Trial · NCT04731298
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.
NCT04731298, Phase 2, Terminated, Swedish Orphan Biovitrum; results first posted 2023-12-28
3 sourcestrials-2026-11-12-0.5.0
Cite this record
ConductScience Library. 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. Trials, record NCT04731298. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04731298
On this page
Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- 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.
- NCT number
- NCT04731298
- Status
- Terminated
- Phases
- Phase 2
- Enrollment
- 2 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Graft Failure
- Interventions
- Emapalumab
- First posted
- 2021-01-29
- Last updated
- 2023-12-28
- Results first posted
- 2023-12-28
- Limitations and caveats
The study was prematurely terminated with only 2 patients enrolled. Both patients were in the non-emapalumab treated arm, i.e. no patients received study treatment (emapalumab). Consequently, the majority of analyses were not performed and no conclusion on the efficacy of emapalumab on this patient population could be drawn from the study.
Source ClinicalTrials.gov
Registered outcomes63
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | CXCL9 in Serum | From start of treatment to EoS Visit, up to 34 weeks | Serum concentration of C-X-C Motif Chemokine Ligand 9 (CXCL9) |
| Primary | Primary Graft Failure (GF) | From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks | Number of participants with primary graft failure (GF) |
| Primary | Secondary GF | From HSCT (Day 0) up to study termination, approximately 46 weeks | Number of participants with secondary GF |
| Secondary | Free & Total Interferon Gamma (IFNγ) in Serum | From start of treatment to EoS Visit, up to 34 weeks | Serum concentration of free and total Interferon gamma (IFNγ) |
| Secondary | Emapalumab in Serum - Peak | From start of treatment to EoS, up to 34 weeks | Peak emapalumab serum concentration |
| Secondary | Ctrough (Emapalumab) | From start of treatment to EoS, up to 34 weeks | Concentration just before administration |
| Secondary | Exploratory Biomarkers: Ferritin | From HSCT (Day 0) up to study termination, approximately 46 weeks | Ferritin - serum concentration |
| Secondary | ADA and nAbs | From Start of treatment until EoS, up to 34 weeks | Number of participants developing antibodies against emapalumab (antidrug antibodies \[ADA\]) and Neutralizing antibodies (nAb) |
| Secondary | Number of Participants With Mixed Donor Chimerism <10% and <20% | From HSCT (Day 0) up to study termination, approximately 46 weeks | Based on unselected leukocytes and based on sorted T cells |
| Secondary | Number of Participants Receiving Thrombopoietic Agents, Stem Cell Boost, Donor Lymphocyte Infusion (DLI) | From HSCT (Day 0) up to study termination, approximately 46 weeks |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 registered outcomesOutcomes63
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | CXCL9 in Serum | From start of treatment to EoS Visit, up to 34 weeks | No efficacy data was collected since no subject received study treatment. | ||
| PRIMARY | Primary Graft Failure (GF) | From Hematopoietic stem-cell transplantation (HSCT) [Day 0] up to study termination, approximately 46 weeks | No efficacy data was collected since no subject received study treatment. | ||
| PRIMARY | Secondary GF | From HSCT (Day 0) up to study termination, approximately 46 weeks | No efficacy data was collected since no subject received study treatment. | ||
| SECONDARY | Free & Total Interferon Gamma (IFNγ) in Serum | From start of treatment to EoS Visit, up to 34 weeks | No efficacy data was collected since no subject received study treatment. | ||
| SECONDARY | Emapalumab in Serum - Peak | From start of treatment to EoS, up to 34 weeks | No patients enrolled in the emapalumab arm. | ||
| SECONDARY | Ctrough (Emapalumab) | From start of treatment to EoS, up to 34 weeks | No patients enrolled in the emapalumab arm. | ||
| SECONDARY | Exploratory Biomarkers: Ferritin | From HSCT (Day 0) up to study termination, approximately 46 weeks | No efficacy data was collected since no subject received study treatment. | ||
| SECONDARY | ADA and nAbs | From Start of treatment until EoS, up to 34 weeks | No patients enrolled in the emapalumab arm. | ||
| SECONDARY | Number of Participants With Mixed Donor Chimerism <10% and <20% | From HSCT (Day 0) up to study termination, approximately 46 weeks | No patients enrolled in the emapalumab arm. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants Receiving Thrombopoietic Agents, Stem Cell Boost, Donor Lymphocyte Infusion (DLI) | From HSCT (Day 0) up to study termination, approximately 46 weeks | No efficacy data was collected since no subject received study treatment. |
1 to 10 of 63Source ClinicalTrials.gov
See all 63 outcomesResults by group13
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Number of Participants With Mixed Donor Chimerism <10% and <20% | Emapalumab Treated | 0 | Participants | ||
| Number of Participants With Mixed Donor Chimerism <10% and <20% | Non-emapalumab Treated | 0 | Participants | ||
| Number of Participants With Event Free Engraftment | Emapalumab Treated | 0 | Participants | ||
| Number of Participants With Event Free Engraftment | Non-emapalumab Treated | 2 | Participants | ||
| Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD) | Emapalumab Treated | 0 | Participants | ||
| Number of Participants With Acute and/or Chronic Mild to Severe Graft Versus Host Disease (GvHD) | Non-emapalumab Treated | 1 | Participants | ||
| Engraftment Syndrome | Emapalumab Treated | 0 | Participants | ||
| Engraftment Syndrome | Non-emapalumab Treated | 1 | Participants | ||
| Survival Rate | Non-emapalumab Treated | 2 | Participants | ||
| Number of Subjects With Change in Donor Chimerism | Emapalumab Treated | Change = 100% | 0 | Participants |
1 to 10 of 13Source ClinicalTrials.gov
See all 13 results by groupParticipant flow24
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Allogeneic HSCT (Day 0) | STARTED | Emapalumab Treated | 0 | |
| Allogeneic HSCT (Day 0) | STARTED | Non-emapalumab Treated | 2 | |
| Allogeneic HSCT (Day 0) | COMPLETED | Emapalumab Treated | 0 | |
| Allogeneic HSCT (Day 0) | COMPLETED | Non-emapalumab Treated | 2 | |
| Allogeneic HSCT (Day 0) | NOT COMPLETED | Emapalumab Treated | 0 | |
| Allogeneic HSCT (Day 0) | NOT COMPLETED | Non-emapalumab Treated | 0 | |
| Monitoring - Primary GF (Day 1 to 42) | STARTED | Emapalumab Treated | 0 | |
| Monitoring - Primary GF (Day 1 to 42) | STARTED | Non-emapalumab Treated | 2 | |
| Monitoring - Primary GF (Day 1 to 42) | COMPLETED | Emapalumab Treated | 0 | |
| Monitoring - Primary GF (Day 1 to 42) | COMPLETED | Non-emapalumab Treated | 2 |
1 to 10 of 24Source ClinicalTrials.gov
See all 24 participant flowAdverse events80
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Hypoxia | Respiratory, thoracic and mediastinal disorders | Emapalumab Treated | 0 | ||
| other | Hypoxia | Respiratory, thoracic and mediastinal disorders | Non-emapalumab Treated | 2 | 2 | 2 |
| other | Flushing | Vascular disorders | Emapalumab Treated | 0 | ||
| other | Flushing | Vascular disorders | Non-emapalumab Treated | 1 | 2 | 1 |
| other | Rash | Skin and subcutaneous tissue disorders | Emapalumab Treated | 0 | ||
| other | Rash | Skin and subcutaneous tissue disorders | Non-emapalumab Treated | 2 | 2 | 3 |
| other | Constipation | Gastrointestinal disorders | Emapalumab Treated | 0 | ||
| other | Constipation | Gastrointestinal disorders | Non-emapalumab Treated | 2 | 2 | 3 |
| other | Hyperglycemia | Metabolism and nutrition disorders | Emapalumab Treated | 0 | ||
| other | Hyperglycemia | Metabolism and nutrition disorders | Non-emapalumab Treated | 1 | 2 | 1 |
1 to 10 of 80Source ClinicalTrials.gov
See all 80 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Emapalumab Treated | 0 | 0 |
| Total, all-cause mortality | Non-emapalumab Treated | 0 | 2 |
| Total, other adverse events | Emapalumab Treated | 0 | 0 |
| Total, other adverse events | Non-emapalumab Treated | 2 | 2 |
| Total, serious adverse events | Emapalumab Treated | 0 | 0 |
| Total, serious adverse events | Non-emapalumab Treated | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04731298
GET https://conductscience.org/api/trials/v1/NCT04731298/registered?page=2