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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 belowSelect...
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
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 moreHURCULES 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.
Research Highlights
Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
The most common treatments for MPS-IH (Hurler Syndrome) include gene therapy and hematopoietic stem cell transplantation (HSCT). Gene therapy works by introducing or altering genetic material within a patient's cells to correct the genetic defect, leading to the production of the missing or defective enzyme needed to break down glycosaminoglycans (GAGs).
HSCT involves transplanting healthy donor stem cells that can produce the necessary enzyme to reduce GAG accumulation. Both treatments aim to restore enzyme activity, which is essential for preventing the progressive organ and tissue damage characteristic of MPS-IH.
Trends in haematopoietic cell transplantation for inborn errors of metabolism.
Trends in haematopoietic cell transplantation for inborn errors of metabolism.
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Who is running the clinical trial?
Orchard TherapeuticsLead Sponsor
22 Previous Clinical Trials
181,406 Total Patients Enrolled
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