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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT01818492
Recruitment Status : Recruiting
First Posted : March 26, 2013
Last Update Posted : August 27, 2018
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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 32 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
Estimated Primary Completion Date : December 2018

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

Contact: Cristina de Min, MD +41 61 201 1322

United States, Colorado
Children's Hospital Colorado Recruiting
Aurora, Colorado, United States, 80045-7106
Contact: Timothy Garrington, MD    720-777-6740   
Principal Investigator: Timothy Garrington, MD         
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   
Principal Investigator: Jonathan Powell, MD         
United States, Georgia
Children's Healthcare of Atlanta Recruiting
Atlanta, Georgia, United States, 30329
Contact: Shanmuganathan Chandrakasan, MD    404-727-8877   
Principal Investigator: Shanmuganathan Chandrakasan, MD         
United States, Massachusetts
Dana-Farber Cancer Institute (DFCI) Recruiting
Boston, Massachusetts, United States, 02115
Contact: Barbara A Degar, MD    617-632-5186   
Principal Investigator: Barbara A Degar, MD         
United States, North Carolina
University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Kimberly Kasow, MD    919-962-8733   
Principal Investigator: Kimberly Kasow, 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   
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   
Contact: Maria Diaz    1-832-824-1520   
Principal Investigator: Carl Allen, MD, PhD         
United States, Utah
Primary Children's Hospital Recruiting
Salt Lake City, Utah, United States, 84113
Contact: Mark N Fluchel, MD    801-662-4710   
Principal Investigator: Mark N Fluchel, MD         
University Children's Hospital Recruiting
Münster, Germany, 48149
Contact: Claudia Rössig, Prof. Dr. Med.    0049 2518347742   
Principal Investigator: Claudia Rössig, Prof. Dr. Med.         
Azienda Ospedaliero Universitaria Meyer Recruiting
Florence, Italy, 50139
Contact: Elena Sieni, MD    +39 055-5662 ext 524   
Principal Investigator: Elena Sieni, MD         
Istituto Giannina Gaslini Recruiting
Genoa, Italy, 16147
Contact: Concetta Micalizzi, MD    0039_010_5636 ext 715   
Principal Investigator: Concetta Micalizzi, MD         
Azienda Ospedaliera San Gerardo Recruiting
Monza, Italy, 20900
Contact: Carmelo Rizzari, MD    0039_039_233 ext 3513   
Principal Investigator: Carmelo Rizzari, MD         
Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica Recruiting
Padua, Italy, 35128
Contact: MariaCaterina Putti, MD    0039 049 8213535   
Principal Investigator: Mariacaterina Putti, MD         
Ospedale Pediatrico Bambino Gesu' Recruiting
Roma, Italy, 00165
Contact: Franco Locatelli, MD    0039 06 68592678   
Principal Investigator: Franco Locatelli, MD         
Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica Recruiting
Verona, Italy, 37126
Contact: Simone Cesaro, MD    0039 045 812 7874   
Principal Investigator: Simone Cesaro, MD         
Hospital Universitario Vall d'Hebron Recruiting
Barcelona, Spain, 119-129
Contact: Jose L Dapena, MD    0034 934893093      
Principal Investigator: Jose L Dapena, MD         
Hospital Sant Joan de Déu Recruiting
Esplugues De Llobregat (Barcelona), Spain, 08950
Contact: Laia Alsina Manrique de Lara, MD    0034 932804000 ext 3330   
Principal Investigator: Laia Alsina, MD         
Hospital Universitario Niño Jesús Recruiting
Madrid, Spain, 28009
Contact: Julian Sevilla, MD    0034 915035938   
Principal Investigator: Julian Sevilla, MD         
Karolinska University Hospital Recruiting
Stockholm, Sweden, 17176
Contact: Jan-Inge Henter, MD    0046851772484      
Principal Investigator: Jan-Inge Henter         
United Kingdom
Great Ormond Street Hospital - Department of Haematology Recruiting
London, United Kingdom, WC1N 3JH
Contact: Anupama Rao, MD    00442074059200 ext 7918   
Principal Investigator: Anupama Rao, MD         
Sponsors and Collaborators
NovImmune SA
Seventh Framework Programme

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: August 27, 2018
Last Verified: August 2018

Keywords provided by NovImmune SA:

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