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NCT03312751 · ClinicalTrials.gov registry record · Phase 3
Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis
A Phase 3 study, sponsored by Swedish Orphan Biovitrum.
- Completed
- Registry status
- Phase 3
- Development phase
- 35
- Enrollment target
NCT03312751: Completed Phase 3 study, sponsored by Swedish Orphan Biovitrum.
NCT03312751 is a Phase 3 study that has completed, run by Swedish Orphan Biovitrum. The registered enrollment target is 35 participants, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower). According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03312751, a Phase 3 study, has completed, sponsored by Swedish Orphan Biovitrum.
- COMPLETED
- Registry status
- Phase 3
- Development phase
- 35 participants
- Enrollment target
Study Summary
The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term outcomes and quality of life assessments. Emapalumab 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 available standard of care. Emapalumab is to be administered until the start of conditioning for hematopoietic stem cell transplantation (HSCT), with an anticipated duration ranging from a minimum of 4 weeks to approximately 12 weeks and not exceeding 6 months. After treatment completion, patients will continue in the study for long-term follow-up until 1 year after either HSCT or last emapalumab infusion (if HSCT is not performed).
Primary Outcome
The overall response rate (ORR) of patients achieving either Complete or Partial Response or HLH Improvement, at Week 8 or EOT (whichever occurs earlier).
Interventions
- DRUG Emapalumab
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2019-02-06 |
| Est. Completion | 2022-09-14 |
| Phase | Phase 3 |
What the finished NCT03312751 record still lists
NCT03312751 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 772-participant average among 15,282 other Phase 3 trials with a reported enrollment target (95% lower).
The record links to 0 conditions, and to 1 intervention - of which Emapalumab is the first listed.
NCT03312751 does not publish any study locations in the registry export this page uses.
Frequently Asked Questions
What is clinical trial NCT03312751 about?
NCT03312751 is a clinical study titled "Study to Assess the Efficacy and Safety of Emapalumab in Primary Haemophagocytic Lymphohistiocytosis". The purpose of this study is to expand the knowledge on the efficacy and safety of emapalumab (previously known as NI-0501) as a treatment for primary haemophagocytic lymphohistiocytosis (HLH) patients, including on long-term outcomes and quality of life assessments. Emapalumab can be administered a...
What is the current status of trial NCT03312751?
This trial is currently completed. It is a Phase 3 study. The enrollment target is 35 participants. The study started on 2019-02-06. Estimated completion is 2022-09-14.
What interventions are being tested in trial NCT03312751?
The interventions under investigation include: Emapalumab (DRUG).
Who is sponsoring clinical trial NCT03312751?
This trial is sponsored by Swedish Orphan Biovitrum, which has 23 total clinical trials registered on ClinicalTrials.gov.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT03312751's enrollment target sits among peer trials
35 1903rd of 2000 higher than 96 of 2,000 other Phase 3 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 3 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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