Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
Conditions
Keywords
MPS IH/S, MPS IS
Brief summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Detailed description
This is a Phase 1, first-in-human, open-label, single-arm study in which patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie are treated with autologous plasmablasts engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty transposon system (ISP-001). This study will evaluate the safety and tolerability of ISP-001.
Interventions
BIOLOGICALAutologous Plasmablasts (B cells)
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
Sponsors
Immusoft of CA, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
10 Years to No maximum
Healthy volunteers
No
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/
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events and serious adverse events | 24 Weeks | Incidence of Adverse Events as assessed by CTCAE (v 5.0) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of participants with treatment-related adverse events and serious adverse events | 48 Weeks | Incidence of Adverse Events as assessed by CTCAE (v 5.0) |
| Determination of Absolute Numbers of B and T cell populations | 1Year | Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion. |
| Concentration of IDUA | 1 Year | Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion. |
| Assessment of Storage Material (glycosaminoglycan, or GAG) | 1 Year | Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion. |
| Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE) | 1 Year | Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion. |
| Analysis of PBMCs | 1 Year | PBMCs will be analyzed at baseline and at scheduled time points post infusion. |
Countries
United States
Contacts
CONTACTJake Wesley, PharmD, MS
STUDY_DIRECTORImmusoft Clinical Development
Immusoft of CA, Inc.
Outcome results
None listed