ClinicalTrials.gov record NCT06519552

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 Given by a Single Intravenous Infusion in Patients With Mucopolysaccharidosis Type I

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.The registry record was last updated on Jul 25, 2024, about 2 years ago, so the status shown may be out of date. The study team or the registry can confirm.
Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.
PhasePhase 1An early-stage study, usually small, mainly to check safety, side effects and how much of a treatment can be given. Whether it works is not the main question yet.
Age18 years old and older
SexAll
LocationChina
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 1, open-label, single-arm dose-escalation trial tests one intravenous infusion of JWK008 in adults (age ≥18) with Mucopolysaccharidosis Type I (MPS I). Six participants will be enrolled using a '3+3' dose-escalation with two dose levels; each participant receives a single infusion. Participants will be followed and assessed for up to five years for safety, laboratory measures, imaging, and functional tests.

What it is studying. The study tests a one-time intravenous gene therapy called JWK008 that delivers an IDUA gene designed for liver-directed expression; the expressed protein is intended to reach the brain. The trial aims to collect initial safety, tolerability, and biological activity data after a single infusion.

What the researchers hope to learn. Scientific goals: to evaluate safety and tolerability (adverse events per CTCAE v5.0) and to gather initial efficacy and biological data such as IDUA enzyme activity in blood and cerebrospinal fluid, glycosaminoglycan (GAG) levels in blood/urine/CSF, six-minute walk distance, joint range of motion, liver and spleen size by CT, and vector shedding.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This study is a single-center, single-arm, non-randomized, open-label, non controlled, dose-escalation, prospective clinical trial designed to assess the safety, tolerability, and preliminary efficacy of JWK008 injection in patients with MPS I.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Genetic
  • JWK008 Single intravenous infusion administration

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Age≥ 18 years old;
  • Diagnosis of MPS type I;
  • Be able to understand the purpose, content, and possible risks of this clinical study, voluntarily participate in and sign the informed consent form;
  • If the subject is female, her sexual partner must agree to use reliable contraception until 2 consecutive tests of vector sequence in the blood are negative; If the subject is male, the subject agrees to use reliable contraception until the semen sample is negative for 2 consecutive tests of the vector sequence.
  • Magnetic resonance imaging (MRI) of the liver mass read by the radiologist is negative.

Exclusion criteria

  • Known to be unresponsive to ERT; or those who have been treated with intrathecal or intravenous laronidase and have serious adverse reactions, such as significant infusion-related reactions (IARs) or anaphylactic shock.
  • Serum AAV5 neutralizing antibody titer is greater than 1:100.
  • Has contraindications for Corticosteroids.
  • Current treatment with systemic (intravenous or oral) immunomodulators or steroid use (topical treatments such as asthma or eczema are allowed).
  • Has contraindications for lumbar puncture.
  • When filtering, one of the following situations exists:
  • Hepatitis B surface antigen (HBsAg) is positive, and the copy number of hepatitis B virus deoxyribonucleic acid (HBV-DNA) is>the upper limit of normal value (ULN);
  • Hepatitis C virus antibody (HCV-Ab) is positive, and the copy number of hepatitis C virus ribonucleic acid (HCV-RNA) is>ULN;
  • Receiving antiviral treatment for hepatitis B or C;
  • The human immunodeficiency virus (HIV) test is positive and the CD4+T lymphocyte count is ≤ 200/mm3;
  • Abnormal laboratory values considered clinically significant (ALT and/or AST >3× upper limit of normal (ULN), total bilirubin > 1.5× ULN, serum creatinine > 1.5× ULN, etc.).
  • Have a history of chronic infections or other chronic diseases that researchers believe pose an unacceptable risk;
  • Active severe infection or any other significant accompanying, uncontrolled medical condition(except for those caused by MPS I), including but not limited to kidney, liver, blood, gastrointestinal, endocrine, lung, nervous system, brain or mental illness, alcoholism, drug dependence, or any psychological disorder assessed by the researcher that may interfere with adherence to experimental protocol procedures or tolerance to JWK008 injection;
  • History of active malignancy within the past 5 years (non-melanotic skin cancer or carcinoma in situ of the cervix is allowed).
  • Circulating alpha-fetoprotein (AFP) is elevated or abnormal.
  • Previously received gene therapy or participated in interventional clinical studies within the past 12 weeks;
  • Pregnant or lactating females.
  • The researcher believes that the subject is not suitable to participate in any concurrent clinically significant major diseases or other situations in the study;
  • Unable or unwilling to comply with the visit and study evaluation schedule described in the clinical protocol.

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

MPS I is a rare autosomal recessive disease caused by deficiency of the α-L-iduronidase (IDUA) gene, which encodes a lysosomal enzyme required for degradation of glycosaminoglycans (GAGs).While currently available therapies, enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), provide clinical benefit over untreated disease progression,they still have significant limitations. ERT does not cross the blood-brain barrier and, therefore, does not treat the central nervous system (CNS) effects of the disease. And HSCT, although it can prevent cognitive decline in patients, has a high mortality rate and morbidity. The investigators have designed a novel IDUA fusion protein with the ability to cross the blood-brain barrier through the addition of the brain-targeting peptide Mtfp. On this basis, the investigators constructed an IDUA gene expression cassette for liver-targeted expression, and used a highly efficient liver-specific promoter to make the IDUA gene specifically and efficiently expressed in liver tissue, and the expressed protein can enter the central nervous system to exert therapeutic effects.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Each participant receives a single intravenous infusion of JWK008 at one of two dose levels as part of a small 3+3 dose-escalation (six participants total). Study visits include blood and urine samples, spinal taps (lumbar punctures) to collect cerebrospinal fluid, liver imaging (CT and MRI), physical tests such as the Six-Minute Walk Test and joint range-of-motion measurements, and ongoing safety monitoring for adverse events; vector shedding will be measured in CSF, serum, and urine. Follow-up and testing continue for up to five years to track safety, enzyme activity, glycosaminoglycan (GAG) levels, and other outcomes. Participants must follow contraception requirements and the study visit schedule.

How the study is structured. Phase 1 interventional trial, single-group and open-label (no masking), non-randomized. A '3+3' dose-escalation design will enroll six participants into two dose levels (5.0×10^12 vg/kg and 2.0×10^13 vg/kg). Each participant receives one IV infusion of JWK008 and is followed with scheduled safety and effectiveness assessments for up to five years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an early-phase study, mainly about safety and dose. What is already known about this treatment?
  • The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Hurler syndrome

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Important dates

Study start
Jul 22, 2024 (estimated)
Primary completion
Jun 22, 2026 (estimated)
Study completion
Jun 22, 2029 (estimated)
Target enrollment
6 participants (estimated)
Last updated on registry
Jul 25, 2024
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
West China Hospital
Principal investigator
Xingchen Peng, PhD, Professor
West China Hospital

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

Terms used on this page · explanations by MedicalUpdates.ai

single-arm
All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
randomized
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
open-label
No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
dose-escalation
Groups of participants receive increasing doses of a treatment, one group after another, to find a dose that can be given safely.
informed consent form
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
Magnetic resonance imaging
A scan that uses magnets and radio waves, not X-rays, to make detailed pictures of the inside of the body. Some implants and metal objects mean a person cannot have one.
intrathecal
Given by injection into the fluid around the spinal cord, usually through a lumbar puncture.
contraindications
A reason a particular test or treatment should not be used for a person, for example a metal implant that rules out an MRI scan.
Corticosteroids
Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
lumbar puncture
A procedure in which a needle is placed between the bones of the lower back to collect spinal fluid or to give a treatment.
upper limit of normal
The highest value of a blood test that a laboratory considers normal. Criteria such as '≤ 3 × ULN' mean the result must be no more than three times that value.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
HSCT
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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