Clinical trial • Phase III • Rare Disease

PABINAFUSP ALFA for Mucopolysaccharidosis type II | Hunter syndrome

Phase III trial of PABINAFUSP ALFA for Mucopolysaccharidosis type II | Hunter syndrome. open-label, none/not specified-controlled. 47 participants.

Overview

Trial Therapeutic Area
Rare Disease
Trial Disease
Mucopolysaccharidosis type II | Hunter syndrome
Trial Stage
Phase III
Drug Modality
Peptide/protein/enzyme
Paediatric Trial
Yes
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
08-09-2023
First CTIS Authorization Date
25-03-2024

Trial design

open-label, none/not specified-controlled Phase III trial in Italy, Spain, Poland and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
47
Trial Duration For Participant
Cohort A: 1460 days; Cohort B: 1825 days

Eligibility

Recruits 47 paediatric patients.

Vulnerable Population
Vulnerable populations are explicitly considered: if the subject is aged under 18 years (under 16 years in the UK) or if willingness to participate cannot be confirmed due to MPS II–related intellectual disability, the subject’s legally acceptable representative (e.g., parents or guardians) may sign the ICF on behalf of the subject. Written informed assent should be obtained from the subject wherever possible. Country-specific assent and caregiver/parent ICF documents are provided (examples: assent documents for under/above 12 years in France, Spain, Germany, Poland, Italy). The principal investigator or designee retains original signed consent/assent documents.

Inclusion criteria

  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- 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

  • {"criterion_text":"- A subject who changed treatment from JR-141 to idursulfase during the treatment period in the Parent Study (JR-141-GS31)."}
  • {"criterion_text":"- 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."}
  • {"criterion_text":"- [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 endpoints

  • {"endpoint_text":"- CSF HS (Total HS and HS disaccharide), dermatan sulfate (DS), and neurofilament light chain (NfL) concentrations, and opening pressure","definition_or_measurement_approach":"Measured concentrations and opening pressure in cerebrospinal fluid (CSF); specific assays and units not detailed in the public record."}
  • {"endpoint_text":"- Neuropsychological assessment","definition_or_measurement_approach":"Neuropsychological testing as per protocol (multiple standardized questionnaires and tests listed in protocol documents); exact instruments referenced in protocol questionnaires (e.g., KABC-II, WAIS/WISC/PGI scales) available in documentation."}
  • {"endpoint_text":"- 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","definition_or_measurement_approach":"Serial quality-of-life (QoL) assessments, sleep disturbance measures, global impression scales and the Toileting Abilities Survey (TAS) performed over time points in Cohorts A and B; measurement instruments referenced in protocol questionnaires."}

Secondary endpoints

  • {"endpoint_text":"- Liver and spleen volume","definition_or_measurement_approach":"Organ volumes measured (likely by imaging) as per protocol; specific modality not specified in the public JSON."}
  • {"endpoint_text":"- Shoulder range of motion","definition_or_measurement_approach":"Clinical assessment of shoulder range-of-motion per protocol."}
  • {"endpoint_text":"- Six-minute walk test","definition_or_measurement_approach":"Standard 6-minute walk test distance measurement."}
  • {"endpoint_text":"- Serum HS (Total), DS, and NfL concentrations","definition_or_measurement_approach":"Measured concentrations in serum; assay details not provided in the public JSON."}
  • {"endpoint_text":"- Urinary HS (Total) and DS concentrations","definition_or_measurement_approach":"Measured concentrations in urine; assay details not provided in the public JSON."}
  • {"endpoint_text":"- Pulmonary function (Cohort B only, if deemed feasible by principal investigator)","definition_or_measurement_approach":"Pulmonary function testing in Cohort B if feasible, per investigator judgment."}
  • {"endpoint_text":"- 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","definition_or_measurement_approach":"Serial cardiac function measures including LVMI, IVST, PWT and serial T.O.V.A.® testing in Cohort B as specified in protocol."}
  • {"endpoint_text":"- 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","definition_or_measurement_approach":"Continuous adverse event monitoring including infusion-related reactions (IARs); periodic laboratory testing, vital signs, 12-lead ECGs, anti-drug antibody testing, physical/neurological exams, and anthropometrics per schedule of assessments."}

Recruitment

Planned Sample Size
47
Recruitment Window Months
60
Consent Approach
Written IRB/IEC-approved informed consent (ICF) required and must be voluntarily signed. For subjects under 18 years (under 16 in the UK) or when participation willingness cannot be confirmed due to MPS II–related intellectual disability, a legally acceptable representative (e.g., parents/guardians) may sign the ICF on behalf of the subject; written informed assent should be obtained from the subject where possible. The principal investigator or designee retains the original signed consent and assent. Country-specific ICF and assent documents are provided in multiple languages (English, Spanish, French, Italian, Polish, German) as indicated by the uploaded country ICF and assent documents.

Geography

Total Number Of Sites
12
Total Number Of Participants
32

Italy

Earliest CTIS Part Ii Submission Date
10-07-2024
Latest Decision Or Authorization Date
31-07-2024
Processing Time Days
21
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Ospedale Pediatrico Bambino Gesu
Department Name
901:Child Neuorology, U.O.C Malattie Metaboliche ed Epatologia
Contact Person Name
Federica Deodato
Contact Person Email
federica.deodato@opbg.net
Site Name
Fondazione IRCCS San Gerardo Dei Tintori
Department Name
902:Centro malattie Metaboliche, Dipartimento Pediatria
Contact Person Name
Serena Gasperini

Spain

Earliest CTIS Part Ii Submission Date
12-03-2024
Latest Decision Or Authorization Date
25-03-2024
Processing Time Days
13
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
University Hospital Virgen Del Rocio S.L.
Department Name
603:Neurocirugía Infantil y General
Contact Person Name
Antonio Gonzalez Meneses
Site Name
Sant Joan De Deu Barcelona Hospital
Department Name
602:Neurología Pediátrica
Contact Person Name
Leticia Pias Peleteiro
Contact Person Email
leticiadiana.pias@sjd.es

Poland

Earliest CTIS Part Ii Submission Date
15-03-2024
Latest Decision Or Authorization Date
02-04-2024
Processing Time Days
18
Number Of Sites
1
Number Of Participants
6

Sites

Site Name
Uniwersytecki Szpital Dzieciecy W Krakowie
Department Name
801:Oddzial Pediatrii, Reumatologii i Chorob Rzadkich
Contact Person Name
Przemko Kwinta
Contact Person Email
przemko.kwinta@uj.edu.pl

France

Earliest CTIS Part Ii Submission Date
12-04-2024
Latest Decision Or Authorization Date
16-04-2024
Processing Time Days
4
Number Of Sites
3
Number Of Participants
10

Sites

Site Name
Hospices Civils De Lyon
Department Name
201: Chirurgie uro-génitale, viscér
Contact Person Name
Nathalie GUFFON
Site Name
Centre Hospitalier Universitaire De Montpellier
Department Name
203:Neuropédiatrie
Contact Person Name
Agathe Roubertie
Contact Person Email
a-roubertie@chu-montpellier.fr
Site Name
Trousseau Hospital
Department Name
202:Service Neuropédiatrie et Path
Contact Person Name
Benedicte HERON
Contact Person Email
benedicte.heron@trs.aphp.fr

Germany

Earliest CTIS Part Ii Submission Date
12-03-2024
Latest Decision Or Authorization Date
03-04-2024
Processing Time Days
22
Number Of Sites
4
Number Of Participants
7

Sites

Site Name
Universitaetsklinikum Giessen und Marburg GmbH
Department Name
104:Pneumology
Contact Person Name
Christina Lampe
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
101:Kinder- und Jugendmedizin
Contact Person Name
Nicole Muschol
Contact Person Email
muschol@uke.uni-hamburg.de
Site Name
SphinCS GmbH
Department Name
102:
Contact Person Name
Eugen Mengel
Contact Person Email
eugen.mengel@sphincs.de
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
103: Zentrum für Kinder- und Jugendmedizin
Contact Person Name
Julia Hennermann

Sponsor

Primary sponsor

Full Name
Jcr Pharmaceuticals Co. Ltd.
Organisation Type
Pharmaceutical company
Country Of Registered Address
Japan

Contract research organisations

Name
PPD Global Limited
Responsibilities
sponsorDuties codes: 9
Name
Parexel International (IRL) Limited
Responsibilities
sponsorDuties codes: 1, 12, 13, 2, 5
Name
Statistics & Data Corp.
Responsibilities
sponsorDuties codes: 10, 6, 7
Name
Medidata Solutions Inc.
Responsibilities
sponsorDuties code: 7
Name
Eresearchtechnology Inc.
Responsibilities
Neurocognitive assessment training and review

Third parties

  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Subject and caregiver travel bookings and reimbursements","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United Kingdom","full_name":"PPD Global Limited","duties_or_roles":"sponsorDuties codes: 9","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Primevigilance Limited","duties_or_roles":"sponsorDuties code: 8","organisation_type":"Pharmaceutical company"}
  • {"country":"Japan","full_name":"Shin Nippon Biomedical Laboratories Ltd.","duties_or_roles":"Antibody testing; sponsorDuties code: 4","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Neurocognitive assessment training and review","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"sponsorDuties code: 7","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"Japan","full_name":"EPS Corporation","duties_or_roles":"sponsorDuties code: 3","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Statistics & Data Corp.","duties_or_roles":"sponsorDuties codes: 10, 6, 7","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Medical Research Network Limited","duties_or_roles":"home trial support provider","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Duke University Health System (DUHS) Clinical Laboratories","duties_or_roles":"CRIM status","organisation_type":"Industry"}
  • {"country":"Ireland","full_name":"Parexel International (IRL) Limited","duties_or_roles":"sponsorDuties codes: 1, 12, 13, 2, 5","organisation_type":"Pharmaceutical company"}
  • {"country":"Japan","full_name":"Micron Inc.","duties_or_roles":"Medical image analysis/review - X-ray, MRI, ultrasound, etc.","organisation_type":"Pharmaceutical company"}
  • {"country":"Japan","full_name":"Shin Nippon Biomedical Laboratories Ltd.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}
  • {"country":"Japan","full_name":"Advanced Medical Laboratory of National Center for Child Health and Development","duties_or_roles":"Gene testing","organisation_type":"Industry"}
  • {"country":"United States","full_name":"Frontage Laboratories Inc.","duties_or_roles":"sponsorDuties code: 4","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
JR-141
Active Substance
PABINAFUSP ALFA
Modality
Peptide/protein/enzyme
Routes Of Administration
INTRAVENOUS INJECTION
Route
INTRAVENOUS INJECTION
Authorisation Status
prodAuthStatus 1
Orphan Designation
Yes
Maximum Dose
2.0 mg/kg

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