Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis
NCT ID: NCT01818492
Last Updated: 2023-02-21
Study Results
Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.
View full resultsBasic Information
Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.
COMPLETED
PHASE2/PHASE3
45 participants
INTERVENTIONAL
2013-07-31
2019-01-31
Brief Summary
Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.
Related Clinical Trials
Explore similar clinical trials based on study characteristics and research focus.
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
NCT03312751
Evaluation of IFNγ and Inflammatory Mediators in Patients With Hemophagocytic Lymphohistiocytosis
NCT06047210
Study of Immune Globulin Intravenous (Human) GC5107 in Pediatric Subjects With Primary Humoral Immunodeficiency
NCT04565015
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
NCT00634569
Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety, and Tolerability of Inebilizumab in Pediatric Participants With IgG4-RD
NCT07222553
Detailed Description
Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.
All participants in the NI-0501-04 study (NCT01818492) were invited to participate in the long-term follow-up study NI-0501-05 (NCT02069899). For the primary completion date, mentioned here, we refer to the NI-0501-04 study, even though in accordance with the NI-0501-04 study objectives, namely the assessment of long-term efficacy and safety endpoints, the study analyses also included data collected in the long-term follow-up study NI-0501-05. Hence these data are reported together. Study NI-0501-05 accepts patients from NI-0501-04 and NI-0501-06. Data collection for the patients from NI-0501-04 is completed.
The primary efficacy and safety analyses are based on the regulatory cut-off date of 20 July 2017. Refer to the publication in N Engl J Med 2020 May 7; 382 (19):1811-1822. Follow-on analyses have been conducted on all patients enrolled in the study, i.e. including the patients enrolled after the cut-off date of 20 July 2017. The results reported here refer to the totality of the 45 patients enrolled.
Conditions
See the medical conditions and disease areas that this research is targeting or investigating.
Study Design
Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.
NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
Review each arm or cohort in the study, along with the interventions and objectives associated with them.
NI-0501
NI-0501 administered by IV infusion at a starting dose of 1 mg/kg.
NI-0501
Interventions
Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.
NI-0501
Eligibility Criteria
Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.
Inclusion Criteria
* 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
* 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
18 Years
ALL
No
Sponsors
Meet the organizations funding or collaborating on the study and learn about their roles.
Seventh Framework Programme
OTHER
Swedish Orphan Biovitrum
INDUSTRY
Responsible Party
Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.
Principal Investigators
Learn about the lead researchers overseeing the trial and their institutional affiliations.
Radmila Kanceva, MD
Role: STUDY_DIRECTOR
Swedish Orphan Biovitrum
Locations
Explore where the study is taking place and check the recruitment status at each participating site.
Children's Hospital Colorado
Aurora, Colorado, United States
Alfred I. duPont Hospital for Children - Nemours Center for Cancer and Blood Disorders - Division of Pediatric Hematology Oncology
Wilmington, Delaware, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Dana-Farber Cancer Institute (DFCI)
Boston, Massachusetts, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States
Cincinnati Children's Hospital - Division of Immunobiology - Department of Pediatrics
Cincinnati, Ohio, United States
Texas Children's Cancer Center
Houston, Texas, United States
Primary Children's Hospital
Salt Lake City, Utah, United States
University Children's Hospital
Münster, , Germany
Azienda Ospedaliero Universitaria Meyer
Florence, , Italy
Istituto Giannina Gaslini
Genoa, , Italy
Azienda Ospedaliera San Gerardo
Monza, , Italy
Azienda Ospedaliera Padova - Clinica di Oncoematologia Pediatrica
Padua, , Italy
Ospedale Pediatrico Bambino Gesu'
Roma, , Italy
Ospedale Donna Bambino - U.O.C. Oncoematologia Pediatrica
Verona, , Italy
Hospital Sant Joan de Déu
Barcelona, , Spain
Hospital Universitario Vall d'Hebron
Barcelona, , Spain
Hospital Universitario Niño Jesús
Madrid, , Spain
Karolinska University Hospital
Stockholm, , Sweden
Great Ormond Street Hospital - Department of Haematology
London, , United Kingdom
Countries
Review the countries where the study has at least one active or historical site.
References
Explore related publications, articles, or registry entries linked to this study.
Brossard P, Laveille C. Population Pharmacokinetics of the Anti-Interferon-Gamma Monoclonal Antibody Emapalumab: An Updated Analysis. Rheumatol Ther. 2024 Jun;11(3):869-880. doi: 10.1007/s40744-024-00669-y. Epub 2024 Apr 25.
Locatelli F, Jordan MB, Allen C, Cesaro S, Rizzari C, Rao A, Degar B, Garrington TP, Sevilla J, Putti MC, Fagioli F, Ahlmann M, Dapena Diaz JL, Henry M, De Benedetti F, Grom A, Lapeyre G, Jacqmin P, Ballabio M, de Min C. Emapalumab in Children with Primary Hemophagocytic Lymphohistiocytosis. N Engl J Med. 2020 May 7;382(19):1811-1822. doi: 10.1056/NEJMoa1911326.
Provided Documents
Download supplemental materials such as informed consent forms, study protocols, or participant manuals.
Document Type: Study Protocol: US protocol
Document Type: Study Protocol: EU protocol
Document Type: Statistical Analysis Plan
Related Links
Access external resources that provide additional context or updates about the study.
NI-0501-05, long-term follow-up study description on clinicaltrials.gov
Other Identifiers
Review additional registry numbers or institutional identifiers associated with this trial.
2012-003632-23
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
NI-0501-04
Identifier Type: -
Identifier Source: org_study_id
More Related Trials
Additional clinical trials that may be relevant based on similarity analysis.