Clinical trial • Phase III • Haematology
Dexamethasone for Immune thrombocytopenia (ITP) | Primary immune thrombocytopenia | Secondary immune thrombocytopenia
Phase III trial of Dexamethasone for Immune thrombocytopenia (ITP) | Primary immune thrombocytopenia | Secondary immune thrombocytopenia.
Overview
- Trial Therapeutic Area
- Haematology
- Trial Disease
- Immune thrombocytopenia (ITP) | Primary immune thrombocytopenia | Secondary immune thrombocytopenia
- Trial Stage
- Phase III
- Drug Modality
- Small molecule | Other antibody
Key dates
- Initial CTIS Submission Date
- 25-09-2024
- First CTIS Authorization Date
- 13-11-2024
Trial design
Randomised, ivig 1 g/kg on days 1 and 2 in combination with prednisone 1 mg/kg per day for 3 weeks (comparator arm); oral dexamethasone 40 mg on days 1 to 4 (test arm).-controlled Phase III trial in France.
- Randomised
- Yes
- Comparator
- IVIg 1 g/kg on days 1 and 2 in combination with prednisone 1 mg/kg per day for 3 weeks (comparator arm); oral dexamethasone 40 mg on days 1 to 4 (test arm).
- Target Sample Size
- 272
- Trial Duration For Participant
- 180
Eligibility
Recruits 272 Persons deprived of their liberty by judicial or administrative decision and persons under legal protection (guardianship, curatorship) are listed in exclusion criteria. Participation requires written consent from the patient; inability or refusal to understand or to sign informed consent excludes participation..
- Pregnancy Exclusion
- Pregnant or breastfeeding woman or ineffective contraception
- Vulnerable Population
- Persons deprived of their liberty by judicial or administrative decision and persons under legal protection (guardianship, curatorship) are listed in exclusion criteria. Participation requires written consent from the patient; inability or refusal to understand or to sign informed consent excludes participation.
Inclusion criteria
- {"criterion_text":"- Age ≥ 18 years ≤ 80 years"}
- {"criterion_text":"- Diagnosis of ITP whatever the duration of the disease (newly diagnosed or relapsed) according to the standard definition"}
- {"criterion_text":"- Platelet count ≤ 20 x 109/L"}
- {"criterion_text":"- Any cutaneous and/or any mucosal bleeding manifestations"}
- {"criterion_text":"- Affiliated to a social security regime"}
- {"criterion_text":"- Written consent from patient"}
Exclusion criteria
- {"criterion_text":"- Symptomatic COVID-19 disease"}
- {"criterion_text":"- Ongoing viral infection (uncontrolled HIV, Viral hepatitis, herpes, varicella, zona)"}
- {"criterion_text":"- Uncontrolled diabetes (Acido-cetosis)"}
- {"criterion_text":"- Psychotic state not yet controlled by treatment"}
- {"criterion_text":"- Inability or refusal to understand or refusal to sign the informed consent from study participation"}
- {"criterion_text":"- Persons deprived of their liberty by judicial or administrative decision"}
- {"criterion_text":"- Persons under legal protection (guardianship, curatorship)"}
- {"criterion_text":"- Pregnant or breastfeeding woman or ineffective contraception"}
- {"criterion_text":"- Participation in another interventional study involving human participants or being in the exclusion period at the end of a previous study involving human participants"}
- {"criterion_text":"- Life-threatening bleeding defined as Intracranial hemorrhage and/or active organ bleeding (GI tract, urinary tract or menorrhagia with at least a 2 g/dl decrease of hemoglobin value from baseline)."}
- {"criterion_text":"- Ongoing anticoagulation treatment (Therapeutic Low molecular weight heparins (LMWHs), direct oral anticoagulants (DOACs) and vitamin K antagonists (VKAs))"}
- {"criterion_text":"- Previous non-response to IVIg or DEX"}
- {"criterion_text":"- Treatment with prednisone (1 mg/kg per day) for more than 3 days"}
- {"criterion_text":"- Any contraindications to the prescribed Ig IV or prednisone patent medicine and to Neofordex®"}
- {"criterion_text":"- Ongoing severe infection"}
- {"criterion_text":"- Severe Renal insufficiency (DFG < 45 ml.min.1.73m2)"}
- {"criterion_text":"- Severe Cardiac insufficiency (FEVG < 30 %)"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Time (in days) to achieve an initial response (R) within 5 days. - Initial response (R) will be defined according to international guidelines (5) as a platelet count ≥ 30x109/L with at least a doubling of the baseline value in the absence of new bleeding and absence of the use of any other ITP directed therapies (other than the one of the treatment arm)","definition_or_measurement_approach":"Initial response (R) defined according to international guidelines as a platelet count ≥ 30 x 10^9/L with at least a doubling of baseline value, in the absence of new bleeding and absence of use of any other ITP-directed therapies (other than the allocated treatment). Time measured in days up to 5 days."}
Secondary endpoints
- {"endpoint_text":"- Time (in days) to achieve an initial complete response (CR) in both arms (defined by a platelet count > 100 x 109/L in the absence use of any other ITP directed therapies between Day 1 and Day 5) in the two arms.","definition_or_measurement_approach":"Complete response (CR) defined as platelet count > 100 x 10^9/L in the absence of any other ITP-directed therapies between Day 1 and Day 5; time measured in days."}
- {"endpoint_text":"- Duration in time (in days) of overall response from Day 1 to the end of the study (6 months) in the two arms.","definition_or_measurement_approach":"Overall response duration measured in days from Day 1 until loss of response or end of study (study duration specified as 6 months)."}
- {"endpoint_text":"- Proportion of early (before day 5) treatment switches across arms","definition_or_measurement_approach":"Proportion of participants who switch treatment before Day 5 compared between arms."}
- {"endpoint_text":"- Number of new bleeding manifestations between Day 1 and Day 5 in two arms. Bleeding manifestations will be assessed the score reported by Khellaf et al. without taking into account the age of the patient.","definition_or_measurement_approach":"Number of new bleeding manifestations counted between Day 1 and Day 5; assessed using the bleeding score reported by Khellaf et al. (age not considered)."}
- {"endpoint_text":"- Rates of response (R) and complete response (CR) at 28 days, and 6 months in the two arms.","definition_or_measurement_approach":"Rates of R and CR measured at Day 28 and at 6 months post-treatment in each arm."}
- {"endpoint_text":"- Number of bleeding manifestations between Day 5 and Day 28 in the two arms.","definition_or_measurement_approach":"Number of bleeding manifestations counted between Day 5 and Day 28 in each arm."}
- {"endpoint_text":"- Number of days of hospitalization between Day 1 and Day 28 in the two arms.","definition_or_measurement_approach":"Total days of hospitalization per participant between Day 1 and Day 28 compared across arms."}
- {"endpoint_text":"- Number of adverse events in the two arms.","definition_or_measurement_approach":"Count and compare adverse events reported in each arm (standard AE reporting)."}
- {"endpoint_text":"- Incremental (decremental) cost effectiveness ratio expressed in cost per responder at 6 months","definition_or_measurement_approach":"Cost-effectiveness analysis: incremental (or decremental) cost per responder at 6 months comparing DXM vs IVIg."}
- {"endpoint_text":"- Comparison of the number of responders, and outcome at 6 months in patients with positive and negative anti-platelets antibodies in the two arms.","definition_or_measurement_approach":"Compare number of responders and 6-month outcomes stratified by anti-platelet antibody status (positive vs negative) across treatment arms."}
Recruitment
- Planned Sample Size
- 272
- Recruitment Window Months
- 47
- Consent Approach
- Written informed consent from the patient (adult) is required. A subject information sheet and informed consent form for adults is listed (L1_SIS and ICF adults). No paediatric assent/consent procedures are described. Languages of consent documents are not specified.
Geography
- Total Number Of Sites
- 38
- Total Number Of Participants
- 272
France
- Earliest CTIS Part Ii Submission Date
- 14-10-2024
- Latest Decision Or Authorization Date
- 27-05-2025
- Processing Time Days
- 225
- Number Of Sites
- 38
- Number Of Participants
- 272
Sites
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine Interne
- Principal Investigator Name
- Nicolas NOEL
- Principal Investigator Email
- nicolas.noel@aphp.fr
- Contact Person Name
- Nicolas NOEL
- Contact Person Email
- nicolas.noel@aphp.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine Interne
- Principal Investigator Name
- Delphine GOBERT
- Principal Investigator Email
- delphine.gobert@aphp.fr
- Contact Person Name
- Delphine GOBERT
- Contact Person Email
- delphine.gobert@aphp.fr
- Site Name
- Centre Hospitalier Regional De Marseille
- Department Name
- Médecine interne
- Principal Investigator Name
- Mikael EBBO
- Principal Investigator Email
- mikael.ebbo@ap-hm.fr
- Contact Person Name
- Mikael EBBO
- Contact Person Email
- mikael.ebbo@ap-hm.fr
- Site Name
- CHRU De Nancy
- Department Name
- Médecine interne
- Principal Investigator Name
- Thomas MOULINET
- Principal Investigator Email
- t.moulinet@chru-nancy.fr
- Contact Person Name
- Thomas MOULINET
- Contact Person Email
- t.moulinet@chru-nancy.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Immunologie clinique
- Principal Investigator Name
- Jehane FADLALLAH
- Principal Investigator Email
- jehane.fadlallah@aphp.fr
- Contact Person Name
- Jehane FADLALLAH
- Contact Person Email
- jehane.fadlallah@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Bordeaux
- Department Name
- Médecine Interne et maladies infectieuses
- Principal Investigator Name
- Jean-François VIALLARDARD
- Principal Investigator Email
- jean-francois.viallard@chu-bordeaux.fr
- Contact Person Name
- Jean-François VIALLARD
- Contact Person Email
- jean-francois.viallard@chu-bordeaux.fr
- Site Name
- Centre Hospitalier Universitaire D'Angers
- Department Name
- Maladies du sang et médecine interne
- 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 Universitaire De Toulouse
- Department Name
- Médecine interne, UMR
- Principal Investigator Name
- Guillaume MOULIS
- Principal Investigator Email
- moulis.g@chu-toulouse.fr
- Contact Person Name
- Guillaume MOULIS
- Contact Person Email
- moulis.g@chu-toulouse.fr
- Site Name
- Centre Hospitalier Universitaire De Caen Normandie
- Department Name
- Hématologie
- Principal Investigator Name
- Stéphane CHEZE
- Principal Investigator Email
- cheze-s@chu-caen.fr
- Contact Person Name
- Stéphane CHEZE
- Contact Person Email
- cheze-s@chu-caen.fr
- Site Name
- Hopital Saint Joseph
- Department Name
- Médecine interne
- Principal Investigator Name
- Antoine POULET
- Principal Investigator Email
- apoulet@hopital-saint-joseph.fr
- Contact Person Name
- Antoine POULET
- Contact Person Email
- apoulet@hopital-saint-joseph.fr
- Site Name
- Centre Hospitalier Regional Et Universitaire De Brest
- Department Name
- Hématologie
- Principal Investigator Name
- Brigitte PAN-PETESCH
- Principal Investigator Email
- brigitte.pan-petesch@chu-brest.fr
- Contact Person Name
- Brigitte PAN-PETESCH
- Contact Person Email
- brigitte.pan-petesch@chu-brest.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Médecine interne
- Principal Investigator Name
- Isabelle DURIEU
- Principal Investigator Email
- isabelle.durieu@chu-lyon.fr
- Contact Person Name
- Isabelle DURIEU
- Contact Person Email
- isabelle.durieu@chu-lyon.fr
- Site Name
- Centre Hospitalier D Avignon
- Department Name
- Hématologie clinique
- 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 Poitiers
- Department Name
- Médecine Interne maladies infectieuses et tropicales
- Principal Investigator Name
- Frederique ROY-PEAUD
- Principal Investigator Email
- frederique.roy-peaud@chu-poitiers.fr
- Contact Person Name
- Frederique ROY-PEAUD
- Contact Person Email
- frederique.roy-peaud@chu-poitiers.fr
- Site Name
- Centre Hospitalier Universitaire Rouen
- Department Name
- Médecine interne
- Principal Investigator Name
- Gaetan SAUVETRE
- Principal Investigator Email
- gaetan.sauvetre@chu-rouen.fr
- Contact Person Name
- Gaetan SAUVETRE
- Contact Person Email
- gaetan.sauvetre@chu-rouen.fr
- Site Name
- Centre Hospitalier Universitaire De Lille
- Department Name
- Hématologie et médecine interne
- Principal Investigator Name
- Louis TERRIOU
- Principal Investigator Email
- louis.terriou@chru-lille.fr
- Contact Person Name
- Louis TERRIOU
- Contact Person Email
- louis.terriou@chru-lille.fr
- Site Name
- Centre Hospitalier Saint Nazaire
- Department Name
- Hématologie
- Principal Investigator Name
- Julie GRAVELEAU
- Principal Investigator Email
- j.graveleau@ch-saintnazaire.fr
- Contact Person Name
- Julie GRAVELEAU
- Contact Person Email
- j.graveleau@ch-saintnazaire.fr
- Site Name
- Les Hopitaux Universitaires De Strasbourg
- Department Name
- Médecine interne
- Principal Investigator Name
- Emmanuel ANDRES
- Principal Investigator Email
- emmanuel.andres@chru-strasbourg.fr
- Contact Person Name
- Emmanuel ANDRES
- Contact Person Email
- emmanuel.andres@chru-strasbourg.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine Interne
- Principal Investigator Name
- Antoine DOSSIER
- Principal Investigator Email
- antoine.dossier@aphp.fr
- Contact Person Name
- Antoine DOSSIER
- Contact Person Email
- antoine.dossier@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Saint Etienne
- Department Name
- Médecine interne
- Principal Investigator Name
- Martin KILLIAN
- Principal Investigator Email
- martin.killlian@chu-st-etienne.fr
- Contact Person Name
- Martin KILLIAN
- Contact Person Email
- martin.killlian@chu-st-etienne.fr
- Site Name
- Hospital Foch
- Department Name
- Médecine interne
- Principal Investigator Name
- Mathilde ROUMIER
- Principal Investigator Email
- m.roumier@hopital-foch.com
- Contact Person Name
- Mathilde ROUMIER
- Contact Person Email
- m.roumier@hopital-foch.com
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine interne
- Principal Investigator Name
- Edouard FLAMARION
- Principal Investigator Email
- edouard.flamarion@aphp.fr
- Contact Person Name
- Edouard FLAMARION
- Contact Person Email
- edouard.flamarion@aphp.fr
- Site Name
- Centre Hospitalier Universitaire De Dijon
- Department Name
- Médecine interne et immunologie clinique
- Principal Investigator Name
- Bernard BONNOTTE
- Principal Investigator Email
- bernard.bonnotte@chu-dijon.fr
- Contact Person Name
- Bernard BONNOTTE
- Contact Person Email
- bernard.bonnotte@chu-dijon.fr
- Site Name
- Centre Hospitalier Bretagne Atlantique
- Department Name
- Médecine interne et maladies infectieuses
- Principal Investigator Name
- Marie GOUSSEFF
- Principal Investigator Email
- marie.gousseff@ch-bretagne-atlantique.fr
- Contact Person Name
- Marie GOUSSEFF
- Contact Person Email
- marie.gousseff@ch-bretagne-atlantique.fr
- Site Name
- Centre Hospitalier Universitaire Grenoble Alpes
- Department Name
- Médecine interne
- Principal Investigator Name
- Laurence BOUILLET
- Principal Investigator Email
- lbouillet@chu-grenoble.fr
- Contact Person Name
- Laurence BOUILLET
- Contact Person Email
- lbouillet@chu-grenoble.fr
- Site Name
- Centre Hospitalier Intercommunal Creteil
- Department Name
- Médecine Interne
- Principal Investigator Name
- Isabelle DELACROIX-SZMANIA
- Principal Investigator Email
- isabelle.delacroix@chicreteil.fr
- Contact Person Name
- Isabelle DELACROIX-SZMANIA
- Contact Person Email
- isabelle.delacroix@chicreteil.fr
- Site Name
- Centre Hospitalier Universitaire De Nantes
- Department Name
- Médecine Interne
- Principal Investigator Name
- Julie GRAVELEAU
- Principal Investigator Email
- julie.graveleau@chu-nantes.fr
- Contact Person Name
- Julie GRAVELEAU
- Contact Person Email
- julie.graveleau@chu-nantes.fr
- Site Name
- Centre Hospitalier Universitaire Reims
- Department Name
- Médecine interne maladies infectieuses et immunologie
- Principal Investigator Name
- Ailsa ROBBINS
- Principal Investigator Email
- arobbins@chu-reims.fr
- Contact Person Name
- Ailsa ROBBINS
- Contact Person Email
- arobbins@chu-reims.fr
- Site Name
- Centre Hospitalier Universitaire Amiens Picardie
- Department Name
- Hématologie
- Principal Investigator Name
- Clément GOURGUECHON
- Principal Investigator Email
- gourguechon.clement@chu-amiens.fr
- Contact Person Name
- Clément GOURGUECHON
- Contact Person Email
- gourguechon.clement@chu-amiens.fr
- Site Name
- University Hospital Of Clermont-Ferrand
- Department Name
- Médecine interne
- Principal Investigator Name
- Marc RUIVARD
- Principal Investigator Email
- mruivard@chu-clermontferrand.fr
- Contact Person Name
- Marc RUIVARD
- Contact Person Email
- mruivard@chu-clermontferrand.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine Interne
- Principal Investigator Name
- Matthieu MAHEVAS
- Principal Investigator Email
- matthieu.mahevas@aphp.fr
- Contact Person Name
- Matthieu MAHEVAS
- Contact Person Email
- matthieu.mahevas@aphp.fr
- Site Name
- Centre Hospitalier Sud Francilien
- Department Name
- Hématologie
- Principal Investigator Name
- Anne-Marie RONCHETTI
- Principal Investigator Email
- anne-marie.ronchetti@chsf.fr
- Contact Person Name
- Anne-Marie RONCHETTI
- Contact Person Email
- anne-marie.ronchetti@chsf.fr
- Site Name
- Centre Hospitalier Victor Dupouy
- Department Name
- Médecine Polyvalente-Médecine interne
- Principal Investigator Name
- Maud PICHON
- Principal Investigator Email
- maud.pichon@ch-argenteuil.fr
- Contact Person Name
- Maud PICHON
- Contact Person Email
- maud.pichon@ch-argenteuil.fr
- Site Name
- Oncopole Claudius Regaud
- Department Name
- Médecine interne
- Principal Investigator Name
- Thibault COMONT
- Principal Investigator Email
- comont.thibault@iuct-oncopole.fr
- Contact Person Name
- Thibault COMONT
- Contact Person Email
- comont.thibault@iuct-oncopole.fr
- Site Name
- Centre Hospitalier Intercommunal De Poissy Saint Germain
- Department Name
- Médecine interne
- Principal Investigator Name
- Mathilde DEVAUX
- Principal Investigator Email
- mathilde.devaux@ght-yvelinesnord.fr
- Contact Person Name
- Mathilde DEVAUX
- Contact Person Email
- mathilde.devaux@ght-yvelinesnord.fr
- Site Name
- Assistance Publique Hopitaux De Paris
- Department Name
- Médecine interne
- Principal Investigator Name
- Nathalie COSTEDOAT CHALUMEAU
- Principal Investigator Email
- nathalie.costedoat@aphp.fr
- Contact Person Name
- Nathalie COSTEDOAT CHALUMEAU
- Contact Person Email
- nathalie.costedoat@aphp.fr
- Site Name
- Hospices Civils De Lyon
- Department Name
- Médecine interne
- Principal Investigator Name
- Maxime FAUTER
- Principal Investigator Email
- maxime.fauter@chu-lyon.fr
- Contact Person Name
- Maxime FAUTER
- Contact Person Email
- maxime.fauter@chu-lyon.fr
- Site Name
- CHU Besancon
- Department Name
- Médecine interne
- Principal Investigator Name
- Nadine MAGY-BERTRAND
- Principal Investigator Email
- nadine.magy@univ-fcomte.fr
- Contact Person Name
- Nadine MAGY-BERTRAND
- Contact Person Email
- nadine.magy@univ-fcomte.fr
Sponsor
Primary sponsor
- Full Name
- Assistance Publique Hopitaux De Paris
- Organisation Type
- Hospital/Clinic/Other health care facility
- Country Of Registered Address
- France
Investigational products
- Investigational Product Name
- Neofordex 40 mg tablets
- Active Substance
- Dexamethasone
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- Oral
- Authorisation Status
- marketingAuthNumber: EU/1/15/1053/001
- Starting Dose
- 40 mg (oral) on Days 1-4
- Dose Levels
- 40 mg once daily on Days 1-4
- Frequency
- Days 1 to 4
- Maximum Dose
- 40 mg per day
- Investigational Product Name
- IMMUNOGLOBULINS, NORMAL HUMAN, FOR INTRAVASCULAR ADM.
- Active Substance
- Human normal immunoglobulin (IV)
- Modality
- Other antibody
- Routes Of Administration
- INTRAVENOUS
- Route
- Intravenous
- Authorisation Status
- marketingAuthNumber: -
- Starting Dose
- 1 g/kg IV on Days 1 and 2
- Dose Levels
- 1 g/kg per day on Day 1 and Day 2
- Frequency
- Days 1 and 2
- Maximum Dose
- 1000 mg/kg per day; max total 2000 mg/kg
- Investigational Product Name
- PREDNISONE
- Active Substance
- Prednisolone (prednisone)
- Modality
- Small molecule
- Routes Of Administration
- ORAL
- Route
- Oral
- Authorisation Status
- marketingAuthNumber: -
- Starting Dose
- 1 mg/kg per day (oral) for 3 weeks
- Dose Levels
- 1 mg/kg per day for 21 days
- Frequency
- Daily for 3 weeks
- Maximum Dose
- 1 mg/kg per day; max total 21 mg/kg
- Combination Treatment
- Yes
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