part Ii Submission Date
08-11-2024
Latest Decision Or Authorization Date
08-04-2026
Processing Time Days
516
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Assistance Publique Hopitaux De Paris (Creteil Cedex)
Department Name
Service de Nephrologie
Contact Person Name
Vincent AUDARD
Contact Person Email
vincent.audard@aphp.fr
Site Name
Assistance Publique Hopitaux De Paris (Paris)
Department Name
Service de Nephrologie
Contact Person Name
Myriam Dao
Contact Person Email
myriam.dao@aphp.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Nephrology
Contact Person Name
Maxime Teisseyre
Contact Person Email
teisseyre.m@chu-nice.fr

Germany

Earliest CTIS Part Ii Submission Date
07-11-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
518
Number Of Sites
4
Number Of Participants
3

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Nephrologie und Intensivmedizin
Contact Person Name
Eva-Vanessa Schrezenmeier
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
III. Medizinische Klinik und Poliklinik
Contact Person Name
Sydney Elisabeth Gies
Contact Person Email
s.gies@uke.de
Site Name
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
Department Name
Medizinische Klinik III
Contact Person Name
Christian Hugo
Contact Person Email
Christian.Hugo@ukdd.de
Site Name
Medizinische Hochschule Hannover
Department Name
Nephrologie
Contact Person Name
Jessica Kaufeld
Contact Person Email
kaufeld.jessica@mh-hannover.de

Greece

Earliest CTIS Part Ii Submission Date
30-08-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
587
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
University General Hospital Of Ioannina
Department Name
Nephrology Clinic
Contact Person Name
Eleni Stamellou
Contact Person Email
e.stamellou@uoi.gr
Site Name
Hippokration Hospital
Department Name
Nephrology Department
Contact Person Name
Petras Dimitrios
Contact Person Email
nefrologiko@hippocratio.gr
Site Name
University General Hospital Of Heraklion
Department Name
Nephrology Clinic
Contact Person Name
Stylianou Konstantinos
Contact Person Email
nephrol@pagni.gr

Hungary

Earliest CTIS Part Ii Submission Date
25-10-2024
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
532
Number Of Sites
3
Number Of Participants
5

Sites

Site Name
Semmelweis University (Bokay Janos Utca 53)
Department Name
Gyermekgyogyaszati Klinika
Contact Person Name
Attila Jozsef Szabo
Contact Person Email
szabo.attila@semmelweis.hu
Site Name
Semmelweis University (Koranyi Sandor Utca 2/a)
Department Name
Belgyógyászati és Onkológiai Klinika Klinikai Farmakológiai Részleg
Contact Person Name
Akos Petho
Contact Person Email
petho.akos@semmelweis.hu
Site Name
University Of Debrecen
Department Name
Klinikai Központ, Belgyógyászati Intézet
Contact Person Name
Reka P.Szabo
Contact Person Email
pszabo@belklinika.com

Italy

Earliest CTIS Part Ii Submission Date
05-11-2024
Latest Decision Or Authorization Date
10-04-2026
Processing Time Days
521
Number Of Sites
4
Number Of Participants
3

Sites

Site Name
Azienda Socio Sanitaria Territoriale Degli Spedali Civili Di Brescia
Department Name
Unità Operativa di Nefrologia
Contact Person Name
Federico Alberici
Contact Person Email
federico.alberici@unibs.it
Site Name
Careggi University Hospital
Department Name
Nephrology Dialysis and Transplant Unit
Contact Person Name
Calogero Lino Cirami
Contact Person Email
ciramil@aou-careggi.toscana.it
Site Name
Fondazione IRCCS San Gerardo Dei Tintori
Department Name
S.C. Nefrologia
Contact Person Name
Federico Pieruzzi
Site Name
Azienda Ospedaliera Universitaria Federico II Di Napoli
Department Name
U.O. di Nefrologia Dipartimento di Sanità Pubblica
Contact Person Name
Antonio Pisani
Contact Person Email
antonio.pisani13@gmail.com

Spain

Earliest CTIS Part Ii Submission Date
07-11-2024
Latest Decision Or Authorization Date
13-04-2026
Processing Time Days
522
Number Of Sites
7
Number Of Participants
5

Sites

Site Name
Hospital Universitario Reina Sofia
Department Name
Nephrology
Contact Person Name
Cristina Rabasco Ruiz
Site Name
Hospital Universitario Y Politecnico La Fe
Department Name
Nephrology
Contact Person Name
Carmen Ramos Tomas
Contact Person Email
ramos-marton@gva.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Nephrology
Contact Person Name
Maria Jose Soler
Contact Person Email
m.soler@vhebron.net
Site Name
Hospital Universitario Fundacion Jimenez Diaz
Department Name
Nephrology
Contact Person Name
Alberto Ortiz Arduán
Contact Person Email
aortiz@fjd.es
Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
Nephrology
Contact Person Name
Miguel Angel Perez Valdivia
Site Name
Fundacio Puigvert
Department Name
Nephrology
Contact Person Name
Helena Marco Rusiñol
Contact Person Email
hmarco@fundacio-puigvert.es
Site Name
Hospital General Universitario Gregorio Maranon
Department Name
Nephorogy Service
Contact Person Name
Marian Goicoechea
Contact Person Email
marian.goicoechea@gmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
07-11-2024
Latest Decision Or Authorization Date
13-04-2026
Processing Time Days
522
Number Of Sites
1
Number Of Participants
4

Sites

Site Name
Stichting Amsterdam UMC
Department Name
Division of Internal Medicine
Contact Person Name
Marc Hilhorst

Poland

Earliest CTIS Part Ii Submission Date
27-11-2024
Latest Decision Or Authorization Date
09-04-2026
Processing Time Days
498
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Uniwersytecki Szpital Kliniczny W Poznaniu
Department Name
Nephrology
Contact Person Name
Krzysztof Pawlaczyk
Contact Person Email
kpawlac@ump.edu.pl
Site Name
Centralny Szpital Kliniczny Uniwersytetu Medycznego W Lodzi
Department Name
Klinika Nefrologii, Hipertensjologii, Transplantologii i Chorób Wewnętrznych
Contact Person Name
Michal Nowicki
Contact Person Email
nefro@wp.pl
Site Name
Uniwersytecki Szpital Kliniczny W Opolu
Department Name
Oddział nefrologii ze stacją dializ
Contact Person Name
Tomasz Porazko
Contact Person Email
tomasz.porazko@usk.opole.pl

Slovakia

Earliest CTIS Part Ii Submission Date
04-11-2024
Latest Decision Or Authorization Date
08-04-2026
Processing Time Days
520
Number Of Sites
4
Number Of Participants
4

Sites

Site Name
Univerzitna nemocnica L. Pasteura Kosice
Department Name
Transplantacne oddelenie
Contact Person Name
Jaroslav ROSENBERGER
Site Name
Univerzitna Nemocnica Martin
Department Name
Transplantacno-nefrologicke oddelenie
Contact Person Name
Ivana Dedinska
Contact Person Email
ivana.dedinska@uniba.sk
Site Name
University Hospital Bratislava
Department Name
I. Interna klinika SZU a UNB
Contact Person Name
Zuzana ZILINSKA
Contact Person Email
zuzana.zilinska@kr.unb.sk
Site Name
F D Roosevelt University General Hospital Of Banska Bystrica
Department Name
Nefrologicka ambulancia
Contact Person Name
Martina KONKOLOVA
Contact Person Email
konkolova1@gmail.com

Portugal

Earliest CTIS Part Ii Submission Date
31-10-2024
Latest Decision Or Authorization Date
08-04-2026
Processing Time Days
524
Number Of Sites
3
Number Of Participants
3

Sites

Site Name
Unidade Local De Saude De Gaia/Espinho E.P.E.
Department Name
nephrology department
Contact Person Name
Ana Marta Gomes
Contact Person Email
ana.gomes@ulsge.min-saude.pt
Site Name
Hospital Pedro Hispano
Department Name
nephrology department
Contact Person Name
Teresa Santos
Site Name
Unidade Local De Saude De Sao Jose E.P.E.
Department Name
Nefrologia
Contact Person Name
Mario Rui Ventura Góis
Contact Person Email
mariovgois@gmail.com

Sponsor

Primary sponsor

Full Name
Sanofi-Aventis Recherche & Developpement
Organisation Type
Pharmaceutical company
Country Of Registered Address
France

Contract research organisations

Name
IQVIA Limited
Responsibilities
Exit Interviews
Name
Icon Clinical Research Limited
Responsibilities
Home Nursing
Name
Endpoint Clinical Inc.
Name
Labcorp Central Laboratory Services LP

Third parties

  • {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Exit Interviews","organisation_type":"Pharmaceutical company"}
  • {"country":"Spain","full_name":"Alcura Health Espana S.A.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Jumo Health USA Inc.","duties_or_roles":"Recruitment Services","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Nephropathology Associates PLC","duties_or_roles":"Central Biopsy Reading","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Endpoint Clinical Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"ESMS Global Limited","duties_or_roles":"24 Hour Emergency Service","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Icon Clinical Research Limited","duties_or_roles":"Home Nursing","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medpoint Communications Inc.","duties_or_roles":"Study Training","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Greenphire LLC","duties_or_roles":"Patient Reimbursement and Stipend","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Portugal","full_name":"Evidenze Portugal Unipessoal Lda.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"France","full_name":"World Courier France","duties_or_roles":"IP Courier","organisation_type":"Non-Pharmaceutical company"}

Investigational products

Investigational Product Name
frexalimab
Modality
Monoclonal antibody
Investigational Product Name
brivekimig
Modality
Monoclonal antibody
Investigational Product Name
rilzabrutinib
Modality
Small molecule
Orphan Designation
Yes

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