Human clinical trials for NCL

NCL disease

Title

Treatment

Start date and Status

Sponsor

Country of operation

Clinical Trials number

Published report

CLN1

 

Cystagon to treat infantile neuronal ceroid lipofuscinosis

Cystagon

2001-2016

Active, not recruiting

National Institute of Child Health and Human Development

USA

NCT00028262

(Gavin et al., 2013)

CLN1

 

Safety and efficacy study of human central nervous system stem cells (HuCNS-SC) in subjects with neuronal ceroid lipofuscinosis

Stem cells

2010-2011

Withdrawn prior to enrollment

StemCells, Inc

USA

NCT01238315

 

-

CLN1 and CLN2

 

Study of human central nervous system stem cells (HuCNS-SC) cells in patients with infantile or late infantile neuronal ceroid lipofuscinosis (NCL)

Stem cells

2006-2009

Completed

StemCells Inc

USA

NCT00337636

(Selden et al., 2013)

CLN2

 

Safety study of a gene transfer vector for children with late infantile neuronal ceroid lipofuscinosis

AAV2CUhCLN2

2005-2019

Active, not recruiting

 

Weill Cornell Medical College

USA

NCT00151216

(Worgall et al., 2008)

CLN2

 

Safety study of a gene transfer vector (rh.10) for children with late infantile neuronal ceroid lipofuscinosis

AAVrh.10CUhCLN2

2010-2016

Recruiting

Weill Cornell Medical College

USA

NCT01161576

-

CLN2

 

AAVrh.10 administered to children with late infantile neuronal ceroid lipofuscinosis with uncommon genotypes or moderate/severe impairment

AAVrh.10CUhCLN2

2010-2014

Recruiting

Weill Cornell Medical College

USA

NCT01414985

 

-

CLN2

Safety and Efficacy Study of BMN190 for the Treatment of CLN2 Patients

rhTPP1 BMN190

2013-2016

Recruiting

Biomarin Pharmaceutical

Germany, UK

NCT01907087

-

CLN3

 

Cellcept for treatment of juvenile neuronal ceroid lipofuscinosis

Mycophenolate mofetil

2011-2015

Recruiting

University of

Rochester

USA

NCT01399047

 

-

Gavin M, Wen GY, Messing J et al. (2013). Substrate Reduction Therapy in Four Patients with Milder CLN1 Mutations and Juvenile-Onset Batten Disease Using Cysteamine Bitartrate. JIMD Rep 11, 87-92.

Selden NR, Al-Uzri A, Huhn SL et al. (2013). Central nervous system stem cell transplantation for children with neuronal ceroid lipofuscinosis. J Neurosurg Pediatr 11, 643-52.

Worgall S, Sondhi D, Hackett NR et al. (2008). Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA. Hum Gene Ther 19, 463-74.