ClinicalTrials.gov record NCT03725670

Direct Lentiviral Injection Gene Therapy for MLD

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Sep 5, 2026

Direct Lentiviral TYF-ARSA Injection Gene Therapy for Metachromatic Leukodystrophy (MLD)

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.
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.
AgeUnder 1 month old to 50 years old
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 I/II trial studies a lentiviral gene therapy (TYF-ARSA) given by intrathecal (spinal) and intravenous injections for people with metachromatic leukodystrophy (MLD). The main goals are to evaluate safety and to measure biological and imaging changes after treatment, with follow-up reported for up to three years. All participants receive the same investigational treatment in a single-group design.

What it is studying. The study is testing the safety of direct intrathecal and intravenous delivery of an improved self-inactivating lentiviral vector (TYF-ARSA) that carries a functional ARSA gene. It also evaluates biochemical markers, brain MRI findings, the vector's integration profile, and longer-term effects on MLD-related signs.

What the researchers hope to learn. Scientific goals are to evaluate the safety of the TYF-ARSA lentiviral vector and direct injection protocol; to measure changes in degradative metabolites and MRI-based disease markers; to characterize vector integration; and to assess longer-term correction of MLD-related pathological signs.

What this study is about

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

This is a Phase I/II clinical trial of gene therapy for treating Metachromatic leukodystrophy (MLD) using a safety and efficacy improved self-inactivating lentiviral vector TYF-ARSA to functionally correct the genetic defect. The primary objectives are to evaluate the safety and efficacy of the direct gene transfer clinical protocol.

Interventions being studied

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

Genetic
  • Intrathecal and intravenous LV gene therapy

Eligibility

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

Inclusion criteria

  • MLD patient age >= 1 month
  • ARSA gene sequence analysis to confirm MLD mutations
  • Scoring system for brain MR Imaging confirmed MLD
  • Parent / guardian / patient signing informed consent
  • Patients and their families have a strong willingness to participate in clinical trials, and are willing to bear all the consequences caused by the failure of the trial, and sign an informed consent form

Exclusion criteria

  • HIV positive patients
  • Patients who are experiencing uncontrolled viral, bacterial or fungal infections, malignant tumors, heart abnormalities, liver dysfunction, or renal insufficiency
  • Cannot perform an MRI
  • Infection or dermatosis at pre-injection site
  • Any condition that may increase the subjects' risk or interfere with the results of the trial. In addition to MLD, there are other neurological disorders.

Study description and what participation involves

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

Show the detailed description from the registry

Important Regulatory Notice: This trial record is only for global academic information registration on ClinicalTrials.gov. Neither the sponsor Beijing Meikang Jimian Biotechnology Co., Ltd. nor collaborator Shenzhen Geno-Immune Medical Institute has obtained NMPA clinical trial approval or clinical technology filing permission to carry out interventional cell therapy trials in mainland China. ClinicalTrials.gov registration alone does not represent legal approval by Chinese health and drug regulatory authorities. Metachromatic leukodystrophy (MLD) is a rare lysosomal storage disease. This disease is an inherited single gene autosomal recessive defect. MLD is caused by a mutation in the ARSA gene encoding arylsulfatase A which leads to a deficiency in sulfatide degradation, resulting in its accumulation in oligodendrocytes, Schwann cells and neurons. A critical level of sulfatide storage can trigger demyelination, the hallmark of MLD, which results in multiple neurological symptoms. MLD has different onset ages including late infancy (1-2 years), adolescence (4 years-before sexual maturity) and adulthood (after sexual maturity). MLD patients are normally rescued by hematopoietic stem cell transplantation (HSCT) from a matched healthy donor. However, HSCT must be performed at a very early stage of the disease thus restricting its therapeutic opportunies in MLD patients. This trial aims to treat MLD using a safety and efficacy improved self-inactivating lentiviral vector (LV) carrying a functional MLD gene to correct the genetic defect by intrathecal (IT) and intravenous (IV) injections to delivery the lentiviral vector carrying a normal ARSA gene to correct the genetic defect. The primary objectives are to evaluate the safety of the improved LV TYF-ARSA and the direct injection gene transfer clinical protocol, the efficacy of degradative metabolite in patients after treatment, vector integration profile, and finally the long-term correction of the related pathological symptoms.

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.

If you take part, you would receive direct gene therapy with the lentiviral TYF-ARSA vector given by intrathecal (into spinal fluid) and intravenous (into a vein) injections. The study includes MRI brain imaging and biochemical analyses to measure disease-related changes, plus scheduled safety assessments such as physical exams, vital signs, and monitoring and reporting of adverse events. The study will also assess the vector integration profile. Safety is followed for up to 1 year and disease- and imaging-related assessments are followed for up to 3 years after treatment.

How the study is structured. This is an interventional, single-group (all participants receive the same treatment), non-masked study. Participants receive the TYF-ARSA lentiviral vector by intrathecal and intravenous injections. Safety is tracked (reported up to 1 year) and disease-related outcomes are followed up to 3 years after treatment.

Questions you could ask

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

  • The registry says recruitment has not started. When is it expected to open, and can I register interest now?
  • 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 metachromatic leukodystrophy

Clinical trials10 trials listed for metachromatic leukodystrophyEvery trial the registry lists for metachromatic leukodystrophy, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in metachromatic leukodystrophy researchLatest monthly update: September 2026. Includes: Transplant timing. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
May 31, 2027 (estimated)
Primary completion
Jun 1, 2028 (estimated)
Study completion
Dec 31, 2028 (estimated)
Target enrollment
10 participants (estimated)
Last updated on registry
Sep 1, 2026
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
Shenzhen Geno-Immune Medical Institute

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

Phase I/II
A study that combines safety and dose-finding (phase 1) with a first look at whether the treatment has an effect (phase 2).
informed consent
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.
MRI
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.
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.
sponsor
The organisation or person that initiates the study and has authority over it. The sponsor may be a company, hospital, university, government agency or individual, and is not necessarily the organisation that funds the study.
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.
intrathecal
Given by injection into the fluid around the spinal cord, usually through a lumbar puncture.

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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