Clinical trial • Phase III • Nephrology

Dapagliflozin for Alport syndrome

Phase III trial of Dapagliflozin for Alport syndrome.

Overview

Trial Therapeutic Area
Nephrology
Trial Disease
Alport syndrome
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes

Key dates

Initial CTIS Submission Date
09-11-2023
First CTIS Authorization Date
07-02-2024

Trial design

Randomised, placebo (p-tabletten weiß 7 mm lichtenstein) oral tablet; matching placebo arm. no explicit dose or dosing schedule for placebo stated in the available record.-controlled Phase III trial in Germany.

Randomised
Yes
Comparator
Placebo (P-Tabletten weiß 7 mm Lichtenstein) oral tablet; matching placebo arm. No explicit dose or dosing schedule for placebo stated in the available record.
Target Sample Size
102
Trial Duration For Participant
336

Eligibility

Recruits 102 paediatric patients.

Pregnancy Exclusion
Pre-menopausal women, who are nursing or pregnant, or who are not practicing an acceptable method of birth control
Vulnerable Population
Vulnerable population selected (isVulnerablePopulationSelected = true). The trial includes adolescents (age ranges include 10–<18 years). Age-specific subject information and informed consent documents are provided (documents titled: DoubleProtect-ALPORT_PIC 10-11 years; DoubleProtect-ALPORT_PIC 12-15 years; DoubleProtect-ALPORT_PIC 16 years-full of age; DoubleProtect-ALPORT_PIC Sorgeberechtigte for guardians). Signed and dated written informed consent is required; assent and parental/guardian consent arrangements are documented via age-specific forms.

Inclusion criteria

  • {"criterion_text":"- Early stages of CKD with established diagnosis of Alport syndrome at visit 1 (screening)\n- adolescents ≥ 10 to < 18 years with albuminuria (UACR ≥ 300mg/g Crea) OR adults ≥ 18 to < 40 years with albuminuria (UACR ≥ 500mg/g Crea) AND eGFR ≥ 60 ml/min/1.73 m2\n- Molecular-genetic diagnosis (any type of Alport gene variant) or diagnosis established by kidney biopsy\n- Stable RAS blockade as standard of care background therapy (defined as no adjustment to dose in the last three months prior the baseline visit).\n- Signed and dated written informed consent"}

Exclusion criteria

  • {"criterion_text":"- Medical history that might limit the individual’s ability to take trial treatments\n- Lack of basic understanding of German language (required for patient questionnaires)\n- Treatment with any SGLT2 inhibitor or within 4 weeks prior to Visit 1\n- Requiring dialysis or after kidney-transplantation\n- Uncontrolled arterial hypertension (blood pressure >145/95 mmHg)\n- Type 1 diabetes OR type 2 diabetes with previous history of diabetic ketoacidosis\n- Known hypersensitivity or allergy to the investigational product (contains lactulose)\n- Any previous or current alcohol or drug abuse\n- Participation in another trial with an investigational drug ongoing\n- Pre-menopausal women, who are nursing or pregnant, or who are not practicing an acceptable method of birth control"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline to 48 weeks in (log transformed) UACR.","definition_or_measurement_approach":"Change from baseline to 48 weeks in (log transformed) urine albumin-to-creatinine ratio (UACR)."}

Secondary endpoints

  • {"endpoint_text":"- eGFR change from baseline to Week 52","definition_or_measurement_approach":"Change from baseline to Week 52 in estimated glomerular filtration rate (eGFR)."}
  • {"endpoint_text":"- In adults, change from baseline to Week 48 in SF- 36.","definition_or_measurement_approach":"Change from baseline to Week 48 in SF-36 health survey scores in adults."}
  • {"endpoint_text":"- In adolescents, change from baseline to Week 48 in PedsQL","definition_or_measurement_approach":"Change from baseline to Week 48 in Pediatric Quality of Life Inventory (PedsQL) scores in adolescents."}
  • {"endpoint_text":"- Difference between treatment groups in eGFR change from Week 4 eGFR to Week 48 measurement","definition_or_measurement_approach":"Between-group difference in eGFR change from Week 4 measurement to Week 48 measurement."}

Recruitment

Planned Sample Size
102
Recruitment Window Months
20
Consent Approach
Signed and dated written informed consent is required. Age-specific information and consent/assent forms are available: subject information and informed consent forms for ages 10-11, 12-15, 16 years/full of age, and for legal guardians (Sorgeberechtigte). The sponsor provides public and scientific contact details; language availability of documents is not specified in the available record.

Geography

Total Number Of Sites
15
Total Number Of Participants
102

Germany

Earliest CTIS Part Ii Submission Date
14-12-2023
Latest Decision Or Authorization Date
16-10-2025
Processing Time Days
672
Number Of Sites
15
Number Of Participants
102

Sites

Site Name
Ludwig Maximilian University Of Munich
Department Name
Kinderklinik und Kinderpoliklinik im Dr. von Haunerschen Kinderspital, Pädiatrische Nephrologie
Principal Investigator Name
Bärbel Lange-Sperandio
Contact Person Name
Bärbel Lange-Sperandio
Site Name
Universitaet Leipzig
Department Name
Klinik für Kinder- und Jugensmedizin, Pädiatrische Nephrologie
Principal Investigator Name
Katalin Dittrich
Principal Investigator Email
katalin.dittrich@medizin.uni-leipzig.de
Contact Person Name
Katalin Dittrich
Site Name
Buergerhospital und Clementine Kinderhospital gGmbH
Department Name
Clementine Kinderhospital
Principal Investigator Name
Matthias Hansen
Principal Investigator Email
Matthias.Hansen@kfh-dialyse.de
Contact Person Name
Matthias Hansen
Contact Person Email
Matthias.Hansen@kfh-dialyse.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Pädiatrische Nephrologie
Principal Investigator Name
Burkhard Tönshoff
Principal Investigator Email
burkhard.toenshoff@med.uni-heidelberg.de
Contact Person Name
Burkhard Tönshoff
Site Name
University Hospital Cologne AöR
Department Name
Innere Medizin II - Nephrologie, Diabetologie und Allgemeine Innere Medizin
Principal Investigator Name
Roman-Urlich Müller
Principal Investigator Email
roman-ulrich.mueller@uk-koeln.de
Contact Person Name
Roman-Urlich Müller
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Fachambulanz für Nephrologie
Principal Investigator Name
Jan Halbritter
Principal Investigator Email
jan.halbritter@charite.de
Contact Person Name
Jan Halbritter
Contact Person Email
jan.halbritter@charite.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik für Nephrologie und Rheumatologie
Principal Investigator Name
Oliver Gross
Principal Investigator Email
gross.oliver@med.uni-goettingen.de
Contact Person Name
Oliver Gross
Site Name
Universitaetsklinikum Leipzig AöR
Department Name
Klinik und Poliklinik für Endokrinologie, Nephrologie, Rheumatologie
Principal Investigator Name
Jan Kowald
Principal Investigator Email
jan.kowald@medizin.uni-leipzig.de
Contact Person Name
Jan Kowald
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Klinik für Pädiatrie mit Schwerpunkten Gastroenterologie, Nephrologie und Stoffwechselmedizin
Principal Investigator Name
Julia Thumfart
Principal Investigator Email
julia.thumfart@charite.de
Contact Person Name
Julia Thumfart
Contact Person Email
julia.thumfart@charite.de
Site Name
Universitaetsmedizin Goettingen
Department Name
Klinik für Kinder- und Jugendmedizin
Principal Investigator Name
Matthias Kettwig
Principal Investigator Email
matthias.kettwig@med.uni-goettingen.de
Contact Person Name
Matthias Kettwig
Site Name
Klinikum der Universitaet Muenchen AöR
Department Name
Medizinische Klinik und Poliklinik IV
Principal Investigator Name
Volker Vielhauer
Principal Investigator Email
volker.vielhauer@med.uni-muenchen.de
Contact Person Name
Volker Vielhauer
Site Name
Universitaetsklinikum Muenster AöR
Department Name
Klinik für Kinder- und Jugendmedizin - Allgemeine Pädiatrie - Pädiatrische Nephrologie
Principal Investigator Name
Martin Konrad
Principal Investigator Email
konradma@uni-muenster.de
Contact Person Name
Martin Konrad
Contact Person Email
konradma@uni-muenster.de
Site Name
University Hospital Cologne AöR
Department Name
Klinik und Poliklinik für Kinder- und Jugendmedizin
Principal Investigator Name
Lutz Weber
Principal Investigator Email
lutz.weber@uk-koeln.de
Contact Person Name
Lutz Weber
Contact Person Email
lutz.weber@uk-koeln.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Zentrum für Geburtshilfe , Kinder- und Jugendmedizin
Principal Investigator Name
Jun Oh
Principal Investigator Email
j.oh@uke.de
Contact Person Name
Jun Oh
Contact Person Email
j.oh@uke.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
III. Medizinische Klinik und Poliklinik
Principal Investigator Name
Markus Gödel
Principal Investigator Email
m.goedel@uke.de
Contact Person Name
Markus Gödel
Contact Person Email
m.goedel@uke.de

Sponsor

Primary sponsor

Full Name
Universitaetsmedizin Goettingen
Organisation Type
Hospital/Clinic/Other health care facility
Country Of Registered Address
Germany

Third parties

  • {"country":"","full_name":"Deutsche Forschungsgemeinschaft","duties_or_roles":"Source of monetary support","organisation_type":""}

Investigational products

Investigational Product Name
Forxiga 5 mg film-coated tablets
Active Substance
Dapagliflozin
Modality
Small molecule
Routes Of Administration
ORAL
Route
Oral
Authorisation Status
Authorised (marketing authorisation EU/1/12/795/001)
Starting Dose
5 mg (product strength)
Maximum Dose
10 mg
Investigational Product Name
P-Tabletten weiß 7 mm Lichtenstein
Active Substance
Placebo
Modality
Other
Routes Of Administration
ORAL
Route
Oral
Authorisation Status
Authorised (MIA DE_RP_01_MIA_2016_0018 / marketing authorization reference present)
Starting Dose
0 mg (placebo)
Maximum Dose
0 mg

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