NCT06519552 A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of JWK008 in Patients With Mucopolysaccharidosis Type I
| NCT ID | NCT06519552 |
| Status | Recruiting |
| Phase | Phase 1 |
| Sponsor | West China Hospital |
| Condition | Mucopolysaccharidosis Type I |
| Study Type | INTERVENTIONAL |
| Enrollment | 6 participants |
| Start Date | 2024-07-22 |
| Primary Completion | 2026-06-22 |
Eligibility & Interventions
Eligibility Fast-Check
Enter your details for a quick preliminary check. This does not replace medical advice.
What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 1 is the earliest stage of human testing — safety and dosage are the primary focus. Visits are frequent and medical supervision is intensive. You will be among the first people to receive this treatment.
This trial targets 6 participants in total. It began in 2024-07-22 with a primary completion date of 2026-06-22.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
Eligibility Criteria
Inclusion Criteria: 1. Age≥ 18 years old; 2. Diagnosis of MPS type I; 3. Be able to understand the purpose, content, and possible risks of this clinical study, voluntarily participate in and sign the informed consent form; 4. 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. 5. Magnetic resonance imaging (MRI) of the liver mass read by the radiologist is negative. Exclusion Criteria: 1. 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. 2. Serum AAV5 neutralizing antibody titer is greater than 1:100. 3. Has contraindications for Corticosteroids. 4. Current treatment with systemic (intravenous or oral) immunomodulators or steroid use (topical treatments such as asthma or eczema are allowed). 5. Has contraindications for lumbar puncture. 6. When filtering, one of the following situations exists: 1. 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); 2. Hepatitis C virus antibody (HCV-Ab) is positive, and the copy number of hepatitis C virus ribonucleic acid (HCV-RNA) is\>ULN; 3. Receiving antiviral treatment for hepatitis B or C; 4. The human immunodeficiency virus (HIV) test is positive and the CD4+T lymphocyte count is ≤ 200/mm3; 7. 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.). 8. Have a history of chronic infections or other chronic diseases that researchers believe pose an unacceptable risk; 9. 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; 10. History of active malignancy within the past 5 years (non-melanotic skin cancer or carcinoma in situ of the cervix is allowed). 11. Circulating alpha-fetoprotein (AFP) is elevated or abnormal. 12. Previously received gene therapy or participated in interventional clinical studies within the past 12 weeks; 13. Pregnant or lactating females. 14. The researcher believes that the subject is not suitable to participate in any concurrent clinically significant major diseases or other situations in the study; 15. Unable or unwilling to comply with the visit and study evaluation schedule described in the clinical protocol.
Contact & Investigator
Xingchen Peng, Ph.D
PRINCIPAL INVESTIGATOR
West China Hospital
Frequently Asked Questions
Who can join the NCT06519552 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Mucopolysaccharidosis Type I. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT06519552 trial and what does that mean for participants?
Phase 1 trials are the first stage of human testing. The primary goal is to assess safety and determine appropriate dosage levels. Participants are closely monitored. These trials typically involve a small number of volunteers.
Is NCT06519552 currently recruiting?
Yes, NCT06519552 is actively recruiting participants. Contact the research team at pxx2014@163.com for enrollment information.
Where is the NCT06519552 trial being conducted?
This trial is being conducted at Chengdu, China.
Who is sponsoring the NCT06519552 clinical trial?
NCT06519552 is sponsored by West China Hospital. The principal investigator is Xingchen Peng, Ph.D at West China Hospital. The trial plans to enroll 6 participants.