G-CSF (filgrastim) treatment for amyotrophic lateral sclerosis: protocol for a phase II randomised, double-blind, placebo-controlled, parallel group, multicentre clinical study (STEMALS-II trial).
GCS-F
amyotrophic lateral sclerosis
haematopoietic stem cells
randomised clinical trial
Journal
BMJ open
ISSN: 2044-6055
Titre abrégé: BMJ Open
Pays: England
ID NLM: 101552874
Informations de publication
Date de publication:
24 03 2020
24 03 2020
Historique:
entrez:
27
3
2020
pubmed:
27
3
2020
medline:
14
4
2021
Statut:
epublish
Résumé
Amyotrophic lateral sclerosis (ALS) is a fatal progressive neurological disorder characterised by a selective degeneration of motor neurons (MNs). Stem cell transplantation is considered as a promising strategy in neurological disorders therapy and the possibility of inducing bone marrow cells (BMCs) to circulate in the peripheral blood is suggested to investigate stem cells migration in degenerated ALS nerve tissues where potentially repair MN damage. Granulocyte-colony stimulating factor (G-CSF) is a growth factor which stimulates haematopoietic progenitor cells, mobilises BMCs into injured brain and it is itself a neurotrophic factor for MN. G-CSF safety in humans has been demonstrated and many observations suggest that it may affect neural cells. Therefore, we decided to use G-CSF to mobilise BMCs into the peripheral circulation in patients with ALS, planning a clinical trial to evaluate the effect of G-CSF administration in ALS patients compared with placebo. STEMALS-II is a phase II multicentre, randomised double-blind, placebo-controlled, parallel group clinical trial on G-CSF (filgrastim) and mannitol in ALS patients. Specifically, we investigate safety, tolerability and efficacy of four repeated courses of intravenous G-CSF and mannitol administered in 76 ALS patients in comparison with placebo (indistinguishable glucose solution 5%). We determine increase of G-CSF levels in serum and cerebrospinal fluid as CD34 The study protocol was approved by the Ethics Committee of Azienda Ospedaliera Universitaria 'Città della Salute e della Scienza', Torino, Italy. Results will be presented during scientific symposia or published in scientific journals. Eudract 2014-002228-28.
Identifiants
pubmed: 32209625
pii: bmjopen-2019-034049
doi: 10.1136/bmjopen-2019-034049
pmc: PMC7202695
doi:
Substances chimiques
Filgrastim
PVI5M0M1GW
Banques de données
EudraCT
['2014-002228-28']
Types de publication
Clinical Trial Protocol
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
e034049Investigateurs
A Bombaci
(A)
M Brunetti
(M)
S Cammarosano
(S)
A Canosa
(A)
C Calvo
(C)
M Daviddi
(M)
G De Marco
(G)
P Cugnasco
(P)
M Grassano
(M)
B Iazzolino
(B)
A Ilardi
(A)
C Lauritano
(C)
A Lomartire
(A)
U Manera
(U)
L Solero
(L)
M C Torrieri R Vasta
(MC)
M Gilestro
(M)
V E Muccio P Omedé
(VE)
F Gerardi
(F)
C Cabona
(C)
G Novi
(G)
E Bersano
(E)
F De Marchi
(F)
R Spataro
(R)
R Scimè
(R)
A Fasano
(A)
N Fini
(N)
A Gessani
(A)
E D'Errico
(E)
A Scarafino
(A)
Informations de copyright
© Author(s) (or their employer(s)) 2020. Re-use permitted under CC BY-NC. No commercial re-use. See rights and permissions. Published by BMJ.
Déclaration de conflit d'intérêts
Competing interests: None declared.
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