Is NCT07748403 recruiting in New Haven, CT?

Yes, NCT07748403 is currently recruiting participants at its New Haven, CT location. This wilson disease clinical trial is actively enrolling qualified participants.

How do I join the wilson disease clinical trial in New Haven?

To join this clinical trial in New Haven, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this New Haven clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Wilson Disease Clinical Trial in New Haven, CT

About This Wilson Disease Study in New Haven

The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM577a, an investigational Prime Editing therapy, in adults and adolescents with Wilson disease (WD). Wilson disease is caused by changes (mutations) in the ATP7B gene that prevent the body from removing excess copper normally. PM577a is designed to precisely correct one of the most common disease-causing ATP7B mutations (p.H1069Q) in liver cells with the goal of restoring normal copper metabolism. This is the first study of PM577a in people. Participants will receive a single intravenous (IV) infusion of PM577a and will be monitored closely to evaluate safety, how the body responds to treatment, whether copper metabolism improves, and whether treatment may improve signs and symptoms of Wilson disease.

Sponsor: Prime Medicine, Inc.

Who Can Participate

Inclusion Criteria

Confirmed Wilson Disease (WD) diagnosis as determined by medical history consistent with WD

Historical genetic analysis demonstrating biallelic pathogenic, likely pathogenic, or suspected pathogenic ATP7B variants, including at least one p.H1069Q allele.

Treated and stable on standard of care therapy for WD for the past 6 months prior to signing ICF, as documented by a history of adherence to SOC medications (i.e., penicillamine, trientine, and/or zinc) without significant medication or dose/frequency changes, per Investigator judgement.

Demonstrated Adequate Copper Control confirmed at screening

Willingness to maintain a stable, copper-conscious diet and avoid copper-containing supplements, from signing of the ICF until the physician determines that it is no longer necessary post-infusion.

Willingness to abstain from alcohol use from start of the screening period through 3 months after PM577a infusion and adhere to recommendations of moderate alcohol consumption (as defined in this protocol) through primary follow-up period.

Participants are expected to enroll in a separate long-term follow-up study for a total of approximately 15 years of follow-up following PM577a administration.

Exclusion Criteria

Known prior medically significant reactions (e.g., severe hypersensitivity, myocarditis) to an LNP-based or PEG-containing product (e.g., mRNA-based COVID vaccinations, MiraLAX) or any medication required as part of the clinical study protocol

Receipt of any prior gene therapy for WD, including AAV-based therapy

Receipt of any liver-directed LNP gene therapy (including siRNA or ASO therapies) or gene editing treatment.

Unstable neurological conditions within the prior 12 months which may impact participant safety or participation in the study, including ability to complete study requirements or procedures as outlined in the clinical study protocol in the opinion of the Investigator

In individuals with psychiatric involvement, current or fluctuant clinical instability with new or changing diagnoses or substantial medication regimen changes in the past 12 months that could limit their participation, in the opinion of the Investigator.

Receipt of prior liver transplantation or listed for transplantation

Body Mass Index ≥ 35 kg/m2

Evidence of Severe Hepatic Impairment or uncontrolled liver disease within 6 months before screening.

Evidence of Moderate or Severe Renal Impairment in the last year

History of significant liver disease other than WD

Clinically significant coagulopathy or disorder of platelet function

Active infectious disease including:

Known or suspected active systemic infection

Receipt of systemic antimicrobial therapy within 30 days of screening.

Positive for presence of human immunodeficiency virus (HIV)-1 or HIV-2 (evidence of infection, regardless of viral load).

History of known autoimmune or genetic causes of myopathy or myositis

Prior or current malignancy or myeloproliferative disorder (excluding Stage 1 or lower, fully treated/excised malignant and pre-malignant disease of the skin, cervix or colon. Additionally, any other malignant and pre-malignant disease that the Investigator in consultation with the treating oncologist and study Medical Monitor deem has been fully treated/excised for \> 5 years).

Any condition, laboratory abnormality, or reason that could adversely affect participant safety or study results, as determined by the Investigator, including, but not limited to:

Clinical evidence of unstable coronary disease as defined by history of myocardial infarction in the past 24 months, history of unstable angina

Any severe clinical condition of limited life expectancy

Pregnancy or breastfeeding in a postpartum female or absence of adequate contraception for fertile participants. Females of childbearing potential and non-sterile male participants who are or may become sexually active with female partners of childbearing potential are required to use highly effective contraception from Screening through at least 84 days after drug product infusion.

History of moderate or severe Alcohol Use Disorder (AUD) or Drug Use Disorder (DUD) with \< 3 years of continuous abstinence preceding the date of screening

Participation in another clinical study with an investigational drug within 30 days of Screening or at least 5 times the half-life of the investigational drug (whichever is longer).

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in New Haven?

Yes, this clinical trial (NCT07748403) has an active research site in New Haven, CT that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Wilson Disease Treatment Options in New Haven, CT

If you're searching for wilson disease treatment options in New Haven, CT, this clinical trial (NCT07748403) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our New Haven research site is actively enrolling participants for this phase 1/2 clinical trial. You'll receive care from experienced wilson disease specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all wilson disease clinical trials near you to find additional studies recruiting in your area.

Wilson Disease Clinical Trial in New Haven, CT

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