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A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis in Whom the Disease Has Reactivated or an Unsatisfactory Response Has Been Achieved

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by NovImmune SA
Sponsor:
Collaborator:
7th Framework Programme
Information provided by (Responsible Party):
NovImmune SA
ClinicalTrials.gov Identifier:
NCT01818492
First received: March 21, 2013
Last updated: October 24, 2014
Last verified: May 2014
  Purpose

The purpose of this study is to assess the safety, tolerability and preliminary efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug will be administered on top of a glucocorticosteroid, which is usually part of the current recommended treatment.


Condition Intervention Phase
Primary Hemophagocytic Lymphohistiocytosis
Biological: NI-0501
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Pilot, Open-label, Single Arm, Multicentre Study to Explore Safety, Tolerability, Pharmacokinetics and Efficacy of Intravenous Multiple Administrations of NI-0501, an Anti-interferon Gamma (Anti-IFNγ) Monoclonal Antibody, in Paediatric Patients With Primary Haemophagocytic Lymphohistiocytosis in Whom the Disease Has Reactivated or an Unsatisfactory Response Has Been Achieved

Resource links provided by NLM:


Further study details as provided by NovImmune SA:

Primary Outcome Measures:
  • Safety and tolerability of NI-0501 in haemophagocytic lymphohistiocytosis [ Time Frame: 12 weeks ] [ Designated as safety issue: Yes ]
    • Vital signs
    • Physical examination
    • Recording of adverse Events
    • Routine laboratory assessments


Other Outcome Measures:
  • Preliminary efficacy [ Time Frame: 8 weeks ] [ Designated as safety issue: No ]
    Evolution of clinical signs (such as fever, splenomegaly, central nervous system symptoms) and laboratory parameters (such as complete blood cell count, fibrinogen, serum triglycerides, ferritin, soluble Interleukin-2 receptor levels), which characterize the disease, to assess response and time to response.


Estimated Enrollment: 10
Study Start Date: January 2013
Estimated Primary Completion Date: December 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: NI-0501 Biological: NI-0501

  Eligibility

Ages Eligible for Study:   up to 18 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Gender: male and female
  • Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis
  • Primary HLH patients who have previously achieved at least partial response to conventional induction therapy and who present a reactivation of the disease or an unsatisfactory response has been achieved
  • Patient (if ≥ 18 years old), or patient's legal representative(s) must have signed informed consent

Exclusion Criteria:

  • Diagnosis of secondary Haemophagocytic Lymphohistiocytosis consequent to a proven rheumatic or neoplastic disease.
  • Body weight < 3 kg.
  • Patients treated with biologics within a specific timeframe
  • Reactivation associated with isolated or multiple acute organ failure(s) (heart, lung or kidney) requiring aggressive therapy such as high dose of inotropic drugs, circulatory assistance, hemofiltration or haemodialysis, artificial ventilation.
  • Active infections.
  • History of malignancy.
  • Concomitant disease or malformation
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01818492

Contacts
Contact: Cristina de Min, MD +41-22-593- ext 5116 cdemin@novimmune.com

Locations
United States, Delaware
Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology Recruiting
Wilmington, Delaware, United States, 19803
Contact: Jonathan Powell, MD    302-651-5505    Jonathan.Powell@nemours.org   
Principal Investigator: Jonathan Powell, MD         
United States, Massachusetts
Dana-Farber Cancer Institute (DFCI) Recruiting
Boston, Massachusetts, United States, 02115
Contact: Barbara A Degar, MD    617-632-5186    Barbara_Degar@dfci.harvard.edu   
Principal Investigator: Barbara A Degar, MD         
United States, Ohio
Cincinnati Children's Hospital - Division of Immunobiology - Department of Pediatrics Recruiting
Cincinnati, Ohio, United States, 45229-3039
Contact: Michael Jordan, MD    513-636-7287    Michael.Jordan@cchmc.org   
Principal Investigator: Michael Jordan, MD         
United States, Texas
Texas Children's Cancer Center Recruiting
Houston, Texas, United States, 77030
Contact: Carl Allen, MD, PhD    832-824-4312    ceallen@txch.org   
Principal Investigator: Carl Allen, MD, PhD         
Austria
St. Anna Children's Hospital Recruiting
Vienna, Austria, A-1090
Contact: Wolfgang Holter, MD    0043 1 40 170 1250    wolfgang.holter@stanna.at   
Principal Investigator: Wolfgang Holter, MD         
Czech Republic
Charles University Prague University Hospital Motol - Department of Pediatric Hematology and Oncology Recruiting
Prague, Czech Republic, 150 06
Contact: Petr Sedlacek, MD    0042 0224436401    petr.sedlacek@lfmotol.cuni.cz   
Principal Investigator: Petr Sedlacek, MD         
Germany
University Children's Hospital Recruiting
Munster, Germany, 48149
Contact: Herbert Jurgens, MD    0049 2518347941    jurgh@uni-muenster.de   
Principal Investigator: Herbert Jurgens, MD         
Italy
Azienda Ospedaliero Universitaria Meyer Recruiting
Florence, Italy, 50139
Contact: Franco Bambi, MD    +39 055-5662 ext 991    f.bambi@meyer.it   
Principal Investigator: Franco Bambi, MD         
Istituto Giannina Gaslini Recruiting
Genoa, Italy, 16147
Contact: Concetta Micalizzi, MD    0039_010_5636 ext 715    concettamicalizzi@ospedale-gaslini.ge.it   
Principal Investigator: Concetta Micalizzi, MD         
Azienda Ospedaliera San Gerardo Recruiting
Monza, Italy, 20900
Contact: Carmelo Rizzari, MD    0039_039_233 ext 3513    c.rizzari@hsgerardo.org   
Principal Investigator: Carmelo Rizzari, MD         
Azienda Ospedaliera Santobono Pausilipon Recruiting
Naples, Italy, 80123
Contact: Carmen De Fusco, MD    0039_081_2205410    carmendefusco@iol.it   
Principal Investigator: Carmen De Fusco, MD         
Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica Recruiting
Padua, Italy, 35128
Contact: MariaCaterina Putti, MD    0039 049 8213535    mariacaterina.putti@unipd.it   
Principal Investigator: Mariacaterina Putti, MD         
Ospedale Pediatrico Bambino Gesu' Recruiting
Roma, Italy, 00165
Contact: Franco Locatelli, MD    0039 06 68592678    franco.locatelli@opbg.net   
Principal Investigator: Franco Locatelli, MD         
Policlinico G.B. Rossi - U.O.C. Oncoematologia Pediatrica Recruiting
Verona, Italy, 37134
Contact: Simone Cesaro, MD    0039 045 8124931    simone.cesaro@ospedaleuniverona.it   
Principal Investigator: Simone Cesaro, MD         
Spain
Hospital Universitario Vall d'Hebron Recruiting
Barcelona, Spain, 119-129
Contact: Jose Sanchez de Toledo, MD    0034 934893093    jossanchez@vhebron.net   
Principal Investigator: Jose Sanchez de Toledo, MD         
Hospital Universitario Cruces Recruiting
Bilbao, Spain, 48903
Contact: Itziar Astigarraga, MD    0034 946006357    itziar.astigarraga@osakidetza.net   
Principal Investigator: Itziar Astigarraga, MD         
Hospital Sant Joan de Déu Recruiting
Esplugues de Llobregat (Barcelona), Spain, 08950
Contact: Laia Alsina Manrique de Lara, MD    0034 932804000 ext 3330    lalsina@hsjdbcn.org   
Principal Investigator: Laia Alsina, MD         
Hospital Universitario Niño Jesús Recruiting
Madrid, Spain, 28009
Contact: Alvaro Lassaletta Atienza, MD    0034 915035938    lassaalvaro@yahoo.com   
Principal Investigator: Alvaro Lassaletta, MD         
Turkey
Hacettepe University - Paediatric Hematology Recruiting
Ankara, Turkey, 06100
Contact: Sule Unal, MD    0090 532 526 37 49    suleunal@hacettepe.edu.tr   
Principal Investigator: Sule Unal, MD         
Sponsors and Collaborators
NovImmune SA
7th Framework Programme
  More Information

No publications provided

Responsible Party: NovImmune SA
ClinicalTrials.gov Identifier: NCT01818492     History of Changes
Other Study ID Numbers: NI-0501-04, 2012-003632-23
Study First Received: March 21, 2013
Last Updated: October 24, 2014
Health Authority: Italy: The Italian Medicines Agency
Austria: Federal Office for Safety in Health Care
Czech Republic: State Institute for Drug Control
Germany: Paul Ehrlich Institute, PEI
Spain: Agencia Espanola de Medicamentos y Productos Sanitarios AEMPS
United States: Food and Drug Administration

Additional relevant MeSH terms:
Lymphohistiocytosis, Hemophagocytic
Histiocytosis
Histiocytosis, Non-Langerhans-Cell
Lymphatic Diseases

ClinicalTrials.gov processed this record on November 24, 2014