Ignite Creation Date:
2024-07-17 @ 10:47 AM
Last Modification Date:
2024-10-26 @ 3:33 PM
Study NCT ID:
NCT06483802
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-07-03
First Post:
2024-06-26
Brief Title:
A Study of ASP2016 in Adults Who Have Heart Disease Associated With Friedreich Ataxia
Sponsor:
Astellas Gene Therapies
Organization:
Astellas Pharma Inc
Study Overview
Official Title:
A Multicenter Open-Label Dose Escalation Phase 1b Study to Evaluate the Safety Tolerability and Preliminary Efficacy of ASP2016 for Friedreich Ataxia Cardiomyopathy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
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:
Friedreich Ataxia is a rare condition that causes damage to the nervous system and muscles People with Friedreich Ataxia have difficulty walking lose sensation in their arms and legs and have slurred speech It can also affect the heart and many people with Friedrich Ataxia develop serious heart problems Friedreich Ataxia is a genetic condition which means a faulty gene is passed down through families This type of gene therapy treats a genetic condition by providing a healthy copy of the gene
At the time this study started there was no approved treatment for heart problems in people with Friedreich Ataxia
In this study ASP2016 is being tested in humans for the first time The people taking part are adults with Friedreich Ataxia who have heart problems
The main aims of the study are to check the safety of ASP2016 and how people cope with tolerate ASP2016 ASP2016 is given as a slow injection into a vein This is called an infusion People will also take tablets of a medicine called prednisolone This is taken to stop the immune system interfering with ASP2016
Each person in the study will be given 1 single infusion of ASP2016 Different small groups will receive lower or higher doses of ASP2016 Each person will stay overnight in the clinic for at least 1 night after their infusion
For the first few months people will visit the clinic regularly There may be the option of home visits by a study nurse at some visits At the 6-month and 12-month visits extra tests procedures and scans will be done One of these is an ECHO echocardiogram scan This is like an ultrasound scan for the heart Another is an endomyocardial biopsy A tiny piece of their heart tissue is removed biopsy A flexible hollow tube catheter goes into the blood vessels up to the heart Then a small device on the end of the catheter takes a tiny piece of heart tissue about the size of a pencil tip Another is a cardiac MRI This takes pictures of the inside of the heart using a powerful magnet Another is a cardiopulmonary exercise test CPET This involves moving a specially designed set of bicycle pedals using hands and arms This will check how the lungs heart and muscles are affected during exercise
After the 12-month visit people will visit the clinic every few months for up to a few years
At the time this study started there was no approved treatment for heart problems in people with Friedreich Ataxia
In this study ASP2016 is being tested in humans for the first time The people taking part are adults with Friedreich Ataxia who have heart problems
The main aims of the study are to check the safety of ASP2016 and how people cope with tolerate ASP2016 ASP2016 is given as a slow injection into a vein This is called an infusion People will also take tablets of a medicine called prednisolone This is taken to stop the immune system interfering with ASP2016
Each person in the study will be given 1 single infusion of ASP2016 Different small groups will receive lower or higher doses of ASP2016 Each person will stay overnight in the clinic for at least 1 night after their infusion
For the first few months people will visit the clinic regularly There may be the option of home visits by a study nurse at some visits At the 6-month and 12-month visits extra tests procedures and scans will be done One of these is an ECHO echocardiogram scan This is like an ultrasound scan for the heart Another is an endomyocardial biopsy A tiny piece of their heart tissue is removed biopsy A flexible hollow tube catheter goes into the blood vessels up to the heart Then a small device on the end of the catheter takes a tiny piece of heart tissue about the size of a pencil tip Another is a cardiac MRI This takes pictures of the inside of the heart using a powerful magnet Another is a cardiopulmonary exercise test CPET This involves moving a specially designed set of bicycle pedals using hands and arms This will check how the lungs heart and muscles are affected during exercise
After the 12-month visit people will visit the clinic every few months for up to a few years
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