Phase 3 N=45 Treatment

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

Primary Haemophagocytic Lymphohistiocytosis

Bottom Line

View on ClinicalTrials.gov: NCT01818492 ↗

Enrolled (actual)

Serious AEs

64.0%

Results posted

Sep 2020

Primary outcome: Primary: Overall Response Rate (ORR) Second Line — 63.0 percentage of participants — p=0.0134

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: NI-0501 (Biological)
Age: Pediatric, Adult
Sex: All
Sponsor: Swedish Orphan Biovitrum
Primary completion: Jan 2019

Outcome Measures

Outcome	Result	p-value
PRIMARY Overall Response Rate (ORR) Second Line	63.0	0.0134 sig
PRIMARY Overall Response Rate (ORR) All Treated	64.7	0.0031 sig
PRIMARY Overall Response Rate (ORR) Follow-on Analysis Set:	58.8	0.0205 sig
PRIMARY Overall Response Rate (ORR) at End of Treatment in Study NI-0501-04 (EOT 04) Follow-on Analysis Set: All Treated	60.0	0.0053 sig
SECONDARY Time to Response	7.0	—
SECONDARY Durability of First Response	58.0	—
SECONDARY Overall Survival	42	—
SECONDARY Number of Patients Able to Reduce Glucocorticoids	46.7; 11.1	—
SECONDARY Cumulative Duration of Response	78.6	—
SECONDARY Survival Pre-HSCT	0.83; 0.73	—
SECONDARY Survival Post-HSCT	0.82; 0.82	—

Summary

The purpose of this study is to assess the safety, tolerability and efficacy of a new drug aimed at controlling disease activity in patients diagnosed with primary haemophagocytic lymphohistiocytosis. The new drug can be administered as the first-line therapy, to patients not previously treated with the current standard of care, or can be given to patients who have either failed or were unable to tolerate the current standard of care. Administration will be on top of a glucocorticosteroid, which is usually part of the current recommended treatment.

Eligibility Criteria

Inclusion Criteria

Gender: male and female
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

Diagnosis of secondary Haemophagocytic Lymphohistiocytosis consequent to a proven rheumatic or neoplastic disease.
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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01818492). 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.