From: Moving towards effective therapeutic strategies for Neuronal Ceroid Lipofuscinosis
Trial ID | NCL Form(s) | Therapeutic Approach | Proposed Mechanism of Action | Preclinical Studies | Trial Phase |
---|---|---|---|---|---|
NCT01399047 | JNCL |
Anti-inflammatory
Mycophenolate mofetil | Reduction in neuroinflammation and production of autoantibodies | Seehafer 2011 [144] | Recruiting |
NCT01161576 | LINCL |
Gene Therapy
AAVrh.10CUhCLN2 | Genetically engineer cells to produce non-mutated TPP1 | Sondhi 2007 [41], Sondhi 2008, Sondhi 2012 | Recruiting |
NCT01414985 | LINCL |
Gene Therapy
AAVrh.10CUhCLN2 | Genetically engineer cells to produce non-mutated TPP1 | Sondhi 2007 [41], Sondhi 2008 [42], Sondhi 2012 [43] | Recruiting |
NCT01907087 | LINCL |
ERT
BMN-190 | Source of recombinant functional TPP1 in which diseased cell can uptake and utilize | Vuillemenot 2014 [58], Vuillemenot 2014 [59] | Active |
NCT00151216 | LINCL |
Gene Therapy
AAV2CUhCLN2 | Genetically engineer cells to produce non-mutated TPP1 | Sondhi 2005 [39], Passini 2006 [40] | Active |
NCT00337636 | INCL, LINCL |
Stem Cell
Human CNS Stem cells | Similar to ERT but, human CNS stem cells act as the source of functional PPT1 and TPP1 | Tamaki 2009 [85] | Complete |
NCT00028262 | INCL |
Small Molecule
Cystagon | Clears lysosome of storage material | Zhang 2001 [174] | Complete |
NCT01238315 | INCL, LINCL |
Stem Cell
Human CNS Stem cells | Similar to ERT but, human CNS stem cells act the source of functional PPT1 and TPP1 | Tamaki 2009 [85], Selden 2013 [91] | Withdrawn |