Recruiting
Phase 1

ISP-001

Sponsor:

Immusoft of CA, Inc.

Code:

NCT05682144

Conditions

Mucopolysaccharidosis IH/S

Mucopolysaccharidosis IS

Eligibility Criteria

Sex: All

Age: 10+

Healthy Volunteers: Not accepted

Interventions

Autologous Plasmablasts (B cells)

Study Details

Brief summary:

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

Conditions

Mucopolysaccharidosis IH/S

Mucopolysaccharidosis IS

Study ID

NCT05682144

Start date

Apr 12, 2023

Status verified date

Jul, 2026

Completion date

Jun 1, 2044

Anticipated

Primary completion date

Jun 1, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 10+

Healthy Volunteers: Not accepted

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.

Study Design

Enrollment

11 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Autologous Plasmablasts (B cells) - Cohort - Target Dose A

Dose Level: 5 x 10e7 cells/kg on Day 0

experimental: Autologous Plasmablasts (B cells) - Cohort - Target Dose B

Dose Level: Between 1 x 10e8 or to 2 x 10e8 cells/kg on Day 0

Interventions

Autologous Plasmablasts (B cells)

Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.

Primary outcome measure

  • Number of participants with treatment-related adverse events and serious adverse events [ Time Frame: 24 Weeks ]

Central Contacts and Locations

Central contacts

Jake Wesley, PharmD, MS

jake.wesley@immusoft.com

Locations

UCSF Benioff Children's Hospital Oakland

Recruiting

Oakland, California, United States, 94609

Contacts

Daniel Schrader

Daniel.Schrader@ucsf.edu

Principal Investigator:

Paul Harmatz, MD

University of Minnesota

Recruiting

Minneapolis, Minnesota, United States, 55455

Principal Investigator:

Paul Orchard, MD

More Information

Sponsor

Immusoft of CA, Inc.

Last update posted

Jul 17, 2026

Last verified

Jul, 2026

Keywords

  • MPS IH/S
  • MPS IS

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-06. This information was provided to ClinicalTrials.gov by Immusoft of CA, Inc. on 2026-07-17.