Study of YOLT-203 in Children and Adults With Primary Hyperoxaluria Type 1 (PH1)
Purpose
This study will be conducted to evaluate the efficacy and safety of YOLT-203 in children and adults with Primary Hyperoxaluria Type 1. After the initial randomized, 6-month double-blind, placebo-controlled period, participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion. For patients interested in additional information on how to participate, please follow this link: https://mytomorro.ws/ph1trial.
Conditions
- Primary Hyperoxaluria Type 1
- PH1
Eligibility
- Eligible Ages
- Over 6 Years
- Eligible Sex
- All
- Accepts Healthy Volunteers
- No
Inclusion Criteria
- You are 6 years old or older. - You have been diagnosed with PH1 (primary hyperoxaluria type 1) through genetic testing. - Urine tests show that your oxalate levels are not within certain limits (based on at least two 24-hour urine collections). - If you take vitamin B6, your dose has been stable for at least 3 months, and you are willing to keep it the same during the study. - You (and/or your parent or legal guardian, if you are under 18) understand the study and agree to follow all study requirements, including signing a consent form.
Exclusion Criteria
- You have signs that oxalate has affected parts of your body outside the kidneys. - Your liver blood tests are not within certain limits. - You have certain abnormal blood clotting results (unless controlled with medication like warfarin). - You have HIV, or active or chronic hepatitis C. - You are pregnant, planning to become pregnant, or breastfeeding. - You are not willing to use birth control during the study and for 6 months after (if applicable). - You are unable to limit alcohol intake to no more than about 2 drinks per day. - You have had alcohol or drug abuse in the past year. - You are unable to follow standard treatments for PH1 (such as drinking plenty of fluids, taking prescribed medications like potassium citrate, or following dietary advice).
Study Design
- Phase
- Phase 2
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Crossover Assignment
- Intervention Model Description
- After the initial 6-month double-blind period, participants will enter a 6-month treatment extension period. Participants who were initially assigned to placebo will receive a single-dose of YOLT-203 treatment whereas participants in the YOLT-203 group will receive a single-dose of placebo infusion.
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
| Arm | Description | Assigned Intervention |
|---|---|---|
|
Experimental YOLT-203 |
Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive YOLT-203 during the double-blind treatment period and will cross over to the placebo treatment in the subsequent treatment period, followed by safety and efficacy follow-up. |
|
|
Placebo Comparator Placebo |
Participants will receive both study treatments in sequential treatment periods according to randomized assignment. Participants who initially receive placebo during the double-blind treatment period and will cross over to the YOLT-203 treatment in the subsequent treatment period, followed by safety and efficacy follow-up. |
|
Recruiting Locations
University of Alabama at Birmingham (UAB Hospital)
Birmingham, Alabama 35249
Birmingham, Alabama 35249
More Details
- Status
- Recruiting
- Sponsor
- YolTech Therapeutics Co., Ltd