CompletedPhase 1/2

Safety and Dose Ranging Study of Human Insulin Receptor MAb-IDUA Fusion Protein in Adults and Children With MPS I

Brazil21 participantsStarted 2015-10

Plain-language summary

AGT-181 is a fusion protein containing alpha-L-iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously. This is a safety and tolerability study to obtain safety and exposure data as well as information on the biological activity of the investigational drug. This is a two-stage, sequential, single and multi-dose study of AGT-181 in patients with MPS I. The first stage will be an open-label, single-dose, dose-escalation cohort study and the second stage will be an open-label, multi dose, adaptive dose escalation cohort study.

Who can participate

Age range

2 Years

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: * Written consent and assent as required * Diagnosis of MPS 1 confirmed by clinical signs and symptoms, documented fibroblast or leukocyte IDUA enzyme activity of less than 10% the lower limit of normal * Female patients must not be pregnant, willing to utilize appropriate birth control methods and undergo pregnancy testing during the study * if taking standard ERT, must be willing to discontinue for 1 week prior to dosing and for the study duration Additional Inclusion Criteria, Stage 1: * 18 years of age or older * must have a diagnosis of Hurler-Scheie or Scheie syndrome Additional Inclusion Criteria, Stage 2: * 2 years of age or older (and less than 18) * must be willing to undergo CNS testing, including assessment of CSF via lumbar puncture, MRI scans and neurocognitive testing * must have evidence of Hurler-Scheie or Scheie with CNS involvement, as evidence by: * score of 1 to 3 standard deviations below mean on IQ testing (i.e. IQ=55 or more) or in one domain of neuropsychological function (language, memory, non-verbal ability) OR * documented historical evidence of a decline greater than 1 standard deviation on sequential testing, OR * score between 0.75 and 1 standard deviation below the mean, AND cognitive deficit affects daily performance Exclusion Criteria: * Refusal to complete all assessments * Pregnant or Lactating * Received investigational drug within 1 year prior to study enrollment * Medical condition or extenuating circumstance t…

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.

What they're measuring

Stage 1: number of patients with adverse events as a measure of safety and tolerability of a single dose

Timeframe: 4 weeks

Stage 2: number of patients with adverse events as a measure of safety and tolerability of repeat weekly doses

Timeframe: 26 weeks

Trial details

NCT IDNCT03053089

SponsorArmaGen, Inc

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2018-02-01

View on ClinicalTrials.gov More Mucopolysaccharidosis I trials