NI-0501-05

Principal Investigator: Mark Fluchel
Keywords: Treatment , NI0501 , Monoclonal Department: Pediatric Administration
IRB Number: 00082348 Co Investigator:  
Specialty: Clinical Immunology/Immunodeficiency
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Deveree Partridge
deveree.partridge@hsc.utah.edu
801-662-4710

Brief Summary

  • To monitor the long-term safety profile of NI-0501
  • To assess HLH patients' survivalafter NI-0501 treatment
  • To assess duration of response to NI-0501 treatment (i.e. maintenance of HLH control)
  • To assess post-HSCT outcome measures, if applicable
  • To assess background disease activity, in patients with secondary forms of HLH
  • To study the elimination profile of NI-0501
  • To evaluate the pharmacodynamics (PD) effects
  • To assess the profile of relevant HLH biomarkers, e.g, sCD25
  • To assess the immunogenicity of NI-0501

Inclusion Criteria

The study population comprises of HLH patients who have received at least one dose of NI-0501 in the context of the clinical development program for NI-0501.

Patients must fulfill the following inclusion criteria:

1. Having received at least one dose of NI-0501 during a previous NI-0501 study or under a CU treatment protocol

2. Having signed the Informed Consent of this study by the patient or the patient’s legal representative(s), as applicable, with the assent of patients who are legally capable of providing it.

3. Having agreed to continue using adequate methods of birth control until 6 months after the last administered dose of NI-0501, when relevant. Males with partner(s) of childbearing potential must agree to take appropriate precautions to avoid fathering a child until 6 months after receiving last dose of NI-0501.

Exclusion Criteria

There are no exclusion criteria.