Download phase 3 trials in multiple sclerosis - National Multiple Sclerosis Society

PHASE 3 TRIALS IN MULTIPLE SCLEROSIS
Updated December 2016. This list highlights notable Phase 3 trials but is not inclusive of all studies.
Abbreviations Key:
ECTRIMS – European Committee of Treatment and Research in MS
IV – IV, injected into vein
PP - primary progressive
RR - relapsing-remitting
SC – SC, injected under the skin
SP - secondary progressive
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
ALKS 8700
(monomethyl
fumarate,
Alkermes plc)
test long-term
safety and
tolerability
Upregulates Th2
response, immunemodulatory
oral
RR/600
Recruiting, read more
https://clinicaltrials.gov/ct2/show/NCT02634307
BG00012
(dimethyl
fumarate,
Tecfidera®,
Biogen)
test
effectiveness
and safety in
children
Upregulates Th2
response, immunemodulatory
oral
RR/172
Recruiting, read more
https://clinicaltrials.gov/ct2/show/NCT02428218
Daclizumab High
Yield Process
(DAC HYP,
Biogen and
Abbvie)
test use of a
prefilled
syringe, also
known as the
OBSERVE
study
Limits T cell
expansion by
blocking signaling
of cytokine IL-2
SC
RR/100
Ongoing, not recruiting; read more
http://clinicaltrials.gov/ct2/show/NCT01462318
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
Fingolimod
(Gilenya®,
Novartis AG)
control
disease in
children
Retaining certain
white blood cells in
the lymph nodes,
thereby preventing
those cells from
crossing the bloodbrain barrier into the
central nervous
system
oral
10-17
years/190
Recruiting, read more:
http://clinicaltrials.gov/show/NCT01892722
Fingolimod
(Gilenya®,
Novartis AG)
test safety
and
effectiveness,
also known as
INFORMS
study
Sphingosine-1phosphate receptor
binder; prevents
lymphocytes from
exiting lymphatic
tissue
oral
PP/970
Completed; did not slow MS progression over three
years; read more
http://www.nationalmssociety.org/About-theSociety/News/Disappointing-Results-AnnouncedFrom-Trial-of-Gile
Hematopoietic
stem cell therapy
control
disease
progression
IV
RR, failing
alternate
therapy/110
Recruiting, read more:
http://clinicaltrials.gov/show/NCT00273364
oral
RR/2199
Ongoing, not recruiting, read more
http://clinicaltrials.gov/ct2/show/NCT01707992.
(Higher dose discontinued due to occurrence of
cardiovascular events, per company press release,
January 4, 2016.)
Laquinimod
(Teva
Pharmaceutical
Industries and
Active Biotech)
Autologous
hematopoietic
(blood cellproducing) stem cell
transplantation to
"reboot" immune
system
reduce time to Immunomodulatory
confirmed
disease
progression
on EDSS,
also known as
CONCERTO
study
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
Masitinib (AB
Science)
determine
safety and
effectiveness
in reducing
disease
activity
determine
effectiveness
in reducing
disability
progression
Inhibits the survival,
migration and
activity of mast cells
oral
SP,PP/450
Recruiting, read more
http://clinicaltrials.gov/ct2/show/NCT01433497
activates main route
for energy
production that
protects against
axonal
degeneration
Closes damaged
blood-brain barrier,
reducing
inflammation in
CNS
oral
SP,PP/300
Not yet recruiting, read more
https://www.clinicaltrials.gov/ct2/show/NCT02936037
oral, IV
RR/199
Completed; oral administration not inferior to
intravenous for improvement of disability scores 1
month after treatment and had similar safety profile.
Read more
http://www.ncbi.nlm.nih.gov/pubmed/26135706
Interferes with
movement of
immune cells
across the bloodbrain barrier by
attaching to alpha
4-integrin
intravenous SP/856
MD1003 (biotin,
MedDay
Pharmaceuticals
SA)
Methylprednisolone
Natalizumab
(Tysabri®,
Biogen)
compare oral
versus
intravenous
delivery to
control
development
of brain
lesions and
treat disease
relapses, also
known as
COPOUSEP
study
determine
effectiveness
in reducing
disability
progression,
also known as
ASCEND
study
Terminated; did not achieve statistical significance
on primary or secondary endpoints. Read more
http://clinicaltrials.gov/ct2/show/NCT01416181
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
Ocrelizumab
(Genentech, a
member of the
Roche Group)
vs. interferon
beta-1a (Rebif®,
EMD Serono)
evaluate
safety and
effectiveness
in reducing
disease
activity, also
known as
OPERA I and
OPERA II
study
Ocrelizumab
(Genentech, a
member of the
Roche Group)
Ofatumumab
(Novartis
Pharmaceuticals)
vs. teriflunomide
(Aubagio®,
Sanofi Genzyme)
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
Binds to CD20
IV, SC
antigen on B cells
and induces B-cell
lysis
(ocrelizumab)/Slows
down immune
response, possibly
by interfering with T
cell activation and
movement across
blood-brain barrier,
and inducing
suppressive T cells
(Rebif)
RR/1656
Completed; significantly reduced annualized relapse
rate compared with Rebif over 2 years (ECTRIMS
2015, Abstract #190).
evaluate time
to disease
progression,
also known as
ORATORIO
study
Binds to CD20
antigen on B cells
and induces B-cell
lysis
IV
PP/732
Completed; slowed progression of disability
compared to placebo. Read more
http://www.nationalmssociety.org/About-theSociety/News/More-Results-Released-from-PositivePhase-III-Clin
comparing the
effectiveness
and safety,
also known as
ASCLEPIOS I
study
Fully human
monoclonal
antibody which
targets CD20 and
works by inducing B
cell depletion in
lymphatic tissues
SC, oral
Relapsing/900 Recruiting, read more
https://www.clinicaltrials.gov/ct2/show/NCT02792218
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
Ozanimod
(Receptos, Inc.,
formerly RPC1063)
control lesion
development,
disease
progression
and relapses,
also known as
RADIANCE
study
Retaining certain
white blood cells in
the lymph nodes,
thereby preventing
those cells from
crossing the bloodbrain barrier into the
central nervous
system
oral
RR/1200
Ongoing, not recruiting. Read more:
http://clinicaltrials.gov/show/NCT02047734
Ponesimod
(Actelion) vs.
teriflunomide
(Aubagio®,
Sanofi Genzyme)
reduce
relapses
Retaining certain
white blood cells in
the lymph nodes,
thereby preventing
those cells from
crossing the bloodbrain barrier into the
central nervous
system
oral
RR/1100
Recruiting, read more
https://clinicaltrials.gov/ct2/show/NCT02425644
Siponimod
evaluate
safety and
effectiveness
Sphingosine-1phosphate receptor
binder; prevents
lymphocytes from
exiting lymphatic
tissue
oral
SP, 1530
Recruiting, read more:
http://clinicaltrials.gov/ct2/show/NCT01665144
Teriflunomide
(Aubagio®,
Sanofi Genzyme)
control
disease in
children, also
known as
TERIKIDS
study
Modulates
responses of T-cells
within the immune
system by impairing
DNA synthesis
oral
Relapsing,
10-17 years
old/165
Recruiting, read more:
http://clinicaltrials.gov/show/NCT02201108
AGENT
PURPOSE
OF STUDY
POSSIBLE
MECHANISM
Vitamin D +
glatiramer
acetate
(Copaxone®,
Teva
Pharmaceutical
Industries, Ltd.)
Supports immune
test safety
function
and
effectiveness
in reducing
disease
activity
HOW
THERAPY
IS GIVEN
TYPE OF
MS/NUMBER
OF
SUBJECTS
STATUS/RESULTS
oral, SC
RR/172
Recruiting; read more
http://clinicaltrials.gov/ct2/show/NCT01490502