A study to investigate the long-term safety and efficacy of a new drug, NI-0501, in children with a disease that is called Haemophagocytic Lymphohistiocytosis.
- Conditions
- Haemophagocytic lymphohistiocytosisMedDRA version: 20.0Level: LLTClassification code 10071583Term: Haemophagocytic lymphohistiocytosisSystem Organ Class: 100000004851Therapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2012-005753-23-FR
- Lead Sponsor
- ovimmune SA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 55
1. Having received at least one dose of NI-0501 during a previous NI-0501 study or under a CU treatment protocol
2. 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, whenever relevant. Males with partners(s) of child-bearing potential must agree to take appropriate precautions to avoid fathering a child until 6 months after receiving last dose of NI-0501.
Are the trial subjects under 18? yes
Number of subjects for this age range: 55
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
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 NI-0501 <br>;Secondary Objective: • To assess HLH patients’ survival after NI-0501 treatment<br>• To assess duration of response to NI-0501 treatment (i.e. maintenance of HLH control)<br>• To assess post-HSCT outcome measures, if applicable<br>• To assess background disease activity, in patients with secondary forms of HLH<br>• To study the elimination profile of NI-0501<br>• To evaluate the pharmacodynamic (PD) effects (levels of circulating Total IFN?, CXCL9, CXCL10)<br>• To assess the profile of relevant HLH biomarkers, e.g., sCD25<br>• To assess the immunogenicity of NI-0501.<br>;Primary end point(s): Safety will be assessed as follows:<br>o Incidence, seriousness, intensity, relationship to NI-0501 and outcome of adverse events (AEs) <br>o Evolution over time of vital signs, physical examination and laboratory values<br><br>;Timepoint(s) of evaluation of this end point: See E.5.1
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Efficacy (as relevant, depending on the different patients’ characteristics):<br>o Duration of Response after completion of NI-0501 treatment (assessed according to the definitions set in the parent study)<br>o Survival time up to one year post-HSCT (including survival to HSCT and survival post-HSCT) or one year after last NI-0501 infusion (if transplant is not performed)<br>o Post-HSCT outcome indices, e.g. engraftment rate, donor chimerism, incidence of acute and chronic Graft versus Host Disease (GvHD) (it applies to patients who receive HSCT)<br>o Monitoring of background disease activity (it applies to patients with secondary forms of HLH) <br><br>Pharmacokinetics: NI-0501 elimination profile<br><br>Pharmacodynamics: IFN? total<br><br>Immunogenicity: presence of ADAs<br><br>Exploratory PD parameters/endpoints: e.g. additional markers of disease activity<br><br>sCD25, CXCL9, CXCL10<br>;Timepoint(s) of evaluation of this end point: See E.5.2