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Sodium Acetate (1-13C)

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.

Table of Contents

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[1]. 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[1]. 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[1]. 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[1]. 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[1]. 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[1]:

  1. Screening Period: This lasts up to 50 days and involves various tests to determine if a person is eligible for the study.
  2. 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.
  3. 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[1]. 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.

Aspect Details
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

FAQ

  • What is the purpose of using Sodium Acetate (1-13C) in these clinical trials? Sodium Acetate (1-13C) is used as a chemical isotopic tracer in the clinical trials. It helps researchers measure and track metabolic processes related to OTC deficiency and the effectiveness of the gene therapy treatment.
  • Who can participate in these clinical trials? The trials are open to male and female patients aged 12 years and older with a confirmed clinical diagnosis of late-onset OTC deficiency. Participants must meet specific criteria, including a history of hyperammonemia episodes and current treatment with ammonia scavenger therapy and/or a protein-restricted diet.
  • What is the main objective of the clinical trials? The main objective is to evaluate the efficacy of DTX301, a gene therapy treatment, in improving OTC function by maintaining safe plasma ammonia levels in patients with late-onset OTC deficiency.
  • How long do the clinical trials last? The trials include a screening period, a 64-week randomized treatment period, and a follow-up period. The total duration can extend up to 324 weeks (about 6 years) for some participants.
  • What are some of the key measurements taken during the trials? Key measurements include plasma ammonia levels over 24 hours, the rate of hyperammonemic crises, changes in disease management, patient-reported outcomes, and safety assessments including adverse events and laboratory values.

Ongoing Clinical Trials on Sodium Acetate (1-13C)

  • Study of CMP-CPS-001 injection in healthy volunteers and patients with abnormal ornithine transcarbamylase (OTC) genotype for treating urea cycle disorders

    Not yet recruiting

    1
    Investigated diseases:
    Investigated drugs:
    The Netherlands

  • Study of Avalotcagene Ontaparvovec for Patients Aged 12 and Older with Late-onset Ornithine Transcarbamylase Deficiency

    Not recruiting

    1 1 1
    Investigated drugs:
    France Germany Italy The Netherlands Portugal Spain

Glossary

  • Ornithine Transcarbamylase (OTC) deficiency: A rare genetic disorder that affects the urea cycle, leading to the body's inability to properly break down and remove ammonia from the blood.
  • Gene therapy: A technique that uses genes to treat or prevent disease. In this case, DTX301 is a gene therapy designed to introduce a functional OTC gene into patients' cells.
  • Hyperammonemia: A condition characterized by high levels of ammonia in the blood, which can be toxic to the brain and other organs.
  • Ammonia scavenger therapy: A treatment that uses medications to help remove excess ammonia from the body in patients with urea cycle disorders.
  • Protein-restricted diet: A diet that limits the intake of protein to help manage conditions like OTC deficiency by reducing the amount of ammonia produced in the body.
  • Plasma ammonia: The amount of ammonia present in the blood plasma, which is measured to assess the severity of OTC deficiency and the effectiveness of treatments.
  • AUC0-24: Area Under the Curve from 0 to 24 hours, a measurement used to quantify the total exposure to a substance (in this case, ammonia) over a 24-hour period.
  • Adeno-associated Virus Serotype 8 (AAV8): A type of virus used as a vector in gene therapy to deliver the corrective gene to the patient's cells.
  • Ureagenesis: The process of producing urea in the body, which is impaired in patients with OTC deficiency.
  • Hyperammonemic crisis (HAC): A medical emergency characterized by a sudden, severe increase in blood ammonia levels, which can be life-threatening for patients with OTC deficiency.

References

  1. http://clinicaltrials.eu/trial/study-of-avalotcagene-ontaparvovec-for-patients-aged-12-and-older-with-late-onset-ornithine-transcarbamylase-deficiency/