A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Recruiting
Phase 2
Phase 3
Interventional Study
Mucopolysaccharidosis Type II
Metabolic Diseases
No Placebo Group
Every participant receives an active treatment — no one gets a placebo.
Pivotal Trial
This treatment is in the last trial phase before FDA approval.
Prior Safety Data
This treatment has already been tested in at least one earlier human trial.
At a Glance
- Sex
- Male
- Trial phase
- Phase 2/3
- Study type
- Interventional
- Purpose
- Treatment
- Participants needed
- 32 (estimated)
- Sponsor
- AO GENERIUM · Industry
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About This Trial
This is phase 2/3 study to evaluate the safety, pharmacokinetics, pharmacodynamics, and efficacy of the investigational product GNR-055 in MPS II (Hunter syndrome) patients of different age groups.
Trial Locations
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Eligibility Criteria
Inclusion Criteria:
* Signed inform consent;
* Verified diagnosis of MPS II (Hunter syndrome);
* Naïve patients or patients who have received standard ERT whit idursulfase products;
* No contraindications for lumbar puncture as judged by the Investigator;
* Willingness and ability to follow study p…
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