Trials / Recruiting
RecruitingNCT06458010
Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)
- Status
- Recruiting
- Phase
- EARLY_Phase 1
- Study type
- Interventional
- Enrollment
- 20 (estimated)
- Sponsor
- RenJi Hospital · Academic / Other
- Sex
- All
- Age
- 18 Years – 75 Years
- Healthy volunteers
- Not accepted
Summary
This study is a single arm, open, single dose escalation trial aimed at evaluating the safety and tolerability of YOLT-101 administration in patients with familial hypercholesterolemia; Determination of YOLT-101 OBD; Preliminary evaluation of the effects of single administration of YOLT-101 on plasma lipid and lipoprotein levels. Note: OBD is defined as the dosage at which plasma PCSK9 protein levels decrease between 60% and 95% from baseline on the 28th day after YOLT-101 administration. OBD ≤ Maximum Tolerable Dose (MTD). In this study, the longest screening period for the main study was 42 days, the treatment day was Day 1 (D1), and the safe follow-up period was up to 52 weeks after medication. In the main study, when OBD occurs, additional subjects will be added to the dose group (specific number of cases will be negotiated between the cooperating organization and investigators) for further validation. In addition, subjects in the first dose group can voluntarily receive a second drug administration of OBD level. After the completion of the main study, participants will undergo long-term follow-up. According to the Technical Guidelines for Long term Follow up Clinical Research of Gene Therapy Products (Trial) released by CDE, a long-term follow-up until 15 years after the medicine administration is required .
Detailed description
Protocol (V1.2, 6 May 2024) has been approved on 14 May 2024 Protocol (V1.3 ,23 May 2024) has been approved on 12 Jun 2024 Protocol (V1.4 ,24 Jun 2024) has been approved on 15 Jul 2024 Protocol (V1.5 ,18 Oct 2024) has been approved on 28 Oct 2024 Protocol (V1.6 ,27 Nov 2024) has been approved on 27 Dec 2024 Protocol (V1.7 ,6 Jan 2025) has been approved on 27 Jan 2025 Protocol (V1.8 ,19 Feb 2025) has been approved on 24 Mar 2025
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | YOLT-101 | The IP is administered intravenously at the predetermined dose. |
Timeline
- Start date
- 2024-05-24
- Primary completion
- 2026-01-25
- Completion
- 2026-01-25
- First posted
- 2024-06-13
- Last updated
- 2025-04-30
Locations
1 site across 1 country: China
Source: ClinicalTrials.gov record NCT06458010. Inclusion in this directory is not an endorsement.