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Tests treatment safety and results for Mucopolysaccharidosis Type I
Official title A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
ClinicalTrials.gov ID: NCT06519552
What this study is testing
What is JWK008 Single intravenous infusion administration?
JWK008 Single intravenous infusion administration is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for mucopolysaccharidosis type i.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 1: an early, usually small safety study
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- 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...
- Magnetic resonance imaging (MRI) of the liver mass read by the radiologist is negative.
You likely can't join if
- Known to be unresponsive to ERT; or those who have been treated with intrathecal or intravenous laronidase and have serious adverse reactions, such...
- 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:
See the full eligibility 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...
- Magnetic resonance imaging (MRI) of the liver mass read by the radiologist is negative.
- 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...
- 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 treatment 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.
The study team makes the final eligibility decision.
Where it's taking place
- Chengdu, Sichuan, China
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, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Chengdu, Sichuan, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.