NCT03274375

Brief Summary

The purpose of the study is to assess the efficacy of immunoadsorption therapy (IA) on improving the neurological status of severe pediatric anti-NMDAR encephalitis patients.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
20

participants targeted

Target at below P25 for phase_2

Timeline
1mo left

Started Jun 2021

Longer than P75 for phase_2

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress99%
Jun 2021Jun 2026

First Submitted

Initial submission to the registry

July 24, 2017

Completed
1 month until next milestone

First Posted

Study publicly available on registry

September 6, 2017

Completed
3.8 years until next milestone

Study Start

First participant enrolled

June 23, 2021

Completed
3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2024

Completed
1.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

June 1, 2026

Expected
Last Updated

July 13, 2023

Status Verified

July 1, 2023

Enrollment Period

3 years

First QC Date

July 24, 2017

Last Update Submit

July 12, 2023

Conditions

Anti-NMDAR Encephalitis

Keywords

Anti-NMDAR encephalitisIA adsorptionPaediatric

Outcome Measures

Primary Outcomes (2)

  • Change in Neurological status evaluated with the Pediatric Cerebral Performance Category Scale (PCPCS)

    at least reduction of 1 point in PCPCS between the two evaluations is expected

    before and after the 10 IA sessions, 28 days maximum

  • Change in Neurological status evaluated with the modified Rankin Scale (mRS)

    at least reduction of 1 point in mRS between the two evaluations is expected

    before and after the 10 IA sessions, 28 days maximum

Secondary Outcomes (72)

  • Need of hospitalization in ICU and pediatric neurology unit

    28 days

  • Duration of hospitalization in ICU and pediatric neurology unit

    28 days

  • Need for mechanical ventilation

    28 days

  • Need for vasopressive treatment

    28 days

  • Time of recovery of independent daily-life activities

    28 days

  • +67 more secondary outcomes

Study Arms (1)

IA session

EXPERIMENTAL

* 4 Rituximab injections * 10 IA sessions

Drug: IA sessionDrug: Rituximab

Interventions

IA sessionDRUG

10 IA sessions performed in 28 days maximum, using TherasorbTM adsorbers which contain sheep derived polyvalent antihuman-immunoglobulin coupled to SepharoseTM CL-4B.

IA session
RituximabDRUG

Concomitantly, Rituximab will be given each week for 4 weeks (one injection by week +/- 3 days): * at least 1 day before each IA session * the last injection will occur after the last session IA (minimum one day after)

IA session

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age: 0-18 years inclusive
  • Autoimmune encephalitis with positive anti-NMDAR antibodies in CSF (definite anti-NMDAR encephalitis according to Graus's criteria (Graus et al., 2016).
  • Parents or legal guardians signed the Informed consent form
  • Social insurance affiliation

You may not qualify if:

  • Autoimmune encephalitis without NMDAR antibodies
  • PCPCS and mRS scores under 4 after first-line therapy
  • Contraindication to perform central vascular access
  • Pregnancy, breastfeeding or absence of effective contraception (including abstinence) in a pubertal patient.
  • Contraindication to perform IA therapy :
  • Clinical conditions that prohibit transitory volume changes
  • Indications that prohibit anticoagulation using Heparin and/or ACD-A solutions
  • History of hypercoagulability
  • Generalized viral, bacterial and/or mycotic infections
  • Severe immune deficiencies (e.g. AIDS)
  • Suspected allergies against sheep antibodies or agarose

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Hôpital Necker Enfants-Malades

Paris, 75015, France

RECRUITING

MeSH Terms

Conditions

Anti-N-Methyl-D-Aspartate Receptor Encephalitis

Interventions

Rituximab

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous SystemNervous System NeoplasmsNeoplasms by SiteNeoplasmsParaneoplastic SyndromesEncephalitisBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesNeurodegenerative DiseasesNeuroinflammatory DiseasesAutoimmune Diseases of the Nervous SystemAutoimmune DiseasesImmune System Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Murine-DerivedAntibodies, MonoclonalAntibodiesImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Rémi SALOMON, Md, PhD

    Assistance Publique - Hôpitaux de Paris

    STUDY CHAIR

Central Study Contacts

Isabelle DESGUERRE, MD, PhD

CONTACT

+33 1.44.49.41.42isabelle.desguerre@aphp.fr

Aminata TRAORE

CONTACT

+33 1 48 19 27 34aminata.traore6@aphp.fr

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

July 24, 2017

First Posted

September 6, 2017

Study Start

June 23, 2021

Primary Completion

July 1, 2024

Study Completion (Estimated)

June 1, 2026

Last Updated

July 13, 2023

Record last verified: 2023-07

Data Sharing

IPD Sharing
Will not share

Locations

FR(1)