Clinical trial • Phase II • Oncology
Venetoclax for Blastic plasmacytoid dendritic cell neoplasm
Phase II trial of Venetoclax for Blastic plasmacytoid dendritic cell neoplasm. open-label, none/not specified-controlled. 33 participants.
Overview
- Trial Therapeutic Area
- Oncology
- Trial Disease
- Blastic plasmacytoid dendritic cell neoplasm
- Trial Stage
- Phase II
- Drug Modality
- Small molecule|Peptide/protein/enzyme
Key dates
- Initial CTIS Submission Date
- 29-01-2025
- First CTIS Authorization Date
- 26-05-2025
Trial design
open-label, none/not specified-controlled Phase II trial across 34 sites in France.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 33
Eligibility
Recruits 33 No vulnerable populations selected. Inclusion criterion requires "Ability to understand the protocol and to sign an informed consent". Trial enrols adults (>18 years); no assent procedure or other special consent arrangements are described..
- Pregnancy Exclusion
- 6.Pregnant or breastfeeding woman ;
- Vulnerable Population
- No vulnerable populations selected. Inclusion criterion requires "Ability to understand the protocol and to sign an informed consent". Trial enrols adults (>18 years); no assent procedure or other special consent arrangements are described.
Inclusion criteria
- {"criterion_text":"- 1.Patients with a confirmed BPDCN diagnosis according to WHO 2022 revised criteria and have not received previous treatment ;\n- 2.Age >18 years ;\n- 3.Ability to understand the protocol and to sign an informed consent ;\n- 4.Possibility of follow-up ;\n- 5.ECOG < 3 ;\n- 6.Adequate renal function as demonstrated by a calculated creatinine clearance ≥ 45 mL/min by the Cockcroft-Gault formula ;\n- 7.\tAdequate cardiac function defined by LVEF >/= 50% by MUGA or ECHO and no clinically significant abnormalities on a 12-lead ECG ;\n- 8.Albumin level≥3,2g/dL ;\n- 9.Adequate liver function ;\n- 10.Men, and women of childbearing potential must be using a highly effective method of contraception ;\n- 11.Negative urine/blood pregnancy test within 1 week prior to the initiation of treatment"}
Exclusion criteria
- {"criterion_text":"- 1.\tParticipation to another clinical trial with any investigative drug within 30 days prior to study enrolment ;\n- 2.Previous treatment with venetoclax or tagraxofusp ;\n- 3.Concomitant immunosuppressive therapy –except for low-dose prednisone (≤10 mg/day) ;\n- 4.Concomitant treatment with medications prohibited in association with venetoclax ;\n- 5.Known allergy or sensitivity to tagraxofusp, venetoclax, and any of its components or excipients ;\n- 6.Pregnant or breastfeeding woman ;\n- 7.Peripheral neuropathy grade > 2 ;\n- 8.\tKnown positivity for hepatitis B or C infection except for those subjects with an undetectable viral load or subjects with serologic evidence of prior vaccination to HBV ;\n- 9.Known HIV-positivity ;\n- 10.Evidence of uncontrolled systemic infection requiring therapy (viral, bacterial, or fungal) ;\n- 11.Subject has any history of clinically significant condition(s) that in the opinion of the investigator would adversely affect his/her participating in this study ;\n- 12.Subject with a history of other malignancies prior to study ;\n- 13.Malabsorption syndrome or other conditions that preclude enteral route of administration"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Proportion of participants who achieve a cCR after 3 TAGVEN cycles.","definition_or_measurement_approach":"cCR (composite complete remission) defined as complete remission (CR) or complete remission with incomplete marrow recovery (Cri) according to European Leukemia Net 2022 definitions adapted for BPDCN, or complete response with minimal residual skin abnormality (CRc[clinical]) defined by ≥75% clearance of a patient’s skin lesions from baseline and a complete metabolic response after 3 cycles of TAGVEN."}
Secondary endpoints
- {"endpoint_text":"- Objective response rate including cCR and partial remission after 3 TAGVEN cycles\n- Safety profile of the tagraxofusp and venetoclax combination\n- Proportion of patients with MRD\n- Negative responses measured by flow cytometry\n- Progression-free Survival\n- Duration of response\n- Overall Survival\n- Proportion of patients bridged to allogeneic stem cell transplantation\n- Possible predictors of response with respect to cytogenetics and mutational status","definition_or_measurement_approach":"Objective response rate (ORR) defined as cCR + partial response (PR) after 3 TAGVEN cycles; MRD assessed by flow cytometry (negative responses measured by flow cytometry; protocol notes sensitivity of 0.1% in secondary objectives); Safety assessed by standard adverse event reporting; PFS, Duration of response, Overall Survival measured using standard time-to-event analyses; proportion bridged to allogeneic transplantation recorded; predictors assessed by cytogenetic and molecular status analyses."}
Recruitment
- Planned Sample Size
- 33
- Recruitment Window Months
- 72
- Consent Approach
- Informed consent must be provided and signed by the participant (adult >18 years). Inclusion requires the "Ability to understand the protocol and to sign an informed consent". Subject information and informed consent form documents are included in study documents. Protocol synopsis documents are available in French and English. No assent process or other special consent arrangements are described.
Geography
- Total Number Of Sites
- 34
- Total Number Of Participants
- 33
France
- Latest Decision Or Authorization Date
- 03-07-2025
- Number Of Sites
- 34
- Number Of Participants
- 33
Sites
- Site Name
- Institut Paoli Calmettes
- Department Name
- Hematology
- Principal Investigator Name
- Valerio MAISANO
- Principal Investigator Email
- maisanov@ipc.unicancer.fr
- Contact Person Name
- Valerio MAISANO
- Contact Person Email
- maisanov@ipc.unicancer.fr
- Site Name
- Groupe Hospitalier De La Region De Mulhouse Et Sud Alsace
- Department Name
- Hematology
- Principal Investigator Name
- Mario OJEDA-URIBE
- Principal Investigator Email
- ojeda-uribem@ghrmsa.fr
- Contact Person Name
- Mario OJEDA-URIBE
- Contact Person Email
- ojeda-uribem@ghrmsa.fr
- Site Name
- Institut De Cancerologie Strasbourg Europe
- Department Name
- Hematology
- Principal Investigator Name
- Célestine SIMAND
- Principal Investigator Email
- c.simand@icans.eu
- Contact Person Name
- Célestine SIMAND
- Contact Person Email
- c.simand@icans.eu
- Site Name
- Centre Hospital Region Metz Thionville
- Department Name
- Hematology
- Principal Investigator Name
- Houria DEBARRI
- Principal Investigator Email
- h.debarri@chr-metz-thionville.fr
- Contact Person Name
- Houria DEBARRI
- Contact Person Email
- h.debarri@chr-metz-thionville.fr
- Site Name
- Centre Hospitalier Sud Francilien
- Department Name
- Hematology
- Principal Investigator Name
- Célia SALANOUBAT
- Principal Investigator Email
- celia.salanoubat@chsf.fr
- Contact Person Name
- Célia SALANOUBAT
- Contact Person Email
- celia.salanoubat@chsf.fr
- Site Name
- CHRU De Nancy
- Department Name
- Hematology
- Principal Investigator Name
- Caroline BONMATI
- Principal Investigator Email
- c.bonmati@chru-nancy.fr
- Contact Person Name
- Caroline BONMATI
- Contact Person Email
- c.bonmati@chru-nancy.fr
- Site Name
- Hopital Henri Mondor - 1 rue Gustave Eiffel
- Department Name
- Hematology
- Principal Investigator Name
- Mathieu LECLER
- Principal Investigator Email
- mathieu.lecler@aphp.fr
- Contact Person Name
- Mathieu LECLER
- Contact Person Email
- mathieu.lecler@aphp.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Hematology
- Principal Investigator Name
- Mathilde HUNAULT
- Principal Investigator Email
- MaHunault@chu-angers.fr
- Contact Person Name
- Mathilde HUNAULT
- Contact Person Email
- MaHunault@chu-angers.fr
- Site Name
- Centre Hospitalier D Avignon
- Department Name
- Hematology
- Principal Investigator Name
- Safia CHEBREK
- Principal Investigator Email
- chebrek.safia@ch-avignon.fr
- Contact Person Name
- Safia CHEBREK
- Contact Person Email
- chebrek.safia@ch-avignon.fr
- Site Name
- Centre Hospitalier Universitaire De Toulouse
- Department Name
- Hematology
- Principal Investigator Name
- Christian RECHER
- Principal Investigator Email
- recher.christian@iuctoncopole.fr
- Contact Person Name
- Christian RECHER
- Contact Person Email
- recher.christian@iuctoncopole.fr
- Site Name
- Centre Hospitalier Universitaire Amiens Picardie
- Department Name
- Hematology
- Principal Investigator Name
- Jean-Pierre MAROLLEAU
- Principal Investigator Email
- marolleau.jean-pierre@chu-amiens.fr
- Contact Person Name
- Jean-Pierre MAROLLEAU
- Contact Person Email
- marolleau.jean-pierre@chu-amiens.fr
- Site Name
- Centre Hospitalier Universitaire De Montpellier
- Department Name
- Hematology
- Principal Investigator Name
- Sarah BONNET
- Principal Investigator Email
- sarah.bonnet@chu-montpellier.fr
- Contact Person Name
- Sarah BONNET
- Contact Person Email
- sarah.bonnet@chu-montpellier.fr
- Site Name
- CHU Rennes Pontchaillou Hospital
- Department Name
- Hematology
- Principal Investigator Name
- Jean-Baptiste MEAR
- Principal Investigator Email
- jean-baptiste.mear@chu-rennes.fr
- Contact Person Name
- Jean-Baptiste MEAR
- Contact Person Email
- jean-baptiste.mear@chu-rennes.fr
- Site Name
- Centre Hospitalier Universitaire Reims
- Department Name
- Hematology
- Principal Investigator Name
- Chantal HIMBERLIN
- Principal Investigator Email
- chimberlin@chu-reims.fr
- Contact Person Name
- Chantal HIMBERLIN
- Contact Person Email
- chimberlin@chu-reims.fr
- Site Name
- Centre Hospitalier Victor Dupouy
- Department Name
- Hematology
- Principal Investigator Name
- Benjamin PAPOULAR
- Principal Investigator Email
- Benjamin.papoular@ch-argenteuil.fr
- Contact Person Name
- Benjamin PAPOULAR
- Contact Person Email
- Benjamin.papoular@ch-argenteuil.fr
- Site Name
- Oncoradio Centre Oncogard
- Department Name
- Hematology
- Principal Investigator Name
- Samy CHRAIBI
- Principal Investigator Email
- samy.chraibi@chu-nimes.fr
- Contact Person Name
- Samy CHRAIBI
- Contact Person Email
- samy.chraibi@chu-nimes.fr
- Site Name
- Hopital Saint Louis
- Department Name
- Hematology
- Principal Investigator Name
- Lionel ADES
- Principal Investigator Email
- lionel.ades@aphp.fr
- Contact Person Name
- Lionel ADES
- Contact Person Email
- lionel.ades@aphp.fr
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- Hematology
- Principal Investigator Name
- Martin CARRE
- Principal Investigator Email
- MCarre1@chu-grenoble.fr
- Contact Person Name
- Martin CARRE
- Contact Person Email
- MCarre1@chu-grenoble.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Hematology
- Principal Investigator Name
- Céline BERTHON
- Principal Investigator Email
- Celine.BERTHON@chu-lille.fr
- Contact Person Name
- Céline BERTHON
- Contact Person Email
- Celine.BERTHON@chu-lille.fr
- Site Name
- Centre Hospitalier De Versailles
- Department Name
- Hematology
- Principal Investigator Name
- Fatiha MERABET
- Principal Investigator Email
- fmerabet@ght78sud.fr
- Contact Person Name
- Fatiha MERABET
- Contact Person Email
- fmerabet@ght78sud.fr
- Site Name
- Hopital Necker-Enfants Malades
- Department Name
- Hematology
- Principal Investigator Name
- Felipe SUAREZ
- Principal Investigator Email
- felipe.suarez@aphph.fr
- Contact Person Name
- Felipe SUAREZ
- Contact Person Email
- felipe.suarez@aphph.fr
- Site Name
- CHU Besancon
- Department Name
- Hematology
- Principal Investigator Name
- Eric DECONINCK
- Principal Investigator Email
- edeconinck@chu-besancon.fr
- Contact Person Name
- Eric DECONINCK
- Contact Person Email
- edeconinck@chu-besancon.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Hematology
- Principal Investigator Name
- Alice GARNIER
- Principal Investigator Email
- alice.garnier@chu-nantes.fr
- Contact Person Name
- Alice GARNIER
- Contact Person Email
- alice.garnier@chu-nantes.fr
- Site Name
- Centre Henri Becquerel
- Department Name
- Hematology
- Principal Investigator Name
- Emilie LEMASLE
- Principal Investigator Email
- emilie.lemasle@chb.unicancer.fr
- Contact Person Name
- Emilie LEMASLE
- Contact Person Email
- emilie.lemasle@chb.unicancer.fr
- Site Name
- Hopital D'Instruction Des Armees Percy
- Department Name
- Hematology
- Principal Investigator Name
- Justine SIAVELLIS
- Principal Investigator Email
- justine.siavellis@intradef.gouv.fr
- Contact Person Name
- Justine SIAVELLIS
- Contact Person Email
- justine.siavellis@intradef.gouv.fr
- Site Name
- CHRU Tours Hopital Bretonneau
- Department Name
- Hematology
- Principal Investigator Name
- Albian VILLATE
- Principal Investigator Email
- a.villate@chu-tours.fr
- Contact Person Name
- Albian VILLATE
- Contact Person Email
- a.villate@chu-tours.fr
- Site Name
- Centre Hospitalier Universitaire De Saint Etienne
- Department Name
- Hematology
- Principal Investigator Name
- Emmanuelle TAVERNIER
- Principal Investigator Email
- emmanuelle.tavernier@chu-st-etienne.fr
- Contact Person Name
- Emmanuelle TAVERNIER
- Contact Person Email
- emmanuelle.tavernier@chu-st-etienne.fr
- Site Name
- CHU d'Estaing
- Department Name
- Hematology
- Principal Investigator Name
- Aurelie RAVINET
- Principal Investigator Email
- aravinet@chu-clermontferrand.fr
- Contact Person Name
- Aurelie RAVINET
- Contact Person Email
- aravinet@chu-clermontferrand.fr
- Site Name
- Centre Hospitalier Et Universitaire De Limoges
- Department Name
- Hematology
- Principal Investigator Name
- Pascal TURLURE
- Principal Investigator Email
- pascal.turlure@chu-limoges.fr
- Contact Person Name
- Pascal TURLURE
- Contact Person Email
- pascal.turlure@chu-limoges.fr
- Site Name
- Assistance Publique Hopitaux de Paris – Hopital Cochin
- Department Name
- Hematology
- Principal Investigator Name
- Justine DECROOCQ
- Principal Investigator Email
- ustine.decroocq@aphp.fr
- Contact Person Name
- Justine DECROOCQ
- Contact Person Email
- ustine.decroocq@aphp.fr
- Site Name
- Institut Gustave Roussy
- Department Name
- Hematology
- Principal Investigator Name
- David GHEZ
- Principal Investigator Email
- David.GHEZ@gustaveroussy.fr
- Contact Person Name
- David GHEZ
- Contact Person Email
- David.GHEZ@gustaveroussy.fr
- Site Name
- Centre Hospitalier Universitaire De Poitiers
- Department Name
- Hematology
- Principal Investigator Name
- Maria-Pilar GALLEGOHERNANZ
- Principal Investigator Email
- maria-pilar.gallegohernanz@chu-poitiers.fr
- Contact Person Name
- Maria-Pilar GALLEGOHERNANZ
- Contact Person Email
- maria-pilar.gallegohernanz@chu-poitiers.fr
- Site Name
- Hopital Haut Leveque
- Department Name
- Hematology
- Principal Investigator Name
- Pierre-Yves DUMAS
- Principal Investigator Email
- pierre-yves.dumas@chu-bordeaux.fr
- Contact Person Name
- Pierre-Yves DUMAS
- Contact Person Email
- pierre-yves.dumas@chu-bordeaux.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Hematology
- Principal Investigator Name
- Mael HEIBLIG
- Principal Investigator Email
- mael.heiblig@chu-lyon.fr
- Contact Person Name
- Mael HEIBLIG
- Contact Person Email
- mael.heiblig@chu-lyon.fr
Sponsor
Primary sponsor
- Full Name
- French Innovative Leukemia Organization
- Organisation Type
- Laboratory/Research/Testing facility
- Country Of Registered Address
- France
Third parties
- {"country":"France","full_name":"For Drug Consulting","duties_or_roles":"sponsorDuties codes: 8","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Oxmo Cdm","duties_or_roles":"sponsorDuties codes: 6","organisation_type":"Non-Pharmaceutical company"}
- {"country":"France","full_name":"Quanticsoft","duties_or_roles":"sponsorDuties codes: 7","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Besancon University Hospital Center","duties_or_roles":"sponsorDuties codes: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"France","full_name":"Eurofins Clinical Trial Supplies France","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Fisher Clinical Services GmbH","duties_or_roles":"sponsorDuties codes: 14","organisation_type":"Pharmaceutical company"}
- {"country":"France","full_name":"Silicon Marketing","duties_or_roles":"sponsorDuties codes: 15 (DPO)","organisation_type":"Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Venetoclax
- Active Substance
- Venetoclax
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- ORAL
- Maximum Dose
- 400 mg (max daily/total amount as listed)
- Investigational Product Name
- ELZONRIS 1 mg/mL concentrate for solution for infusion
- Active Substance
- Tagraxofusp
- Modality
- Peptide/protein/enzyme
- Routes Of Administration
- IV INFUSION
- Route
- IV
- Authorisation Status
- Marketing authorisation number EU/1/20/1504/001 (product info present)
- Maximum Dose
- 12 µg/Kg (max daily/total amount as listed)
- Combination Treatment
- Yes
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