Trial · NCT03922308
Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
NCT03922308, Phase 2, Completed, Shire; results first posted 2022-12-01
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP). Trials, record NCT03922308. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03922308
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
- NCT number
- NCT03922308
- Status
- Completed
- Phases
- Phase 2
- Enrollment
- 28 participants, actual
- Lead sponsor
- Shire
- Conditions
- Acquired Thrombotic Thrombocytopenic Purpura (aTTP)
- Interventions
- Placebo, SHP655, Standard of Care
- First posted
- 2019-04-19
- Last updated
- 2022-12-01
- Other registries' IDs
- eudract:2018-003775-35
- Results first posted
- 2022-12-01
Source ClinicalTrials.gov
Registered outcomes32
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | ADAMTS-13 Activity Levels | Up to Days 11 or 12 | ADAMTS-13 Activity Levels was assessed by fluorescence resonance energy transfer (FRETS) ADAMTS13 activity, with or without SHP655 Supplementation. Schedule A (Days 1, 2, 3, 4, 6, 8, 11, and every 3 days thereafter) or Schedule B (Days 1, 2, 3, 5, 7, 9, 12, and every 3 days thereafter). Data is reported for multiple timepoints as Within 15 minutes pre-PEX and post-PEX; Within 15 minutes, 0.5-3 hours, 4-6 hours post end of investigational product (IP) infusion 1; Within 15 minutes, 0.5-3 hours post end IP infusion 2; 30 minutes pre-IP infusion 2 of Schedule A and Schedule B (up to Day 11 or 12). |
| Primary | Platelet Count | Baseline and end of study (EOS) (up to approximately 15 months) | The platelet counts are reported in units of 10\^9 per liter blood. |
| Primary | Lactate Dehydrogenase (LDH) Levels | Baseline and EOS (up to approximately 15 months) | The lactate dehydrogenase levels are reported. |
| Secondary | Dose(s) of SHP655 Needed to Achieve and Maintain Adequate Plasma Levels of rADAMTS-13 | From start of study drug administration up to 13 weeks (following remission up to 6 months) | Dose(s) of SHP655 needed to achieve and maintain adequate plasma levels of rADAMTS-13 in order to support induction of remission and to reduce the number of PEX procedures needed for the treatment of acute aTTP episodes was assessed. |
| Secondary | PK/PD Temporal Relationship of Safety and Efficacy Parameter as a Function of ADAMTS-13 Activity | Up to 6 months | Parameters included platelet and LDH counts. |
| Secondary | Number of Participants With ADAMTS-13 Binding Antibodies Per Titer | Baseline and EOS (up to approximately 15 months) | Antibody titer indicates the level of the antibodies in a blood sample, defined as the greatest dilution (or lowest concentration) of the blood sample at which an antibody assay (such as ELISA for e.g.), still produces a detectable positive result. Data is presented per titer for ADA positive participants only. |
| Secondary | Inhibitory Autoantibodies (Nab) Titer Levels | Baseline and EOS (up to approximately 15 months) | NAb titers were summarized (median, minimum and maximum) at baseline and EOS per treatment arms, in those subjects with NAb positive results (NAb positive is defined as titer value \>=0.6 BU/mL). |
| Secondary | ADAMTS-13 Activity Levels in Participants Receiving Additional SHP655 for up to 30 Days After Resolution by Using FRETS | At Days 3, 7, 10, 21, 28, 42, 56 and 84 | ADAMTS-13 activity levels in participants receiving additional SHP655 for up to 30 days after the resolution of the thrombotic thrombocytopenic purpura (TTP) episode were assessed. Resolution was defined as a normal platelet count and LDH \<2 ULN for at least 48 hours following initial normalization of platelet count (acute episode period). |
| Secondary | Relationship Between ADAMTS-13 Activity and End-organ Disease Status | Up to 6 months | End-organ disease status were evaluated for renal, cardiac and neurological diseases. |
| Secondary | Predose Concentration (Cpre) to Maximum Plasma Concentration (Cmax) Ratio | Pre-PEX and post-PEX at multiple timepoints at Days 1, 2, 3, 4 or 5, 6 or 7, 8 or 9, and 11 or 12 |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 registered outcomesOutcomes32
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | ADAMTS-13 Activity Levels | Up to Days 11 or 12 | Pharmacokinetic (PK) Set included all enrolled participants with confirmed aTTP diagnosis who received at least 1 dose of investigational product and who had at least 1 evaluable post-dose PK value (ADAMTS-13 antigen and ADAMTS-13 activity). Overall number analyzed are the number of participants available for analyses. Number analyzed are the number of participants with data available for analyses at the given timepoint. | international units(IU)/ml | MEAN |
| PRIMARY | Platelet Count | Baseline and end of study (EOS) (up to approximately 15 months) | SAS included all participants randomized, who received any dose of investigational product. Overall number analyzed are the number of participants with data evaluable for analyses. Number analyzed is the number of participants available for analysis. | 10^9 platelets/L | MEAN |
| PRIMARY | Lactate Dehydrogenase (LDH) Levels | Baseline and EOS (up to approximately 15 months) | SAS included all participants randomized, who received any dose of investigational product. Overall number analyzed are the number of participants with data evaluable for analyses. Number analyzed is the number of participants available for analysis. | international units (IU)/L | MEAN |
| SECONDARY | Dose(s) of SHP655 Needed to Achieve and Maintain Adequate Plasma Levels of rADAMTS-13 | From start of study drug administration up to 13 weeks (following remission up to 6 months) | Data could not be analyzed due to sparse sample collections and confounding dosing inputs with daily sequential PEX + SHP655 dosing. | ||
| SECONDARY | PK/PD Temporal Relationship of Safety and Efficacy Parameter as a Function of ADAMTS-13 Activity | Up to 6 months | Data could not be analyzed due to sparse sample collections and confounding dosing inputs with daily sequential PEX + SHP655 dosing. | ||
| SECONDARY | Number of Participants With ADAMTS-13 Binding Antibodies Per Titer | Baseline and EOS (up to approximately 15 months) | SAS included all participants randomized, who received any dose of investigational product. Overall number analyzed are the number of participants are ADA positive participants. Number analyzed is the number of participants available for analysis. | Participants | COUNT_OF_PARTICIPANTS |
| SECONDARY | Inhibitory Autoantibodies (Nab) Titer Levels | Baseline and EOS (up to approximately 15 months) | SAS included all participants randomized, who received any dose of investigational product. Overall number analyzed are the number of participants with data evaluable for analyses. Number analyzed is the number of participants available for analysis. | BU/mL | MEDIAN |
| SECONDARY | ADAMTS-13 Activity Levels in Participants Receiving Additional SHP655 for up to 30 Days After Resolution by Using FRETS | At Days 3, 7, 10, 21, 28, 42, 56 and 84 | PK Set included all enrolled participants with confirmed aTTP diagnosis who received at least 1 dose of investigational product and who had at least 1 evaluable post-dose PK value (ADAMTS-13 antigen and ADAMTS-13 activity). Overall number analyzed are the number of participants available for analyses. Number analyzed is the number of participants with data available for analyses at given time point. | IU/mL | MEAN |
| SECONDARY | Relationship Between ADAMTS-13 Activity and End-organ Disease Status | Up to 6 months | Data could not be analyzed due to sparse sample collections and confounding dosing inputs with daily sequential PEX + SHP655 dosing. | ||
| SECONDARY | Predose Concentration (Cpre) to Maximum Plasma Concentration (Cmax) Ratio | Pre-PEX and post-PEX at multiple timepoints at Days 1, 2, 3, 4 or 5, 6 or 7, 8 or 9, and 11 or 12 | PK Set included all enrolled participants with confirmed aTTP diagnosis who received at least 1 dose of investigational product and who had at least 1 evaluable post-dose PK value (ADAMTS-13 antigen and ADAMTS-13 activity). Overall number analyzed are the number of participants available for analyses. Number analyzed is the number of participants with data available for analyses at given time point. | ratio | MEAN |
1 to 10 of 32Source ClinicalTrials.gov
See all 32 outcomesResults by group380
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| ADAMTS-13 Activity Levels | Standard of Care (SoC) + Placebo | FRETS: Day 1: ≤15 min Pre-PEX | 0.1439 | Standard Deviation 0.19247 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 + Placebo | FRETS: Day 1: ≤15 min Pre-PEX | NA | Standard Deviation NA | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 | FRETS: Day 1: ≤15 min Pre-PEX | NA | Standard Deviation NA | international units(IU)/ml |
| ADAMTS-13 Activity Levels | Standard of Care (SoC) + Placebo | FRETS: Day 1: ≤15 min Post-PEX | 0.5418 | Standard Deviation 0.18345 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 + Placebo | FRETS: Day 1: ≤15 min Post-PEX | 0.5011 | Standard Deviation 0.19236 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 | FRETS: Day 1: ≤15 min Post-PEX | 0.3029 | Standard Deviation 0.29020 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | Standard of Care (SoC) + Placebo | FRETS: Day 1: ≤15 min Post End of IP 1 | 0.4571 | Standard Deviation 0.19125 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 + Placebo | FRETS: Day 1: ≤15 min Post End of IP 1 | 2.012 | Standard Deviation 0.82832 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | SoC + SHP655 | FRETS: Day 1: ≤15 min Post End of IP 1 | 1.352 | Standard Deviation 0.93502 | international units(IU)/ml |
| ADAMTS-13 Activity Levels | Standard of Care (SoC) + Placebo | FRETS: Day 1: 0.5-3 Hour (hr) Post End of IP 1 | 0.4420 | Standard Deviation 0.20895 | international units(IU)/ml |
1 to 10 of 380Source ClinicalTrials.gov
See all 380 results by groupParticipant flow12
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Standard of Care (SoC) + Placebo | 10 | |
| Overall Study | STARTED | SoC + SHP655 + Placebo | 9 | |
| Overall Study | STARTED | SoC + SHP655 | 9 | |
| Overall Study | Pharmacokinetic Population | Standard of Care (SoC) + Placebo | 10 | |
| Overall Study | Pharmacokinetic Population | SoC + SHP655 + Placebo | 8 | |
| Overall Study | Pharmacokinetic Population | SoC + SHP655 | 9 | |
| Overall Study | COMPLETED | Standard of Care (SoC) + Placebo | 9 | |
| Overall Study | COMPLETED | SoC + SHP655 + Placebo | 6 | |
| Overall Study | COMPLETED | SoC + SHP655 | 8 | |
| Overall Study | NOT COMPLETED | Standard of Care (SoC) + Placebo | 1 |
1 to 10 of 12Source ClinicalTrials.gov
See all 12 participant flowAdverse events360
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 discomfort | Gastrointestinal disorders | Placebo Post PEX + Placebo 12H Post PEX | 1 | 10 | |
| other | Abdominal discomfort | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 0 | 9 | |
| other | Abdominal discomfort | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 0 | 9 | |
| other | Abdominal distension | Gastrointestinal disorders | Placebo Post PEX + Placebo 12H Post PEX | 1 | 10 | |
| other | Abdominal distension | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 0 | 9 | |
| other | Abdominal distension | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 0 | 9 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo Post PEX + Placebo 12H Post PEX | 0 | 10 | |
| other | Abdominal pain upper | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 1 | 9 | |
| other | Abdominal pain upper | Gastrointestinal disorders | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 0 | 9 | |
| other | Abnormal weight gain | Metabolism and nutrition disorders | Placebo Post PEX + Placebo 12H Post PEX | 0 | 10 |
1 to 10 of 360Source ClinicalTrials.gov
See all 360 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Placebo Post PEX + Placebo 12H Post PEX | 0 | 10 |
| Total, all-cause mortality | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 0 | 9 |
| Total, all-cause mortality | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 0 | 9 |
| Total, other adverse events | Placebo Post PEX + Placebo 12H Post PEX | 10 | 10 |
| Total, other adverse events | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 9 | 9 |
| Total, other adverse events | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 6 | 9 |
| Total, serious adverse events | Placebo Post PEX + Placebo 12H Post PEX | 4 | 10 |
| Total, serious adverse events | SHP655 40 IU/kg Post PEX + Placebo 12H Post PEX | 1 | 9 |
| Total, serious adverse events | SHP655 40 IU/kg Post PEX + SHP655 40 IU/kg 12H Post PEX | 3 | 9 |
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.
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