RECRUITING

ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I

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Conditions

Mucopolysaccharidosis IH/SMucopolysaccharidosis ISMPS IH/SMPS IS

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

A first-in-human study using ISP-001 in adult patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.

Official Title

A Phase I Open Label Study to Evaluate the Safety and Tolerability of ISP-001 in Adult Patients With Mucopolysaccharidosis Type I Hurler-Scheie and Scheie

Quick Facts

Study Start:2023-04-12
Study Completion:2039-06-01
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT05682144

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:18 Years
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:No
Standard Ages:ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Diagnosis of Mucopolysaccharidosis type I Hurler-Scheie or Scheie syndrome.
  2. * Age ≥ 18 years at time of study registration.
  3. * Creatinine clearance, calculated or measured directly, that is \>60ml/min/1.73m2.
  4. * Ejection fraction ≥ 40% by echocardiogram.
  5. * Must commit to traveling to the study site for the necessary follow-up evaluations.
  6. * Must agree to stay \<45-minute drive from the study site for a minimum of 5 days after cell infusion.
  1. * Known familial inherited cancer syndrome. Suspected cases will be investigated, per the physicians discretion, using relevant genetic tests to determine presence of germline mutations.
  2. * History of B cell related cancer, EBV lymphoproliferative disease or autoimmune disorders.
  3. * Evidence of active graft-vs-host disease.
  4. * Underwent a previous hematopoietic stem cell transplant (HSCT).
  5. * Requirement for systemic immune suppression.
  6. * Requirement for continuous supplemental oxygen.
  7. * Any medical condition likely to interfere with assessment of safety or efficacy of the study treatment.
  8. * 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.

Contacts and Locations

Study Contact

Jake Wesley, PharmD, MS
CONTACT
jake.wesley@immusoft.com

Principal Investigator

Immusoft Clinical Development
STUDY_DIRECTOR
Immusoft of CA, Inc.

Study Locations (Sites)

University of Minnesota
Minneapolis, Minnesota, 55455
United States

Collaborators and Investigators

Sponsor: Immusoft of CA, Inc.

  • Immusoft Clinical Development, STUDY_DIRECTOR, Immusoft of CA, Inc.

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2023-04-12
Study Completion Date2039-06-01

Study Record Updates

Study Start Date2023-04-12
Study Completion Date2039-06-01

Terms related to this study

Keywords Provided by Researchers

  • MPS IH/S
  • MPS IS

Additional Relevant MeSH Terms

  • Mucopolysaccharidosis IH/S
  • Mucopolysaccharidosis IS