Mucopolysaccharidosis IH/SPhase 1NCT05682144
ISP-001: Sleeping Beauty Transposon-Engineered B Cells for MPS I
A Phase 1 study testing Autologous Plasmablasts (B cells) in people with Mucopolysaccharidosis IH/S and Mucopolysaccharidosis IS, run by Immusoft of CA, Inc. at 2 sites in United States.
Trial summary
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Mucopolysaccharidosis IH/S, Mucopolysaccharidosis IS
- What is being tested?
- Autologous Plasmablasts (B cells)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 2 sitesUnited States
- Study dates
- 12 Apr 2023to 1 Jun 2044
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Show all 9Show fewer
- 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.
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Jake Wesley, PharmD, MS contact
United States2 sites, 2 recruiting
- Recruiting
University of Minnesota
Minneapolis, Minnesota, 55455
Paul Orchard, MD
- Recruiting
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Daniel Schrader, Daniel.Schrader@ucsf.edu
Paul Harmatz, MD
About this study
Official title: 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
A first-in-human study using ISP-001 in patients with Mucopolysaccharidosis Type I Hurler-Scheie and Scheie.
Read the 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.
- Being tested
- Autologous Plasmablasts (B cells)
- Keywords
- MPS IH/S, MPS IS
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Plasmablasts (B cells)Biological
Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system.
| Study group | Role | What they receive |
|---|---|---|
| Autologous Plasmablasts (B cells) - Cohort - Target Dose A | Experimental | Dose Level: 5 x 10e7 cells/kg on Day 0 Receives: Biological: Autologous Plasmablasts (B cells) |
| Autologous Plasmablasts (B cells) - Cohort - Target Dose B | Experimental | Dose Level: Between 1 x 10e8 or to 2 x 10e8 cells/kg on Day 0 Receives: Biological: Autologous Plasmablasts (B cells) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Measured: 24 Weeks
Secondary outcomes (6)
Number of participants with treatment-related adverse events and serious adverse events
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
Measured: 48 Weeks
Determination of Absolute Numbers of B and T cell populations
Determination of Absolute Numbers of B and T cell populations in peripheral blood at baseline and at scheduled time points post infusion.
Measured: 1Year
Concentration of IDUA
Determine IDUA concentration in plasma at baseline and at scheduled time points post infusion.
Measured: 1 Year
Assessment of Storage Material (glycosaminoglycan, or GAG)
Assessment of Storage Material (glycosaminoglycan, or GAG) in urine at baseline and at scheduled time points post infusion.
Measured: 1 Year
Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)
Determine levels of circulating antibodies (IgG, IgM, IgA, and IgE) at baseline and at scheduled time points post infusion.
Measured: 1 Year
Analysis of PBMCs
PBMCs will be analyzed at baseline and at scheduled time points post infusion.
Measured: 1 Year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jan 2023
- Study start12 Apr 2023
- Primary completion1 Jun 2029 (estimated)
- Study completion1 Jun 2044 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis IH/SStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 11 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Immusoft of CA, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Immusoft Clinical Development, Study director, Immusoft of CA, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05682144
- Sponsor's protocol ID
- MT2021-24
- Status last verified
- Jul 2026
- First submitted
- 12 Dec 2022
- Last updated
- 17 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05682144
- Acronym / sponsor ID
- MT2021-24
- Keywords
- MPS IH/S, MPS IS
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 11 participants (estimated)
- Lead sponsor
- Immusoft of CA, Inc.Industry (pharma, biotech, devices)
- Study start
- 12 Apr 2023 (actual)
- Primary completion
- 1 Jun 2029 (estimated)
- Study completion
- 1 Jun 2044 (estimated)
- First posted
- 12 Jan 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Autologous Plasmablasts (B cells) - Cohort - Target Dose A | Experimental | Biological: Autologous Plasmablasts (B cells)Dose Level: 5 x 10e7 cells/kg on Day 0 |
| Autologous Plasmablasts (B cells) - Cohort - Target Dose B | Experimental | Biological: Autologous Plasmablasts (B cells)Dose Level: Between 1 x 10e8 or to 2 x 10e8 cells/kg on Day 0 |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (9 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Autologous Plasmablasts (B cells)Autologous plasmablasts (B cells) engineered to express α-L-iduronidase (IDUA) using the Sleeping Beauty (SB) transposon system. | Biological | Autologous Plasmablasts (B cells) - Cohort - Target Dose A, Autologous Plasmablasts (B cells) - Cohort - Target Dose B |
Primary outcome
Number of participants with treatment-related adverse events and serious adverse events
Time frame: 24 Weeks
Incidence of Adverse Events as assessed by CTCAE (v 5.0)
6 secondary outcomes
Number of participants with treatment-related adverse events and serious adverse events
Time frame: 48 Weeks
Determination of Absolute Numbers of B and T cell populations
Time frame: 1Year
Concentration of IDUA
Time frame: 1 Year
Assessment of Storage Material (glycosaminoglycan, or GAG)
Time frame: 1 Year
Levels of Circulating Antibodies (IgG, IgM, IgA, and IgE)
Time frame: 1 Year
Analysis of PBMCs
Time frame: 1 Year
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Jul 2026.
- Individual participant dataSharing planned: No
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Jake Wesley, PharmD, MS contact
Immusoft Clinical Development study director
Immusoft of CA, Inc.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Autologous Plasmablasts (B cells) in people with Mucopolysaccharidosis IH/S and Mucopolysaccharidosis IS, run by Immusoft of CA, Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 17 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jake Wesley, PharmD, MS (jake.wesley@immusoft.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with treatment-related adverse events and serious adverse events (time frame: 24 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Jul 2026.