Using engineered B cells to treat Mucopolysaccharidosis Type I
A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie
This study is testing a new treatment using modified B cells to see if it can help adults with Mucopolysaccharidosis Type I feel better by addressing a missing enzyme in their bodies.
Quick facts
| Phase | Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 11 (estimated) |
| Ages | 10 Years and up |
| Sex | All |
| Sponsor | Immusoft of CA, Inc. Industry-sponsored |
| Locations | 2 sites (Oakland, California and 1 other locations) |
| Trial ID | NCT05682144 on ClinicalTrials.gov |
What this trial studies
This Phase 1, first-in-human, open-label, single-arm study aims to evaluate the safety and tolerability of ISP-001, which involves treating adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie using autologous plasmablasts engineered to express α-L-iduronidase through the Sleeping Beauty transposon system. Participants will receive their own modified B cells to potentially address the underlying enzyme deficiency associated with their condition. The study will monitor participants for safety outcomes and any adverse effects following the treatment.
Who should consider this trial
Good fit: Ideal candidates are adults aged 18 and older diagnosed with Mucopolysaccharidosis Type I Hurler-Scheie or Scheie syndrome.
Not a fit: Patients with a history of B cell-related cancers, autoimmune disorders, or those requiring systemic immune suppression may not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could provide a novel therapeutic option for patients with Mucopolysaccharidosis Type I, potentially improving their quality of life.
How similar studies have performed: While this approach is novel, similar gene therapy strategies have shown promise in other conditions, indicating potential for success.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome. * Age ≥ 10 years at time of study registration. * Creatinine clearance, calculated or measured directly, that is \>60ml/min/1.73m2. * Ejection fraction ≥ 40% by echocardiogram. * Must commit to traveling to the study site for the necessary follow-up evaluations. * Must agree to stay \<45-minute drive from the study site for a minimum of 5 days after cell infusion. Exclusion Criteria: * Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations. * History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders. * Evidence of active graft-vs-host disease. * Underwent a previous hematopoietic stem cell transplant (HSCT). * Requirement for systemic immune suppression. * Requirement for continuous supplemental oxygen. * Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment. * In the investigator's judgement, the subject is unlikely to complete all protocol-required study visits or procedures, including follow up visits, or comply with the study requirements for participation. Other protocol defined inclusion/exclusion criteria may apply.
Where this trial is running
Oakland, California and 1 other locations
- UCSF Benioff Children's Hospital Oakland — Oakland, California, United States (Recruiting)
- University of Minnesota — Minneapolis, Minnesota, United States (Recruiting)
Study contacts
- Study coordinator: Jake Wesley, PharmD, MS
- Email: jake.wesley@immusoft.com
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.