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Phase I/II study of weekly infusions of JR-441 in patients with mucopolysaccharidosis type IIIA
EU CTIS ID: 2024-517045-14-00
What this study is testing
To evaluate the safety and explore efficacy of JR-441 in development for the treatment of MPS IIIA patients.
- Human Pharmacology (Phase I)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- (1) Chronological age of ≥1 year and ≤18 years at the time of signing ICF.
- (2) A participant who voluntarily signs an IRB or IEC-approved written ICF. If the participant is aged 1year to <18 years at the time of informed consent, or willingness to participate in the study cannot be confirmed due to MPS IIIA-related intellectual disability, the participant’s legally acceptable representative (e.g., his/her parents or guardians) may sign the ICF on behalf of the participant. Written informed assent must be obtained from the participant, wherever possible..
- (3) A participant with a confirmed diagnosis of MPS IIIA, based on all the following criteria: ○ Activity of the N-sulphoglucosamine sulphohydrolase (SGSH) enzyme below 10% of the lower reference level in white blood cells or cultured skin fibroblasts. ○ A normal enzyme activity level of at least one other sulfatase (to rule out multiple sulfatase deficiency) as measured in leukocytes. ○ Presence of a pathological mutation in each of the individual alleles of the SGSH gene. Note: if SGSH enzyme activity results are abnormal (i.e., below the normal range of the assay) but still above the threshold of 10% of the lower reference level, MPS IIIA diagnosis may be confirmed based on family history and genotype following discussion and approval from the sponsor’s Medical Monitor.
- (4) Study participants should have a minimal body weight of 10 kg.
- (5) Female participants of childbearing potential or participants whose female partner is of child-bearing potential agree to use a medically accepted, highly effective method of contraception as described in Section 10.5, from the time of signing the ICF. The method of contraception must be used during the study until 90 days for male participants, and 30 days for female participants after the final study drug administration or vasectomy at least 13 weeks prior to signing ICF..
- (6) For participants with hearing impairment requiring hearing aid(s), every effort has been made to encourage compliance with the use of functioning hearing aid(s) before baseline neurodevelopmental assessments, and parent/legally acceptable representative or participant agrees to encourage wearing them during the study and on neurodevelopmental function test days.
You likely can't join if
- (1) A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
- (10) A participant who has had a ventriculoperitoneal (VP) shunt placed or any other brain surgery
- (11) A participant has a history of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
- (12) A participant who has a history of poorly controlled seizures.
- (13) Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or C (HepC) infection.
- (14) A participant who has lab abnormalities with CTCAE grade ≥ II for liver function test, bilirubin, creatinine, hemoglobin, white blood cell count, platelet count, prothrombin time, and activated partial thromboplastin time (aPTT), except subject whose bilirubin elevated due to Gilbert’s Syndrome.
See the full eligibility criteria
Who can join
- (1) Chronological age of ≥1 year and ≤18 years at the time of signing ICF.
- (2) A participant who voluntarily signs an IRB or IEC-approved written ICF. If the participant is aged 1year to <18 years at the time of informed consent, or willingness to participate in the study cannot be confirmed due to MPS IIIA-related intellectual disability, the participant’s legally acceptable representative (e.g., his/her parents or guardians) may sign the ICF on behalf of the participant. Written informed assent must be obtained from the participant, wherever possible..
- (3) A participant with a confirmed diagnosis of MPS IIIA, based on all the following criteria: ○ Activity of the N-sulphoglucosamine sulphohydrolase (SGSH) enzyme below 10% of the lower reference level in white blood cells or cultured skin fibroblasts. ○ A normal enzyme activity level of at least one other sulfatase (to rule out multiple sulfatase deficiency) as measured in leukocytes. ○ Presence of a pathological mutation in each of the individual alleles of the SGSH gene. Note: if SGSH enzyme activity results are abnormal (i.e., below the normal range of the assay) but still above the threshold of 10% of the lower reference level, MPS IIIA diagnosis may be confirmed based on family history and genotype following discussion and approval from the sponsor’s Medical Monitor.
- (4) Study participants should have a minimal body weight of 10 kg.
- (5) Female participants of childbearing potential or participants whose female partner is of child-bearing potential agree to use a medically accepted, highly effective method of contraception as described in Section 10.5, from the time of signing the ICF. The method of contraception must be used during the study until 90 days for male participants, and 30 days for female participants after the final study drug administration or vasectomy at least 13 weeks prior to signing ICF..
- (6) For participants with hearing impairment requiring hearing aid(s), every effort has been made to encourage compliance with the use of functioning hearing aid(s) before baseline neurodevelopmental assessments, and parent/legally acceptable representative or participant agrees to encourage wearing them during the study and on neurodevelopmental function test days.
- (7) Medically stable and able to accommodate the protocol requirements, including travel without placing an undue burden on the participant/participant’s family, as determined by the principal investigator.
What rules you out
- (1) A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.
- (10) A participant who has had a ventriculoperitoneal (VP) shunt placed or any other brain surgery
- (11) A participant has a history of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.
- (12) A participant who has a history of poorly controlled seizures.
- (13) Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or C (HepC) infection.
- (14) A participant who has lab abnormalities with CTCAE grade ≥ II for liver function test, bilirubin, creatinine, hemoglobin, white blood cell count, platelet count, prothrombin time, and activated partial thromboplastin time (aPTT), except subject whose bilirubin elevated due to Gilbert’s Syndrome.
- (15) A participant with known iron-metabolism disorder.
- (16) A participant with visual or hearing impairment sufficient, in the clinical judgment of the principal investigator or sub-investigator, to preclude cooperation with neurodevelopmental testing.
- (17) A participant currently receiving psychotropic or other medications which in the principal investigator’s or sub-investigator’s opinion, would be likely to substantially confound test results.
- (18) A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant’s ability to comply with protocol requirements, the participant’s well-being or safety, or the interpretability of the participant’s clinical data.
- (19) A participant who is ineligible to participate in the study in the opinion of the principal investigator or sub-investigator.
- (2) A participant who is pregnant or breast feeding.
- (3) A participant who has received another investigational drug or product within 4 months or 5 half-lives (whichever is longer) before the time of providing informed consent.
- (4) A participant who is participating concurrently or who has participated prior (within 30 days of enrolment into this study) in a study involving invasive procedures.
- (5) A participant who has received Genistein within 4 months before the time of providing informed consent.
- (6) A participant who has received KINERET® (anakinra) within 4 months before the time of providing informed consent.
- (7) A participant who has developed serious drug allergy or hypersensitivity to any components of JR-441 or medications likely prescribed during the study, which, in the opinion of the principal investigator or sub-investigator, would be an impediment towards completion of the study.
- (8) A participant unable to undergo lumbar puncture.
- (9) A participant unable to undergo MRI.
The study team makes the final eligibility decision.
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.