Mucopolysaccharidosis II Clinical Trial
Official title:
An Open-Label, Phase 1 and Extension Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ Who Have Central Nervous System Involvement and Are Receiving Treatment With Intravenous Drug
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of intracerebroventricular GC1123 in patients with MPS Ⅱ who have central nervous system involvement and are receiving treatment with intravenous drug
Status | Recruiting |
Enrollment | 12 |
Est. completion date | June 2026 |
Est. primary completion date | June 2026 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 18 Months to 18 Years |
Eligibility | Inclusion Criteria: 1. Patient who has been diagnosed with severe MPS ? (Hunter syndrome) 2. Patient, aged 1.5 years (18 months) to 18 years at the time of the screening 3. Patient who has received and tolerated a minimum of 12 weeks of treatment with weekly intravenous treatment, and who has received 80% of the total planned infusions within that time frame. 4. Patient who is capable of undergoing neurosurgery, which has been confirmed by neurosurgeons and anesthesiologist. 5. Patient eligible to execute patient evaluation activities during the clinical trial period, as assessed by the investigator 6. Patient whose parents or legal representative are willing to participate in this clinical trial and provide written informed consent form Exclusion Criteria: 1. Patient who has been administered with intrathecal Idursulfase in the past 2. Patient with a history of bone marrow transplantation or cord blood transplant 3. Patient with a history of ventriculoperitoneal shunt or other intracranial surgeries 4. Patient with end-stage multiple organ dysfunction syndrome or other severe diseases 5. Patient who is exposed to malignant neoplasm 6. Patient who has received treatment with any investigational drug or device within 30 days prior to study entry 7. Patient who have experience of hypersensitivity or anaphylaxis to ingredients of the investigational product at the time of screening 8. Patient with a history of bronchotomy/tracheostomy, or patient with acute respiratory disease at the time of screening 9. Patient who is ineligible to participate in the clinical trial due to laboratory test results or other reasons, as determined by the investigator |
Country | Name | City | State |
---|---|---|---|
Korea, Republic of | Pusan National University Yangsan Hospital | Pusan | |
Korea, Republic of | Samsung Medical Center | Seoul | |
Korea, Republic of | Seoul National University | Seoul |
Lead Sponsor | Collaborator |
---|---|
GC Biopharma Corp |
Korea, Republic of,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Other | Development Function assessed by Bayley Scales of Infant and Toddler Development-III and/or Kaufman Assessment Battery for Children-II (BSID-III/KABC-II) | All children will be tested for BSID-III, and children over the age of 3 will be also tested for KABC-II. | Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110) | |
Other | Adaptive Function assessed by Vineland Adaptive Behavior Scales 2nd Ed. (VABS-II) | Children under the age of 19 years will be tested for VABS-II. | Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110) | |
Other | Quality of Life (Survey) assessed by Infant and Toddler Quality of Life Questionnaire (ITQOL) and/or Childhood Health Questionnaire parent form (CHQ-PF50) | Children from the age of 2 months to 5 years will be tested for ITQOL, and children over the age of 5 will be tested for CHQ-PF50. The test performed during screening will be continued to be performed for each patient throughout the study period. | Approximately every 6 months (Week 1 [baseline], Week 26, Week 54, Week 82, Week 110) | |
Other | Liver and Spleen volume | Liver and Spleen volume measured by MRI -phase I only | Week 1 to phase I study completion (about 26 weeks) | |
Primary | Incidence and frequency of serious adverse events (SAEs) | Incidence and frequency of serious adverse events (SAEs) after administration of ICV-Hunterase (GC1123) | Every 28 days from Week 1 through study completion (about 110 weeks) | |
Primary | Frequency and characteristics (severity, outcome, etc.) of adverse events | Frequency and characteristics (severity, outcome, etc.) of adverse events after administration of ICV-Hunterase (GC1123) | Every 28 days from Week 1 through study completion (about 110 weeks) | |
Primary | Presence of clinically significant abnormal echocardiography results | Presence of clinically significant abnormal echocardiography results after administration of ICV-Hunterase (GC1123); phase I only | Week 1 to Phase I study completion (about 26 weeks) | |
Secondary | Pharmacokinetic (PK) parameters - Cmax | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - Tmax | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - AUClast | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - AUCinf | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - t1/2 | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - CL/F (or CL) | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - Vd/F (or Vd) | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacokinetic (PK) parameters - Bioavailability (F) | Pharmacokinetic (PK) parameters of ICV-Hunterase (GC1123) in serum and CSF | Week 2 to Week 22 | |
Secondary | Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in CSF | Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123) | Every 28 days from Week 1 through study completion (about 110 weeks) | |
Secondary | Pharmacodynamic (PD) parameters - Heparan Sulfate (HS) in serum | Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123) | Every 28 days from Week 1 through study completion (about 110 weeks) | |
Secondary | Pharmacodynamic (PD) parameters - Urine Glycosaminoglycan (GAG) | Pharmacodynamic (PD) parameters of ICV-Hunterase (GC1123) | Every 28 days from Week 1 through study completion (about 110 weeks) | |
Secondary | Presence of anti-drug antibodies (ADAs) | Presence of anti-drug antibodies (ADAs) in CSF and serum, and neutralizing antibodies of ICV-Hunterase (GC1123) | Approximately every 6 months (Week 2 [baseline], Week 18, Week 26, Week 54, Week 82, Week 110) |
Status | Clinical Trial | Phase | |
---|---|---|---|
Withdrawn |
NCT05238324 -
Safety and Efficacy of HMI-203 in ERT-Treated Adults With MPS II
|
Phase 1 | |
Completed |
NCT03529786 -
Mucopolysaccharidosis Type II Natural History
|
||
Recruiting |
NCT02254863 -
UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells
|
Phase 1 | |
Terminated |
NCT01675674 -
Study to Detect Unrecognized Mucopolysaccharidosis in Children Visiting Rheumatology, Hand or Skeletal Dysplasia Clinics
|
N/A | |
Enrolling by invitation |
NCT06075537 -
An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007
|
Phase 2/Phase 3 | |
Completed |
NCT00069641 -
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
|
Phase 2/Phase 3 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Active, not recruiting |
NCT04348136 -
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
|
Phase 2/Phase 3 | |
Completed |
NCT04007536 -
A Study of Potential Treatment-Responsive Biomarkers and Clinical Outcomes in Hunter Syndrome
|
||
Completed |
NCT00004454 -
Phase I/II Study of Retroviral-Mediated Transfer of Iduronate-2-Sulfatase Gene Into Lymphocytes of Patients With Mucopolysaccharidosis II (Mild Hunter Syndrome)
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT04628871 -
Long Term Follow-up (LTFU) of Subjects Who Received SB-318, SB-913, or SB-FIX
|
||
Terminated |
NCT00748969 -
Clinical Trial of Growth Hormone in MPS I, II, and VI
|
Phase 2/Phase 3 | |
Recruiting |
NCT05619900 -
Registry of Patients Diagnosed With Lysosomal Storage Diseases
|
||
Completed |
NCT01301898 -
To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients
|
Phase 1/Phase 2 | |
Terminated |
NCT03041324 -
Ascending Dose Study of Genome Editing by the Zinc Finger Nuclease (ZFN) Therapeutic SB-913 in Subjects With MPS II
|
Phase 1/Phase 2 | |
Enrolling by invitation |
NCT05368038 -
ScreenPlus: A Comprehensive, Flexible, Multi-disorder Newborn Screening Program
|
||
Completed |
NCT03128593 -
A Study of JR-141 in Patients With Mucopolysaccharidosis Type II
|
Phase 1/Phase 2 | |
Withdrawn |
NCT04591834 -
Mucopolysaccharidosis Type II Observational
|
||
Enrolling by invitation |
NCT04597385 -
Long-term Follow-Up for RGX-121
|
||
Active, not recruiting |
NCT03153319 -
Study to Evaluate the Safety and Efficacy of Adalimumab in MPS I, II, and VI
|
Phase 1/Phase 2 |