Early immunotherapy with intravenous immunoglobulin, cyclophosphamide and methylprednisolone in patients with anti-Hu-associated paraneoplastic sensory neuropathy (NESPA).

Phase 1

• Conditions: paraneoplastic sensory neuropathy with anti-Hu antibodies; MedDRA version: 20.0Level: PTClassification code: 10072106Term: Paraneoplastic neurological syndrome Class: 100000004852; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: CTIS2023-506942-22-01
• Lead Sponsor: Assistance Publique Hopitaux De Paris

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2023-11-10
• Last Update Posted: 22 July 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 21

Inclusion Criteria

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), Start of neurological symptoms less than 3 months ago, Inform consent Free, written and signed, Registered with a social security scheme or beneficiary (except AME)

Exclusion Criteria

Known hypersensitivity to one of the treatments under study, to their metabolites, or to one of the excipients, 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, 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: 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 Objective: 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;Primary end point(s): Percentage of patients with clinical improvement on the Overall Neuropathy Limitations Scale (ONLS) at 3 months

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s):Percentage of patients with clinical improvement on the ONLS scale at 3 and 6 months;Secondary end point(s):Percentage of patients with improvement of the ataxic component on the Score of Ataxia scale at 3 and 6 months;Secondary end point(s):Percentage of patients with improvement in neuropathic pain on the Numeric Rating Scale (NRS) at 3 and 6 months;Secondary end point(s):Percentage of patients with functional improvement on the modified Rankin Score (mRS) at 3 and 6 month;Secondary end point(s):Percentage of patients with functional improvement on the Barthel Index (BI) at 3 and 6 months;Secondary end point(s):Percentage of patients alive and without tumour progression at 6 months;Secondary end point(s):Tolerability of treatment will be assessed by the frequency and severity of expected and unexpected adverse events recorded during treatment.