Overview
Sponsor-declared trial summary
Phase
Therapeutic exploratory (Phase II)
Status
Ongoing, recruiting
Participants planned
21
Countries
1
Sites
8
paraneoplastic sensory neuropathy with anti-Hu antibodies
Evaluation of the efficacy at 3 months of an early immunotherapy based on intravenous immunoglobulins, Cyclophosphamide and Methylprednisolone in patients with paraneoplastic sensitive neuronopathy with anti-Hu antibodies.
Key facts
- Sponsor
- Assistance Publique Hopitaux De Paris
- Participant type
- Patients
- Age range
- 18-64 years, 65+ years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nervous System Diseases [C10]
- Trial duration
- 10 Jan 2025 → ongoing
- Decision date (initial)
- 2024-03-18
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
- Funding sources
- ministry of health · AP-HP
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Therapy, Efficacy
Evaluation of the efficacy at 3 months of an early immunotherapy based on intravenous immunoglobulins, Cyclophosphamide and Methylprednisolone in patients with paraneoplastic sensitive neuronopathy with anti-Hu antibodies.
Secondary objectives 3
- Evaluation efficacy of the immunotherapy under study at 3 and 6 months
- Evaluation the percentage of patients alive and without tumour progression at 6 months
- Evaluation treatment tolerance
Conditions and MedDRA coding
paraneoplastic sensory neuropathy with anti-Hu antibodies
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10072106 | Paraneoplastic neurological syndrome | 100000004852 |
Regulatory references
- Plan to share IPD
- No
- IPD plan description
- Non Applicable
| EU CT number | Title | Sponsor |
|---|---|---|
| 2023-506942-22-00 | Early immunotherapy with intravenous immunoglobulin, cyclophosphamide and methylprednisolone in patients with anti-Hu-associated paraneoplastic sensory neuropathy (NESPA). | Assistance Publique Hopitaux De Paris |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 9
- Adult patients with anti-Hu antibody paraneoplastic sensory neuropathy
- Age ≥ 18 years old
- "Possible" sensory neuropathy according to the criteria of Camdessanché et al.
- dominant picture of sensitive ataxia (damage to the central nervous system and/or neuromuscular junction is accepted, provided that it has a minor impact on the patient's disability)
- Positive anti-Hu antibodies in blood and/or cerebrospinal fluid
- Outpatient (Modified Rankin Score (mRS) 2 or 3)
- Electroneuromyogram (ENMG) leading to diagnosis of sensory neuronopathy less than 3 months old
- Inform consent Free, written and signed
- Registered with a social security scheme or beneficiary (except AME)
Exclusion criteria 12
- Known hypersensitivity to one of the treatments under study, to their metabolites, or to one of the excipients
- Absolute contraindications to IVIg: selective IgA deficiency, known thrombophilia, patients suffering from type I or II hyperprolinaemia, hypersensitivity to human immunoglobulins
- Absolute contraindications to cyclophosphamide: yellow fever vaccination within three months of inclusion, acute urinary tract infection, acute bone marrow failure
- More than two courses of intravenous Immunoglobulins administered in the 3 months prior to recruitment
- Other concomitant immunotherapy
- Other causes of immunodepression (acquired or congenital)
- Treatment with checkpoint inhibitors in progress or completed less than 3 months previously
- Women of childbearing age without effective contraception, pregnant or breastfeeding
- History of psychiatric or general illness that may contraindicate treatment
- Patients unable to complete the follow-up required by the study
- Patients under tutorship or curatorship
- Patient deprived of liberty by a judicial or administrative decision
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- Percentage of patients with clinical improvement on the Overall Neuropathy Limitations Scale (ONLS) at 3 months
Secondary endpoints 7
- Percentage of patients with clinical improvement on the ONLS scale at 3 and 6 months
- Percentage of patients with improvement of the ataxic component on the Score of Ataxia scale at 3 and 6 months
- Percentage of patients with improvement in neuropathic pain on the Numeric Rating Scale (NRS) at 3 and 6 months
- Percentage of patients with functional improvement on the modified Rankin Score (mRS) at 3 and 6 month
- Percentage of patients with functional improvement on the Barthel Index (BI) at 3 and 6 months
- Percentage of patients alive and without tumour progression at 6 months
- Tolerability of treatment will be assessed by the frequency and severity of expected and unexpected adverse events recorded during treatment.
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 3
CYCLOPHOSPHAMIDE SANDOZ 1000 mg, poudre pour solution injectable ou pour perfusion
PRD5386164 · Product
- Active substance
- Cyclophosphamide
- Pharmaceutical form
- SOLUTION FOR INJECTION/INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 1 g gram(s)
- Max total dose
- 6 g gram(s)
- Max treatment duration
- 6 Day(s)
- Authorisation status
- Authorised
- ATC code
- L01AA01 — CYCLOPHOSPHAMIDE
- Marketing authorisation
- 34009 550 014 8 5
- MA holder
- SANDOZ
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
SOLUMEDROL 1 g, poudre et solvant pour solution injectable
PRD457536 · Product
- Active substance
- Methylprednisolone Hemisuccinate
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 1 g gram(s)
- Max total dose
- 18 g gram(s)
- Max treatment duration
- 18 Day(s)
- Authorisation status
- Authorised
- ATC code
- H02AB04 — METHYLPREDNISOLONE
- Marketing authorisation
- 34009 386 772 2 5
- MA holder
- PFIZER HOLDING FRANCE
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
CLAIRYG 50 mg/ml, solution pour perfusion
PRD491313 · Product
- Active substance
- Human Normal Immunoglobulin (IV)
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 200 g gram(s)
- Max total dose
- 2200 g gram(s)
- Max treatment duration
- 11 Day(s)
- Authorisation status
- Authorised
- ATC code
- J06BA02 — IMMUNOGLOBULINS, NORMAL HUMAN, FOR INTRAVASCULAR ADM.
- Marketing authorisation
- 34009 576 190 4 6
- MA holder
- LFB BIOMEDICAMENTS
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Auxiliary 1
MESNA EG 100 mg/ml, solution injectable pour perfusion
PRD513855 · Product
- Active substance
- Mesna
- Pharmaceutical form
- SOLUTION FOR INFUSION
- Route of administration
- INTRAVENOUS
- Max daily dose
- 1.2 g gram(s)
- Max total dose
- 7.2 g gram(s)
- Max treatment duration
- 6 Day(s)
- Authorisation status
- Authorised
- ATC code
- V03AF01 — MESNA
- Marketing authorisation
- NL 29384
- MA holder
- EG LABO LABORATOIRES EUROGENERICS - DO NOT USE
- MA country
- France
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Assistance Publique Hopitaux De Paris
- Sponsor organisation
- Assistance Publique Hopitaux De Paris
- Address
- Porte 23, 1 Avenue Claude Vellefaux 1 Avenue Claude Vellefaux
- City
- Paris Cedex 10
- Postcode
- 75475
- Country
- France
Scientific contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Dimitri PSIMARAS
Public contact point
- Organisation
- Assistance Publique Hopitaux De Paris
- Contact name
- Dimitri PSIMARAS
Locations
1 EU/EEA country · 8 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| France | Ongoing, recruiting | 21 | 8 |
| Rest of world | — | 0 | — |
Investigational sites
Centre Hospitalier Universitaire De Saint Etienne
Neurology, Avenue Albert Raimond, 42270, Saint Priest En Jarez
Centre Hospitalier Universitaire De Nantes
Rare neuromuscular diseases reference centre, 1 Place Alexis Ricordeau, 44000, Nantes
Les Hopitaux Universitaires De Strasbourg
Neurology, 1 Avenue Moliere, Bp 49, Strasbourg Cedex 2
Assistance Publique Hopitaux De Paris
Neurology, 47 Boulevard De L Hopital, 75651, Paris Cedex 13
Assistance Publique Hopitaux De Marseille
Neuromuscular diseases and ALS, 264 Rue Saint Pierre, 13005, Marseille
Hospices Civils De Lyon
neuro-oncology, 59 Boulevard Pinel, 69500, Bron
Centre Hospitalier Universitaire De Toulouse
Neurology, 1 Place Du Docteur Joseph Baylac, 31300, Toulouse
Centre Hospitalier Et Universitaire De Limoges
Neurology, 2 Avenue Martin Luther King, 87000, Limoges
Country notifications
Trial-start, recruitment-start, end and early-termination notifications submitted per Member State
| Country | Trial start | Trial end | Recruitment start | Recruitment end | Early termination |
|---|---|---|---|---|---|
| France | 2025-01-10 | 2025-01-10 |
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 12 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | D1_Protocol 2023-506942-22-00_v1-0_20230922_Public | 1 |
| Protocol (for publication) | D1_Protocol-Annexe-liste traitements concomitants_formulaire EIG_FR_2023-506942-22-00 | 1 |
| Protocol (for publication) | D1_Protocol-Formulaire cancers secondaires_2023-506942-22-00 | 1 |
| Protocol (for publication) | D1_Protocol-formulaire EIG_2023-506942-22-00 | 1 |
| Protocol (for publication) | D1_Protocol-Formulaire notification grossesse_2023-506942-22-00 | 1 |
| Recruitment arrangements (for publication) | K1_Recruitment Arrangements | 1 |
| Subject information and informed consent form (for publication) | L1_SIS-ICF_Patient adults | 2-0 |
| Subject information and informed consent form (for publication) | L2_Carte patient | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_ SmPC SOLUMEDROL 1g | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC CLAIRYG | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | E2_SmPC Cyclophosphamide SANDOZ 1000 mg | 1 |
| Synopsis of the protocol (for publication) | D1_Protocol synopsis_FR_2023-506942-22-00 | 1 |
Application history
3 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2023-11-10 | France | Acceptable 2024-03-11
|
2024-03-18 |
| 2 | NON SUBSTANTIAL MODIFICATION | NSM-1 | 2024-06-26 | France | Acceptable 2024-03-11
|
2024-06-26 |
| 3 | SUBSTANTIAL MODIFICATION | SM-2 | 2025-02-21 | France | Acceptable 2025-04-17
|
2025-04-23 |