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A Study to Investigate the Safety and Efficacy of an Anti-IFNγ mAb in Children Affected by Primary Haemophagocytic Lymphohistiocytosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01818492
Recruitment Status : Active, not recruiting
First Posted : March 26, 2013
Last Update Posted : February 26, 2019
Seventh Framework Programme
Information provided by (Responsible Party):
NovImmune SA

Brief Summary:
The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.

Condition or disease Intervention/treatment Phase
Primary Haemophagocytic Lymphohistiocytosis Biological: NI-0501 Phase 2 Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 45 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2/3, Open-label, Single Arm, Multicentre Study to Assess 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
Study Start Date : January 2013
Actual Primary Completion Date : January 2019

Arm Intervention/treatment
Experimental: NI-0501 Biological: NI-0501

Primary Outcome Measures :
  1. Overall response rate [ Time Frame: end of treatment ]

Other Outcome Measures:
  1. Time to response [ Time Frame: any time during the study ]
  2. Durability of response [ Time Frame: any time during the study ]
  3. Survival [ Time Frame: end of the study and beyond ]
  4. Glucocorticoid tapering [ Time Frame: any time during the study ]
  5. Safety and tolerability of NI-0501 in haemophagocytic lymphohistiocytosis [ Time Frame: up to end of the study ]
    • Vital signs
    • Physical examination
    • Recording of adverse events
    • Routine laboratory assessments

Information from the National Library of Medicine

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Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Gender: male and female
  • Age: up to and including 18 years at diagnosis of Haemophagocytic Lymphohistiocytosis
  • Primary HLH patients
  • 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
  • Active Mycobacteria, Histoplasma Capsulatum, Shigella, Salmonella, Campylobacter and Leishmania infections.
  • Presence of malignancy.
  • Concomitant disease or malformation severely affecting the cardiovascular, pulmonary, liver or renal function

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01818492

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United States, Colorado
Children's Hospital Colorado
Aurora, Colorado, United States, 80045-7106
United States, Delaware
Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology
Wilmington, Delaware, United States, 19803
United States, Georgia
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
United States, Massachusetts
Dana-Farber Cancer Institute (DFCI)
Boston, Massachusetts, United States, 02115
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cincinnati Children's Hospital - Division of Immunobiology - Department of Pediatrics
Cincinnati, Ohio, United States, 45229-3039
United States, Texas
Texas Children's Cancer Center
Houston, Texas, United States, 77030
United States, Utah
Primary Children's Hospital
Salt Lake City, Utah, United States, 84113
University Children's Hospital
Münster, Germany, 48149
Azienda Ospedaliero Universitaria Meyer
Florence, Italy, 50139
Istituto Giannina Gaslini
Genoa, Italy, 16147
Azienda Ospedaliera San Gerardo
Monza, Italy, 20900
Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica
Padua, Italy, 35128
Ospedale Pediatrico Bambino Gesu'
Roma, Italy, 00165
Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica
Verona, Italy, 37126
Hospital Sant Joan de Déu
Barcelona, Spain, 08950
Hospital Universitario Vall d'Hebron
Barcelona, Spain, 119-129
Hospital Universitario Niño Jesús
Madrid, Spain, 28009
Karolinska University Hospital
Stockholm, Sweden, 17176
United Kingdom
Great Ormond Street Hospital - Department of Haematology
London, United Kingdom, WC1N 3JH
Sponsors and Collaborators
NovImmune SA
Seventh Framework Programme

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Responsible Party: NovImmune SA Identifier: NCT01818492     History of Changes
Other Study ID Numbers: NI-0501-04
2012-003632-23 ( EudraCT Number )
First Posted: March 26, 2013    Key Record Dates
Last Update Posted: February 26, 2019
Last Verified: February 2019

Keywords provided by NovImmune SA:

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