FAQs

To participate in this study, you or a loved one must:

• Have uncontrolled classical PKU disease due to PAH deficiency in which the PAH gene is not working properly
• Be an adult aged 18 to 55 years
• Be willing and able to maintain a stable Phe-restricted diet
• Be willing to commit to required study visits and tests for the duration of the trial
• Not have contraindications to immunosuppressant therapy

There are additional criteria that are reviewed by the study team during your first appointment.

You will meet with a study coordinator at a study site location closest to you. You will learn more about the research study and the benefits and risks of participation.

The study coordinator or study doctor will review the Informed Consent Form with you. You will have the chance to ask any questions about this form, and sign once comfortable and willing to participate in the study. Different procedures will be performed to see if you qualify for the clinical study.

A patient would receive a single intravenous (I.V.) administration of the Investigational gene editing candidate known as HMI-103.

This study will occur in a series of stages for 115 weeks (this includes screening, observation, and follow-up visit time). In every stage of the study, a variety of standard and specific medical tests and exams such as genotyping, vital sign review, and ECG, will be performed. The full list of all medical tests, exams, and procedures can be discussed with the site staff.

Interested patients will have two separate planned clinic visits during the screening period, which is the portion of the study dedicated to learning more about the medical history and status of the patient. The screening period also assists in determining the eligibility of patients for the study. The screening period can last up to 47 days.

Participants can expect 4 separate planned clinic visits during the run-in period. The run-in period of the study continues to learn more about the medical status of the patient, while preparing for the upcoming investigational gene editing treatment. The run-in period will last approximately 35 days.

Enrolled patients will enter the treatment period after the run-in period. The treatment period is approximately one day where the patient will receive a single I.V. administration of HMI-103 over approximately 4 hours.

Enrolled patients will then continue into the next stage of the study in order to investigate how the patient is responding to HMI-103. This stage of the study will last 104 weeks. The week of receiving the treatment (week 1) will require the enrolled patient to follow up every day, followed by progressively longer intervals between visits until the end of the study. Certain patients will be able to undergo some visits at home.

The study center is located in the United States.

You do not have to stop taking your medication unless you are advised to do so by the study center staff or your primary care physician.

No, there will be no cost to you for the study therapy or study procedures.

No, health insurance is not a requirement to participate in this study.

Clinical trials, also known as clinical studies or research studies are conducted by doctors and researchers to, among other things, evaluate whether investigational medications or treatments are safe and effective before being approved for use by the general public. Participation in a clinical trial is completely confidential.

You can find out more information about clinical trials by browsing through www.clinicaltrials.gov. This is an online, government database that is managed by the National Library of Medicine. It provides information about both federally and privately supported clinical research studies.

All research studies generally require that doctors and/or researchers give interested participants accurate information about the potential risks, benefits and activities of a research study. Interested participants will sign an Informed Consent Form prior to enrolling in the study. This form shows that a participant understands what will happen during the study and that they can leave the study at any time.