RGX-111 Gene Therapy in Patients With MPS I (NCT03580083) | Clinical Trial Compass
SuspendedPhase 1/2
RGX-111 Gene Therapy in Patients With MPS I
Stopped: The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.
United States, Brazil21 participantsStarted 2019-04-03
Plain-language summary
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.
Who can participate
Age range
4 Months
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. A male or female ≥ 4 months of age.
. Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study-related procedures. If the participant is unable to provide informed consent, then informed assent will be obtained, and informed consent must be provided by the participant's legal guardian.
. Having previously documented diagnosis of MPS I confirmed by molecular genetic testing, or IDUA deficiency in leucocytes or fibroblasts.
. Has documented evidence of CNS involvement due to MPS I based on one of the following criteria, if not explainable by any other neurologic or psychiatric factors:
. A score of ≥ 1 standard deviation below mean on neurodevelopmental testing or in 1 domain of neuropsychological function.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
. A decline of \> 1 standard deviation on sequential neurodevelopmental testing or in 1 domain of neuropsychological function administered between 3 to 36 months apart.
. Has a documented diagnosis of severe MPS I confirmed by biallelic mutations predictive of the severe phenotype or has a relative clinically diagnosed with severe MPS I and the same IDUA mutations. This participant is not required to have documented evidence of neurocognitive deficit.
. Participants who have had HSCT may be enrolled in the study if the investigator, Medical Monitor, and Sponsor agree that he/she can safely and successfully participate in the study.
Exclusion criteria
. Has any contradiction to MRI, contrast or to general anesthesia
. Has any contradiction to gadolinium
. Has renal insufficiency as determined by an estimated glomerular filtration rate (eGFR) \< 30 mL/min/1.73 m2, based on creatinine. If the laboratory determines that the creatinine level is less than the lower limit of assay validation or detection, then the lowest limit cutoff value will be used for the eGFR.
. Has a contradiction for an IC and IVR infusion, including any of the following:
. Review of baseline MRI testing by the team of neuroradiologists/neurosurgeons participating in the study (1 per site) shows a contraindication for an IC and an IVR infusion.
. History of prior head/neck surgery, which resulted in a contraindication to IC and IVR infusion, based on review of available information by the team of neuroradiologists/neurosurgeons participating in the study.
. Has previously experienced a clinically significant intracranial bleed that, in the opinion of the investigator and team of neuroradiologists/neurosurgeons, is a contraindication to IC and IVR infusion.
. Has any neurocognitive deficit not attributable to MPS I or has a diagnosis of a neuropsychiatric condition that may, in the opinion of the investigator, confound interpretation of study results.