Ignite Creation Date:
2024-05-06 @ 7:19 PM
Last Modification Date:
2024-10-26 @ 3:04 PM
Study NCT ID:
NCT05972629
Status:
RECRUITING
Last Update Posted:
2024-02-12
First Post:
2023-06-23
Brief Title:
A Phase 1Phase 2 Open-label Study to Evaluate the Safety Tolerability and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria
Sponsor:
Sanofi
Organization:
Sanofi
Study Overview
Official Title:
A Phase 1Phase 2 Open-label Dose-escalation and Dose Expansion Study to Evaluate the Safety Tolerability and Efficacy of SAR444836 an Adeno-associated Viral Vector-mediated Gene Transfer of Human Phenylalanine Hydroxylase in Adult Participants With Phenylketonuria
Status:
RECRUITING
Status Verified Date:
2024-10
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:
This is a single group Phase 1Phase 2 1-arm open-label study with SAR444836 an adeno-associated virus AAV vector-mediated gene transfer of human phenylalanine hydroxylase PAH for the treatment of adult participants with phenylketonuria PKU on a chronic stable diet The purpose of the study is to evaluate the safety and efficacy of SAR444836 in reducing phenylalanine Phe levels and in the elimination of a Phe restricted diet
Participants will receive a one-time intravenous IV administration of SAR444836
The study is constituted of 2 separate parts a dose escalation part and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part In both study parts clinical and laboratory assessments will be collected to a assess the incidence of adverse events and b evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet
The study duration will be approximately 102 weeks approximately 2 years for each participant and includes a 6-week screening phase and 96-week follow-up period after SAR444836 administration
There will be a total of 41 study visits Many study visits may occur as remote visits and be performed by a qualified in-home service provider
Actual study duration for an individual participant may be longer than 102 weeks due to the administration of SAR444836 to participants in Stage 1A in a serial fashion or other factors such as delays in scheduling study visits
Participants will receive a one-time intravenous IV administration of SAR444836
The study is constituted of 2 separate parts a dose escalation part and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part In both study parts clinical and laboratory assessments will be collected to a assess the incidence of adverse events and b evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet
The study duration will be approximately 102 weeks approximately 2 years for each participant and includes a 6-week screening phase and 96-week follow-up period after SAR444836 administration
There will be a total of 41 study visits Many study visits may occur as remote visits and be performed by a qualified in-home service provider
Actual study duration for an individual participant may be longer than 102 weeks due to the administration of SAR444836 to participants in Stage 1A in a serial fashion or other factors such as delays in scheduling study visits
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 |
|---|---|---|---|
| 2022-501200-98 | REGISTRY | CTIS | None |
| U1111-1271-1293 | REGISTRY | None | None |