NCT07193615

Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

Clinical Exploratory Study of YOLT-202 in the Treatment of Alpha-1 Antitrypsin Deficiency (AATD)

• Status: Active Not Recruiting
• Phase: EARLY_Phase 1
• Study type: Interventional
• Enrollment: 18 (estimated)
• Sponsor: RenJi Hospital · Academic / Other
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This is a single-arm, open-label, single-dose, dose-escalation exploratory study to evaluate the safety and tolerability of a single dose of YOLT-202 in patients with AATD and determine the optimal biologically active dose (OBD) of YOLT-202.

Detailed description

This is a single-arm, open-label, single-dose, dose-escalation exploratory study to evaluate the safety and tolerability of a single dose of YOLT-202 in patients with AATD, determine the OBD of YOLT-202, and preliminarily assess the impact of a single dose of YOLT-202 on changes in FEV1 and FEV1/FVC ratio from baseline after bronchodilator use. Note: The OBD is defined as the dose achieving either a blood functional-AAT/total-AAT protein ratio of 50%-95% at Day 21 post-dose, or a blood total-AAT protein concentration ≥ 11 μM at Day 21 post-dose. OBD ≤ maximum tolerated dose (MTD). In this study, the maximum screening period of the main study is 60 days, the treatment day is D0, and the safety follow-up period is up to Week 52 after administration. In the main study, when OBD is achieved, additional subjects will be added to the dose group (the sponsor and the investigator will jointly negotiate the specific number of subjects) for further verification. After completing safety follow-up, subjects may voluntarily receive a second dose of the investigational drug at the OBD level based on comprehensive assessments of efficacy, safety, and PK/PD data. After the end of the main study, the subjects will undergo long-term follow-up.

Conditions

• Alpha-1 Antitrypsin Deficiency (AATD)

Interventions

Type	Name	Description
DRUG	YOLT-202	The IP is administered intravenously at the predetermined dose; Administration frequency: Once

Timeline

• Start date: 2025-06-18
• Primary completion: 2027-01-01
• Completion: 2027-03-01
• First posted: 2025-09-26
• Last updated: 2025-09-26

Locations

1 site across 1 country: China