Trial · NCT05148299
A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
NCT05148299, Phase 2, Completed, Swedish Orphan Biovitrum; results first posted 2025-11-28
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation. Trials, record NCT05148299. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05148299
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of Pegcetacoplan for Patients With Transplant-associated Thrombotic Microangiopathy After Hematopoietic Stem Cell Transplantation
- NCT number
- NCT05148299
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 12 participants, actual
- Lead sponsor
- Swedish Orphan Biovitrum
- Conditions
- Transplant-Associated Thrombotic Microangiopathy
- Interventions
- Pegcetacoplan
- First posted
- 2021-12-08
- Last updated
- 2025-11-28
- Other registries' IDs
- eudract:2021-003157-27
- Results first posted
- 2025-11-28
- Limitations and caveats
- The small sample size limits the generalizability of the findings.
Source ClinicalTrials.gov
Registered outcomes39
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Week 1 | Area under the concentration-time curve limited to the end of the dosing interval. The samples included in the calculation of AUC0-tau were collected at the following times: on dosing Days 1, 3 and 5, PK samples were taken up to 30 minutes pre-dose and at 15 minutes (± 5 min), 30 minutes (± 5 min), 1 hour (± 10 min), 4 hours (± 10 min), 8 hours (± 30 min), and 24 hours (± 30 min) post-dose as well as on Day 8 pre-dose. |
| Primary | Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Week 1 | Maximum observed serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5. |
| Primary | Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Week 1 | Time of maximum measured serum concentration. Determined using the samples collected post-dose on dosing Days 1, 3 and 5. |
| Primary | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 1 up to Week 14 | Observed serum concentration pre-dose. From Day 8 (Week 1) and onwards, PK samples were taken pre-dose at each visit. |
| Secondary | Absolute Levels and Change From Baseline in sC5b-9 | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation sC5b-9 |
| Secondary | Absolute Levels and Change From Baseline in C3a | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3a |
| Secondary | Absolute Levels and Change From Baseline in C3 | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C3 |
| Secondary | Absolute Levels and Change From Baseline in Bb | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation Bb |
| Secondary | Absolute Levels and Change From Baseline in C4a | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation C4a |
| Secondary | Absolute Levels and Change From Baseline in Classical Pathway (CH50) | Week 24 | Absolute levels and change from baseline to Week 24 in biomarker of complement activation CH50 |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 registered outcomesOutcomes39
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Week 1 | On Day 1 and 5 the AUC0-tau parameters did not meet lambda-z acceptance criteria for 2 patients, so they were not included. On Day 8 the AUC0-tau parameter did not meet lambda-z acceptance criteria for 1 patient, so they were not included. | µg*h/mL | MEAN |
| PRIMARY | Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Week 1 | On Day 1, Cmax could not be estimated for 2 patients due to a) no sample provided; and b) Cmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Cmax could not be estimated for 1 patient due to a limited number of samples provided. | µg/mL | MEAN |
| PRIMARY | Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Week 1 | On Day 1, Tmax could not be estimated for 2 patients due to a) no sample provided; and b) Tmax considered unreliable due to pegcetacoplan positive pre-dose sample. On Day 3 and Day 5, Tmax could not be estimated for one patient due to a limited number of samples provided. | Hour | MEDIAN |
| PRIMARY | Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Week 1 up to Week 14 | The number of patients in the analysis population for Ctrough decreased over the course of the study, from 11 at Week 1 to 7 at Week 14, reflecting the limited number of samples available due to study discontinuation of patients. | µg/mL | GEOMETRIC_MEAN |
| SECONDARY | Absolute Levels and Change From Baseline in sC5b-9 | Week 24 | The pharmacodynamic (PD) set includes patients in the intent-to-treat (ITT) set who receive investigational medicinal product (IMP) and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. There were also missing samples. | µg/L | MEAN |
| OTHER_PRE_SPECIFIED | Number of Participants With Treatment-emergent Adverse Events | From treatment start to end of study, up to 6 months (8 weeks since last dose of IMP) | Participants | COUNT_OF_PARTICIPANTS | |
| OTHER_PRE_SPECIFIED | Change From Baseline in Platelets | Week 24 | The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included. | 10^9 platelets/L | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Hemoglobin | Week 24 | The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included. | g/L | MEAN |
| OTHER_PRE_SPECIFIED | Change From Baseline in Lactate Dehydrogenase (LDH) | Week 24 | The number of patients in the analysis population reflects the limited number of samples available due to study discontinuation of patients. Patients with an available measure are included. | U/L | MEAN |
| SECONDARY | Absolute Levels and Change From Baseline in C3a | Week 24 | The PD set includes all patients in the ITT set who receive IMP and have at least 1 evaluable post-dose PD measurement. The number of patients in the analysis population decreased over the course of the study reflecting the limited number of samples available due to study discontinuation of patients. Patients with an available measure both at the analyzed time point and at baseline are included. In some cases, there were also missing samples. | µg/L | MEAN |
1 to 10 of 39Source ClinicalTrials.gov
See all 39 outcomesResults by group451
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Pegcetacoplan | Day 1-Day 3 | 14934.65 | Standard Deviation 4448.067 | µg*h/mL |
| Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Pegcetacoplan | Day 3-Day 5 | 20089.79 | Standard Deviation 5162.653 | µg*h/mL |
| Pegcetacoplan Pharmacokinetic (PK) Parameter Area Under the Curve Limited to the End of Dosing Interval (AUC0-tau) | Pegcetacoplan | Day 5-Day 8 | 36208.37 | Standard Deviation 10685.626 | µg*h/mL |
| Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Pegcetacoplan | Day 1 | 508.1 | Standard Deviation 134.86 | µg/mL |
| Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Pegcetacoplan | Day 3 | 579.6 | Standard Deviation 142.11 | µg/mL |
| Pegcetacoplan PK Parameter Maximal Serum Concentration (Cmax) | Pegcetacoplan | Day 5 | 705.2 | Standard Deviation 203.04 | µg/mL |
| Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Pegcetacoplan | Day 1 | 1.0 | Full Range 0.3 to 48.0 | Hour |
| Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Pegcetacoplan | Day 3 | 1.02 | Full Range 0.3 to 8.5 | Hour |
| Pegcetacoplan PK Parameter Time to Cmax (Tmax) | Pegcetacoplan | Day 5 | 1.05 | Full Range 0.5 to 24.0 | Hour |
| Pegcetacoplan PK Parameter Observed Serum Concentration Pre-dose (Ctrough) | Pegcetacoplan | Week 1 | 365.22 | 95% Confidence Interval 308.37 to 432.55 | µg/mL |
1 to 10 of 451Source ClinicalTrials.gov
See all 451 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period | STARTED | Pegcetacoplan | 12 | |
| Treatment Period | COMPLETED | Pegcetacoplan | 9 | |
| Treatment Period | NOT COMPLETED | Pegcetacoplan | 3 | |
| Follow-up Period | STARTED | Pegcetacoplan | 9 | |
| Follow-up Period | COMPLETED | Pegcetacoplan | 6 | |
| Follow-up Period | NOT COMPLETED | Pegcetacoplan | 3 |
Source ClinicalTrials.gov
Adverse events51
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 | Diarrhea | Gastrointestinal disorders | Pegcetacoplan | 3 | 12 | 4 |
| other | Vomiting | Gastrointestinal disorders | Pegcetacoplan | 2 | 12 | 3 |
| other | Abdominal pain | Gastrointestinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Hemoperitoneum | Gastrointestinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Nausea | Gastrointestinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Oral pain | Gastrointestinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Pleural effusion | Respiratory, thoracic and mediastinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Bronchospasm | Respiratory, thoracic and mediastinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Hemoptysis | Respiratory, thoracic and mediastinal disorders | Pegcetacoplan | 1 | 12 | 1 |
| other | Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | Pegcetacoplan | 1 | 12 | 1 |
1 to 10 of 51Source ClinicalTrials.gov
See all 51 adverse eventsAdverse event totals3
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Pegcetacoplan | 6 | 12 |
| Total, other adverse events | Pegcetacoplan | 5 | 12 |
| Total, serious adverse events | Pegcetacoplan | 8 | 12 |
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/NCT05148299
GET https://conductscience.org/api/trials/v1/NCT05148299/registered?page=2