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Gene therapy study using a frozen formulation of OTL-200 in patients with Metachromatic Leukodystrophy (MLD)
EU CTIS ID: 2024-511970-66-00
What this study is testing
To evaluate the clinical efficacy of the cryopreserved formulation of OTL200
- Therapeutic confirmatory (Phase III)
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
- Documented biochemical and molecular diagnosis of MLD, based on ARSA activity below the normal range and identification of two disease-causing ARSA alleles, either known or novel mutations. Novel mutations will be analyzed with in silico prediction tools and excluded from being known common polymorphisms. In the case of a novel mutation(s), a 24-hour urine collection must show elevated sulfatide levels.
- Eligible participants must have EITHER: a) an older sibling affected by MLD (index case), whose age of symptom onset was ≤6 years of age (i.e. had not celebrated 7th birthday). Participants will be classified as Late Infantile, Early Juvenile or Intermediate LI/EJ based on age of symptom onset in the index case and their ARSA genotype: i. LI: symptom onset in index case ≤30 months of age; genotype typically 0/0 ii. EJ: symptom onset in index case >30 months and ≤6 years of age; genotype typically 0/R iii. Intermediate LI/EJ: symptom onset in index case ≤6 years of age but unable to unambiguously characterize index case as LI or EJ OR b) If MLD is diagnosed in a pre-symptomatic child without an older affected sibling, (e.g. incidentally or via newborn screening) and the totality of the data available to the investigator strongly suggest that the patient has an early onset variant of MLD likely to benefit from gene therapy, and the patient is ≤6 years of age (i.e. has not celebrated 7th birthday), the patient may be considered eligible after discussion and approval by the Orchard Therapeutics (Europe) Ltd. Medical Monitor (Orchard-MM)
You likely can't join if
- If LI MLD variant, clinical manifestations of the disease defined as EITHER of the following: i. Delay in expected achievement of independent standing or independent walking, together with abnormal signs at neurological evaluation OR ii. Documented neurological signs and symptoms of MLD associated with cognitive, motor, or behavioral functional impairment or regression (substantiated by neurological examination and/or neuropsychological tests appropriate for age)
- If EJ MLD variant, symptoms of MLD resulting in the loss of capacity of walking independently as defined by a GMFC level ≥2 or symptoms consistent with cognitive impairment as defined by an IQ<85 using age-appropriate neurocognitive instruments
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.