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OPPUS: A Non-Interventional National Study in Pediatric Patients With Unexplained Enlarged Spleen

Sponsor
Sanofi (Industry)
Overall Status
Recruiting
CT.gov ID
NCT04845958
Collaborator
(none)
150
Enrollment
40
Locations
39
Anticipated Duration (Months)
3.8
Patients Per Site
0.1
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Primary Objective:

To assess prevalence of Gaucher disease (GD) diagnosed in pediatric patients presenting with unexplained splenomegaly (SMG) after exclusion of first intention-diagnoses (e.g. portal hypertension, haematological malignancy, hemolytic anemia, infection) based on clinical examination and routine biological tests (full blood count, reticulocytes, liver tests, abdominal ultrasound, Coombs test and Epstein Barr virus serology).

Secondary Objectives:

  • To describe the rate of each identified disease category and the rate of patients with no final diagnosis at the end of the study in pediatric patients with unexplained SMG after exclusion of first intention diagnoses

  • To describe the characteristics (clinical, lab, genetics) of all pediatric patients included in the study and to describe the characteristics subdivided by identified disease category and absence of final diagnosis at the end of the study

Condition or Disease Intervention/Treatment Phase

    Detailed Description

    The planned duration of this study is 39 months, which includes 36 months of patient recruitment.

    Study Design

    Study Type:
    Observational
    Anticipated Enrollment :
    150 participants
    Observational Model:
    Cohort
    Time Perspective:
    Cross-Sectional
    Official Title:
    An Observational National Pediatric Study on Prevalence of Unexplained Splenomegaly
    Actual Study Start Date :
    Jun 30, 2021
    Anticipated Primary Completion Date :
    Sep 30, 2024
    Anticipated Study Completion Date :
    Sep 30, 2024

    Arms and Interventions

    Arm Intervention/Treatment
    Pediatric Patients with Unexplained Enlarged Spleen

    Outcome Measures

    Primary Outcome Measures

    1. Percentage of patients diagnosed with GD among enrolled patients [Up to 3 months after inclusion]

      Diagnosis of GD based on deficient β-glucocerebrosidase activity in peripheral blood leukocytes or other nucleated cells, or genetic analysis.

    Secondary Outcome Measures

    1. Rate of each identified disease category at the end of the study among enrolled patients [Up to 3 months after inclusion]

    2. Rate of patients with no final diagnosis at the end of the study among enrolled patients [Up to 3 months after inclusion]

    3. Number of patients based on specific char. (clinical, lab, genetics) [Up to 3 months after inclusion]

      Detailed characteristics of all patients included in the study (clinical, lab, genetics) will be evaluated

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    0 Years to 18 Years
    Sexes Eligible for Study:
    All
    Accepts Healthy Volunteers:
    No
    Inclusion criteria:

    • Patient under the age of 18 years

    • Patient with unexplained SMG (SMG defined as a palpable spleen, already known or discovered for the first time) and who has undergone tests to eliminate obvious causes of SMG

    Exclusion criteria:

    Patient with any obvious cause of SMG as described by clinical examination and/or lab or imaging test available in medical records and/or having been diagnosed with any of the following conditions:

    1. hemolytic anemia

    2. hematological malignancy

    3. portal hypertension

    4. infectious disease associated with SMG (Cytomegalovirus, Epstein Barr virus, leishmaniasis or other obvious infectious cause revealed by the medical history)

    The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Investigational Site Number 2500036 Amiens France 80054
    2 Investigational Site Number 2500014 Angers France 49933
    3 Investigational Site Number 2500034 Argenteuil France 95107
    4 Investigational Site Number 2500039 Bayonne France 64100
    5 Investigational Site Number 2500015 Besançon France 25030
    6 Investigational Site Number 2500003 Bordeaux France 33000
    7 Investigational Site Number 2500038 Brest France 29609
    8 Investigational Site Number 2500026 Caen France 14033
    9 Investigational Site Number 2500018 Chambéry France 73000
    10 Investigational Site Number 2500009 Clermont-Ferrand France 63000
    11 Investigational Site Number 2500008 Colombes France 92700
    12 Investigational Site Number 2500028 Créteil France 94000
    13 Investigational Site Number 2500006 Dijon France 21079
    14 Investigational Site Number 2500010 Gleizé France 69400
    15 Investigational Site Number 2500031 Jossigny France 77600
    16 Investigational Site Number 2500040 La Rochelle France 17000
    17 Investigational Site Number 2500007 La Tronche France 38700
    18 Investigational Site Number 2500030 Limoges France 87042
    19 Investigational Site Number 2500001 Lyon France 69008
    20 Investigational Site Number 2500032 Mantes-la-Jolie France 78200
    21 Investigational Site Number 2500005 Marseille France 13005
    22 Investigational Site Number 2500027 Meaux France 77100
    23 Investigational Site Number 2500002 Montpellier France 34090
    24 Investigational Site Number 2500021 Nantes France 44000
    25 Investigational Site Number 2500019 Nice France 06200
    26 Investigational Site Number 2500022 Nîmes France 30029
    27 Investigational Site Number 2500016 Paris France 75012
    28 Investigational Site Number 2500013 Perpignan France 66000
    29 Investigational Site Number 2500035 Poissy France 78300
    30 Investigational Site Number 2500011 Poitiers France 86000
    31 Investigational Site Number 2500029 Reims France 51100
    32 Investigational Site Number 2500023 Rennes France 35203
    33 Investigational Site Number 2500037 Roubaix France 59056
    34 Investigational Site Number 2500024 Rouen France 76031
    35 Investigational Site Number 2500017 Saint-Priest-en-Jarez France 42270
    36 Investigational Site Number 2500004 Strasbourg France 67200
    37 Investigational Site Number 2500033 Toulouse France
    38 Investigational Site Number 2500020 Tours France
    39 Investigational Site Number 2500025 Valenciennes France 59300
    40 Investigational Site Number 2500012 Vandœuvre-lès-Nancy France 54500

    Sponsors and Collaborators

    • Sanofi

    Investigators

    • Study Director: Clinical Sciences & Operations, Sanofi

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    Sanofi
    ClinicalTrials.gov Identifier:
    NCT04845958
    Other Study ID Numbers:
    • OBS16092
    First Posted:
    Apr 15, 2021
    Last Update Posted:
    Aug 22, 2022
    Last Verified:
    Aug 1, 2022
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Additional relevant MeSH terms:
    Gaucher Disease
    Niemann-Pick Disease, Type A
    Niemann-Pick Diseases
    Niemann-Pick Disease, Type C
    Splenomegaly

    Study Results

    No Results Posted as of Aug 22, 2022