www.BattenTreatmentReport.com
https://clinicaltrials.gov/ct2/show/NCT04480476?cond=Batten+Disease&lupd_s=07%2F07%2F2020&lupd_d=14&sort=nwst
Condition : Late-infantile Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Intervention :
Sponsor : Regenxbio Inc.
Not yet recruiting
A Retrospective, Natural History Study in Children With CLN2
NCT04480476
Tue, 21 Jul 2020 12:00:00 EDT
https://clinicaltrials.gov/ct2/show/NCT04476862
Brineura (cerliponase alfa)
Brief Summary:
This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are currently being treated with cerliponase alfa. Patients receiving or expected to receive cerliponase alfa within 60 days of signing the informed consent form (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites that have agreed to participate and protocol inclusion criteria are met. Data may be collected for all or some of the assessments as outlined in the protocol, dependent upon the clinic’s and/or individual patient’s standard of care.
Condition or disease Intervention/treatment
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Drug: Cerliponase Alfa
Device: Administration Kit
Study Design
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Study Type : Observational
Estimated Enrollment : 35 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Cerliponase Alfa Observational Study
Estimated Study Start Date : August 24, 2020
Estimated Primary Completion Date : August 24, 2030
Estimated Study Completion Date : August 24, 2030
https://clinicaltrials.gov/ct2/show/NCT04462692
https://clinicaltrials.gov/ct2/show/NCT04462692?cond=Batten+Disease&lupd_s=06%2F24%2F2020&lupd_d=14&sort=nwst
Condition : Neuronal Ceroid Lipofuscinosis Type 2 (CLN2)
Intervention :
Sponsor : Regenxbio Inc.
Not yet recruiting
An Observational Study in Children With CLN2 Batten Disease
NCT04462692
Wed, 08 Jul 2020 12:00:00 EDT
https://clinicaltrials.gov/ct2/show/NCT04273243
Long-Term Follow Up of CLN6 Batten Disease Subjects Following Gene Transfer
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT04273243
Recruitment Status : Recruiting
First Posted : February 18, 2020
Last Update Posted : February 26, 2020
See Contacts and Locations
Sponsor:
Amicus Therapeutics
Information provided by (Responsible Party):
Amicus Therapeutics
Study DetailsTabular ViewNo Results PostedDisclaimerHow to Read a Study Record
Study Description
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Brief Summary:
This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501.
Condition or disease Intervention/treatment
CLN6
Batten Disease
Genetic: AT-GTX-501
Detailed Description:
This is a long-term safety and efficacy study in subjects with CLN6 Batten disease who previously received a single intrathecal administration of AT-GTX-501. The assessments described in this long-term follow-up (LTFU) study (AT GTX 501 02) are performed following and in addition to the initial 2 years of assessments in the treatment study (AT-GTX-501-01). In this LTFU study, subjects complete safety and efficacy assessments throughout the study’s 13-year duration. Combining the duration of the initial treatment study and this LTFU study, the overall duration reflects a follow-up period up-to 15 years since gene transfer via AT-GTX-501.
The primary outcome for this study is to assess the long-term safety of AT-GTX-501 in subjects with CLN6 Batten disease.
The secondary outcome measure of this study is to assess the long-term efficacy of AT-GTX-501 in subjects with CLN6 Batten disease.
Study Design
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Study Type : Observational
Estimated Enrollment : 13 participants
Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Long-Term Follow-Up of AT-GTX-501 scAAV9 Gene Transfer in Subjects With CLN6 Batten Disease
Actual Study Start Date : January 24, 2020
Estimated Primary Completion Date : January 2035
Estimated Study Completion Date : January 2035
