Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT03922308 | Study of rADAMTS-13 (SHP655) in the Treatment of Participants With Acquired Thrombotic Thrombocytopenic Purpura (aTTP) | PHASE2 | COMPLETED | 28 | — | — | Oct 9, 2019 | Aug 5, 2021 | Dec 1, 2022 | 24 | United States, Canada +5 |
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).
The platelet counts are reported in units of 10\^9 per liter blood.
The lactate dehydrogenase levels are reported.
| Arm | Type | Description |
|---|---|---|
| Standard of Care (SoC) + Placebo | PLACEBO_COMPARATOR | Participants received SoC daily PEX followed by placebo immediately and 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months). |
| SoC + SHP655 + Placebo | EXPERIMENTAL | Participants received SoC daily PEX and SHP655 40 +/- 4 international units per kilogram (IU/kg), IV injection, QD, immediately after PEX and placebo 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months). |
| SoC + SHP655 | EXPERIMENTAL | Participants received SoC daily PEX and SHP655 40 +/- 4 IU/kg, IV injection, BID, immediately after PEX and 12 +/- 1 hours after completion of PEX until remission was achieved (up to approximately 6 months). |
| Name | Type | Description |
|---|---|---|
| Placebo | OTHER | Participants will receive injection of placebo matched to SHP655. |
| SHP655 | DRUG | Participants will receive injection of SHP655. |
| Standard of Care | OTHER | Participants will receive PEX as Standard of Care (SOC). |
Inclusion Criteria: * Participant or legally authorized representative voluntarily signs informed consent. For participants unable to provide consent, a fully recognized medical proxy may be used according to local laws. * Participant is 18 to 75 years old at the time of screening. * Participant ha...