CTX310 for High Cholesterol
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial tests CTX310, a new treatment for individuals with high cholesterol unresponsive to standard treatments. The goal is to assess how the body processes different doses of CTX310 through IV infusion. Participants should have struggled with high cholesterol levels despite using other medications for at least 12 weeks. This trial may suit those with persistent high cholesterol that hasn't improved with other treatments. As a Phase 1 trial, participants will be among the first to receive this new treatment, aiding researchers in understanding its effects in people.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications. However, it mentions that participants should be refractory to standard lipid-lowering therapies, which suggests you might need to continue your current treatment.
Is there any evidence suggesting that CTX310 is likely to be safe for humans?
Research has shown that CTX310 is generally safe for people. In a previous study, participants who took CTX310 did not experience harmful reactions, even at higher doses, indicating the treatment did not cause serious medical problems.
Administered as a single intravenous (IV) infusion, the treatment has demonstrated promising safety results. Specifically, no participants experienced serious side effects in the studies reviewed. These findings suggest that CTX310 might be a safe option for individuals with high cholesterol that does not improve with other treatments.12345Why do researchers think this study treatment might be promising?
CTX310 is unique because it is designed to lower high cholesterol through an intravenous (IV) infusion, which is different from the oral medications like statins that are typically used. Most treatments for high cholesterol work by inhibiting cholesterol production in the liver, but CTX310 may have a different mechanism of action that targets cholesterol in a novel way. Researchers are excited about CTX310 because this innovative delivery method could offer an alternative for patients who cannot tolerate oral medications or do not respond well to current therapies.
What evidence suggests that CTX310 might be an effective treatment for high cholesterol?
Research has shown that CTX310, the investigational treatment in this trial, could be promising for high cholesterol. Studies indicate that CTX310, which uses the gene-editing technology CRISPR, safely reduced LDL cholesterol (often called "bad" cholesterol) by up to 60%. It also lowered triglycerides (a type of fat in the blood) by about 55%. These improvements appeared just two weeks after a single treatment. The treatment targets a protein called ANGPTL3, which is linked to cholesterol and triglyceride levels. Although these findings are early, they suggest that CTX310 might offer a new way to manage difficult-to-control cholesterol levels.13567
Are You a Good Fit for This Trial?
This trial is for adults aged 18–75 with high cholesterol or abnormal blood fats that haven't improved despite using the strongest available treatments. Participants must have stable organ function, not be pregnant, and agree to use birth control if needed.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive a dose of CTX310 via intravenous (IV) infusion
Follow-up
Participants are monitored for safety and effectiveness after treatment
What Are the Treatments Tested in This Trial?
Interventions
- CTX310
Trial Overview
The study tests CTX310, a new medication given in increasing doses to see how safe and tolerable it is for people whose cholesterol problems haven’t responded to standard therapies. All participants receive the drug; there’s no placebo group.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Subjects will receive an intravenous (IV) infusion.
Find a Clinic Near You
Who Is Running the Clinical Trial?
CRISPR Therapeutics AG
Lead Sponsor
Citations
CRISPR Therapeutics Announces Positive Phase 1…
Overall, CTX310 demonstrated a well-tolerated safety and tolerability profile that supports continued advancement of the program. Efficacy ...
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ...
Results: A total of 15 participants received CTX310 and had at least 60 days of follow-up. No dose-limiting toxic effects related to CTX310 ...
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ...
We conducted an ascending-dose phase 1 trial to assess the safety and efficacy of CTX310, a lipid-nanoparticle–encapsulated clustered ...
CRISPR Therapeutics Reports Positive Additional…
-New Phase 1 clinical data for CTX310™ continues to demonstrate dose-dependent reductions in triglycerides (TG) and low-density lipoprotein ...
CRISPR gene editing of angiopoietin-like 3: toward one- ...
Individuals with natural loss-of-function variants in ANGPTL3 display profoundly reduced triglycerides and LDL cholesterol, along with markedly ...
Phase 1 Trial of CRISPR-Cas9 Gene Editing Targeting ...
Primary Outcome- Safety: Incidence of Adverse Events. No dose-limiting toxic effects or serious adverse events related to CTX310. Serious ...
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newsroom.heart.org
newsroom.heart.org/news/first-in-human-trial-of-crispr-gene-editing-therapy-safely-lowered-cholesterol-triglyceridesFirst-in-human trial of CRISPR gene-editing therapy safely ...
In this trial, both LDL cholesterol and triglyceride levels dropped within two weeks after treatment and stayed low for at least 60 days.
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