Clinical trial • Phase III • Infectious Disease | Rare Disease

BRINCIDOFOVIR for Adenovirus viremia

Phase III trial of BRINCIDOFOVIR for Adenovirus viremia.

Overview

Trial Therapeutic Area
Infectious Disease | Rare Disease
Trial Disease
Adenovirus viremia
Trial Stage
Phase III
Drug Modality
Small molecule
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
27-06-2025
First CTIS Authorization Date
02-10-2025

Trial design

Randomised, open-label, intravenous brincidofovir (bcv) versus intravenous cidofovir (cdv); routes: infusion. dose and schedule not specified in the provided ctis data.-controlled Phase III trial across 17 sites in France, Germany, Italy and others.

Randomised
Yes
Open Label
Yes
Comparator
Intravenous Brincidofovir (BCV) versus Intravenous Cidofovir (CDV); routes: infusion. Dose and schedule not specified in the provided CTIS data.
Target Sample Size
108

Eligibility

Recruits 108 paediatric patients.

Pregnancy Exclusion
Subject is non-pregnant, and either not breast feeding or willing to discontinue breast feeding prior to randomization.
Vulnerable Population
Includes paediatric subjects (age range from 2 months and older). Consent must be provided by the subject or guardian: 'Subject/Guardian willing and able to understand and provide written informed consent to participate in the study.' Assent and parent information/ICF documents are provided (documents listed include SIS and ICF Parent, Assent_Aged 12-17, Assent_Child) in multiple country-specific packs.

Inclusion criteria

  • {"criterion_text":"- Male and female, post-allo-HCT within last 180 days, aged 2 months and older at time of signing informed consent form.\n- Subject/Guardian willing and able to understand and provide written informed consent to participate in the study.\n- In the investigator’s judgement, the subject’s clinical condition justifies treatment with IV BCV or IV CDV for AdV infection.\n- Has adenoviremia\n- Men and women of childbearing potential (WOCBP) must be willing to use acceptable method(s) of contraception during the study and for at least 6 months after the last dose of IV BCV or at least 6 months after the last dose of IV CDV.\n- Women of childbearing potential (WOCBP) must agree to use two (2) acceptable forms of contraception (one of which must be a barrier method) during heterosexual intercourse. Males capable of fathering a child must agree to use acceptable method(s) of contraception during heterosexual intercourse.\n- Subject is non-pregnant, and either not breast feeding or willing to discontinue breast feeding prior to randomization."}

Exclusion criteria

  • {"criterion_text":"- Subject received an allo-HCT with a matched sibling donor\n- Subject has any other disease, or laboratory abnormality, or clinical finding that, in the judgment of the investigator, would put the subject at unacceptable risk for participation or interfere with study assessments or data quality.\n- Subject is expected to die from a non-adenovirus cause within 30 days from the date of ICF.\n- Subject is critically ill, for example with sepsis on high dose vasopressors and mechanical ventilation.\n- Subject is unable to comply with protocol visits and procedures.\n- Subject received more than 5 mg/kg of CDV for any reason in the 21 days prior to first dose of study drug.\n- Subject is allergic or hypersensitive to IV BCV or IV CDV or any of their components.\n- Subject received anti-AdV-specific cell-based therapy within 3 weeks prior to W1D1 or an anti-AdV vaccine at any time.\n- Subject has participated in any other investigational study within 30 days (or within 5.5 half-lives of the investigational product, whichever is longer) before signing the informed consent form (ICF), is currently participating in another interventional treatment trial with an investigational agent or is using an investigational device at the time of Screening.\n- Subject has NIH Stage 3 or higher acute GVHD of the gut within 7 days prior to W1D1.\n- Subject has NIH Stage 2 or higher acute GVHD of the liver within 7 days prior to W1D1 (i.e., bilirubin>3 mg/dL [International System, SI: >51 μmol/L]).\n- Subject has exclusionary hepatic parameters within 7 days prior to W1D1: •\tTotal bilirubin >3 mg/dL (SI: >51 μmol/L) except for subjects with Gilbert’s Disease, •\tProthrombin time-international normalized ratio (PT INR) >2x ULN, unless attributed to AdV. •\tALT or AST >5x upper limit of normal (ULN), except if it is judged by the PI to be due to the AdV infection. Note: Subjects with elevated serum transaminases >5x ULN (CTCAE Grade 3 or higher) due to AdV will be required to demonstrate improvement and must stop study drug if the elevated values have not improved by W3D1: either at least one CTCAE grade or clinical improvement based on the physician’s assessment.\n- Subject has uncontrolled viral (other than AdV), bacterial, or fungal infection(s) leading to hemodynamic instability or radiologic or laboratory evidence attributable to worsening disease."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Proportion of subjects with AdV virological success","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- Proportion of subjects with overall success","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Proportion of subjects with clinical success","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Proportion of subjects with AdV virological success","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Correlation of virologic success and clinical response","definition_or_measurement_approach":""}
  • {"endpoint_text":"- AdV-free survival","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Time to AdV virological success","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Rate of AdV recurrence","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Length of hospitalization and length of ICU stay","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Desirability Of Outcome Ranking (DOOR) Analysis for benefit-risk estimation","definition_or_measurement_approach":""}
  • {"endpoint_text":"- All-cause mortality","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Adenovirus attributed mortality, as adjudicated by the EAC","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Primary malignancy relapse-free survival","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Incidence and severity of treatment-emergent adverse events (TEAEs)","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
108
Recruitment Window Months
32
Consent Approach
Subject or guardian must provide written informed consent ('Subject/Guardian willing and able to understand and provide written informed consent to participate in the study.'). Paediatric assent documents available (Assent_Child, Assent_Aged 12-17) and parent ICFs and adult ICFs are provided in country packs. Documents available in multiple languages as indicated in CTIS (French, German, Italian, Spanish, English).

Geography

Total Number Of Sites
17
Total Number Of Participants
72

France

Earliest CTIS Part Ii Submission Date
29-09-2025
Latest Decision Or Authorization Date
03-03-2026
Processing Time Days
155
Number Of Sites
3
Number Of Participants
15

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
Pediatric immunohematology and rheumatology department
Contact Person Name
Bénédicte Neven
Contact Person Email
benedicte.neven@aphp.fr
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Hematology and Immunology Pediatric Department
Contact Person Name
Jean-Hugues Dalle
Site Name
Assistance Publique Hopitaux De Paris
Department Name
Hematology and Bone Marrow Transplant Department
Contact Person Name
Alienor Xhaard
Contact Person Email
alienor.xhaard@aphp.fr

Germany

Earliest CTIS Part Ii Submission Date
17-09-2025
Latest Decision Or Authorization Date
04-03-2026
Processing Time Days
168
Number Of Sites
7
Number Of Participants
19

Sites

Site Name
Goethe University Frankfurt
Department Name
Department of Pediatrics
Contact Person Name
Peter Bader
Contact Person Email
Peter.Bader@unimedizin-ffm.de
Site Name
Medizinische Hochschule Hannover
Department Name
Klinik für Pädiatrische Hämatologie und Onkologie
Contact Person Name
Martin Sauer
Contact Person Email
kinderonkologie@mh-hannover.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Klinik für Kinderheilkunde und Jugendmedizin
Contact Person Name
Tim Flaadt
Site Name
Universitaet Muenster
Department Name
Klinik für Kinder- und Jugendmedizin - Pädiatrische Hämatologie und Onkologie
Contact Person Name
Andreas Groll
Site Name
Universitaetsklinikum Essen AöR
Department Name
Institut für Virologie
Contact Person Name
Sebastian Voigt
Contact Person Email
Paedonko-Studien@uk-essen.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Klinik für Pädiatrische Hämatologie und Onkologie
Contact Person Name
Ingo Müller
Contact Person Email
paed-bmt@uke.de
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Campus Virchow-Klinikum
Contact Person Name
Felix Zirngibl
Contact Person Email
0@0

Italy

Earliest CTIS Part Ii Submission Date
15-07-2025
Latest Decision Or Authorization Date
03-03-2026
Processing Time Days
231
Number Of Sites
5
Number Of Participants
19

Sites

Site Name
Fondazione IRCCS Policlinico San Matteo
Department Name
Oncoematologia Pediatrica
Contact Person Name
Marco Zecca
Contact Person Email
m.zecca@smatteo.pv.it
Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
Clinical Oncohaematology and Cell Therapy
Contact Person Name
Franco Locatelli
Contact Person Email
franco.locatelli@opbg.net
Site Name
IRCCS Istituto Giannina Gaslini
Department Name
Unità di Trapianto di cellule Staminali emopoietiche
Contact Person Name
Maura Faraci
Contact Person Email
maurafaraci@gaslini.org
Site Name
Ospedale San Raffaele S.r.l.
Department Name
UO Ematologia e Trapiando di Midollo Osseo
Contact Person Name
Raffaella Greco
Contact Person Email
greco.raffaella@hsr.it
Site Name
Hospital Santa Maria Della Misericordia
Department Name
Oncoematologia Pediatrica
Contact Person Name
Katia Perruccio

Spain

Earliest CTIS Part Ii Submission Date
07-10-2025
Latest Decision Or Authorization Date
04-03-2026
Processing Time Days
148
Number Of Sites
2
Number Of Participants
19

Sites

Site Name
Clinica Universidad De Navarra
Department Name
Hematology
Contact Person Name
Sara Villar Fernández
Contact Person Email
ensayoscun@unav.es
Site Name
Hospital Universitari Vall D Hebron
Department Name
Hematology
Contact Person Name
Ana Pérez González
Contact Person Email
aperez@vhio.net

Sponsor

Primary sponsor

Full Name
Symbio Pharmaceuticals Ltd.
Organisation Type
Pharmaceutical company
Country Of Registered Address
Japan

Contract research organisations

Name
Certara USA Inc.
Responsibilities
Data analysis: PK parameter report
Name
Cti Clinical Trial Services Inc.
Responsibilities
Regulatory/operational roles (codes: 13,4,5)
Name
CTI Clinical Trial and Consulting Services Europe GmbH
Responsibilities
Regulatory/operational roles (codes: 1,12,2,5)
Name
Syneos Health Inc.
Responsibilities
Safety reporting (code: 8)
Name
CTI Laboratory Services Spain S.L.
Responsibilities
Laboratory services (code: 4)

Third parties

  • {"country":"Spain","full_name":"Taxi Travel Ticket S.L.","duties_or_roles":"15: Travel/Reimbursement Managament","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Certara USA Inc.","duties_or_roles":"15: Data analysis: PK parameter report","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Cti Clinical Trial Services Inc.","duties_or_roles":"13, 4, 5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"8","organisation_type":"Pharmaceutical company"}
  • {"country":"Spain","full_name":"CTI Laboratory Services Spain S.L.","duties_or_roles":"4","organisation_type":"Pharmaceutical company"}
  • {"country":"Ireland","full_name":"Verasafe Ireland Limited","duties_or_roles":"15: Data Protection Officer","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Unisphere Travel Ltd. Inc.","duties_or_roles":"15: Travel/Reimbursement Managament","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Germany","full_name":"CTI Clinical Trial and Consulting Services Europe GmbH","duties_or_roles":"1, 12, 2, 5","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
Brincidofovir
Active Substance
BRINCIDOFOVIR
Modality
Small molecule
Routes Of Administration
INFUSION
Route
INFUSION
Authorisation Status
Authorised
Orphan Designation
Yes
Investigational Product Name
Cidofovir
Active Substance
ANHYDROUS CIDOFOVIR
Modality
Small molecule
Routes Of Administration
INFUSION
Route
INFUSION
Authorisation Status
Authorised

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