Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)NCT04462692
An Observational Study in Children With CLN2 Batten Disease
An observational study of Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), run by REGENXBIO Inc..
Trial summary
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 31 Mar 2021to Oct 2023
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Children with CLN2 Batten disease undergoing current standard of care for their condition.
Who may be able to join
- A participant is eligible to be included in the study only if all of the following criteria apply:
- His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
- Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
- Is currently receiving biweekly ERT treatment with cerliponase alfa
Who cannot join
- A participant is excluded from the study if any of the following criteria apply:
- Has had prior treatment with an adeno-associated virus-based AAV gene therapy
- Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease
About this study
Official title: A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
This is a prospective, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
Read the detailed description
CLN2 is a rare disease with limited available ocular natural history data. While current standard of care slows motor degeneration, it is not known to treat the ocular manifestations of disease. This study is planned to document, through prospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2 Batten disease undergoing current standard of care for their condition. No investigational product is administered in this observational study.
- Keywords
- CLN2, Batten Disease
- Medical subject headings
- Neuronal Ceroid-Lipofuscinoses
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in retinal structure in children with CLN2 Batten disease
As assessed by SD-OCT measures over time.
Measured: 96 weeks
Secondary outcomes (2)
Change in visual function
As assessed by visual acuity over time.
Measured: 96 weeks
Change in visual function
As assessed by pupillary light reflex over time.
Measured: 96 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2020
- Study start31 Mar 2021 (estimated)
- Primary completionOct 2023 (estimated)
- Study completionOct 2023 (estimated)
Why it stopped: study stopped due to company decision
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- REGENXBIO Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04462692
- Sponsor's protocol ID
- RGX-381-9101
- Status last verified
- Oct 2021
- First submitted
- 2 Jul 2020
- Last updated
- 1 Nov 2021
- Results posted
- No
- Data monitoring committee
- No
- 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 1 Nov 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Observational Study to Evaluate Ocular Disease Progression in Children With CLN2 Batten Disease
- ClinicalTrials.gov ID
- NCT04462692
- Acronym / sponsor ID
- RGX-381-9101
- Keywords
- CLN2, Batten Disease
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: study stopped due to company decision
- Enrolment
- 0 participants (actual)
- Lead sponsor
- REGENXBIO Inc.Industry (pharma, biotech, devices)
- Study start
- 31 Mar 2021 (estimated)
- Primary completion
- Oct 2023 (estimated)
- Study completion
- Oct 2023 (estimated)
- First posted
- 8 Jul 2020 (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, longitudinal natural history study to document the progression of ocular manifestations of CLN2 disease among a community-dwelling population of pediatric participants affected by this disease.
Detailed description
CLN2 is a rare disease with limited available ocular natural history data. While current standard of care slows motor degeneration, it is not known to treat the ocular manifestations of disease. This study is planned to document, through prospective data collection, ocular disease progression in children with a clinical presentation consistent with CLN2 Batten disease undergoing current standard of care for their condition. No investigational product is administered in this observational study.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Children with CLN2 Batten disease undergoing current standard of care for their condition.
Key inclusion criteria (4 listed)
- A participant is eligible to be included in the study only if all of the following criteria apply:
- His or her legal guardian(s) is(are) willing and able to provide them written, signed informed consent.
- Has documented diagnosis of CLN2 disease due to TPP1 deficiency, or has a relative clinically diagnosed with CLN2 with the same mutation as the participant
- Is currently receiving biweekly ERT treatment with cerliponase alfa
Key exclusion criteria (3 listed)
- Has had prior treatment with an adeno-associated virus-based AAV gene therapy
- Is currently participating in a clinical trial of investigational product for the treatment of CLN2 disease
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in retinal structure in children with CLN2 Batten disease
Time frame: 96 weeks
As assessed by SD-OCT measures over time.
2 secondary outcomes
Change in visual function
Time frame: 96 weeks
Change in visual function
Time frame: 96 weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Nov 2021.
- 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.
Questions about this trial
What is this clinical trial studying?
An observational study of Neuronal Ceroid Lipofuscinosis Type 2 (CLN2), run by REGENXBIO Inc..
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 4 inclusion and 3 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: withdrawn, as of the sponsor's last update on 1 Nov 2021. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
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?
Change in retinal structure in children with CLN2 Batten disease (time frame: 96 weeks). 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 1 Nov 2021.