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New treatment option for Mucopolysaccharidosis IH/S
Official title ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
ClinicalTrials.gov ID: NCT05682144
What this study is testing
What is Autologous Plasmablasts (B cells)?
Autologous Plasmablasts (B cells) is an investigational medicine, being studied as a potential treatment for mucopolysaccharidosis ih/s.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 10 and older
You may be able to join if
- 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.
You likely can't join if
- Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to...
- 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.
See the full eligibility 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.
- 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 how well it works 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...
The study team makes the final eligibility decision.
Where it's taking place
- Oakland, California, United States
- Minneapolis, Minnesota, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 10 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Oakland, California, United States; Minneapolis, Minnesota, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.