Trial · NCT03393975
A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
NCT03393975, Phase 3, Completed, Baxalta now part of Shire; results first posted 2025-03-19
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP). Trials, record NCT03393975. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03393975
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of BAX 930 in Children, Teenagers, and Adults Born With Thrombotic Thrombocytopenic Purpura (TTP)
- NCT number
- NCT03393975
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 52 participants, actual
- Lead sponsor
- Baxalta now part of Shire
- Conditions
- Thrombotic Thrombocytopenic Purpura (TTP)
- Interventions
- TAK-755, Standard of care
- First posted
- 2018-01-09
- Last updated
- 2026-02-09
- Other registries' IDs
- eudract:2017-000858-18
- Results first posted
- 2025-03-19
Source ClinicalTrials.gov
Registered outcomes44
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment | Up to 74.5 months | As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Percentage of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events Responding to TAK-755 | Up to 79.6 months | Percentage of acute TTP events responding to TAK-755, was defined as not requiring the use of another human disintegrin and metalloprotease with a thrombospondin type 1 motif, member 13 (ADAMTS13)-containing agent. As per planned analysis, data for this outcome measure were collected and reported only for the TAK-755 treatment arm of both the prophylaxis (irrespective of the prophylaxis periods) and on demand cohorts. |
| Secondary | Time to Resolution of Acute TTP Events | Up to 79.6 months | Time to resolution of acute TTP events following initiation of treatment with TAK-755 or SoC agent was assessed. Acute TPP events were considered resolved when: (a) Platelet count was \>150,000 per microliter (μL) or drop of platelet count was within 25 percent (%) of baseline, whichever occurred first, and (b) Elevation of lactate dehydrogenase (LDH) \<1.5 x baseline or \<1.5 x upper limit of normal (ULN). As per planned analysis, data for this outcome measure were collected and reported in a combined manner irrespective of the prophylaxis treatment Periods, partitioned per treatment received (TAK-755 and SoC) for the on demand and prophylactic cohorts. |
| Secondary | Number of Participants With Thrombocytopenia During Prophylactic Treatment | Up to 79.6 months | Thrombocytopenia was defined as a decrease in platelet count ≥25 % of baseline or a platelet count \<150,000/μL, reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Participants With Microangiopathic Hemolytic Anemia During Prophylactic Treatment | Up to 79.6 months | Microangiopathic hemolytic anemia was defined as an elevation of LDH \>1.5\* of baseline or \>1.5\*ULN (with a possible evidence of schistocytes on blood smear) and was reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Participants With Neurological Symptoms During Prophylactic Treatment | Up to 79.6 months | Neurological symptoms (TTP related) (e.g., confusion, dysphonia, dysarthria, focal or general motor symptoms including seizures), were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Participants With Renal Dysfunction During Prophylactic Treatment | Up to 79.6 months | Renal dysfunction was defined as an increase in serum creatinine \>1.5\*baseline. Number of participants with renal dysfunction were reported by treatment arm for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Participants With Abdominal Pain During Prophylactic Treatment | Up to 79.6 months | Number of participants with abdominal pain (TTP related) were reported by treatment arm for the prophylaxis cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Supplemental Doses Prompted by Subacute TTP Event During Prophylactic Treatment | Up to 79.6 months | Number of supplemental doses prompted by subacute TTP events were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
| Secondary | Number of Participants With Dose Modification Not Prompted by an Acute TTP Event During Prophylactic Treatment | Up to 79.6 months | Number of participants with dose modification not prompted by an acute TTP event were reported by treatment for the prophylactic cohort. As per planned analysis, data for this outcome measure were collected and reported only for the prophylaxis cohorts, in a combined manner for Periods 1 and 2 for TAK-755 and SoC treatments respectively, and separately for Period 3 in which all participants received TAK-755. |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 registered outcomesOutcomes44
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment | Up to 74.5 months | Modified Full Analysis Set(mFAS): all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 because TAK-755 was unavailable.For participants enrolled prior to Nov 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants analyzed:number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Percentage of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events Responding to TAK-755 | Up to 79.6 months | mFAS:all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov.2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants: participants with acute TTP events that were confirmed by central lab data treated with TAK-755. | percentage of events | NUMBER |
| SECONDARY | Time to Resolution of Acute TTP Events | Up to 79.6 months | mFAS: all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017,only first 6 months of SoC treatment in period 1 was included in analysis.Overall number of participants analyzed:number of participants with acute TTP events that were confirmed by central lab data. | days | MEDIAN |
| SECONDARY | Number of Participants With Thrombocytopenia During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Microangiopathic Hemolytic Anemia During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Neurological Symptoms During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Renal Dysfunction During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Participants With Abdominal Pain During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Number of Supplemental Doses Prompted by Subacute TTP Event During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | supplemental doses | NUMBER |
| SECONDARY | Number of Participants With Dose Modification Not Prompted by an Acute TTP Event During Prophylactic Treatment | Up to 79.6 months | mFAS included all FAS participants who were treated according to their randomized treatment sequence with exclusion of those enrolled prior to Nov. 2017 who were treated with SoC instead of randomized treatment of TAK-755 in period 1 as TAK-755 was unavailable. For participants enrolled prior to Nov. 2017, only first 6 months of SoC treatment in period 1 was included in analysis. Overall number of participants analyzed indicates the number of participants with data available for analyses. | Participants | COUNT_OF_PARTICIPANTS |
1 to 10 of 44Source ClinicalTrials.gov
See all 44 outcomesResults by group293
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 Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment | Prophylaxis Cohort: TAK-755 (Periods 1 and 2) | 0 | Participants | ||
| Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment | Prophylaxis Cohort: TAK-755 (Period 3) | 0 | Participants | ||
| Number of Participants With Acute Thrombotic Thrombocytopenic Purpura (TTP) Events During Prophylactic Treatment | Prophylaxis Cohort: SoC (Periods 1 and 2) | 1 | Participants | ||
| Percentage of Acute Thrombotic Thrombocytopenic Purpura (TTP) Events Responding to TAK-755 | On Demand Cohort I: TAK-755 | 100 | percentage of events | ||
| Time to Resolution of Acute TTP Events | Prophylaxis Cohort: SoC | 14.8 | 95% Confidence Interval NA to NA | days | |
| Time to Resolution of Acute TTP Events | On Demand Cohort I: TAK-755 | 3.0 | 95% Confidence Interval NA to NA | days | |
| Time to Resolution of Acute TTP Events | On Demand Cohort II: SoC | 1.5 | 95% Confidence Interval NA to NA | days | |
| Number of Participants With Thrombocytopenia During Prophylactic Treatment | Prophylaxis Cohort: TAK-755 (Periods 1 and 2) | 13 | Participants | ||
| Number of Participants With Thrombocytopenia During Prophylactic Treatment | Prophylaxis Cohort: TAK-755 (Period 3) | 11 | Participants | ||
| Number of Participants With Thrombocytopenia During Prophylactic Treatment | Prophylaxis Cohort: SoC (Periods 1 and 2) | 21 | Participants |
1 to 10 of 293Source ClinicalTrials.gov
See all 293 results by groupParticipant flow72
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Urgent Treatment Period | STARTED | Prophylaxis Cohort I: TAK-755 Then SoC | 0 | |
| Urgent Treatment Period | STARTED | Prophylaxis Cohort II: SoC Then TAK-755 | 0 | |
| Urgent Treatment Period | STARTED | On Demand Cohort I: TAK-755 | 2 | |
| Urgent Treatment Period | STARTED | On Demand Cohort II: SoC | 4 | |
| Urgent Treatment Period | Safety Analysis Set | Prophylaxis Cohort I: TAK-755 Then SoC | 0 | |
| Urgent Treatment Period | Safety Analysis Set | Prophylaxis Cohort II: SoC Then TAK-755 | 0 | |
| Urgent Treatment Period | Safety Analysis Set | On Demand Cohort I: TAK-755 | 2 | |
| Urgent Treatment Period | Safety Analysis Set | On Demand Cohort II: SoC | 4 | |
| Urgent Treatment Period | Full Analysis Set | Prophylaxis Cohort I: TAK-755 Then SoC | 0 | |
| Urgent Treatment Period | Full Analysis Set | Prophylaxis Cohort II: SoC Then TAK-755 | 0 |
1 to 10 of 72Source ClinicalTrials.gov
See all 72 participant flowAdverse events248
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 | Abdominal pain | Gastrointestinal disorders | Prophylaxis Cohort: TAK-755 | 8 | 47 | |
| other | Abdominal pain | Gastrointestinal disorders | Prophylaxis Cohort: SoC | 6 | 48 | |
| other | Abdominal pain | Gastrointestinal disorders | On Demand Cohort I: TAK-755 | 0 | 2 | |
| other | Abdominal pain | Gastrointestinal disorders | On Demand Cohort II: SoC | 0 | 4 | |
| other | Allergic transfusion reaction | Injury, poisoning and procedural complications | Prophylaxis Cohort: TAK-755 | 0 | 47 | |
| other | Allergic transfusion reaction | Injury, poisoning and procedural complications | Prophylaxis Cohort: SoC | 7 | 48 | |
| other | Allergic transfusion reaction | Injury, poisoning and procedural complications | On Demand Cohort I: TAK-755 | 0 | 2 | |
| other | Allergic transfusion reaction | Injury, poisoning and procedural complications | On Demand Cohort II: SoC | 1 | 4 | |
| other | Anaemia | Blood and lymphatic system disorders | Prophylaxis Cohort: TAK-755 | 3 | 47 | |
| other | Anaemia | Blood and lymphatic system disorders | Prophylaxis Cohort: SoC | 2 | 48 |
1 to 10 of 248Source ClinicalTrials.gov
See all 248 adverse eventsAdverse event totals12
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Prophylaxis Cohort: TAK-755 | 0 | 47 |
| Total, all-cause mortality | Prophylaxis Cohort: SoC | 0 | 48 |
| Total, all-cause mortality | On Demand Cohort I: TAK-755 | 0 | 2 |
| Total, all-cause mortality | On Demand Cohort II: SoC | 0 | 4 |
| Total, other adverse events | Prophylaxis Cohort: TAK-755 | 39 | 47 |
| Total, other adverse events | Prophylaxis Cohort: SoC | 37 | 48 |
| Total, other adverse events | On Demand Cohort I: TAK-755 | 0 | 2 |
| Total, other adverse events | On Demand Cohort II: SoC | 3 | 4 |
| Total, serious adverse events | Prophylaxis Cohort: TAK-755 | 6 | 47 |
| Total, serious adverse events | Prophylaxis Cohort: SoC | 8 | 48 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 adverse event totalsPapers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 38692292 | DERIVED |
Source PubMed
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 comparisons4
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Prophylaxis Cohort: TAK-755 vs Prophylaxis Cohort: SoC: Number of Participants With Inhibitory Antibodies to ADAMTS13 | Trial comparisons |
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Neutralizing Antibodies to ADAMTS13 During Prophylactic Treatment | Trial comparisons |
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Anti-Chinese Hamster Ovary (Anti-CHO) Protein Antibodies During Prophylactic Treatment | Trial comparisons |
| Prophylaxis Cohort I: TAK-755 Then SoC vs Prophylaxis Cohort II: SoC Then TAK-755: Number of Participants With Total Binding Antibodies to ADAMTS13 During Prophylactic Treatment | Trial comparisons |
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.
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Read this trial from software.
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