Ignite Creation Date:
2024-05-06 @ 10:58 AM
Last Modification Date:
2024-10-26 @ 12:38 PM
Study NCT ID:
NCT03393975
Status:
COMPLETED
Last Update Posted:
2024-06-04
First Post:
2017-10-05
Brief Title:
A Study of BAX 930 in Children Teenagers and Adults Born With Thrombotic Thrombocytopenic Purpura TTP
Sponsor:
Baxalta now part of Shire
Organization:
Takeda
Study Overview
Official Title:
A Phase 3 Prospective Randomized Controlled Open-label Multicenter 2 Period Crossover Study With a Single Arm Continuation Evaluating the Safety And Efficacy of BAX 930 rADAMTS13 in the Prophylactic And On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura cTTP Upshaw-Schulman Syndrome USS Hereditary Thrombotic Thrombocytopenic Purpura hTTP
Status:
COMPLETED
Status Verified Date:
2024-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Thrombotic thrombocytopenic purpura or TTP for short is a condition where blood clots form in small blood vessels throughout the body The clots can limit or block the flow of oxygen-rich blood to the bodys organs such as the brain kidneys and heart As a result serious health problems can develop The increased clotting that occurs in TTP uses up the cells that help the blood to clot called platelets With fewer platelets available in the blood bleeding problems can occur People who have TTP may bleed underneath the skin forming purple bruises or purpura or from the surface of the skin TTP also can cause anemia a condition in which red blood cells break apart faster than the body can replace them leading to lower than normal number of red blood cells
A lack of activity in the ADAMTS13 enzyme a protein in the blood involved in blood clotting causes TTP The enzyme breaks up another blood protein called von Willebrand factor that clumps together with platelets to form blood clots Some people are born with this condition others get the condition during their life Many people who born with TTP experience frequent flareups that need to be treated right away If not treated It can be fatal or cause lasting damage such as brain damage or a stroke BAX 930 is a medicine that replaces ADAMTS13 and can prevent or control TTP flareups called TTP events
The main aim of this study is to compare the number of TTP events in people born with severe TTP when they treated with BAX 930 versus when they are treated with the standard treatment Treatment will be given in 2 ways
BAX 930 or standard treatment given to prevent TTP events from happening
BAX 930 or standard treatment given to control an acute TTP event when it happens according to the clinics standard practice
Both BAX 930 and standard treatment are given slowly through a vein infusion
At the first visit the study doctor will check if you can participate in the study If you are eligible and enter the study you will follow an assigned schedule and either start with BAX 930 Period 1 and then switch to standard treatment Period 2 or start with standard treatment Period 1 and then switch to BAX 930 Period 2 Everyone will be treated with BAX 930 again for Period 3 Each Period will last approximately 6 months
If you enter the study to control an acute TTP event you will follow a schedule receiving either BAX 930 or standard care to treat your acute TTP event Once the acute TTP event has gotten better you can decide to continue in the study and be given treatment to prevent TTP events from happening following the schedule above
Another studys aim is to assess side effects from treatment with BAX 930 and standard treatment To do that the study doctor will ask you questions about your health at each study visit
The study doctors will also check how long BAX 930 stays in the blood of the participants over time They will do this from blood samples taken after participants receive their specific infusions of BAX 930 This will happen at different times during the study
1 month after all treatment has been completed participants will visit the clinic for a final check-up
A lack of activity in the ADAMTS13 enzyme a protein in the blood involved in blood clotting causes TTP The enzyme breaks up another blood protein called von Willebrand factor that clumps together with platelets to form blood clots Some people are born with this condition others get the condition during their life Many people who born with TTP experience frequent flareups that need to be treated right away If not treated It can be fatal or cause lasting damage such as brain damage or a stroke BAX 930 is a medicine that replaces ADAMTS13 and can prevent or control TTP flareups called TTP events
The main aim of this study is to compare the number of TTP events in people born with severe TTP when they treated with BAX 930 versus when they are treated with the standard treatment Treatment will be given in 2 ways
BAX 930 or standard treatment given to prevent TTP events from happening
BAX 930 or standard treatment given to control an acute TTP event when it happens according to the clinics standard practice
Both BAX 930 and standard treatment are given slowly through a vein infusion
At the first visit the study doctor will check if you can participate in the study If you are eligible and enter the study you will follow an assigned schedule and either start with BAX 930 Period 1 and then switch to standard treatment Period 2 or start with standard treatment Period 1 and then switch to BAX 930 Period 2 Everyone will be treated with BAX 930 again for Period 3 Each Period will last approximately 6 months
If you enter the study to control an acute TTP event you will follow a schedule receiving either BAX 930 or standard care to treat your acute TTP event Once the acute TTP event has gotten better you can decide to continue in the study and be given treatment to prevent TTP events from happening following the schedule above
Another studys aim is to assess side effects from treatment with BAX 930 and standard treatment To do that the study doctor will ask you questions about your health at each study visit
The study doctors will also check how long BAX 930 stays in the blood of the participants over time They will do this from blood samples taken after participants receive their specific infusions of BAX 930 This will happen at different times during the study
1 month after all treatment has been completed participants will visit the clinic for a final check-up
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| TAK-755-281102 | OTHER | Sponsor | None |
| 2017-000858-18 | EUDRACT_NUMBER | None | None |