Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
PHASE1/PHASE2
7 participants
INTERVENTIONAL
2018-11-13
2029-09-30
Brief Summary
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Detailed Description
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This study consists of a one-time injection of CLN-301 with follow-up visits on Day 7, 14, 21, and 30, followed by every 3 months through 1 year post-dose, and then every 6 months through the fifth year. There are two Cohorts with a low dose and a high dose.
The primary outcome for this clinical study is to evaluate safety. The co-primary objective is to determine the efficacy of CLN-301 as measured by United Batten Disease Rating Scale (UBDRS) physical subscale.
The secondary outcome measures include Pediatric Quality of Life (PedsQL) inventory, seizure subscale of the UBDRS and global impression subscale of the UBDRS.
The exploratory outcome measures include visual impairment assessment, cognitive evaluations, Brain magnetic resonance imaging (MRI), electroencephalogram (EEG), electrocardiogram (ECG) and echocardiogram (ECHO).
For more information about this study, please contact Alcyone Therapeutics at [email protected]
Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
Dose escalation in this study will begin with low-dose, determined to be the minimal efficacious dose as determined in non-clinical studies. Dose escalation to a high-dose (2x the minimally effective dose (MED) as evaluated in Cohort 1) will proceed as part of Cohort 2 of the study upon demonstration of safety of the low-dose in Cohort 1 of the study.
TREATMENT
NONE
Study Groups
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Cohort 1: CLN-301 Low-Dose
No more than 5 mL of 6 x 1013 vg CLN-301 administered via intrathecal injection
Low dose CLN-301
Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at low dose
Cohort 2: CLN-301 High-Dose
No more than 10 mL of 1.2 x 1014 vg of CLN-301 administered via intrathecal injection
High dose CLN-301
Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at high dose
Interventions
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Low dose CLN-301
Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at low dose
High dose CLN-301
Subjects with diagnosis of CLN3 Batten disease will receive a single dose of CLN-301 at high dose
Eligibility Criteria
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Inclusion Criteria
2. Aged ≥ 3 to \< 11 years
3. UBDRS physical impairment score of ≤ 7
4. Able to walk independently at least 50 feet
Exclusion Criteria
2. Presence of another neurological illness that may have caused cognitive decline (eg, trauma, meningitis, hemorrhage) before screening
3. Active viral infection (includes HIV or serology positive for hepatitis B or C)
4. Subjects with 2 consecutive aminotransaminase liver tests \> 3 times the upper limit of normal or \> 1.5 times the upper limit of normal if taking valproic acid at Visit 1 (screening/baseline)
5. Subjects with anti-AAV9 antibody titers \> 1:400 as determined by ELISA (enzyme-linked immunosorbent assay) binding immunoassay
6. Abnormal laboratory values considered clinically significant
7. Presence of immunologic disease
8. Has received stem cell or bone marrow transplantation
9. Has received any form of organ transplant
10. History of or current chemotherapy, radiotherapy, or other immunosuppression therapy within the past 30 days (corticosteroid treatment may be permitted at the discretion of the investigator)
11. Current use of cannabinoids and any by-products
12. Contraindications for intrathecal administration of the product or lumbar puncture (for collection of CSF), such as bleeding disorders or other medical conditions (eg, spina bifida, meningitis, or clotting abnormalities)
13. Contraindications for MRI scans (eg, cardiac pacemaker, metal fragment or chip in the eye, aneurysm clip in the brain)
14. Poorly controlled seizures - intractable epilepsy
15. Episode of generalized motor status epilepticus within 4 weeks before the Gene Transfer visit
16. History of corneal or intraocular surgery
17. Severe infection (eg, upper respiratory tract infection, pneumonia, pyelonephritis, or meningitis) within 4 weeks before the Gene Transfer visit (Enrollment may be postponed.)
18. Has received any investigational medication within 30 days before the infusion of study drug
19. Has a medical condition or extenuating circumstance that, in the opinion of the investigator, might compromise the subject's ability to comply with the protocol required testing or procedures or compromise the subject's wellbeing, safety, or clinical interpretability
20. Pregnancy at screening or Day 0. Any female subject judged by the investigator to be of childbearing potential will be tested for pregnancy.
21. Family does not want to disclose subject's study participation with primary care physician and other medical providers
3 Years
10 Years
ALL
No
Sponsors
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Alcyone Therapeutics, Inc
INDUSTRY
Responsible Party
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Principal Investigators
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Kathrin C Meyer, PhD
Role: PRINCIPAL_INVESTIGATOR
Alcyone Therapeutics
Locations
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Nationwide Children's Hospital
Columbus, Ohio, United States
Countries
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References
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Schulz A, Kohlschutter A, Mink J, Simonati A, Williams R. NCL diseases - clinical perspectives. Biochim Biophys Acta. 2013 Nov;1832(11):1801-6. doi: 10.1016/j.bbadis.2013.04.008. Epub 2013 Apr 17.
Phillips SN, Benedict JW, Weimer JM, Pearce DA. CLN3, the protein associated with batten disease: structure, function and localization. J Neurosci Res. 2005 Mar 1;79(5):573-83. doi: 10.1002/jnr.20367.
Munroe PB, Mitchison HM, O'Rawe AM, Anderson JW, Boustany RM, Lerner TJ, Taschner PE, de Vos N, Breuning MH, Gardiner RM, Mole SE. Spectrum of mutations in the Batten disease gene, CLN3. Am J Hum Genet. 1997 Aug;61(2):310-6. doi: 10.1086/514846.
Drack AV, Mullins RF, Pfeifer WL, Augustine EF, Stasheff SF, Hong SD. Immunosuppressive Treatment for Retinal Degeneration in Juvenile Neuronal Ceroid Lipofuscinosis (Juvenile Batten Disease). Ophthalmic Genet. 2015;36(4):359-64. doi: 10.3109/13816810.2014.886271. Epub 2014 Feb 19.
Kwon JM, Adams H, Rothberg PG, Augustine EF, Marshall FJ, Deblieck EA, Vierhile A, Beck CA, Newhouse NJ, Cialone J, Levy E, Ramirez-Montealegre D, Dure LS, Rose KR, Mink JW. Quantifying physical decline in juvenile neuronal ceroid lipofuscinosis (Batten disease). Neurology. 2011 Nov 15;77(20):1801-7. doi: 10.1212/WNL.0b013e318237f649. Epub 2011 Oct 19.
Adams HR, Mink JW; University of Rochester Batten Center Study Group. Neurobehavioral features and natural history of juvenile neuronal ceroid lipofuscinosis (Batten disease). J Child Neurol. 2013 Sep;28(9):1128-36. doi: 10.1177/0883073813494813.
Ostergaard JR, Rasmussen TB, Molgaard H. Cardiac involvement in juvenile neuronal ceroid lipofuscinosis (Batten disease). Neurology. 2011 Apr 5;76(14):1245-51. doi: 10.1212/WNL.0b013e31821435bd.
Cotman SL, Vrbanac V, Lebel LA, Lee RL, Johnson KA, Donahue LR, Teed AM, Antonellis K, Bronson RT, Lerner TJ, MacDonald ME. Cln3(Deltaex7/8) knock-in mice with the common JNCL mutation exhibit progressive neurologic disease that begins before birth. Hum Mol Genet. 2002 Oct 15;11(22):2709-21. doi: 10.1093/hmg/11.22.2709.
Johnson TB, Brudvig JJ, Likhite S, Pratt MA, White KA, Cain JT, Booth CD, Timm DJ, Davis SS, Meyerink B, Pineda R, Dennys-Rivers C, Kaspar BK, Meyer K, Weimer JM. Early postnatal administration of an AAV9 gene therapy is safe and efficacious in CLN3 disease. Front Genet. 2023 Mar 24;14:1118649. doi: 10.3389/fgene.2023.1118649. eCollection 2023.
Other Identifiers
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CLN-301
Identifier Type: -
Identifier Source: org_study_id
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