In short
This article discusses the use of Sodium Acetate (1-13C) in clinical trials for patients with late-onset Ornithine Transcarbamylase (OTC) deficiency. The trials aim to evaluate the effectiveness of a gene therapy treatment called DTX301 in improving OTC function and maintaining safe plasma ammonia levels. The study involves patients aged 12 and older and explores various aspects of the treatment's impact on patient health outcomes and safety.
At a glance
- Study Type
- Phase 3, randomized, double-blind, placebo-controlled
- Condition
- Late-onset Ornithine Transcarbamylase (OTC) deficiency
- Intervention
- DTX301 (gene therapy) vs. Placebo
- Primary Endpoints
- Plasma ammonia levels at Week 64, Percentage of patients achieving complete response
- Key Secondary Endpoints
- Response categories, Patient-reported outcomes, Rate of hyperammonemic crises, Safety assessments
- Eligibility
- Patients 12 years and older with confirmed late-onset OTC deficiency
- Study Duration
- Up to 324 weeks (including follow-up)
- Role of Sodium Acetate (1-13C)
- Chemical isotopic tracer for metabolic measurements
What is SODIUM ACETATE (1-13C)?
SODIUM ACETATE (1-13C) is a special form of sodium acetate that contains a specific type of carbon atom (carbon-13). It's not a treatment itself, but rather a diagnostic tool used in medical research. This compound is being used in a clinical trial to help study a rare genetic disorder called Ornithine Transcarbamylase (OTC) Deficiency.
Understanding OTC Deficiency
Ornithine Transcarbamylase (OTC) Deficiency is a rare genetic disorder that affects the body's ability to break down protein. People with this condition lack an important enzyme that helps remove ammonia from the body. As a result, ammonia can build up in the blood, which can be very dangerous. The late-onset form of OTC deficiency, which is the focus of this study, typically appears later in life and can vary in severity.
Clinical Trial Information
The clinical trial using SODIUM ACETATE (1-13C) is part of a larger study investigating a potential gene therapy treatment for late-onset OTC deficiency. The main goals of this study are:
- To see if the gene therapy (called DTX301) can improve OTC function and keep ammonia levels in the blood at safe levels
- To evaluate how well DTX301 works in treating OTC deficiency
- To assess the safety of DTX301
- To understand how DTX301 affects the overall health of patients with OTC deficiency
How SODIUM ACETATE (1-13C) is Used
In this study, SODIUM ACETATE (1-13C) is given as an oral solution, which means it's a liquid that patients drink. It's used as a tracer, helping researchers track certain processes in the body. The maximum daily dose is 50 milliliters, and the maximum total dose over the study period is 300 milliliters.
This compound is not a treatment for OTC deficiency. Instead, it helps researchers measure how well the body is processing certain substances, which can give important information about how the gene therapy is working.
Who Can Participate in the Study?
The study is looking for participants who meet certain criteria. Some key points include:
- Patients must be 12 years of age or older
- They must have a confirmed diagnosis of late-onset OTC deficiency
- They must have a history of at least one episode of high ammonia levels in the blood
- They must be currently receiving treatment to manage their condition
There are also several factors that would prevent someone from participating, such as having had a liver transplant or certain other medical conditions.
Study Design and Procedures
The study is divided into several phases:
- Screening Period: This lasts up to 50 days and involves various tests to determine if a person is eligible for the study.
- Treatment Period: This lasts 64 weeks. Participants are randomly assigned to receive either the gene therapy (DTX301) or a placebo. After 64 weeks, they switch to the other treatment.
- Follow-up Period: This continues for several years after treatment to monitor long-term effects and safety.
Throughout the study, participants will need to undergo various tests and procedures, including blood tests, urine tests, and questionnaires about their health and quality of life.
Potential Benefits and Risks
While the potential benefits of the gene therapy being studied are significant, it's important to note that clinical trials also come with risks. The study is designed to carefully monitor participants for any side effects or adverse reactions. The use of SODIUM ACETATE (1-13C) as a diagnostic tool is generally considered safe, but as with any medical procedure, there may be some risks involved.
It's crucial for anyone considering participating in this or any clinical trial to discuss the potential risks and benefits thoroughly with their healthcare provider and the research team.