Mucopolysaccharidosis I – European Clinical Trials Information Network https://clinicaltrials.eu Bridging Patients with Clinical Trials Fri, 12 Jun 2026 10:37:30 +0000 en-US hourly 1 https://wordpress.org/?v=7.0 https://clinicaltrials.eu/wp-content/uploads/2024/12/cropped-EU_icon-32x32.png Mucopolysaccharidosis I – European Clinical Trials Information Network https://clinicaltrials.eu 32 32 Study on Gene Therapy with Autologous Stem Cells and Drug Combination for Children with Mucopolysaccharidosis Type I Hurler Variant https://clinicaltrials.eu/trial/study-on-gene-therapy-with-autologous-stem-cells-and-drug-combination-for-children-with-mucopolysaccharidosis-type-i-hurler-variant/ Wed, 29 Apr 2026 15:01:34 +0000 https://clinicaltrials.eu/trial/study-on-gene-therapy-with-autologous-stem-cells-and-drug-combination-for-children-with-mucopolysaccharidosis-type-i-hurler-variant/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis Type I, Hurler variant (MPS I Hurler). This condition affects the body’s ability to break down certain sugars, leading to various health issues. The study is testing a new treatment called OTL-203, which involves using the patient’s own blood stem cells that have been genetically modified. These cells are altered to include a gene that helps produce an enzyme called alpha-L-iduronidase, which is missing or not working properly in people with MPS I Hurler.

The purpose of the study is to evaluate the safety and tolerability of this gene therapy in children with MPS I Hurler. Participants will receive a conditioning treatment to prepare their body, followed by an infusion of the modified stem cells. The study will monitor the participants over time to see how well they tolerate the treatment and to check for any side effects. The study will also look at how well the treatment works in increasing the levels of the missing enzyme in the blood.

In addition to the main treatment, the study involves other medications such as Lenograstim, Plerixafor, Busulfan, Fludarabine, and Rituximab, which are used to support the treatment process. Some participants may receive a placebo instead of the active treatment to help compare the results. The study is expected to continue until 2027, with regular follow-ups to ensure the safety and effectiveness of the treatment.

]]> Study on the Effectiveness and Safety of OTL-203 for Patients with Hurler Syndrome (MPS-IH) Compared to Standard Treatment with Stem Cell Transplantation https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-otl-203-for-patients-with-hurler-syndrome-mps-ih-compared-to-standard-treatment-with-stem-cell-transplantation/ Wed, 29 Apr 2026 14:30:00 +0000 https://clinicaltrials.eu/trial/study-on-the-effectiveness-and-safety-of-otl-203-for-patients-with-hurler-syndrome-mps-ih-compared-to-standard-treatment-with-stem-cell-transplantation/ This clinical trial is focused on studying a rare genetic disorder called Mucopolysaccharidosis type I, Hurler Syndrome (MPS-IH). This condition affects the body’s ability to break down certain sugars, leading to various health problems. The study is comparing a new treatment called OTL-203 with the current standard treatment, which is a type of stem cell transplant known as allogeneic hematopoietic stem cell transplantation (allo-HSCT). OTL-203 is a gene therapy that involves using a patient’s own stem cells, which are modified to help produce an enzyme that people with MPS-IH lack.

The purpose of the study is to evaluate the safety and effectiveness of OTL-203 compared to the standard treatment. Participants in the study will receive either the new gene therapy or the standard stem cell transplant. The study will monitor participants over time to see how well they do with each treatment. This includes looking at survival without major health events, changes in enzyme activity, and other health measures like cognitive function, joint movement, and quality of life.

The study will also track any side effects or complications that may arise from the treatments. This includes monitoring for immune responses, infections, or other serious health issues. The goal is to determine if OTL-203 can provide a safer and more effective treatment option for people with Mucopolysaccharidosis type I, Hurler Syndrome.

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