Ignite Creation Date:
2024-05-06 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 1:52 PM
Study NCT ID:
NCT04683003
Status:
RECRUITING
Last Update Posted:
2024-04-29
First Post:
2020-12-10
Brief Title:
A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Sponsor:
Takeda
Organization:
Takeda
Study Overview
Official Title:
A Phase 3b Prospective Open-label Multicenter Single Treatment Arm Continuation Study of the Safety and Efficacy of TAK-755 rADAMTS13 Also Known as BAX 930SHP655 in the Prophylactic and On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura cTTP Upshaw-Schulman Syndrome or Hereditary Thrombotic Thrombocytopenic Purpura
Status:
RECRUITING
Status Verified Date:
2024-04
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 also occur People who have TTP may bleed underneath the skin forming purple bruises or purpura TTP also can cause anemia a condition in which red blood cells break apart faster than the body can replace them leading to fewer red blood cells than in normal
TTP is caused by a lack of activity in the ADAMTS13 enzyme a protein in the blood involved in controlling clotting of the blood The ADAMTS13 enzyme breaks up another blood protein called von Willebrand factor that forms blood clots by clumping together with platelets Some people are born with this condition while others develop the condition during their life Many people who are born with TTP experience frequent flare-ups that need to be treated right away TAK-755 is a medicine that replaces ADAMTS13 and may prevent or control TTP flare-ups called acute TTP events
The main aim of the study is to check for side effects of long-term treatment with TAK-755 Treatment will be given in 2 ways
1 TAK-755 treatment given either every week or every other week to prevent acute TTP events from happening the prophylactic cohort
2 TAK-755 treatment given to control an acute TTP event when it happens the on-demand cohort
Participants in the prophylactic cohort will receive treatment in the clinic or at home for up to approximately 3 years They will visit the clinic at least every 12 weeks Participants in the on-demand cohort will receive daily treatment for the acute TTP event until the flare-up has gotten better They will have a follow-up visit at the clinic 4 weeks later
TTP is caused by a lack of activity in the ADAMTS13 enzyme a protein in the blood involved in controlling clotting of the blood The ADAMTS13 enzyme breaks up another blood protein called von Willebrand factor that forms blood clots by clumping together with platelets Some people are born with this condition while others develop the condition during their life Many people who are born with TTP experience frequent flare-ups that need to be treated right away TAK-755 is a medicine that replaces ADAMTS13 and may prevent or control TTP flare-ups called acute TTP events
The main aim of the study is to check for side effects of long-term treatment with TAK-755 Treatment will be given in 2 ways
1 TAK-755 treatment given either every week or every other week to prevent acute TTP events from happening the prophylactic cohort
2 TAK-755 treatment given to control an acute TTP event when it happens the on-demand cohort
Participants in the prophylactic cohort will receive treatment in the clinic or at home for up to approximately 3 years They will visit the clinic at least every 12 weeks Participants in the on-demand cohort will receive daily treatment for the acute TTP event until the flare-up has gotten better They will have a follow-up visit at the clinic 4 weeks later
Detailed Description:
This is a follow-up study to the Phase 3 pivotal study 281102 NCT03393975 and will be comprised of two treatment cohorts Prophylactic and On-demand consisting of naïve and non-naïve participants who were born with TTP with a total duration of approximately 6 years A maximum of approximately 77 participants will be enrolled in this study approximately 57 participants who have completed study 281102 NCT03393975 non-naïve participants and at least 20 naïve participants participants who are naïve to TAK-755 Participants from Expanded Access Programs participants from study 281102 NCT03393975 who had an allergic reaction to standard of care treatment and participants who completed the Phase 1 study 281101 NCT02216084 but did not participate in Study 281102 will also be eligible for enrollment in this continuation study as naïve participants
TAK-755 is given slowly through a vein intravenous infusion Participants in the prophylactic cohort will be able to opt for treatment in a home setting by caregiver or self-infusion subject to conditions and to local regulatory approval
TAK-755 is given slowly through a vein intravenous infusion Participants in the prophylactic cohort will be able to opt for treatment in a home setting by caregiver or self-infusion subject to conditions and to local regulatory approval
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 |
|---|---|---|---|
| 2020-003348-10 | EUDRACT_NUMBER | None | None |