Mucopolysaccharidosis Type I (MPS I)Phase 1/2NCT03580083
RGX-111 Gene Therapy in Patients With MPS I
A Phase 1/2 study testing RGX-111 in people with Mucopolysaccharidosis Type I (MPS I) and Hurler Syndrome, run by REGENXBIO Inc. at 2 sites in 2 countries.
Trial summary
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I. Read more about this study
- Who is it for?
- Ages 4 months and older
- Conditions the study is for
- Mucopolysaccharidosis Type I (MPS I), Hurler Syndrome, Hurler-Scheie Syndrome
- What is being tested?
- RGX-111
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 3 Apr 2019to 6 Mar 2029
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 4 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- A male or female ≥ 4 months of age.
- Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study-related procedures. If the participant is unable to provide informed consent, then informed assent will be obtained, and informed consent must be provided by the participant's legal guardian.
- Having previously documented diagnosis of MPS I confirmed by molecular genetic testing, or IDUA deficiency in leucocytes or fibroblasts.
- Has documented evidence of CNS involvement due to MPS I based on one of the following criteria, if not explainable by any other neurologic or psychiatric factors:
- A score of ≥ 1 standard deviation below mean on neurodevelopmental testing or in 1 domain of neuropsychological function.
- A decline of > 1 standard deviation on sequential neurodevelopmental testing or in 1 domain of neuropsychological function administered between 3 to 36 months apart.
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- Has a documented diagnosis of severe MPS I confirmed by biallelic mutations predictive of the severe phenotype or has a relative clinically diagnosed with severe MPS I and the same IDUA mutations. This participant is not required to have documented evidence of neurocognitive deficit.
- Participants who have had HSCT may be enrolled in the study if the investigator, Medical Monitor, and Sponsor agree that he/she can safely and successfully participate in the study.
- Has sufficient auditory and visual capacity, with or without aids, to complete the required protocol testing and willing to be compliant with wearing the aid, if applicable, on testing days.
- Females of childbearing potential must have negative serum pregnancy test at the screening visit, have negative results on Day 1, and be willing to have additional pregnancy tests during the study.
- Sexually active male participants must be willing to use a medically accepted method of barrier contraception (eg, condom or female diaphragm) from the screening visit until 24 weeks after vector administration. Cessation of birth control after this point should be discussed with the participant's physician.
- Sexually active females must be willing to use an effective method of birth control from the screening visit until 12 weeks after the last dose of sirolimus or tacrolimus, whichever is later. Cessation of birth control after this point should be discussed with the participant's physician. Effective methods of birth control include a double barrier method (eg, male condom plus a diaphragm), an intrauterine device, or hormonal contraception. Sustained abstinence is an acceptable practice; however, periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of contraception.
Who cannot join
- Has any contradiction to MRI, contrast or to general anesthesia
- Has any contradiction to gadolinium
- Has renal insufficiency as determined by an estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, based on creatinine. If the laboratory determines that the creatinine level is less than the lower limit of assay validation or detection, then the lowest limit cutoff value will be used for the eGFR.
- Has a contradiction for an IC and IVR infusion, including any of the following:
- Review of baseline MRI testing by the team of neuroradiologists/neurosurgeons participating in the study (1 per site) shows a contraindication for an IC and an IVR infusion.
- History of prior head/neck surgery, which resulted in a contraindication to IC and IVR infusion, based on review of available information by the team of neuroradiologists/neurosurgeons participating in the study.
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- Has previously experienced a clinically significant intracranial bleed that, in the opinion of the investigator and team of neuroradiologists/neurosurgeons, is a contraindication to IC and IVR infusion.
- Has any neurocognitive deficit not attributable to MPS I or has a diagnosis of a neuropsychiatric condition that may, in the opinion of the investigator, confound interpretation of study results.
- Has any contradiction to lumbar puncture.
- Has received IT laronidase at any time and experienced a significant AE considered related to IT administration that, in the opinion of the investigator, would put the participant at undue risk.
- Has received IV laronidase at any time and experienced a significant AE considered related to IV administration that, in the opinion of the investigator, would put the participant at undue risk.
- Has imminent plans to receive HSCT within the next year.
- Has any history of lymphoma or history of another cancer other than squamous cell or basal cell carcinoma of the skin that has not been in full remission for at least 3 months before screening.
- Has uncontrolled hypertension (systolic BP > 180 mmHg, diastolic BP > 100 mmHg) despite maximal medical treatment.
- Has alanine aminotransferase (ALT) or aspartate aminotransferase (AST) > 3 × ULN or total bilirubin > 1.5 × ULN at screening, unless the participant has a previously known history of Gilbert's syndrome and a fractionated bilirubin that shows conjugated bilirubin < 35% of total bilirubin.
- Has a history of human immunodeficiency virus (HIV) or hepatitis B or hepatitis C virus infection, or positive screening tests for hepatitis B surface antigen or hepatitis B core antibody, or hepatitis C or HIV antibodies.
- Received any IP within 30 days or 5 half-lives before signing of the Informed consent form (ICF), whichever is longer.
- Is a first-degree family member of a clinical site employee or any other individual involved in the conduct of the study, or is a clinical site employee or any other individual involved in the conduct of the study.
- Is pregnant, < 6 weeks postpartum, breastfeeding at screening, or planning to become pregnant (self or partner) at any time from signing of informed consent through Week 52. Plans to become pregnant after Week 52 should be discussed with the participant's physician.
- Has history of alcohol or substance abuse within 1 year before screening.
- Has a clinically significant ECG or transthoracic echocardiogram abnormality that, in the opinion of the investigator, would compromise the participant's safety.
- Has a serious or unstable medical or psychological condition that, in the opinion of the investigator, would compromise the participant's safety or successful participation in the study or interpretation of study results.
- A history of a hypersensitivity reaction to tacrolimus, sirolimus, or prednisone.
- A history of a severe immunodeficiency (eg, common variable immunodeficiency syndrome), splenectomy, or any underlying condition that predisposes the participant to infection.
- Varicella zoster virus (VZV), herpes zoster (shingles), CMV, or EBV infection that has not completely resolved at least 12 weeks prior to screening.
- Any infection requiring hospitalization or treatment with parenteral anti-infectives not resolved at least 8 weeks prior to Visit 2.
- Any active infection requiring oral anti-infectives (including antivirals) within 10 days prior to Visit 2.
- History of active tuberculosis (TB) or a positive QuantiFERON-TB Gold test during screening.
- Any live vaccine within 4 weeks prior to Day -2.
- Major surgery within 8 weeks before signing the ICF or major surgery planned during the study period.
- Absolute neutrophil count < 1.3 × 103/µL.
- Any condition or laboratory abnormality that the investigator believes would not be appropriate for immunosuppressive therapy.
Trial locations and contacts
2 sites in 2 countries.
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 Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 Gene Therapy in Participants With Mucopolysaccharidosis Type I
RGX-111 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.
Read the detailed description
Mucopolysaccharidosis type I (MPS I) is a rare recessive genetic disease caused by a deficiency of α-L-iduronidase (IDUA) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of patients with MPS I. While currently available therapies, enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), provide clinical benefit over untreated disease progression, they still possess significant limitations. ERT does not cross the blood-brain barrier and, therefore, does not treat the central nervous system (CNS) effects of the disease, and HSCT has clinically relevant morbidity and mortality and is not able to completely treat the CNS effects. RGX-111 is designed to deliver a functioning gene enabling the production of IDUA in the brain. This is a Phase I/II , first-in-human, multicenter, open-label, single-arm dose escalation study of RGX 111. Up to 21 subjects with MPS I will be treated in 2 dose cohorts and will receive a single dose of RGX-111 administered using an investigational device by IC or IVR injection. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period). Following completion of the primary study period, subjects will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-111. At the end of the study, all subjects will be invited to participate in a long-term observational follow-up study.
- Being tested
- RGX-111
- Keywords
- MPS I , gene therapy, Hurler, Hurler- Scheie
- Medical subject headings
- Mucopolysaccharidosis I
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGX-111Genetic
Recombinant adeno-associated virus serotype 9 capsid containing α-L-iduronidase expression cassette
| Study group | Role | What they receive |
|---|---|---|
| Dose 1; 1x10^10 GC/g brain mass of RGX-111 | Experimental | Not described Receives: Genetic: RGX-111 |
| Dose 2; 5x10^10 GC/g brain mass of RGX-111 | Experimental | Not described Receives: Genetic: RGX-111 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety and Tolerability
Number of participants with treatment-related adverse events and serious adverse events. To evaluate safety and tolerability following a single IC or IVR dose administered to participants who have documented CNS involvement due to MPS I.
Measured: 24 Weeks
Secondary outcomes (12)
Long-term Safety and Tolerability
Evaluate safety and tolerability through AE reporting using CTCAE Version 5.0.
Measured: 104 Weeks
Evaluation of the 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 for individuals from ages 6 to 90 years old. The assessment yields composite scores that estimates verbal comprehension and perceptual reasoning abilities. The subtests have different scoring ranges, with higher subtest raw scores indicating completion of more complex items, but can be normalized for cross-subtest comparison.
Measured: 104 Weeks
Evaluation of the impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-II)
Change from baseline in neurodevelopmental parameters as measured by the Bayley Scales of Infant and Toddler Development Third Edition (BSID-III). The BSID-III assesses children with developmental functioning ranging from 1-42 months. In this study, participants will be assessed for the Cognitive, Language, and Motor domains. The domains have different scoring ranges, with higher subtest raw scores indicating better function, but can be normalized for cross-domain comparison.
Measured: 104 Weeks
Evaluation of the 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 cognitive development in young children from ages 30-91 months of age. This study utilizes the General Ability Index (GAI) comprised of 4 core subtests depending on the child's age. The subtests have different scoring ranges, with higher subtest raw scores indicating completion of more complex items but can be normalized for cross-subtest comparison.
Measured: 104 Weeks
Evaluation of the long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III)
Change in 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 comparison
Measured: 104 Weeks
Vector Concentrations
To evaluate vector concentrations in CSF and serum measured by qPCR for RGX-111
Measured: Baseline, Week 1, Week 6,Week 7,Week 9,Week 24,Week 32,Week 40, Week 52
Vector Shedding
To evaluate vector shedding in feces and urine by qPCR for RGX-111
Measured: Baseline, Week 1, Week 6,Week 7,Week 9,Week 24,Week 32,Week 40, Week 52
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS)
To assess the pharmacodynamic effect of RGX-111 on Hepran Sulfate (HS) levels measured by Lumbar Puncture at scheduled time points.
Measured: Baseline, Week 8, Week 16, Week 24, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA)
To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measured by Lumbar Puncture at scheduled time points.
Measured: Baseline, Week 8, Week 16, Week 24, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS)
To assess the pharmacodynamic effect of RGX-111 on Hepran Sulfate (HS) levels measured by plasma collection at scheduled time points.
Measured: Baseline, Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA)
To assess the pharmacodynamic effect of RGX-111 on α-L-iduronidase (IDUA) levels measured by plasma collections at scheduled time points.
Measured: Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Urine Biomarkers
To assess the pharmacodynamic effect of RGX-111 on glycosaminoglycans (GAGs) levels measured by urine analysis at scheduled time points.
Measured: Baseline, Week 1, Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Jul 2018
- Study start3 Apr 2019
- Primary completion7 Jul 2027 (estimated)
- Study completion6 Mar 2029 (estimated)
Why it stopped: The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Mucopolysaccharidosis Type I (MPS I)Studies 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Sequential Assignment Dose escalation
- Masking
- None (open label)
- 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
- NCT03580083
- Sponsor's protocol ID
- RGX-111-002
- Status last verified
- Mar 2026
- First submitted
- 18 Jun 2018
- Last updated
- 12 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
De BP, Rosenberg JB, Selvan N, Wilson I, Yusufzai N, Greco A, Kaminsky SM, Heier LA, Ricart Arbona RJ, Miranda IC, Monette S, Nair A, Khanna R, Crystal RG, Sondhi D. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Hum Gene Ther. 2023 Sep;34(17-18):905-916. doi: 10.1089/hum.2023.067.
PubMed 37624739
Study at a glance
From the registry record, last updated by the sponsor on 12 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Multicenter, Open-Label Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of Intracisternal RGX-111 Gene Therapy in Participants With Mucopolysaccharidosis Type I
- ClinicalTrials.gov ID
- NCT03580083
- Acronym / sponsor ID
- RGX-111-002
- Keywords
- MPS I , gene therapy, Hurler, Hurler- Scheie
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- SuspendedReason stopped: The FDA placed a clinical hold on RGX-111 investigational gene therapy following an initial single case report of neoplasm (intraventricular CNS tumor) in a participant treated in its phase I/II study four years prior.
- Enrolment
- 21 participants (estimated)
- Lead sponsor
- REGENXBIO Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Apr 2019 (actual)
- Primary completion
- 7 Jul 2027 (estimated)
- Study completion
- 6 Mar 2029 (estimated)
- First posted
- 9 Jul 2018 (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 is a gene therapy which is intended to deliver a functional copy of the α-L-iduronidase (IDUA) gene to the central nervous system. This is a safety and dose ranging study to determine whether RGX-111 is safe and tolerated by patients with MPS I.
Detailed description
Mucopolysaccharidosis type I (MPS I) is a rare recessive genetic disease caused by a deficiency of α-L-iduronidase (IDUA) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of patients with MPS I. While currently available therapies, enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), provide clinical benefit over untreated disease progression, they still possess significant limitations. ERT does not cross the blood-brain barrier and, therefore, does not treat the central nervous system (CNS) effects of the disease, and HSCT has clinically relevant morbidity and mortality and is not able to completely treat the CNS effects. RGX-111 is designed to deliver a functioning gene enabling the production of IDUA in the brain. This is a Phase I/II , first-in-human, multicenter, open-label, single-arm dose escalation study of RGX 111. Up to 21 subjects with MPS I will be treated in 2 dose cohorts and will receive a single dose of RGX-111 administered using an investigational device by IC or IVR injection. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period). Following completion of the primary study period, subjects will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-111. At the end of the study, all subjects will be invited to participate in a long-term observational follow-up study.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Sequential Assignment Dose escalation
| Arm | Type | Receives |
|---|---|---|
| Dose 1; 1x10^10 GC/g brain mass of RGX-111 | Experimental | Genetic: RGX-111 |
| Dose 2; 5x10^10 GC/g brain mass of RGX-111 | Experimental | Genetic: RGX-111 |
Who may qualify
- Ages
- 4 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (12 listed)
- A male or female ≥ 4 months of age.
- Willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any study-related procedures. If the participant is unable to provide informed consent, then informed assent will be obtained, and informed consent must be provided by the participant's legal guardian.
- Having previously documented diagnosis of MPS I confirmed by molecular genetic testing, or IDUA deficiency in leucocytes or fibroblasts.
- Has documented evidence of CNS involvement due to MPS I based on one of the following criteria, if not explainable by any other neurologic or psychiatric factors:
- Participants who have had HSCT may be enrolled in the study if the investigator, Medical Monitor, and Sponsor agree that he/she can safely and successfully participate in the study.
Key exclusion criteria (32 listed)
- Has any contradiction to MRI, contrast or to general anesthesia
- Has a contradiction for an IC and IVR infusion, including any of the following:
- Has any neurocognitive deficit not attributable to MPS I or has a diagnosis of a neuropsychiatric condition that may, in the opinion of the investigator, confound interpretation of study results.
- Has any contradiction to lumbar puncture.
- Has received IT laronidase at any time and experienced a significant AE considered related to IT administration that, in the opinion of the investigator, would put the participant at undue risk.
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 |
|---|---|---|
| RGX-111Recombinant adeno-associated virus serotype 9 capsid containing α-L-iduronidase expression cassette | Genetic | Dose 1; 1x10^10 GC/g brain mass of RGX-111, Dose 2; 5x10^10 GC/g brain mass of RGX-111 |
Primary outcome
Safety and Tolerability
Time frame: 24 Weeks
Number of participants with treatment-related adverse events and serious adverse events. To evaluate safety and tolerability following a single IC or IVR dose administered to participants who have documented CNS involvement due to MPS I.
12 secondary outcomes
Long-term Safety and Tolerability
Time frame: 104 Weeks
Evaluation of the impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WASI-II)
Time frame: 104 Weeks
Evaluation of the impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (BSID-II)
Time frame: 104 Weeks
Evaluation of the impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (WPPSI-IV)
Time frame: 104 Weeks
Evaluation of the long-term impact of RGX-111 on neurodevelopmental parameters of cognitive, behavioral, and adaptive function (VABS-III)
Time frame: 104 Weeks
Vector Concentrations
Time frame: Baseline, Week 1, Week 6,Week 7,Week 9,Week 24,Week 32,Week 40, Week 52
Vector Shedding
Time frame: Baseline, Week 1, Week 6,Week 7,Week 9,Week 24,Week 32,Week 40, Week 52
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (HS)
Time frame: Baseline, Week 8, Week 16, Week 24, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on CSF Biomarkers (IDUA)
Time frame: Baseline, Week 8, Week 16, Week 24, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (HS)
Time frame: Baseline, Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Plasma Biomarkers (IDUA)
Time frame: Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Pharmacodynamic Effect of RGX-111 on Urine Biomarkers
Time frame: Baseline, Week 1, Week 2, Week 4, Week 8, Week 16, Week 24, Week 32, Week 40, Week 48, Week 52, Week 56, Week 78, Week 104
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Mar 2026.
- Derived from this study: PubMed 37624739De BP, Rosenberg JB, Selvan N, Wilson I, Yusufzai N, Greco A, Kaminsky SM, Heier LA, Ricart Arbona RJ, Miranda IC, Monette S, Nair A, Khanna R, Crystal RG, Sondhi D. Assessment of Safety and Biodistribution of AAVrh.10hCLN2 Following Intracisternal Administration in Nonhuman Primates for the Treatment of CLN2 Batten Disease. Hum Gene Ther. 2023 Sep;34(17-18):905-916. doi: 10.1089/hum.2023.067.
- 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.
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?
A Phase 1/2 study testing RGX-111 in people with Mucopolysaccharidosis Type I (MPS I) and Hurler Syndrome, 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 12 inclusion and 32 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: paused, as of the sponsor's last update on 12 Mar 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.
What does this combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Safety and Tolerability (time frame: 24 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03580083, which also lists 1 linked publication.
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 12 Mar 2026.