Hematopoietic Cell TransplantationNCT05634512
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
An observational study of Hematopoietic Cell Transplantation and Mucopolysaccharidosis Type I, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant. Sixteen patients will be enrolled over a 24 month period. Read more about this study
- Who is it for?
- Ages 0 to 3 years
- Conditions the study is for
- Hematopoietic Cell Transplantation, Mucopolysaccharidosis Type I
- What is being tested?
- Laronidase therapy and a stem cell transplant
- Type of treatment
- Injection or infusion
- How many people
- 13 participants
- Where
- 1 siteUnited States
- Study dates
- 17 Nov 2022to 1 Oct 2027
- Phase
- Observational
- 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 0 to 3 years
- All sexes
- No healthy volunteers
- Child
Study population: Study entry is open to pediatric patients regardless of gender or ethnic background. While there will be every effort to seek out and include females and minority patients, the patient population is dependent upon the MPS-IH populations at the University of Minnesota.
Who may be able to join
- Between 0 to 3 years of age
- Meet protocol specific eligibility criteria for allogeneic HCT for MPS IH
- Planning to receive laronidase both pre and post-transplant in an inpatient setting as part of standard-of-care treatment. Virtually all patients with MPSIH being considered for transplantation at the University of Minnesota are already receiving enzyme infusions, and it is standard practice to continue to give enzyme infusions to 8 weeks post-transplant. Therefore, participation will not modify the treatment course.
Who cannot join
- Patient's parent/ legal guardians are unable to provide informed consent.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota Masonic Cancer Center
Minneapolis, Minnesota, 55455
About this study
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant.
Sixteen patients will be enrolled over a 24 month period.
- Being tested
- Laronidase therapy and a stem cell transplant
- Keywords
- MPS I, HCT
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Laronidase therapy and a stem cell transplantDrug
To identify key differences leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH. There will be 12 samples per patient (6 pre-transplant and 6-post-transplant). Laronidase will be administered IV per protocol using standard dosing (0.58 mg/kg intravenously on a weekly basis), and six (n=6) blood samples will be collected over 24 hours for the determination of mononuclear cell lysates and plasma laronidase concentrations for a total of 18mL. The second PK monitoring will be obtained using the same PK design but following complete or near-completedonor derived myeloid engraftment which is evaluated at different time pointspost-HCT (day 30, day 42, day 60). The standard is to continue ERT through 8 weeks post-transplant.
| Study group | Role | What they receive |
|---|---|---|
| Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant. | The primary aim is to characterize the PK of IV laronidase in individuals with MPS IH and identify patient specific covariates that impact drug exposure. The secondary aim is to identify key differences pre-and post-HCT leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH. Receives: Drug: Laronidase therapy and a stem cell transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify covariates that impact drug exposure
Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.
Measured: 2 years
Identify key differences pre- and post-HCT leading to variability in PK parameters
Measure endogenous source of enzyme present in relation to the transplanted cells.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Dec 2022
- Study start17 Nov 2022
- Primary completion28 Oct 2025
- Study completion1 Oct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hematopoietic Cell TransplantationStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 13 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Paul Orchard, Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05634512
- Sponsor's protocol ID
- 2021LS144
- Other IDs
- NCI-2022-09774, MT2021-29
- Status last verified
- Nov 2026
- First submitted
- 22 Nov 2022
- Last updated
- 4 Nov 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Nov 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
- ClinicalTrials.gov ID
- NCT05634512
- Acronym / sponsor ID
- 2021LS144
- Keywords
- MPS I, HCT
- Study type
- Observational
- Recruitment status
- Active, not recruiting
- Enrolment
- 13 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- 17 Nov 2022 (actual)
- Primary completion
- 28 Oct 2025 (actual)
- Study completion
- 1 Oct 2027 (estimated)
- First posted
- 2 Dec 2022 (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.
This is a prospective, observational multicenter study to collect blood from patients with mucopolysaccharidosis type IH undergoing laronidase therapy and a stem cell transplant.
Sixteen patients will be enrolled over a 24 month period.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant. | – | Drug: Laronidase therapy and a stem cell transplantThe primary aim is to characterize the PK of IV laronidase in individuals with MPS IH and identify patient specific covariates that impact drug exposure. The secondary aim is to identify key differences pre-and post-HCT leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH. |
Who may qualify
- Ages
- 0 to 3 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Study entry is open to pediatric patients regardless of gender or ethnic background. While there will be every effort to seek out and include females and minority patients, the patient population is dependent upon the MPS-IH populations at the University of Minnesota.
Key inclusion criteria (3 listed)
- Between 0 to 3 years of age
Key exclusion criteria (1 listed)
- Patient's parent/ legal guardians are unable to provide informed consent.
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 |
|---|---|---|
| Laronidase therapy and a stem cell transplantTo identify key differences leading to variability in PK parameters for patients receiving IV laronidase therapy for the treatment of MPS-IH. There will be 12 samples per patient (6 pre-transplant and 6-post-transplant). Laronidase will be administered IV per protocol using standard dosing (0.58 mg/kg intravenously on a weekly basis), and six (n=6) blood samples will be collected over 24 hours for the determination of mononuclear cell lysates and plasma laronidase concentrations for a total of 18mL. The second PK monitoring will be obtained using the same PK design but following complete or near-completedonor derived myeloid engraftment which is evaluated at different time pointspost-HCT (day 30, day 42, day 60). The standard is to continue ERT through 8 weeks post-transplant. | Drug | Collect blood from patients with MPS-IH) undergoing laronidase therapy and stem cell transplant. |
Primary outcome
Identify covariates that impact drug exposure
Time frame: 2 years
Measure indicators for body size and maturation contribute to variability in laronidase exposure in patients with MPS IH.
Identify key differences pre- and post-HCT leading to variability in PK parameters
Time frame: 2 years
Measure endogenous source of enzyme present in relation to the transplanted cells.
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 4 Nov 2026.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Paul Orchard principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hematopoietic Cell Transplantation and Mucopolysaccharidosis Type I, run by Masonic Cancer Center, University of Minnesota at 1 site 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 3 inclusion and 1 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: under way, not looking for participants, as of the sponsor's last update on 4 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Identify covariates that impact drug exposure (time frame: 2 years). The study lists 2 primary outcomes in all. 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 4 Nov 2026.