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Tests treatment safety and results for Mucopolysaccharidosis Type II
Official title A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
ClinicalTrials.gov ID: NCT05208281
What this study is testing
What is GNR-055 1.0-2.0-3.0 mg/kg?
GNR-055 1.0-2.0-3.0 mg/kg is an investigational medicine, given as an once-weekly infusion into a vein, being studied as a potential treatment for mucopolysaccharidosis type ii.
Also referred to as GNR-055.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Adults, men only
You may be able to join if
- 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 procedures.
You likely can't join if
- Clinically pronounced hypersensitivity to ID2S or any other component of the drug product;
- History of hematopoietic stem cell transplantation (HSCT) or bone marrow transplantation;
- Implanted or external non-removable metal devices, a cardiac pacemaker, or other objects sensitive to the magnetic field that may pose a danger to...
- Concomitant diseases and conditions that, in the Investigator's opinion, can put at risk the patient's safety during his/her participation in the...
The study team makes the final eligibility decision.
Where it's taking place
- Moscow, Russia
- Saint Petersburg, Russia
- Simferopol, Russia
- Ufa, Russia
- Yekaterinburg, Russia
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, not specified. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Moscow, Russia; Saint Petersburg, Russia; Simferopol, Russia; Ufa, Russia; Yekaterinburg, Russia. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.