Mucopolysaccharidosis INCT06103487
Long Term Follow-Up for RGX-111
An observational study of Mucopolysaccharidosis I, run by REGENXBIO Inc. at 2 sites in 2 countries.
Trial summary
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Mucopolysaccharidosis I
- How many people
- 21 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 24 Jul 2023to Sep 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Received RGX-111 in a separate parent trial.
Who may be able to join
- To be eligible, a participant must have previously received RGX-111 in a separate parent trial.
- Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent.
Who cannot join
- None.
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
United States1 site
Children's Hospital of Orange County
Orange, California, 92868
Brazil1 site
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
About this study
Official title: A Long-term Follow-up Study to Evaluate the Safety of RGX-111
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
Read the detailed description
This is a prospective, multicenter, long-term observational, follow-up study to evaluate the long-term safety and efficacy of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single intracisternal (IC) or intraventricular (IVR; also referred to as intracerebroventricular [ICV]) infusion of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study). No treatment will be directed under this observational study. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 administration in the parent study.
- Conditions
- Mucopolysaccharidosis I
- Being tested
- No Intervention
- Keywords
- MPS I, Hurler Syndrome, Gene Therapy
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No InterventionGenetic
Observational study
| Study group | Role | What they receive |
|---|---|---|
| RGX-111 Recipients | Subjects who have received RGX-111 in a separate parent study. Receives: Genetic: No Intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluation of the long-term safety of RGX-111
Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time using CTCAE Version 5.0
Measured: 5 years inclusive of parent study
Secondary outcomes (9)
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WASI-II)
Change from baseline in neurodevelopmental parameters as measured by the Wechsler Abbreviated Scale of Intelligence (WASI-II). The WASI-II assesses intelligence in individuals from ages 6 to 90 years old. The assessment will yield composite scores that estimate verbal comprehension and perceptual reasoning abilities. The age equivalent scores are also provided for each of the subtests.
Measured: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WPPSI-IV)
Change from baseline in neurodevelopmental parameters as measured by the Wechsler Preschool and Primary Scale of Intelligence Fourth Edition (WPPSI-IV). The WPPSI-IV measures the cognitive development in young children approximately 30 to 91 months in age. This study utilizes the General Ability Index (GAI) comprised of 4 core subtests depending on the child's age; the subtests include receptive vocabulary, block design, information, and quiet assembly for children 3 to 47 months and block design, information, matrix reasoning and similarities for ages 48 to 91 months. The subtests have different scoring ranges, with higher subtest raw scores indicating completion of more complex items but can be normalized from cross-subtest comparison.
Measured: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-III)
Change from baseline in neurodevelopmental parameters as measured by the Bayley Scales of Infant and Toddler Development, Third Edition. The BSID-III assesses children aged 1 to 42 months across several domains to give a holistic view of the child's development and potential delays. Participants will be assessed for the Cognitive, Language and Motor domains of the BSID-III. The domains have different scoring ranges but can be normalized for cross-domain comparisons.
Measured: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III)
Change from baseline adaptive behavior as measured by Vineland Adaptive Behavior Scales Third Edition (VABS-III). The VABS-III assesses adaptive behavior in individuals from infancy to age 90 years. In this study, 4 domains of Communication, Daily Living Skills, Socialization, and Motor Skills will be assessed. The domains have different scoring ranges, with higher subdomain raw scores indicating greater function, but can be normalized for cross-domain comparisons.
Measured: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS)
To assess the pharmacodynamic effect of RGX-111 on Heparan Sulfate (HS) levels measures by lumbar puncture.
Measured: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA)
To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measures by lumbar puncture.
Measured: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS)
To assess the pharmacodynamic effect of RGX-111 on Heparan Sulfate (HS) levels measures by plasma collection.
Measured: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA)
To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measures by plasma collection.
Measured: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Urine Biomarkers (GAGs)
To assess the pharmacodynamic effect of RGX-111 on glycosaminoglycans (GAGs) levels measured by urine analysis.
Measured: 5 years inclusive of parent study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2023
- Study start24 Jul 2023
- Primary completionSep 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 21 participants (estimated)
Sponsor and investigators
- Lead sponsor
- REGENXBIO Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06103487
- Sponsor's protocol ID
- RGX-111-5101
- Status last verified
- Apr 2026
- First submitted
- 5 Oct 2023
- Last updated
- 29 Apr 2026
- Results posted
- No
- 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 29 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Long-term Follow-up Study to Evaluate the Safety of RGX-111
- ClinicalTrials.gov ID
- NCT06103487
- Acronym / sponsor ID
- RGX-111-5101
- Conditions
- Mucopolysaccharidosis I
- Keywords
- MPS I, Hurler Syndrome, Gene Therapy
- Study type
- Observational
- Recruitment status
- Enrolling by invitation
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- REGENXBIO Inc.Industry (pharma, biotech, devices)
- Study start
- 24 Jul 2023 (actual)
- Primary completion
- Sep 2027 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 26 Oct 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.
RGX-111-5101 is a long-term follow up study that evaluates the long-term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.
Detailed description
This is a prospective, multicenter, long-term observational, follow-up study to evaluate the long-term safety and efficacy of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single intracisternal (IC) or intraventricular (IVR; also referred to as intracerebroventricular [ICV]) infusion of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study). No treatment will be directed under this observational study. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 administration in the parent study.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| RGX-111 Recipients | – | Genetic: No InterventionSubjects who have received RGX-111 in a separate parent study. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Received RGX-111 in a separate parent trial.
Key inclusion criteria (2 listed)
- To be eligible, a participant must have previously received RGX-111 in a separate parent trial.
- Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent.
Key exclusion criteria (1 listed)
- None.
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 |
|---|---|---|
| No InterventionObservational study | Genetic | RGX-111 Recipients |
Primary outcome
Evaluation of the long-term safety of RGX-111
Time frame: 5 years inclusive of parent study
Incidences of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) over time using CTCAE Version 5.0
9 secondary outcomes
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WASI-II)
Time frame: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WPPSI-IV)
Time frame: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-III)
Time frame: 5 years inclusive of parent study
Long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III)
Time frame: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS)
Time frame: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA)
Time frame: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS)
Time frame: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA)
Time frame: 5 years inclusive of parent study
Pharmacodynamic Effect of RGX-111 on Urine Biomarkers (GAGs)
Time frame: 5 years inclusive of parent study
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 29 Apr 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.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Mucopolysaccharidosis I, run by REGENXBIO Inc. at 2 sites in 2 countries.
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 2 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: enrolling by invitation, as of the sponsor's last update on 29 Apr 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, Brazil. 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?
Evaluation of the long-term safety of RGX-111 (time frame: 5 years inclusive of parent study). 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 29 Apr 2026.