Mucopolysaccharidosis Type II (MPS II)Phase 2/3NCT03566043
CAMPSIITE™ RGX-121 Gene Therapy in Subjects With MPS II (Hunter Syndrome)
A Phase 2/3 study testing RGX-121 in people with Mucopolysaccharidosis Type II (MPS II), run by REGENXBIO Inc. at 5 sites in 2 countries.
Trial summary
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II. Read more about this study
- Who is it for?
- Ages 4 months to 5 years, men only
- Conditions the study is for
- Mucopolysaccharidosis Type II (MPS II)
- What is being tested?
- RGX-121
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 5 sitesin 2 countries
- Study dates
- 27 Sep 2018to Aug 2025
- Phase
- Phase 2/3
- 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 4 months to 5 years
- Men only
- No healthy volunteers
- Child
Part 1 Inclusion Criteria:
- The subject's legal guardian(s) is (are) willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures
- Is a male ≥4 months to < 5 years of age on Day 1
- Must meet any of the following criteria:
- Has a documented diagnosis of MPS II and a has a neurocognitive testing score ≤ 77 (Bayley or Kaufman), OR
- Has a documented diagnosis of MPS II AND has a decline of ≥ 1 standard deviation on serial neurocognitive testing administered between 3 to 36 months apart (Bayley or Kaufman) OR
- Has a relative clinically diagnosed with severe MPS II who has the same IDS mutation as the subject AND in the opinion of a geneticist has inherited a severe form of MPS II OR
- Has documented mutation (s) in IDS that in the opinion of a geneticist is always known to result in a neuronopathic phenotype AND in the opinion of a clinician has a severe form of MPS II
Part 2 Inclusion Criteria:
- The subject's legal guardian(s) is (are) willing and able to provide written, signed informed consent after the nature of the study has been explained, and prior to any research-related procedures
- Is a male ≥4 months to < 5 years of age on Day 1
- Has a documented diagnosis of neuronopathic MPS II. Neuronopathic MPS II can be documented with any of the following methods:
- Has a BSID-III Cognitive Composite score at or below -1 SD (85) from normative mean
- Has two consecutive neurodevelopmental assessments that support a decline on MSEL visual receptive, expressive language, or fine motor, or BSID-III cognition, expressive language, or fine motor ≥ 1 SD on serial neurocognitive testing administered between 3 to 36 months apart
- Has a relative clinically diagnosed with neuronopathic MPS II who has the same IDS mutation as the subject AND the subject, in the opinion of a geneticist, has inherited a neuronopathic form of MPS II
- Has documented mutation(s) in IDS known to result in a neuronopathic phenotype
Part 1 Exclusion Criteria:
- Has contraindications for intracisternal (IC) injection, intracerebroventricular (ICV) injection or lumbar puncture
- Has contraindications for immunosuppressive therapy
- Has neurocognitive deficit not attributable to MPS II or diagnosis of a neuropsychiatric condition
- Has a (cerebral) ventricular shunt that may impact the proper dosing of the subject
- Received hematopoietic stem cell transplantation
- Has had prior treatment with an AAV-based gene therapy product
- Received ELAPRASE® via intrathecal (IT) administration within 4 months of signing the ICF or experienced a serious hypersensitivity reaction to ELAPRASE®
- Has received any investigational product within 30 days of Day 1 or 5 half-lives before signing the ICF, whichever is longer
Part 2 Exclusion Criteria:
- Has a contraindication for an IC injection, ICV injection or lumbar puncture
- Has contraindications for immunosuppressive therapy
- Has neurocognitive deficit not attributable to MPS II or diagnosis of a neuropsychiatric condition
- Has a (cerebral) ventricular shunt that may impact the proper dosing of the subject
- Received hematopoietic stem cell transplantation
- Has had prior treatment with an AAV-based gene therapy product
- Is receiving idursulfase (ELAPRASE®) via intrathecal (IT) administration, or a blood brain barrier-crossing enzyme replacement therapy. Subjects receiving IT ELAPRASE® or a blood brain barrier-crossing ERT may enroll if they agree to discontinue these therapies starting at least 3 months prior to dosing with RGX-121, and for the 24 months of follow-up
- Has received any investigational product within 30 days of Day 1 or 5 half-lives before signing the ICF, whichever is longer
Trial locations and contacts
5 sites in 2 countries.
5 sites in 2 countries
United States4 sites
St. Peter's University Hospital
New Brunswick, New Jersey, 08901
University of California San Francisco, Benioff Children's Hospital
Oakland, California, 94609
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh - UPMC: Program for Neurodevelopment in Rare Disorders
Pittsburgh, Pennsylvania, 15224
Brazil1 site
Hospital de Clinicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, 90035-903
About this study
Official title: A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
Read the detailed description
MPS II (Hunter Syndrome) is a rare X-linked recessive genetic disease caused by mutations in the iduronate-2-sulfatase gene (IDS). Enzyme replacement therapy (ERT) with recombinant idursulfase (ELAPRASE®) is the only approved product for the treatment of Hunter syndrome, however, ERT as currently administered does not cross the blood brain barrier and is therefore unable to address the unmet need in MPS II patients with central nervous system (CNS) (neurocognition and behavior) involvement. RGX-121 is designed to deliver a functional gene to cells in the CNS. Iduronate-2-sulfatase (I2S) may then be secreted by transduced cells, which may then cross-correct non-transduced cells by taking up the functional enzyme.
This is a Phase I/II/III study enrolling in two sequential parts. Part 1 is a Phase I/II, first-in-human, multicenter, open-label, single arm dose escalation study of RGX-121. Three one-time doses of RGX-121 will be studied in up to 16 pediatric subjects who have neuronopathic MPS II. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period) whereupon, subjects will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-121. Part 2 is a pivotal expansion, multicenter, open-label, single arm study of RGX-121. A single dose of RGX-121 will be studied in up to 30 pediatric patients who have been diagnosed with neuronopathic MPS II. Subjects will be assessed at various timepoints for 24 months after receiving RGX-121. Subjects will be given the opportunity to enroll in a separate 3-year long-term follow-up study in accordance with the US federal government guidelines for the safety follow-up of patients receiving gene therapy.
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Being tested
- RGX-121
- Keywords
- MPS II, gene therapy, Hunter Syndrome, Lysosomal Storage Disorder
- Medical subject headings
- Mucopolysaccharidosis II, Sudden Infant Death, Lysosomal Storage Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RGX-121Genetic
Recombinant adeno-associated virus serotype 9 capsid containing human iduronate-2-sulfatase expression cassette
| Study group | Role | What they receive |
|---|---|---|
| Part 1: RGX-121 Dose 1 | Experimental | 1.3x10^10 GC/g brain mass of RGX-121 Receives: Genetic: RGX-121 |
| Part 1: RGX-121 Dose 2 | Experimental | 6.5x10^10 GC/g brain mass of RGX-121 Receives: Genetic: RGX-121 |
| Part 1: RGX-121 Dose 2 Expanded Cohort | Experimental | 6.5x10^10 GC/g brain mass of RGX-121 Receives: Genetic: RGX-121 |
| Part 1: RGX-121 Dose 3 | Experimental | 2.0x10^11 GC/g brain mass of RGX-121 Receives: Genetic: RGX-121 |
| Part 1: RGX-121 Dose 3 Expanded Cohort | Experimental | 2.9x10^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) equivalent to, 2.0x10^11 GC/g brain mass of RGX-121 (Poly-A-specific PCR assay) Receives: Genetic: RGX-121 |
| Part 2: RGX-121 Pivotal Expansion | Experimental | 2.9x10^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) Receives: Genetic: RGX-121 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1 Safety
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03).
Measured: 24 Weeks
Part 2 Biomarkers
CSF GAG levels (as measured by D2S6)
Measured: 52 Weeks
Part 2 Biomarkers
CSF GAG levels (as measured by D2S6)
Measured: 104 weeks
Part 2 Neurodevelopmental parameters
Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability, language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
Measured: 52 Weeks
Part 2 Neurodevelopmental parameters
Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
Measured: 104 weeks
Secondary outcomes (8)
Part 1 Safety
Number of participants with treatment-related adverse events as assessed by CTCAE (Version 4.03)
Measured: 104 Weeks
Part 1 Biomarkers
Glycosaminoglycan levels and iduronate-2-sulfatase activity
Measured: 104 Weeks
Part 1 Neurodevelopmental parameters
Neurodevelopment parameters of cognitive, behavioral \& adaptive function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Kaufman Assessment Battery for Children, 2nd Edition (KABC-II) and Mullen Scales of Early Learning (MSEL). The BSID-III evaluates the developmental functioning of infants \& small children 1 to 42 months old to identify developmental delays. The KABC-II measures cognitive skill \& academic knowledge to evaluate knowledge acquired \& level of school learning attained. This test evaluates children 2.5 to 12.5 years old in 4 dimensions: mental, sequential and simultaneous processing, \& knowledge. The MSEL measures cognitive ability language \& motor development \& has 5 scales: gross \& fine motor, visual reception, \& receptive and expressive language. An increase in raw \& age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
Measured: 104 Weeks
Part 1 Change in neurodevelopmental parameters
Neurodevelopment parameters of cognitive, behavioral and adaptive function as measured by the Vineland Adaptive Behavior Scales, 2nd Edition (VABS-II), Comprehensive Interview Form. The Vineland Adaptive Behavior Scale II (VABS-II) is a standardized paediatric functional assessment tool. The VABS-II offers a way to measure personal and social self-sufficiency in real-life situations and to observe how these cognitive abilities impact the autonomy management process when put into practice. The VABS-II consists in a semi-structured interview with the parents. Higher scores mean a better outcome.
Measured: 104 Weeks
Part 2 Change in neurodevelopmental parameters
Change from baseline in neurodevelopment effect on daily living skills as measured by the Vineland Adaptive Behavior Scales, 2nd Edition (VABS-II), Comprehensive Interview Form. The Vineland Adaptive Behavior Scale II (VABS-II) is a standardized paediatric functional assessment tool. The VABS-II offers a way to measure personal and social self-sufficiency in real-life situations and to observe how these cognitive abilities impact the autonomy management process when put into practice. The VABS-II consists in a semi-structured interview with the parents. Higher scores mean a better outcome.
Measured: 52 Weeks
Part 2 Change in brain magnetic resonance imaging (MRI) parameters
Change from baseline in brain size as measured on MRI
Measured: 52 Weeks
Part 2 Safety
Number of participants with treatment-related adverse events as assessed by CTCAE (Version 4.03)
Measured: 24 Months
Part 2 Biomarkers
Change in Glycosaminoglycan levels and iduronate-2-sulfatase activity
Measured: 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Jun 2018
- Study start27 Sep 2018
- Primary completion27 Nov 2023
- Study completionAug 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Mucopolysaccharidosis Type II (MPS II)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 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Dose escalation
- Masking
- None (open label)
- Enrolment
- 48 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
- NCT03566043
- Sponsor's protocol ID
- RGX-121-101
- Status last verified
- Jan 2025
- First submitted
- 1 May 2018
- Last updated
- 28 Jan 2025
- 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 28 Jan 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1/2/3 Multicenter, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of RGX-121 in Pediatric Subjects With MPS II (Hunter Syndrome)
- ClinicalTrials.gov ID
- NCT03566043
- Acronym / sponsor ID
- RGX-121-101
- Conditions
- Mucopolysaccharidosis Type II (MPS II)
- Keywords
- MPS II, gene therapy, Hunter Syndrome, Lysosomal Storage Disorder
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 48 participants (estimated)
- Lead sponsor
- REGENXBIO Inc.Industry (pharma, biotech, devices)
- Study start
- 27 Sep 2018 (actual)
- Primary completion
- 27 Nov 2023 (actual)
- Study completion
- Aug 2025 (estimated)
- First posted
- 21 Jun 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-121 is a gene therapy which is intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study is a safety and efficacy, dose ranging study to determine whether RGX-121 is safe, effective and well-tolerated by patients with MPS II.
Detailed description
MPS II (Hunter Syndrome) is a rare X-linked recessive genetic disease caused by mutations in the iduronate-2-sulfatase gene (IDS). Enzyme replacement therapy (ERT) with recombinant idursulfase (ELAPRASE®) is the only approved product for the treatment of Hunter syndrome, however, ERT as currently administered does not cross the blood brain barrier and is therefore unable to address the unmet need in MPS II patients with central nervous system (CNS) (neurocognition and behavior) involvement. RGX-121 is designed to deliver a functional gene to cells in the CNS. Iduronate-2-sulfatase (I2S) may then be secreted by transduced cells, which may then cross-correct non-transduced cells by taking up the functional enzyme.
This is a Phase I/II/III study enrolling in two sequential parts. Part 1 is a Phase I/II, first-in-human, multicenter, open-label, single arm dose escalation study of RGX-121. Three one-time doses of RGX-121 will be studied in up to 16 pediatric subjects who have neuronopathic MPS II. Safety will be the primary focus for the initial 24 weeks after treatment (primary study period) whereupon, subjects will continue to be assessed (safety and efficacy) for up to a total of 104 weeks following treatment with RGX-121. Part 2 is a pivotal expansion, multicenter, open-label, single arm study of RGX-121. A single dose of RGX-121 will be studied in up to 30 pediatric patients who have been diagnosed with neuronopathic MPS II. Subjects will be assessed at various timepoints for 24 months after receiving RGX-121. Subjects will be given the opportunity to enroll in a separate 3-year long-term follow-up study in accordance with the US federal government guidelines for the safety follow-up of patients receiving gene therapy.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 6
- Model description
- Dose escalation
| Arm | Type | Receives |
|---|---|---|
| Part 1: RGX-121 Dose 1 | Experimental | Genetic: RGX-1211.3x10^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 2 | Experimental | Genetic: RGX-1216.5x10^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 2 Expanded Cohort | Experimental | Genetic: RGX-1216.5x10^10 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 3 | Experimental | Genetic: RGX-1212.0x10^11 GC/g brain mass of RGX-121 |
| Part 1: RGX-121 Dose 3 Expanded Cohort | Experimental | Genetic: RGX-1212.9x10^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) equivalent to, 2.0x10^11 GC/g brain mass of RGX-121 (Poly-A-specific PCR assay) |
| Part 2: RGX-121 Pivotal Expansion | Experimental | Genetic: RGX-1212.9x10^11 GC/g brain mass of RGX-121 (transgene-specific PCR assay) |
Who may qualify
- Ages
- 4 months to 5 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child
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-121Recombinant adeno-associated virus serotype 9 capsid containing human iduronate-2-sulfatase expression cassette | Genetic | Part 1: RGX-121 Dose 1, Part 1: RGX-121 Dose 2, Part 1: RGX-121 Dose 2 Expanded Cohort, Part 1: RGX-121 Dose 3, Part 1: RGX-121 Dose 3 Expanded Cohort, Part 2: RGX-121 Pivotal Expansion |
Primary outcome
Part 1 Safety
Time frame: 24 Weeks
Number of participants with treatment-related adverse events and serious adverse events as assessed by CTCAE (Version 4.03).
Part 2 Biomarkers
Time frame: 52 Weeks
CSF GAG levels (as measured by D2S6)
Part 2 Biomarkers
Time frame: 104 weeks
CSF GAG levels (as measured by D2S6)
Part 2 Neurodevelopmental parameters
Time frame: 52 Weeks
Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability, language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
Part 2 Neurodevelopmental parameters
Time frame: 104 weeks
Neurodevelopmental function as measured by the Bayley Scales of Infant and Toddler Development, 3rd Edition (BSID-III) or Mullen Scales of Early Learning (MSEL). The Bayley Scales of Infant Development, or the BSID-III is an individually administered test, designed to evaluate the developmental functioning of infants and small children, between 1 and 42 months of age. The purpose of the test is to identify infants and children with developmental delay. The Mullen Scales of Early Learning (MSEL) is a developmental test to measure cognitive ability language and motor development. The test has five scales: gross motor, visual reception, fine motor, receptive language, and expressive language. An increase in raw and age equivalent scores indicates neurodevelopmental skill acquisition. Standard scores compare function to age matched normative data.
8 secondary outcomes
Part 1 Safety
Time frame: 104 Weeks
Part 1 Biomarkers
Time frame: 104 Weeks
Part 1 Neurodevelopmental parameters
Time frame: 104 Weeks
Part 1 Change in neurodevelopmental parameters
Time frame: 104 Weeks
Part 2 Change in neurodevelopmental parameters
Time frame: 52 Weeks
Part 2 Change in brain magnetic resonance imaging (MRI) parameters
Time frame: 52 Weeks
Part 2 Safety
Time frame: 24 Months
Part 2 Biomarkers
Time frame: 24 Months
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 28 Jan 2025.
- 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.
5 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing RGX-121 in people with Mucopolysaccharidosis Type II (MPS II), run by REGENXBIO Inc. at 5 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?
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 28 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
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?
Part 1 Safety (time frame: 24 Weeks). The study lists 5 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 28 Jan 2025.