-
Je něco špatně v tomto záznamu ?
Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration
T. Klopstock, A. Videnovic, AT. Bischoff, C. Bonnet, L. Cif, C. Comella, M. Correa-Vela, ML. Escolar, JL. Fraser, V. Gonzalez, N. Hermanowicz, R. Jech, HA. Jinnah, T. Kmiec, A. Lang, MJ. Martí, S. Mercimek-Andrews, M. Monduy, GAM. Nimmo, B....
Jazyk angličtina Země Spojené státy americké
Typ dokumentu časopisecké články, multicentrická studie, randomizované kontrolované studie, práce podpořená grantem
PubMed
33200489
DOI
10.1002/mds.28392
Knihovny.cz E-zdroje
- MeSH
- činnosti denního života MeSH
- dvojitá slepá metoda MeSH
- Hallervordenův-Spatzův syndrom * farmakoterapie genetika MeSH
- kyselina pantothenová analogy a deriváty MeSH
- lidé MeSH
- Check Tag
- lidé MeSH
- Publikační typ
- časopisecké články MeSH
- multicentrická studie MeSH
- práce podpořená grantem MeSH
- randomizované kontrolované studie MeSH
BACKGROUND: Pantothenate kinase-associated neurodegeneration (PKAN) currently has no approved treatments. OBJECTIVES: The Fosmetpantotenate Replacement Therapy pivotal trial examined whether treatment with fosmetpantotenate improves PKAN symptoms and stabilizes disease progression. METHODS: This randomized, double-blind, placebo-controlled, multicenter study evaluated fosmetpantotenate, 300 mg oral dose three times daily, versus placebo over a 24-week double-blind period. Patients with pathogenic variants of PANK2, aged 6 to 65 years, with a score ≥6 on the PKAN-Activities of Daily Living (PKAN-ADL) scale were enrolled. Patients were randomized to active (fosmetpantotenate) or placebo treatment, stratified by weight and age. The primary efficacy endpoint was change from baseline at week 24 in PKAN-ADL. RESULTS: Between July 23, 2017, and December 18, 2018, 84 patients were randomized (fosmetpantotenate: n = 41; placebo: n = 43); all 84 patients were included in the analyses. Six patients in the placebo group discontinued treatment; two had worsening dystonia, two had poor compliance, and two died of PKAN-related complications (aspiration during feeding and disease progression with respiratory failure, respectively). Fosmetpantotenate and placebo group PKAN-ADL mean (standard deviation) scores were 28.2 (11.4) and 27.4 (11.5) at baseline, respectively, and were 26.9 (12.5) and 24.5 (11.8) at week 24, respectively. The difference in least square mean (95% confidence interval) at week 24 between fosmetpantotenate and placebo was -0.09 (-1.69 to 1.51; P = 0.9115). The overall incidence of treatment-emergent serious adverse events was similar in the fosmetpantotenate (8/41; 19.5%) and placebo (6/43; 14.0%) groups. CONCLUSIONS: Treatment with fosmetpantotenate was safe but did not improve function assessed by the PKAN-ADL in patients with PKAN. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
Biostatistics Retrophin Inc San Diego California USA
Child Neurology Department Children's Memorial Health Institute Warsaw Poland
Department of Child Neurology Fondazione IRCCS Istituto Neurologico Carlo Besta Milan Italy
Department of Child Neurology Hospital Universitari Vall d'Hebron Barcelona Spain
Department of Child Neurology Oslo University Hospital Rikshospitalet Oslo Norway
Department of Neurology Children's National Medical Center Washington District of Columbia USA
Department of Neurology Columbia University College of Physicians and Surgeons New York New York USA
Department of Neurology Hospital Clinic of Barcelona Barcelona Spain
Department of Neurology Sorbonne University AP HP Salpêtrière Hospital Paris France
Department of Neurology University of California Irvine Irvine California USA
Department of Neurosurgery CHRU de Montpellier Gui de Chauliac Hospital Montpellier France
Department of Pediatrics University of Pittsburgh School of Medicine Pittsburgh Pennsylvania USA
Departments of Neurology and Human Genetics Emory University School of Medicine Atlanta Georgia USA
German Center for Neurodegenerative Diseases Munich Munich Germany
Munich Cluster for Systems Neurology Munich Munich Germany
Neurology Nicklaus Children's Hospital Miami Florida USA
Research and Development Retrophin Inc San Diego California USA
Citace poskytuje Crossref.org
- 000
- 00000naa a2200000 a 4500
- 001
- bmc21018530
- 003
- CZ-PrNML
- 005
- 20210830100122.0
- 007
- ta
- 008
- 210728s2021 xxu f 000 0|eng||
- 009
- AR
- 024 7_
- $a 10.1002/mds.28392 $2 doi
- 035 __
- $a (PubMed)33200489
- 040 __
- $a ABA008 $b cze $d ABA008 $e AACR2
- 041 0_
- $a eng
- 044 __
- $a xxu
- 100 1_
- $a Klopstock, Thomas $u Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany $u German Center for Neurodegenerative Diseases (DZNE), Munich, Munich, Germany $u Munich Cluster for Systems Neurology (SyNergy), Munich, Munich, Germany
- 245 10
- $a Fosmetpantotenate Randomized Controlled Trial in Pantothenate Kinase-Associated Neurodegeneration / $c T. Klopstock, A. Videnovic, AT. Bischoff, C. Bonnet, L. Cif, C. Comella, M. Correa-Vela, ML. Escolar, JL. Fraser, V. Gonzalez, N. Hermanowicz, R. Jech, HA. Jinnah, T. Kmiec, A. Lang, MJ. Martí, S. Mercimek-Andrews, M. Monduy, GAM. Nimmo, B. Perez-Dueñas, HCV. Pfeiffer, L. Planellas, E. Roze, N. Thakur, L. Tochen, N. Vanegas-Arroyave, G. Zorzi, C. Burns, F. Greblikas
- 520 9_
- $a BACKGROUND: Pantothenate kinase-associated neurodegeneration (PKAN) currently has no approved treatments. OBJECTIVES: The Fosmetpantotenate Replacement Therapy pivotal trial examined whether treatment with fosmetpantotenate improves PKAN symptoms and stabilizes disease progression. METHODS: This randomized, double-blind, placebo-controlled, multicenter study evaluated fosmetpantotenate, 300 mg oral dose three times daily, versus placebo over a 24-week double-blind period. Patients with pathogenic variants of PANK2, aged 6 to 65 years, with a score ≥6 on the PKAN-Activities of Daily Living (PKAN-ADL) scale were enrolled. Patients were randomized to active (fosmetpantotenate) or placebo treatment, stratified by weight and age. The primary efficacy endpoint was change from baseline at week 24 in PKAN-ADL. RESULTS: Between July 23, 2017, and December 18, 2018, 84 patients were randomized (fosmetpantotenate: n = 41; placebo: n = 43); all 84 patients were included in the analyses. Six patients in the placebo group discontinued treatment; two had worsening dystonia, two had poor compliance, and two died of PKAN-related complications (aspiration during feeding and disease progression with respiratory failure, respectively). Fosmetpantotenate and placebo group PKAN-ADL mean (standard deviation) scores were 28.2 (11.4) and 27.4 (11.5) at baseline, respectively, and were 26.9 (12.5) and 24.5 (11.8) at week 24, respectively. The difference in least square mean (95% confidence interval) at week 24 between fosmetpantotenate and placebo was -0.09 (-1.69 to 1.51; P = 0.9115). The overall incidence of treatment-emergent serious adverse events was similar in the fosmetpantotenate (8/41; 19.5%) and placebo (6/43; 14.0%) groups. CONCLUSIONS: Treatment with fosmetpantotenate was safe but did not improve function assessed by the PKAN-ADL in patients with PKAN. © 2020 The Authors. Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.
- 650 _2
- $a činnosti denního života $7 D000203
- 650 _2
- $a dvojitá slepá metoda $7 D004311
- 650 _2
- $a lidé $7 D006801
- 650 12
- $a Hallervordenův-Spatzův syndrom $x farmakoterapie $x genetika $7 D006211
- 650 _2
- $a kyselina pantothenová $x analogy a deriváty $7 D010205
- 655 _2
- $a časopisecké články $7 D016428
- 655 _2
- $a multicentrická studie $7 D016448
- 655 _2
- $a randomizované kontrolované studie $7 D016449
- 655 _2
- $a práce podpořená grantem $7 D013485
- 700 1_
- $a Videnovic, Aleksandar $u Department of Neurology, Massachusetts General Hospital/Harvard Medical School, Boston, Massachusetts, USA
- 700 1_
- $a Bischoff, Almut Turid $u Friedrich Baur Institute at the Department of Neurology, University Hospital, LMU Munich, Munich, Germany
- 700 1_
- $a Bonnet, Cecilia $u Department of Neurology, Sorbonne University, AP-HP Salpêtrière Hospital, Paris, France
- 700 1_
- $a Cif, Laura $u Department of Neurosurgery, CHRU de Montpellier, Gui de Chauliac Hospital, Montpellier, France
- 700 1_
- $a Comella, Cynthia $u Department of Neurosurgery and Neurological Sciences, Rush University Medical Center, Chicago, Illinois, USA
- 700 1_
- $a Correa-Vela, Marta $u Department of Child Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain
- 700 1_
- $a Escolar, Maria L $u Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pennsylvania, USA
- 700 1_
- $a Fraser, Jamie L $u Rare Disease Institute, Division of Genetics and Metabolism, Children's National Medical Center, Washington, District of Columbia, USA
- 700 1_
- $a Gonzalez, Victoria $u Department of Neurosurgery, University Hospital of Montpellier, Gui de Chauliac Hospital, Montpellier, France
- 700 1_
- $a Hermanowicz, Neal $u Department of Neurology, University of California Irvine, Irvine, California, USA
- 700 1_
- $a Jech, Robert $u Department of Neurology, First Faculty of Medicine, Charles University and General Faculty Hospital, Prague, Czech Republic
- 700 1_
- $a Jinnah, Hyder A $u Departments of Neurology and Human Genetics, Emory University School of Medicine, Atlanta, Georgia, USA
- 700 1_
- $a Kmiec, Tomasz $u Child Neurology Department, Children's Memorial Health Institute, Warsaw, Poland
- 700 1_
- $a Lang, Anthony $u Edmond J. Safra Program in Parkinson's Disease and the Department of Medicine (Neurology), Toronto Western Hospital and the University of Toronto, Toronto, Ontario, Canada
- 700 1_
- $a Martí, Maria J $u Movement Disorders Unit, Hospital Clinic of Barcelona, European Reference Network for Rare Neurological Diseases (ERN-RND), Centro de Investigación Biomédica en Red sobre Enfermedades Neurodegenerativas (CIBERNED. CB06/05/0018-ISCIII), Barcelona, Spain
- 700 1_
- $a Mercimek-Andrews, Saadet $u Division of Clinical and Metabolic Genetics, Department of Pediatrics, University of Toronto, The Hospital for Sick Children, Toronto, Ontario, Canada
- 700 1_
- $a Monduy, Migvis $u Neurology, Nicklaus Children's Hospital, Miami, Florida, USA
- 700 1_
- $a Nimmo, Graeme A M $u Division of Clinical and Metabolic Genetics, The Hospital for Sick Children, University of Toronto, Toronto, Ontario, Canada
- 700 1_
- $a Perez-Dueñas, Belen $u Department of Child Neurology, Hospital Universitari Vall d'Hebron, Barcelona, Spain
- 700 1_
- $a Pfeiffer, Helle Cecilie Viekilde $u Department of Child Neurology, Oslo University Hospital-Rikshospitalet, Oslo, Norway
- 700 1_
- $a Planellas, Lluis $u Department of Neurology, Hospital Clinic of Barcelona, Barcelona, Spain
- 700 1_
- $a Roze, Emmanuel $u Department of Neurology, Sorbonne University, AP-HP Salpêtrière Hospital, Brain and Spine Institute, Paris, France
- 700 1_
- $a Thakur, Nivedita $u Department of Pediatrics, Division of Child and Adolescent Neurology, University of Texas at Houston Medical School, Houston, Texas, USA
- 700 1_
- $a Tochen, Laura $u Department of Neurology, Children's National Medical Center, Washington, District of Columbia, USA
- 700 1_
- $a Vanegas-Arroyave, Nora $u Department of Neurology, Columbia University College of Physicians and Surgeons, New York, New York, USA
- 700 1_
- $a Zorzi, Giovanna $u Department of Child Neurology, Fondazione IRCCS Istituto Neurologico Carlo Besta, Milan, Italy
- 700 1_
- $a Burns, Colleen $u Biostatistics, Retrophin, Inc., San Diego, California, USA
- 700 1_
- $a Greblikas, Feriandas $u Research and Development, Retrophin, Inc., San Diego, California, USA
- 773 0_
- $w MED00003420 $t Movement disorders : official journal of the Movement Disorder Society $x 1531-8257 $g Roč. 36, č. 6 (2021), s. 1342-1352
- 856 41
- $u https://pubmed.ncbi.nlm.nih.gov/33200489 $y Pubmed
- 910 __
- $a ABA008 $b sig $c sign $y p $z 0
- 990 __
- $a 20210728 $b ABA008
- 991 __
- $a 20210830100122 $b ABA008
- 999 __
- $a ok $b bmc $g 1689585 $s 1138976
- BAS __
- $a 3
- BAS __
- $a PreBMC
- BMC __
- $a 2021 $b 36 $c 6 $d 1342-1352 $e 20201116 $i 1531-8257 $m Movement disorders $n Mov Disord $x MED00003420
- LZP __
- $a Pubmed-20210728
