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Study to Investigate the Pharmacokinetics, Pharmacodynamics and Assess the Efficacy and Safety to Support Dose Selection of Emapalumab in Pre-empting Graft Failure in Patients at High Risk After HSCT.
Phase 2 trial testing Emapalumab in Graft Failure in 2 participants. Terminated before completion.
| Lead sponsor | Swedish Orphan Biovitrum |
|---|---|
| Phase | Phase 2 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | sequential |
| Masking | none |
| Primary purpose | prevention |
| Enrollment | 2 |
| Start date | 25 May 2021 |
| Primary completion | 21 April 2022 |
| Estimated completion | 21 April 2022 |
| Sites | 5 locations across Canada, Israel, Australia |
Swedish Orphan Biovitrum — full company profile →
1 and older, any sex, with Graft Failure. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Based on unselected leukocytes and based on sorted T cells
| Group | Value | 95% CI |
|---|---|---|
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 0 |
defined as absence of GF or graft support
| Group | Value | 95% CI |
|---|---|---|
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 2 |
(grade I to IV)
| Group | Value | 95% CI |
|---|---|---|
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 1 |
Number of participants with engraftment syndrome
| Group | Value | 95% CI |
|---|---|---|
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 1 |
Number of patients alive at the end of study.
| Group | Value | 95% CI |
|---|---|---|
| Non-emapalumab Treated | 2 |
| Group | Value | 95% CI |
|---|---|---|
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 2 | |
| Emapalumab Treated | 0 | |
| Non-emapalumab Treated | 0 |
Time frame: From Day 1 up to study termination, approximately 46 weeks. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Emapalumab Treated | Non-emapalumab Treated |
|---|---|---|---|
| Hypoxia | Respiratory, thoracic and mediastinal disorders | — | — |
| Rash | Skin and subcutaneous tissue disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| Pyrexia | General disorders | — | — |
| Hypomagnesaemia | Metabolism and nutrition disorders | — | — |
| Headache | Nervous system disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Flushing | Vascular disorders | — | — |
| Hyperglycemia | Metabolism and nutrition disorders | — | — |
| Bradycardia | Cardiac disorders | — | — |
| Hepatic enzyme increased | Investigations | — | — |
| Chills | General disorders | — | — |
| Sepsis | Infections and infestations | — | — |
| Febrile neutropenia | Blood and lymphatic system disorders | — | — |
| Thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Nephropathy toxic | Renal and urinary disorders | — | — |
| Graft versus host disease in skin | Immune system disorders | — | — |
| Acne | Skin and subcutaneous tissue disorders | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
| Hypophosphataemia | Metabolism and nutrition disorders | — | — |
| Hypervolaemia | Metabolism and nutrition disorders | — | — |
| Mucosal inflammation | General disorders | — | — |
| Insomnia | Psychiatric disorders | — | — |
| Perineal abscess | Infections and infestations | — | — |
| Engraftment syndrome | Immune system disorders | — | — |
| Myalgia | Musculoskeletal and connective tissue disorders | — | — |
| Pityriasis rosea | Skin and subcutaneous tissue disorders | — | — |
| Salmonella test positive | Investigations | — | — |
| Injection site pain | General disorders | — | — |
| Hypertrichosis | Skin and subcutaneous tissue disorders | — | — |
| Dysmenorrhoea | Reproductive system and breast disorders | — | — |
| Heavy menstrual bleeding | Reproductive system and breast disorders | — | — |
| Allergy to immunoglobulin therapy | Immune system disorders | — | — |
| Ingrown toe nail | Skin and subcutaneous tissue disorders | — | — |
| Chemokine increase | Investigations | — | — |
| Drug hypersensitivity | Immune system disorders | — | — |
| Non-cardiac chest pain | General disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
Data from ClinicalTrials.gov NCT04731298 adverse events section.
This study is designed as an open-label, single arm, proof of concept study in order to determine the appropriate emapalumab dosing regimen neutralizing IFNγ in patients at risk of GF. Patients presenting CXCL9 levels above a defined threshold and other clinical criteria will be eligible to receive emapalumab. Both children and adults, with malignant and non-malignant underlying diseases, receiving allo-HSCT who are at high risk of GF as defined in the inclusion criteria will be included in the study. The main objective of the study is to determine the appropriate emapalumab dose regimen neutralizing interferon gamma (IFNγ) activity to pre-empt graft failure post allo-HSCT in a population with various underlying diseases and at high risk of graft failure (GF). Maximum 3 cohorts are foreseen to determine the appropriate dose regimen to pre-emptively treat patients at risk of primary GF. Emapalumab will be administered by IV infusion and treatment will last up to 56 days (15 infusions) or until evidence of engraftment. The study is expected to last approximately 3 years from screening to the last follow-up phone call for each patient.
4 peer-reviewed publications reference this trial (live from Europe PMC):
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