A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias

New gene editing therapy CTX310 tested for hard-to-treat cholesterol.

NCT: NCT07491172 · Status: RECRUITING · Phase: Phase 1 · Sponsor: CRISPR Therapeutics AG · Started: 2024-06-21 · Est. Completion: 2028-06

Plain English Summary

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias is a Phase 1 clinical trial sponsored by CRISPR Therapeutics AG studying Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia. This trial tests a new gene editing therapy called CTX310 to see if it's safe and well-tolerated. It is for adults with severe cholesterol problems that haven't improved with existing treatments. Participants will receive CTX310, and their health will be closely monitored. Alternative treatments include various cholesterol-lowering medications and lifestyle changes. The trial aims to enroll 90 participants.

Official Summary

This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.

Who Can Participate

Here is what you need to know about eligibility for this trial. Adults aged 18-75 with specific high cholesterol or triglyceride levels. Must have tried and failed at least 12 weeks of standard cholesterol treatments. Cannot have certain liver, kidney, heart, or blood conditions. Women must be postmenopausal or surgically sterile; men and their partners must use effective contraception. This trial is studying Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia, so participants generally need a confirmed diagnosis. The trial is currently accepting new participants.

What They're Measuring

The primary outcome measures how safe CTX310 is and how well the body tolerates it, meaning researchers will focus on any side effects or adverse reactions to ensure patient well-being. The specific primary outcome measures are: To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments (From CTX310 infusion up to 12 months). These endpoints are how researchers determine whether the treatment is effective and will form the basis of any future regulatory submissions.

About This Phase

This trial is in Phase 1, the first major stage of clinical testing. Phase 1 trials typically involve 20-100 participants and focus on safety, dosage levels, and side effects. The primary goal is not to test whether the treatment works but to establish that it is safe enough for further testing. About 70% of Phase 1 drugs advance to Phase 2. If successful, the treatment will proceed to Phase 2 efficacy testing.

Why This Trial Matters

This trial addresses a significant unmet need for patients with severe, treatment-resistant dyslipidemias by exploring a novel gene editing approach to lower harmful blood lipids. This research targets Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia, where improved treatment options are needed.

Investor Insight

This trial represents an early-stage investment in a potentially groundbreaking gene therapy for a large market of cardiovascular patients, with CRISPR Therapeutics aiming to establish a new treatment Phase 1 trials have approximately a 10% chance of eventually gaining FDA approval.

Is This Trial Right for Me?

Ask your doctor about the potential benefits and risks of CTX310, and how it differs from your current treatments. Participation involves receiving the study drug and attending regular medical appointments for monitoring. You will need to follow specific instructions regarding contraception and avoid certain medications during the trial. This trial is currently recruiting participants. The trial is being conducted at 18 sites. Always discuss clinical trial participation with your healthcare provider before making any decisions. This information is for educational purposes only and is not medical advice.

AI-generated analysis for educational purposes only. This is not medical advice. Discuss clinical trial participation with your doctor. Data sourced from ClinicalTrials.gov.

Study Design

Interventions

Primary Outcomes

Secondary Outcomes

Full Eligibility Criteria

Key Inclusion Criteria:

1. Age of ≥18 and ≤75 years at the time of signing the informed consent.
2. Able to provide written informed consent.
3. Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL.
4. Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening
5. Female participants must be postmenopausal or surgically sterile.
6. All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study.

Exclusion Criteria:

1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
2. Evidence of liver disease, defined as but not limited to:

   LFTS \>2 × upper limit of normal (ULN), or total bilirubin \>2 × ULN, or INR \>1.5 × ULN, or liver stiffness measured by liver elastography
3. Abnormal or compromised function of kidney, heart, blood or liver.
4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
7. Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast).
8. Women of childbearing potential.

Note: Other protocol defined Inclusion/Exclusion criteria may apply.

Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.

Trial Locations

Frequently Asked Questions

What is clinical trial NCT07491172?

NCT07491172 is a Phase 1 INTERVENTIONAL study titled "A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias." It is currently recruiting and is sponsored by CRISPR Therapeutics AG. The trial targets enrollment of 90 participants.

What conditions does NCT07491172 study?

This trial investigates treatments for Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH), Homozygous Familial Hypercholesterolemia (HoFH), Severe Hypertriglyceridemia (sHTG), Mixed Hyperlipemia, Hypercholesterolaemia. The primary condition under study is Cardiovascular.

What treatments are being tested in NCT07491172?

The interventions being studied include: CTX310 (DRUG). CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).

What does Phase 1 mean for NCT07491172?

Phase 1 trials are the first stage of testing a new treatment in humans. They focus on safety, dosage, and side effects, usually involving 20-100 healthy volunteers or patients.

What is the current status of NCT07491172?

This trial is currently "Recruiting." It started on 2024-06-21. The estimated completion date is 2028-06.

Who is sponsoring NCT07491172?

NCT07491172 is sponsored by CRISPR Therapeutics AG. The sponsor is responsible for funding, designing, and overseeing the clinical trial.

How many people can participate in NCT07491172?

The trial aims to enroll 90 participants. The trial is currently recruiting and accepting new participants.

How is NCT07491172 designed?

This is a interventional study, uses na allocation, follows a single_group design, employs none masking.

What are the primary outcomes being measured in NCT07491172?

The primary outcome measures are: To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments (From CTX310 infusion up to 12 months). These are the main endpoints researchers use to determine whether the treatment is effective.

Where is NCT07491172 being conducted?

This trial is being conducted at 18 sites, including Jacksonville, Florida; Orlando, Florida; Port Orange, Florida; Durham, North Carolina and 14 more sites (United States, Australia, New Zealand).

Where can I find official information about NCT07491172?

The official record for NCT07491172 is available on ClinicalTrials.gov at https://clinicaltrials.gov/study/NCT07491172. This government database provides the most up-to-date and detailed information about the trial.

What is NCT07491172 testing in simple terms?

This trial tests a new gene editing therapy called CTX310 to see if it's safe and well-tolerated. It is for adults with severe cholesterol problems that haven't improved with existing treatments.

Why is this trial significant?

This trial addresses a significant unmet need for patients with severe, treatment-resistant dyslipidemias by exploring a novel gene editing approach to lower harmful blood lipids.

What are the potential risks of participating in NCT07491172?

Potential side effects may include reactions at the infusion site, and effects on liver function. As this is a gene editing therapy, long-term effects are still being studied. The therapy targets a specific gene, and unintended effects on other genes are a theoretical risk. As with any clinical trial, participants are closely monitored and can withdraw at any time.

Should I consider participating in NCT07491172?

Ask your doctor about the potential benefits and risks of CTX310, and how it differs from your current treatments. Participation involves receiving the study drug and attending regular medical appointments for monitoring. You will need to follow specific instructions regarding contraception and avoid certain medications during the trial. Always discuss clinical trial participation with your healthcare provider to determine if it is appropriate for your specific situation.

What does NCT07491172 signal from an investment perspective?

This trial represents an early-stage investment in a potentially groundbreaking gene therapy for a large market of cardiovascular patients, with CRISPR Therapeutics aiming to establish a new treatment This is a Phase 1 trial, which is in early development stages.

What happens if the treatment in this trial doesn't work?

Participants will receive CTX310, and their health will be closely monitored. Participants in clinical trials always have the right to withdraw and pursue alternative treatments. The study team will help transition patients to other available options.

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This analysis is AI-generated and does not constitute medical advice. Always consult your healthcare provider before making decisions about clinical trial participation.