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Stem Cell Transplant

Gene Therapy vs Stem Cell Treatment for Hurler Syndrome (HURCULES Trial)

Phase 3

Recruiting

Research Sponsored by Orchard Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed laboratory diagnosis of MPS-IH as demonstrated by biallelic mutation(s) in the gene coding for IDUA enzyme

Be younger than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years post-treatment

Awards & highlights

HURCULES Trial Summary

This trial will compare a gene therapy to stem cell treatment for Hurler Syndrome, a rare inherited disorder.

Who is the study for?

This trial is for patients with Hurler syndrome (MPS-IH) who have a cognitive score of ≥70 and confirmed MPS-IH diagnosis. They must not have uncontrolled seizures, active infections resistant to treatment, severe organ damage, or other conditions that risk safety or data quality. Those previously treated with stem cell transplants or gene therapy, enrolled in another interventional study, unable to follow the protocol, or positive for certain infectious diseases are excluded.Check my eligibility

What is being tested?

The trial compares OTL-203 (a new gene therapy) against the standard allogeneic hematopoietic stem cell transplant (HSCT). It's designed to see which one is more effective and safer for treating Hurler syndrome. Patients will be randomly assigned to receive either the experimental gene therapy or the standard HSCT treatment.See study design

What are the potential side effects?

Potential side effects may include immune system reactions due to genetic modification in OTL-203 and complications related to stem cell transplantation like graft-versus-host disease. Specific side effects depend on individual patient responses and can vary widely.

HURCULES Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have been diagnosed with MPS-IH through genetic testing.

HURCULES Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years post-treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years post-treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years post-treatment for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Event-free survival

Secondary outcome measures

Change from baseline to Year 2 in urinary heparan sulfate levels, defined as ratio to the upper limit of normal

Change from baseline to Year 2 in α-L-iduronidase (IDUA) activity in leukocytes

Immune response against IDUA enzyme

+3 more

HURCULES Trial Design

2Treatment groups

Experimental Treatment

Active Control

Group I: OTL-203Experimental Treatment1 Intervention

Eligible subjects randomized to Arm 1 will receive an intravenous (IV) infusion of OTL-203 gene therapy. Subjects will receive conditioning regimen with busulfan and fludarabine prior to OTL-203 infusion.

Group II: Allo-HSCTActive Control1 Intervention

Eligible subjects randomized to Arm 2 will receive allogeneic hematopoietic stem cell transplantation. Subjects will receive conditioning regimen with busulfan and fludarabine prior to allo-HSCT.

0 / 1Eligibility criteria met

Find a Location

Who is running the clinical trial?

Orchard TherapeuticsLead Sponsor

22 Previous Clinical Trials

181,406 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How widespread is the implementation of this clinical research within the US?

"Potential participants in this medical study can be found at The Children's Hospital of Philadelphia (Philadelphia), UCSF Benioff Children's Hospital Oakland (Oakland) and Ospedale San Raffaele (Milan). Additionally, there are 7 other recruitment centres."

Answered by AI

Is this medical experiment currently recruiting participants?

"Unfortunately, this clinical trial is not presently accepting participants. It was initially posted on December 1st 2023 and most recently revised on November 27th 2023. However, there are still 1547 other studies that are actively recruiting patients at the moment."

Answered by AI

Are there any eligibility criteria I should meet to join this experiment?

"The inclusion criteria for this research requires that volunteers possess a mps-ih diagnosis (Hurler Syndrome) and are between 28 Days to 30 Months old. This investigation is seeking 40 recruits in total."

Answered by AI

Is OTL-203 hazardous to those who use it therapeutically?

"OTL-203 has been accorded a rating of 3 due to its Phase 3 status, signifying there is existing evidence for efficacy and multiple studies that indicate safety."

Answered by AI

Does this test require participants to be at least 18 years old?

"To be considered for this trial, a patient must have an age in the range of 28 days to 30 months. Conversely, there are 414 clinical trials catering exclusively to minors and 1072 studies targeting seniors."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

2023. "A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06149403.

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