What side effects are associated with this type of intervention?

Ovarian tissue cryopreservation typically involves surgical procedures to remove ovarian tissue, followed by freezing and later re-implantation. Common side effects of these procedures include surgical risks such as infection, bleeding, and damage to surrounding tissues. Hormonal treatments used to stimulate the ovaries before tissue removal can cause side effects like ovarian hyperstimulation syndrome (OHSS), which may lead to abdominal pain, bloating, and, in severe cases, more serious complications. While gene therapy treatments like DTX301 for improving OTC function are not directly related to ovarian tissue cryopreservation, they share a focus on advanced medical interventions that may carry risks of immune reactions, infection, and potential off-target effects.

What prior FDA approvals exist?

There are no specific FDA approvals for ovarian tissue cryopreservation as it is primarily a procedure rather than a drug treatment. However, gene therapy treatments similar to DTX301, which aims to improve OTC function, have been explored in other contexts. For instance, gene editing and RNA silencing techniques are being investigated for various genetic conditions, including amyloidosis and hereditary ATTR amyloidosis. These approaches aim to correct or silence defective genes, similar to the mechanism of DTX301.

What other types of research exist?

There are no direct alternatives to DTX301 specifically for ovarian tissue cryopreservation mentioned in the provided context. However, for improving OTC function, other gene therapies or advanced treatments targeting metabolic conditions could be considered. Patients should consult with their healthcare provider to explore the most current and relevant treatment options available in 2024.

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Gene Therapy

Gene Therapy for Ornithine Transcarbamylase Deficiency

Phase 3

Recruiting

Research Sponsored by Ultragenyx Pharmaceutical Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed clinical diagnosis of late-onset OTC deficiency with historical documentation via enzymatic (ie, liver biopsy), biochemical (ie, hyperammonemia in the presence of elevated plasma glutamine, low citrulline, and elevated spot urine orotic acid), or molecular testing (ie, OTC analysis)

Free from symptomatic hyperammonemia and has not required emergent active intervention for hyperammonemia within 4 weeks before screening/baseline

Must not have

Evidence of active hepatitis B virus (HBV) or hepatitis C virus (HCV) infection, documented by current use of antiviral therapy for HBV or HCV or by hepatitis B surface antigen (HBsAg) or HCV RNA positivity

Active infection (viral or bacterial)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Pivotal Trial

Summary

This trial is testing DTX301, a treatment designed to help people with late-onset OTC deficiency, a condition that makes it hard for their bodies to process certain proteins. The treatment aims to improve the function of an enzyme that helps manage ammonia levels in the blood. Participants will be monitored for several years, with some continuing in a follow-up program for additional time.

Who is the study for?

This trial is for people with late-onset OTC deficiency who are on a stable dose of ammonia scavenger therapy and diet, have safe plasma ammonia levels, and agree to use effective contraception. It's not for those in other gene studies, with active hepatitis or significant liver issues, infections, conditions that risk participation or skew results, or detectable antibodies against the AAV8 capsid.

What is being tested?

The study tests DTX301's ability to improve OTC function by maintaining safe ammonia levels without dietary protein restrictions or alternative meds. Participants will receive either DTX301 with oral corticosteroids or placebos for both and their efficacy will be compared.

What are the potential side effects?

Potential side effects may include reactions related to gene transfer such as immune responses against the vector (AAV8), complications from corticosteroid use like increased infection risk, blood sugar changes, bone weakening among others.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with late-onset OTC deficiency through tests.

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I haven't needed urgent treatment for high ammonia levels in the last 4 weeks.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently being treated for or have tested positive for hepatitis B or C.

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I do not have any active infections.

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I have severe liver inflammation or cirrhosis.

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My kidney function is reduced, with a filtration rate below 60 mL/min.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Group I: Placebo, Then DTX301Experimental Treatment5 Interventions

Participants receive single peripheral IV infusion of placebo. At week 64, participants receive single peripheral IV infusion of DTX301 in solution.

Group II: DTX301, Then PlaceboExperimental Treatment5 Interventions

Participants receive single peripheral intravenous (IV) infusion of DTX301 in solution. At week 64, participants receive single peripheral IV infusion of placebo.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Oral Corticosteroids

2015

Completed Phase 4

~1080

0 / 6Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Ovarian tissue cryopreservation involves freezing and storing ovarian tissue for future use, often to preserve fertility in patients undergoing treatments that may harm their ovaries. Common treatments include hormonal therapies to stimulate the ovaries before tissue extraction and cryoprotectants to prevent ice formation during freezing. Gene therapy, like DTX301, aims to correct genetic deficiencies by introducing functional genes into the body. While DTX301 specifically targets OTC deficiency, the principle of gene therapy could be applied to ovarian tissue cryopreservation by potentially correcting genetic issues that affect ovarian function, thereby improving the success rates of tissue transplantation and fertility preservation.