Who can enter
- Children with confirmed laboratory diagnosis of MPS-IH
- Age at enrolment: ≥ 28 days to ≤30 months
Goal
The goal of this study is to compare the current standard treatment allogeneic hematopoietic stem cell transplantation (allo-HSCT) with the new gene therapy drug OTL-203. It is hypothesized that OTL-203 may offer advantages over standard treatment in terms of survival, reduction of complications and symptoms.
Background
Allo HSCT has been used as a standard treatment for MPS-IH by doctors around the world for many years, but it is not considered an approved drug by the regulators of medical treatments.
Treatment with OTL-203 or allo-HSCT is expected to stop or delay the progression of MPS-IH disease. There is the possibility of an improvement in quality of life, fewer MPS-IH symptoms and MPS-IH related complications, and reduction or stopping of other medications for MPS-IH complications.
In this study, the child will be given one of the two treatments and followed for a minimum of five years (up to 15 years with OTL-203 treatment). During the research, the child will also undergo various examinations at different time-points, including (but not limited to) a physical examination, blood tests, heart tests, functional and mobility examinations, and scans of brain or spine. After two years, there will be an interim evaluation of whether OTL-203 treatment can be a similar or improved treatment option.
OTL-203 is a gene therapy drug that consists of an autologous (from your own body), CD34+ cell-enriched population of hematopoietic stem and progenitor cells (HSPCs). The stem cells are modified outside the body to contain and produce the IDUA gene once back in the body. A single dose is administered back to the child via intravenous (i.v.) infusion. Reduced activity of the IDUA gene is considered an important factor in Hurler disease.
Currently, an ongoing phase I/II study shows that OTL-203 is well tolerated, and follow-up results suggest an encouraging outcome.