Actively Recruiting
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
Led by AceLink Therapeutics, Inc. · Updated on 2024-07-30
16
Participants Needed
6
Research Sites
135 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
This is a Phase II, open-label study designed to evaluate the safety, tolerability, PK, PD, and preliminary efficacy of AL01211 in male subjects with classic Fabry disease who have never received any treatment (eg. ERT or chaperone therapy). Eligible subjects will receive 182 days (26 weeks) of study treatment as the primary study period followed by an extension period. The total study duration for a subject is up to at most 2 years including the primary period of 26 weeks.
CONDITIONS
Official Title
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male subjects aged 18 to 60 years at screening
- Never received any Fabry disease-specific treatment (eg, enzyme replacement or chaperone therapy)
- Signed and dated informed consent before any study procedures
- Confirmed diagnosis of Fabry disease by genetic variant and enzyme activity tests
- Estimated glomerular filtration rate (eGFR) of at least 50 mL/min/1.73 m2 at screening
- Willing to comply with all study procedures including observational follow-up beyond 52 weeks
- Stable dose of RAAS inhibitors/blockers or SGLT2 inhibitors for at least 3 months before screening if applicable
- Presence of at least one symptom characteristic of Fabry disease (e.g., neuropathic pain, gastrointestinal, renal, or cardiac symptoms)
- Willing to have eye examinations with photo documentation at baseline and during the study
- Plasma Lyso GL3 level of 25 ng/mL or higher
You will not qualify if you...
- On regular dialysis or has had a kidney transplant
- Clinically significant abnormal liver function
- Scheduled for in-patient hospitalization or elective surgery during the study
- Positive for hepatitis B surface antigen, hepatitis C antibodies, or HIV antibodies
- Significant cataracts affecting more than a quarter of the lens or 30% opacity
- Currently or recently (within past month) taking medications that may cause cataracts
- Male with partner of child-bearing potential not agreeing to use effective contraception during and 3 months after study
- Medical, emotional, behavioral, or psychological conditions interfering with study compliance
- Participation in another investigational drug study within 90 days or 5 half-lives of last dose
- Unwillingness to comply with study protocol
- Major recent cardiovascular events or unstable cardiac disease within 6 months
- Cardiac diseases that mimic Fabry or contraindicate MRI
- Acute kidney injury in past 12 months or specific kidney diseases
- Use of herbal medicines within 14 days prior to screening
- Use of strong or moderate CYP3A4 inducers/inhibitors or grapefruit products near enrollment
- Any other conditions making participation unsuitable as judged by the investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 6 locations
1
The First Affiliated Hospital of Sun Yat sen University
Guangzhou, Guangdong, China, 510062
Actively Recruiting
2
The First Affiliated Hospital of Zhengzhou University
Zhengzhou, Henan, China, 450052
Actively Recruiting
3
Xiangya Hospital of Central South University
Changsha, Hunan, China, 410008
Actively Recruiting
4
West China Hospital, Sichuan University
Chengdu, Sichuan, China, 610044
Actively Recruiting
5
Peking University First Hospital
Beijing, China, 100034
Actively Recruiting
6
Ruijin Hospital, Shanghai Jiaotong University School Of Medicine
Shanghai, China, 200025
Actively Recruiting
Research Team
A
Acelink Clinical Trial
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here