To participate in this study, you or a loved one:
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 therapy, HMI-203 over approximately 4 hours.
There will be three stages to the juMPStart clinical study: a screening period, a study period, and a follow-up period.
The screening period: The study will include a screening period of 8 weeks to make sure participants are able and eligible to receive the single I.V. dose of HMI-203, the investigational gene therapy for adult males with MPS II.
The study period: Following the single dose of HMI-203, patients will be observed periodically for 52 weeks.
The follow-up period: Finally, patients will be seen less frequently for approximately 4 additional years.
The study centers are located throughout the United States and Canada.
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 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.
