A study to investigate the long-term safety and efficacy of a new drug, NI-0501, in children with a disease that is called reactivated Primary Haemophagocytic Lymphohistiocytosis.
Phase 1
- Conditions
- Primary haemophagocytic lymphohistiocytosis which has reactivated.MedDRA version: 14.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2012-005753-23-ES
- Lead Sponsor
- ovImmune SA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 58
Inclusion Criteria
1. Having received at least one dose of NI-0501 during a previous NI-0501 study.
2. Having signed the Informed Consent 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
Are the trial subjects under 18? yes
Number of subjects for this age range: 10
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Exclusion Criteria
None.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: - To monitor the long-term safety profile of patients who received NI-0501.<br>- To assess HLH patients' survival after NI-0501 treatment.<br>- To study the elimination profile of NI-0501.<br>- To assess the immunogenicity of NI-0501.;Secondary Objective: Not applicable;Primary end point(s): * Safety:<br>- Incidence, intensity, relationship to NI-0501 and outcomes of Adverse Events (serious and non-serious)<br>- Evolution of vital signs, physical examination and laboratory values over time<br>* Efficacy:<br>- Clinical Response mostly based on HLH diagnostic criteria and existence of neurological symptomatology<br>- Survival before HSCT, 100 days and one year after post HSCT<br>* Pharmacokinetics: NI-0501 elimination profile (clearance and elimination half-life)<br>* Pharmacodynamics: NI-0501 activity on IFN? neutralization<br>* Immunogenicity: presence of ADA<br>* Exploratory endpoints: e.g. additional markers of disease activity;Timepoint(s) of evaluation of this end point: See E.5.1
- Secondary Outcome Measures
Name Time Method Secondary end point(s): None.;Timepoint(s) of evaluation of this end point: See E.5.2