An Extension Study of JR-171-101 Study in Patients With MPS I

Mucopolysaccharidosis I Clinical Trial

Official title:

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04453085
• Other study ID #: JR-171-102
• Status: Active, not recruiting
• Phase: Phase 1/Phase 2
• Start date: October 28, 2021
• Est. completion date: April 30, 2025

Study information

• Verified date: March 2024
• Source: JCR Pharmaceuticals Co., Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Phase I/II, open label, multicenter, multinational (Japan, Brazil and the US) extension study of JR-171-101 for the treatment of MPS I

Description:

Patients who have completed the Part2 of JR-171-101 study and fulfill all eligibility criteria can be enrolled in this JR-171-102 study. Until the dose determination in the JR-171-102 study, subjects will intravenously receive either low dose or high dose of JR-171 at the same doses received at Week 12 of the JR-171-101 study. Thereafter, all subjects will be transitioned to the optimal dose determined based on the results of JR-171-101 study.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 14
• Est. completion date: April 30, 2025
• Est. primary completion date: April 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Patients who have completed the Part 2 of JR-171-101 study

• A patient from whom written informed consent can be obtained. If the patient is aged under 18 years (20 years in case of Japan) at the time of assent or willingness to participate in the study cannot be confirmed due to MPS I-related intellectual disability, informed permission from the patient's legally acceptable representative (e.g., his/her parents or guardians) need to be obtained instead of his/her consent. Even in this case, written informed consent should be obtained from the patient, wherever possible

• Female patient or male patient whose co-partners is of child-bearing potential agree to use a medically accepted, highly effective method of contraception, such as spermatocidal gel plus condom, an intrauterine device or oral contraceptives until one month after the final administration.

Exclusion Criteria:

• A patient who is unable to perform the study procedures, except for 6-minute walk test, neurocognitive testing, BVMT-R, HVLT-R, and T.O.V.A.

• Judged by the principal investigator or subinvestigator as being unable to undergo lumbar puncture, including those who have difficulties in taking a position for lumbar puncture due to joint contracture or those who are likely to experience difficulty breathing during the lumbar puncture process

• Judged by the principal investigator or subinvestigator to be ineligible to participate in the study due to a history of a serious drug allergy or hypersensitivity to any drugs

• Otherwise judged by the principal investigator or subinvestigator to be ineligible to participate in the study in consideration of patient's safety

Related Conditions & MeSH terms

• Mucopolysaccharidoses
• Mucopolysaccharidosis I

Intervention

Drug:
• JR-171 (lepunafusp alfa)
JR-171 IV infusion

Locations

Country	Name	City	State
Brazil	Hospital de Clínicas de Porto Alegre	Porto Alegre
Brazil	Instituto de Genética e Erros Inatos do Metabolismo - IGEIM	São Paulo
Japan	Fukuoka Children's Hospital	Fukuoka
Japan	National Hospital Organization Kokura Medical Center	Kitakyushu
Japan	Osaka Metropolitan University Hospital	Osaka
United States	UCSF Benioff Children's Hospital Oakland	Oakland	California

Sponsors (1)

Lead Sponsor	Collaborator
JCR Pharmaceuticals Co., Ltd.

Countries where clinical trial is conducted

United States,  Brazil,  Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of participants with Adverse Events	Adverse events	156 weeks
Primary	Incidence of abnormal laboratory test results	Hematology	156 weeks
Primary	Incidence of abnormal laboratory test results	Biochemistry	156 weeks
Primary	Incidence of abnormal laboratory test results	Serum iron tests	156 weeks
Primary	Incidence of abnormal laboratory test results	Urinalysis	156 weeks
Primary	Incidence of abnormal vital signs	Pulse rate	156 weeks
Primary	Incidence of abnormal vital signs	Body temperature	156 weeks
Primary	Incidence of abnormal vital signs	Blood pressure	156 weeks
Primary	Incidence of abnormal vital signs	Body weight	156 weeks
Primary	Incidence of abnormal EKG readings		156 weeks
Primary	Number of participants with Adverse Events	Anti-human-a-L-iduronidase antibodies	156 weeks
Primary	Number of participants with Adverse Events	Anti-JR-171 antibodies	156 weeks
Primary	Number of participants with Adverse Events	Infusion associated reaction (IAR)	156 weeks
Secondary	Change From Baseline in Heparan Sulfate Levels in Cerebrospinal Fluid		Weeks 26, 52, 104, 156
Secondary	Change From Baseline in Dermatan Sulfate Levels in Cerebrospinal Fluid		Weeks 26, 52, 104, 156
Secondary	CSF opening pressure		Weeks 26, 52, 104, 156
Secondary	Change From Baseline in Heparan Sulfate Levels in Urinary		Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Secondary	Change From Baseline in Dermatan Sulfate Levels in Urinary		Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Secondary	Change From Baseline in Heparan Sulfate Levels in Serum		Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Secondary	Change From Baseline in Dermatan Sulfate Levels in Serum		Weeks 13, 26, 39, 52, 65, 78, 91, 104, 130, 156
Secondary	Change From Baseline in Liver Volume		Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Spleen Volume.		Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	Left ventricular posterior wall thickness	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	interventricular septal thickness	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	left ventricular mass index	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	left ventricular ejection fraction	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	left ventricular ejection fraction,	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in Echocardiography.	E/A ratio	Weeks 13, 26, 52, 78, 104, 156
Secondary	Change From Baseline in 6-minute Walk Test Distance.		Weeks 13, 26, 52, 78, 104, 156
Secondary	Changes from baseline in cognitive age equivalent score of neurocognitive testing	Wechsler IQ test or the Bayley scales of infant and toddler development	Weeks 52, 104, 156
Secondary	Changes from baseline in outcome of adaptive behavioral function	Vineland adaptive behavior scales	Weeks 52, 104, 156
Secondary	Changes from baseline in outcome of the Brief Visuospatial Memory Test-Revised		Weeks 13, 26, 52, 78, 104, 130, 156
Secondary	Changes from baseline in outcome of the Hopkins Verbal Learning Test-Revised		Weeks 13, 26, 52, 78, 104, 130, 156
Secondary	Changes from baseline in outcome of the Test of Variables of Attention		Weeks 13, 26, 52, 78, 104, 130, 156
Secondary	Changes from baseline in outcome of the Pediatric Quality of Life Inventory Family Impact Module (PedsQL-FIM)		Weeks 13, 26, 52, 104 156