Clinical trial NCT03568175: A Study of JR-141 in Patients With Mucopolysaccharidosis II
Official title: A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Mucopolysaccharidosis II
Lead sponsor: JCR Pharmaceuticals Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2018-06-26
Enrollment: 28
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A Phase II/ III multicenter, open-label, single-group, designed to evaluate the efficacy and safety of study drug for the treatment of the MPS II.


Eligibility criteria: Inclusion Criteria:

1. Capable of providing written consent by himself (not mandatory for those aged under 20 years at the time of informed consent process, or those who is impossible to obtain consent from the patient himself due to intellectual disabilities associated with MPS II.)
2. In the case of a patient who is under the age of 20 years or from whom it is not possible to obtain consent due to intellectual disabilities associated with MPS II, he may be included if written consent can be provided by legal representative (however, written consent should be obtained from the patient himself too, whenever possible)
3. Males with confirmed diagnosis of MPS II, based on deficient activity of iduronate-2-sulfatase (IDS) in leucocytes, plasma or fibroblasts and/or pathogenic mutations identified in the IDS gene, etc.
4. Naïve patients or patients who are receiving stable enzyme replacement therapy with Elaprase for more than 8 weeks before the observational period starts.

Exclusion Criteria:

1. Previous engrafted HSCT, excluding those who need enzyme replacement therapy even after HSCT.
2. Judged by the investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking position for lumbar puncture due to joint contracture or those who are likely experience difficulty breathing during the lumbar puncture process.
3. Judged by the investigator or subinvestigator to be ineligible to participate in the study due to a history of serious drug allergy or sensitivity.
4. Patients who have received other investigational product within 4 months before enrollment in the study.
5. Otherwise judged by the investigator or subinvestigator to be ineligible to participate in the study.

Primary outcomes:
measure: Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid
timeFrame: Baseline to 52 weeks
Secondary outcomes:
measure: Change From Baseline in Serum Heparan Sulfate Levels.
timeFrame: Baseline, 24-26 weeks, 50-52 weeks
measure: Change From Baseline in Serum Dermatan Sulfate Levels.
timeFrame: Baseline, 24-26 weeks, 50-52 weeks
measure: Change From Baseline in Urinary Heparan Sulfate Levels.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Urinary Dermatan Sulfate Levels.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Liver Volumes.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Spleen Volumes.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Cardiac Function.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in 6-minute Walk Test Distance.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Joint Range of Motion.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid.
timeFrame: Baseline to 25 weeks
measure: Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid.
timeFrame: Baseline, 25 weeks, 52 weeks
measure: Change from Baseline in Neurocognitive Testing (Kyoto Scale of Psychological Development 2001)
timeFrame: 25, 52 weeks
measure: Change from Baseline in Adaptive Behavioral Testing ( Vineland Adaptive Behavior Scales Second Edition. )
timeFrame: 25, 52 weeks
measure: Drug concentration in Cerebrospinal Fluid.
timeFrame: 25, 52*weeks
Other outcomes:
Locations:
city: Fukui
country: Japan
facility: Fukui Clinical site
city: Fukuoka
country: Japan
facility: Fukuoka Clinical site 2
city: Fukuoka
country: Japan
facility: Fukuoka Clinical site
city: Gifu
country: Japan
facility: Gifu Clinical site
city: Hokkaido
country: Japan
facility: Hokkaido Clinical site
city: Kanagawa
country: Japan
facility: Kanagawa Clinical site
city: Kumamoto
country: Japan
facility: Kumamoto Clinical site
city: Okayama
country: Japan
facility: Okayama Clinical site
city: Okayama
country: Japan
facility: Okayama Clinical site 2
city: Okinawa
country: Japan
facility: Okinawa Clinical site
city: Osaka
country: Japan
facility: Osaka Clinical site 3
city: Osaka
country: Japan
facility: Osaka Clinical site 2
city: Osaka
country: Japan
facility: Osaka Clinical site
city: Saitama
country: Japan
facility: Saitama Clinical site
city: Shizuoka
country: Japan
facility: Shizuoka Clinical site
city: Shizuoka
country: Japan
facility: Shizuoka Clinical site 2
city: Tochigi
country: Japan
facility: Tochigi Clinical site
city: Tokyo
country: Japan
facility: Tokyo Clinical site
city: Tottori
country: Japan
facility: Tottori Clinical site
Study officials:
References: