Mucopolysaccharidosis INCT02232477
Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in MPS I
A study testing Intrathecal recombinant human alpha iduronidase in people with Mucopolysaccharidosis I and Cognitive Decline, run by agnes chen at 1 site in United States.
Trial summary
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Mucopolysaccharidosis I, Cognitive Decline
- What is being tested?
- Intrathecal recombinant human alpha iduronidase
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 6 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2014to Feb 2020
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- The subject has completed the MIRC-002 study of intrathecal enzyme replacement therapy for cognitive decline in mucopolysaccharidosis I
- Age six years or older.
- Subject and/or guardian willing and able to provide written informed consent.
- Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)
- Currently using two acceptable methods of birth control as determined by the investigator and willing to continue to use acceptable birth control during their participation in the study (non-sterile females of child-bearing potential who are sexually active only)
- Willing and able to comply with study procedures. For example, the subjects must be able to complete written and computer-based testing. The subjects must be able to lie still in the MRI scanner for at least 40 minutes without sedation.
Who cannot join
- The subject has undergone hematopoietic stem cell transplantation
- Recent initiation of intravenous Aldurazyme® therapy with less than 6 months of therapy. Subjects who have been receiving Aldurazyme® therapy for more than 6 months, and those who have never received Aldurazyme® therapy, will be allowed to enroll
- Pregnant or lactating, or considering pregnancy
- Receipt of an investigational drug or procedure other than intrathecal Aldurazyme® within 30 days of enrollment
- A condition, medical or other, that prevents participation in the study, including severe auditory or visual impairment, significant lumbar pathology, lumbar catheter, or recent major surgery within 6 weeks that would preclude their ability to participate.
- Infusion reactions to intravenous or intrathecal Aldurazyme® therapy that are life-threatening or require emergent intervention such as epinephrine, cardiopulmonary resuscitation, or hospitalization
- The subject has severely impaired spinal CSF flow, demonstrated by failure of appearance of radionuclide in the basal cisterns by 4 hours after intra-lumbar administration.
- The subject has a coagulopathy, as identified by a platelet count of less than 50,000, an INR of 1.5 or greater, or a PTT that is 1.5 times the upper limit of normal for the laboratory from which it was drawn.
Trial locations and contacts
1 site in 1 country.
United States1 site
Los Angeles Biomedical Research Institute at Harbor-UCLA Medical Center
Torrance, California, 90509
About this study
Official title: An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
This is a five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study.
- Conditions
- Mucopolysaccharidosis I, Cognitive Decline
- Being tested
- Intrathecal recombinant human alpha iduronidase
- Medical subject headings
- Mucopolysaccharidosis I, Cognitive Dysfunction
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Intrathecal recombinant human alpha iduronidaseDrug
Also called Aldurazyme
Intrathecal recombinant human alpha iduronidase every 3 months
| Study group | Role | What they receive |
|---|---|---|
| Open-label treatment | Experimental | laronidase 1.74 mg IT q 3 months for five years Receives: Drug: Intrathecal recombinant human alpha iduronidase |
What is being measured
The primary outcome is the main question the study is designed to answer.
Hopkins Verbal Learning Test
Mean intra-subject change in total recall score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit.
The Hopkins Verbal Learning Test measures verbal memory. The subject is read a list of words and asked to memorize as many words as possible. The scale ranges from 0 to 36, with higher scores indicating a better outcome.
Two relevant time points: Baseline and the subject's final visit. Because this study ended prematurely for due to the pandemic, the subject's final visit was at the 12-month time point for one subject, 24-months for one subject, 36-months for two subjects, and 48-months for two subjects.
Calculation: The total recall score at Baseline was subtracted from the total recall score at the subject's final visit and mean change and standard deviation were reported.
Measured: zero to 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Sep 2014 (estimated)
- Study startAug 2014
- Primary completionFeb 2020
- Study completionFeb 2020
Why it stopped: Termination of study by investigator related to COVID pandemic.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Mucopolysaccharidosis IStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- agnes chen
- Sponsor type
- University, hospital or other
- Collaborators
- University of Minnesota, UCSF Benioff Children's Hospital Oakland, University of California, Los Angeles, The Ryan Foundation, Rare Diseases Clinical Research Network, National Institute of Neurological Disorders and Stroke (NINDS)
- Responsible party
- Sponsor-investigator, agnes chen, Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02232477
- Sponsor's protocol ID
- MIRC-002 (100)
- Status last verified
- Oct 2023
- First submitted
- 3 Sep 2014
- Last updated
- 31 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 31 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Extension Study of Intrathecal Enzyme Replacement for Cognitive Decline in Mucopolysaccharidosis I
- ClinicalTrials.gov ID
- NCT02232477
- Acronym / sponsor ID
- MIRC-002 (100)
- Conditions
- Mucopolysaccharidosis I, Cognitive Decline
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Termination of study by investigator related to COVID pandemic.
- Enrolment
- 6 participants (actual)
- Lead sponsor
- agnes chenUniversity, hospital or other
- Collaborators
- University of Minnesota, UCSF Benioff Children's Hospital Oakland, University of California, Los Angeles, The Ryan Foundation, Rare Diseases Clinical Research Network, National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Aug 2014 (actual)
- Primary completion
- Feb 2020 (actual)
- Study completion
- Feb 2020 (actual)
- First posted
- 5 Sep 2014 (estimated)
- Results
- Posted 31 Oct 2023
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 five-year extension study of the pilot study, "Intrathecal Enzyme Replacement for Cognitive Decline in MPS I". Participants must have completed the pilot study to participate in this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Open-label treatment | Experimental | Drug: Intrathecal recombinant human alpha iduronidaselaronidase 1.74 mg IT q 3 months for five years |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- The subject has completed the MIRC-002 study of intrathecal enzyme replacement therapy for cognitive decline in mucopolysaccharidosis I
- Age six years or older.
- Subject and/or guardian willing and able to provide written informed consent.
- Negative urine pregnancy test at screening (non-sterile females of child-bearing potential only)
- Currently using two acceptable methods of birth control as determined by the investigator and willing to continue to use acceptable birth control during their participation in the study (non-sterile females of child-bearing potential who are sexually active only)
Key exclusion criteria (8 listed)
- The subject has undergone hematopoietic stem cell transplantation
- Recent initiation of intravenous Aldurazyme® therapy with less than 6 months of therapy. Subjects who have been receiving Aldurazyme® therapy for more than 6 months, and those who have never received Aldurazyme® therapy, will be allowed to enroll
- Pregnant or lactating, or considering pregnancy
- Receipt of an investigational drug or procedure other than intrathecal Aldurazyme® within 30 days of enrollment
- A condition, medical or other, that prevents participation in the study, including severe auditory or visual impairment, significant lumbar pathology, lumbar catheter, or recent major surgery within 6 weeks that would preclude their ability to participate.
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 |
|---|---|---|
| Intrathecal recombinant human alpha iduronidaseAlso: AldurazymeIntrathecal recombinant human alpha iduronidase every 3 months | Drug | Open-label treatment |
Primary outcome
Hopkins Verbal Learning Test
Time frame: zero to 48 months
Mean intra-subject change in total recall score on the Hopkins Verbal Learning Test between baseline/screening for MIRC-002 and the subject's final visit. The Hopkins Verbal Learning Test measures verbal memory. The subject is read a list of words and asked to memorize as many words as possible. The scale ranges from 0 to 36, with higher scores indicating a better outcome. Two relevant time points: Baseline and the subject's final visit. Because this study ended prematurely for due to the pandemic, the subject's final visit was at the 12-month time point for one subject, 24-months for one subject, 36-months for two subjects, and 48-months for two subjects. Calculation: The total recall score at Baseline was subtracted from the total recall score at the subject's final visit and mean change and standard deviation were reported.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Active, not recruitingObservational study
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
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An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
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Phase I/II/III Gene Transfer Clinical Trial of scAAV9.U1a.hSGSH
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Intrathecal recombinant human alpha iduronidase in people with Mucopolysaccharidosis I and Cognitive Decline, run by agnes chen 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 6 inclusion and 8 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: stopped early, as of the sponsor's last update on 31 Oct 2023. 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?
Yes. Results are posted in the registry (first posted 31 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Hopkins Verbal Learning Test (time frame: zero to 48 months). 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 31 Oct 2023.