Phase 3
N=58
A Study for Long-term Follow-up of Hemophagocytic Lymphohistiocytosis (HLH) Participants Who Received Treatment With Emapalumab (NI-0501), an Anti-interferon Gamma Monoclonal Antibody
Hemophagocytic Lymphohistiocytosis
Bottom Line
View on ClinicalTrials.gov: NCT02069899 ↗Enrolled (actual)
58
Serious AEs
63.8%
Results posted
Jun 2022
Primary outcome: Primary: Number of Participants With Adverse Event (AE) — 37; 12; 7 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Emapalumab (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Swedish Orphan Biovitrum
- Primary completion
- May 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Adverse Event (AE) |
37; 12; 7 | — |
| SECONDARY Cumulative Duration of Response (Enrolled-04 Cohort) |
70.7 | — |
| SECONDARY Duration of First Response (Enrolled-06 Cohort) |
61.0 | — |
| SECONDARY Overall Survival (Enrolled-04 Cohort) |
NA | — |
| SECONDARY Overall Survival (Enrolled-06 Cohort) |
NA | — |
| SECONDARY Percentage of Participants Who Achieved Engraftment (Enrolled-04 Cohort) |
20.7 | — |
| SECONDARY Percentage of Participants Who Achieved Donor Chimerism (Enrolled-04 Cohort) |
72.4 | — |
| SECONDARY Percentage of Participants With Graft-versus-host-disease (Enrolled-04 Cohort) |
24.1 | — |
| SECONDARY MAS Activity Level as Assessed by Visual Analogue Scale (Enrolled-06 Cohort) |
0.0; 0.0; 0.2 | — |
| SECONDARY Circulating Emapalumab Level (Enrolled-04 Cohort) |
165148.4; 157849.0; 80.5 | — |
| SECONDARY Circulating Emapalumab Level (Enrolled-06 Cohort) |
20968.0; 8515.3; 1628.5 | — |
| SECONDARY Total Human Interferon Gamma Levels (Enrolled-04 Cohort) |
5290.4; 3613.6; 447.4 | — |
| SECONDARY Total Human Interferon Gamma Levels (Enrolled-06 Cohort) |
5544.3; 2958.7; 1111.0 | — |
| SECONDARY Number of Participants With Anti-drug Antibody |
1; 3; 0 | — |
Summary
International, multicenter, long-term, follow-up study that will enrol HLH participants who have received emapalumab in previous clinical trials, in the context of the clinical development program for emapalumab or under compassionate use (CU).
Eligibility Criteria
Inclusion Criteria
- Having received at least one dose of emapalumab.
- Having signed the Informed Consent by the participant or the participant's legal representative(s), as applicable, with the assent of participant who are legally capable of providing it.
Exclusion Criteria
- None
Data sourced from ClinicalTrials.gov (NCT02069899). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.