A study to extend treatment with JR-141 after completion of Study JR-141-GS31

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Jcr Pharmaceuticals Co. Ltd.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

6 May 2024 → ongoing

Decision date (initial)

2024-04-16

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

JCR Pharmaceuticals Co., Ltd., ORG-100032023

External identifiers

EU CT number

2022-503142-41-00

ClinicalTrials.gov

NCT05594992

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy, Others

To evaluate the long-term efficacy of JR-141 on CNS symptoms in subjects with MPS II

Secondary objectives 2

1. To evaluate the long-term efficacy of JR-141 on somatic symptoms in subjects with MPS II
2. To evaluate the long-term safety of JR-141 in subjects with MPS II

Conditions and MedDRA coding

Mucopolysaccharidosis type II

Study design 1 period

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. A subject who participated in the Parent Study (JR-141-GS31) and completed the assessments at Week 105 in Cohort A or Week 53 in Cohort B before being administered the study medication of that respective visit, and in the opinion of the investigator, there are no safety concerns.
2. A subject from whom an IRB or IEC-approved written informed consent form (ICF) can be obtained, which is voluntarily signed. If the subject is aged under 18 years (aged under 16 years in the United Kingdom [UK]) at the time of enrollment or willingness to participate in the study cannot be confirmed due to MPS II related intellectual disability, the subject’s legally acceptable representative (e.g., his parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject, wherever possible. The principal investigator or his/her designee will retain the original copy of each subject’s signed consent and assent (if applicable) document.
3. Female subject of child bearing potential or male subject whose female partner is of child-bearing potential , i.e., fertile, following menarche and until becoming post- menopausal unless permanently sterile, agrees to use a medically accepted, highly effective method of contraception, from the time of signing the ICF.

Exclusion criteria 3

1. A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31).
2. A subject who is unable to comply with the protocol (e.g., is unable to return for safety evaluations or is otherwise unlikely to complete the study) as determined by the principal investigator or sub-investigator.
3. [Only in France] Persons deprived of their liberty by a judicial or administrative decision, according to article L. 1121-6 of the Public Health Code (Code dela santé publique, CSP) adults who are the subject of a measure of legal protection or unable to express their consent according to article L. 1121-8 of the CSP.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 3

1. CSF HS (Total HS and HS disaccharide), dermatan sulfate (DS), and neurofilament light chain (NfL) concentrations, and opening pressure
2. Neuropsychological assessment
3. Others: Time course of QoL assessment, sleep disturbance, and global impression in Cohort A and Cohort B, Time course of Toileting Abilities Survey (TAS) in Cohort A and Cohort B

Secondary endpoints 8

1. Liver and spleen volume
2. Shoulder range of motion
3. Six-minute walk test
4. Serum HS (Total), DS, and NfL concentrations
5. Urinary HS (Total) and DS concentrations
6. Pulmonary function (Cohort B only, if deemed feasible by principal investigator)
7. Others: Time course of cardiac function (LVMI, IVST, and PWT) in Cohort A and Cohort B, Time course of T.O.V.A.® in Cohort B
8. Safety endpoints (Cohort A and Cohort B): Continuous assessment of AEs, including IARs, Periodical assessment of laboratory tests (hematology, blood biochemistry, iron-related tests, and urinalysis); vital signs (pulse rate, body temperature, respiratory rate, blood pressure); 12-lead ECG; anti-JR-141 antibodies, physical and neurological examination, weight, and height

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Jcr Pharmaceuticals Co. Ltd.

Sponsor organisation

Jcr Pharmaceuticals Co. Ltd.

Address

3-19 Kasugacho

City

Ashiya

Postcode

659-0021

Country

Japan

Scientific contact point

Organisation

Jcr Pharmaceuticals Co. Ltd.

Contact name

JCR Pharmaceuticals Co., Ltd.

Public contact point

Organisation

Jcr Pharmaceuticals Co. Ltd.

Contact name

JCR Pharmaceuticals Co., Ltd.

Third parties 15

Locations

5 EU/EEA countries · 13 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 243 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

12 submissions — initial application plus substantial / non-substantial modifications