GC1111, Hunterase, Hunter Syndrome, Hunter Syndrome (MPS II), MPS II
Conditions
Keywords
Hunterase, GC1111, GC Biopharma, Long-term study, extension study
Brief summary
The objective of this study is to assess the long-term safety and efficacy of Hunterase for approximately 1 year in subjects who completed the end of study (EOS) visit (Visit 54) tests in the Phase 3 Hunterase study and patients who received Hunterase for more than 6 months
Interventions
DRUGHunterase
0.5mg/kg weekly IV administration
Sponsors
Samsung Medical Center, Sungkyunkwan University School of Medicine
GC Biopharma Corp
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Phase 3 Extension study
Eligibility
Sex/Gender
MALE
Healthy volunteers
No
Inclusion criteria
1. Subject diagnosed with Hunter syndrome (MPS II) who completed activities at the EOS visit (Visit 54) in the GC1111\_P3 study 2. (Subject who did not participate in the GC1111\_P3 study) Subject diagnosed with Hunter syndrome (MPS II) who received Hunterase for more than 6 months 3. (Subjects who did not participate in the GC1111\_P3 study) Males aged ≥ 5 4. Informed consent form voluntarily signed by the subject or by a legally acceptable representative 5. Subject who agrees to use contraception
Exclusion criteria
1. Subject with hypersensitivity to any of the ingredients of the investigational product 2. Subject impossible to perform follow-up observation of the safety 3. Subject who received the treatment with another investigational product within 14 days prior to the start of study drug 4. Subject who plans to be treated with another investigational product during the study period 5. Subject who has history of tracheostomy, bone marrow transplant, or cord blood transplant 6. Any other inappropriate conditions for study participation at the investigator's discretion
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Laboratory tests (CBC, Chemistry, Urinanalysis) | Every visit until 12months | Abnormality of Laboratory tests results |
| Physical examination | Every visit until 12months | Abnormality of physical examination |
| Adverse Event | Every visit until 12months | occurence, grade |
| Electrocardiography | Every 6 months until 12months | Abnormality of ECG results |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 6-minute walk test | Every 4months until 12months | Mean, Change and percent change of 6-minute walk test |
| Urine GAG (Heparan sulfate, Dermatan sulfate) | Every visit until 12months | Mean, Change and percent change of Urine HS/DS |
Countries
South Korea
Outcome results
None listed