PRO-51 Phase II/III Trial
http://www.prosensa.eu/news/TREAT-NMD_Prosensa.pdf
Prosensa and TREAT-NMD enter into strategic collaboration
for PRO-051 clinical trial planning
Leiden, August 31, 2009 – Prosensa, the Dutch based biopharmaceutical
company focusing on RNA modulating therapeutics, announces the successful
completion of a feasibility enquiry using the TREAT-NMD Global Database for
DMD and the TREAT-NMD Care and Trial Sites Registry for the planning of the
phase II/III study for its lead compound PRO-051.
Prosensa recently completed a phase I/II clinical trial for PRO-051, its lead compound
for the treatment of Duchenne Muscular Dystrophy (DMD), and the company
anticipates starting a phase II/III clinical study early next year. PRO-051 is directed to a
specific mutation in the dystrophin gene that occurs in approximately 13% of the DMD
patient population. In order to set up the pivotal study, patients with a genetic mutation
amenable to exon skipping by PRO-051 need to be selected.
TREAT-NMD has developed a global database that contains precise genetic and
clinical information from patients with DMD, including age, ambulation status and
medication use. Using the TREAT-NMD Global Database for DMD, Prosensa has
identified around 300 patients from 21 countries who meet the inclusion criteria for the
upcoming trial. The TREAT-NMD database holds up-to-date information about all these
patients that will allow them to be contacted for trial recruitment purposes through the
national registries. These patients were matched to 50 potential trial sites and selected
patients and sites will be contacted for participation in the upcoming clinical trial.
“Patient recruitment for clinical studies is a very expensive and time-consuming
process, in particular for rare diseases such as DMD” comments Dr. Giles Campion,
CMO and VP R&D of Prosensa. “Therefore, this successful collaboration with TREATNMD
is extremely valuable for us as it facilitates the acceleration of the recruitment
process and hopefully allows us to bring this treatment faster to patients.”
“We are delighted that our global patient database is providing such useful results for
pharmaceutical companies like Prosensa,” said Professor Hanns Lochmüller, leader of
the TREAT-NMD patient registries initiative. “Our approach is unique because the data
we are giving companies isn’t just generic statistical information. It is up-to-date
information about real patients who can be recruited into clinical trials through the
registry or contacted when a treatment is available. At the same time, it’s safe for
patients, since we don’t give out identifiable information to companies but act as a
trusted intermediary and all our registries have full ethical approval and comply with
data protection laws.”
Prosensa and TREAT-NMD will both be attending and presenting at the World Muscle
Society Conference in Geneva from September 9th till 12th.
Prosensa and TREAT-NMD enter into strategic collaboration
for PRO-051 clinical trial planning
Leiden, August 31, 2009 – Prosensa, the Dutch based biopharmaceutical
company focusing on RNA modulating therapeutics, announces the successful
completion of a feasibility enquiry using the TREAT-NMD Global Database for
DMD and the TREAT-NMD Care and Trial Sites Registry for the planning of the
phase II/III study for its lead compound PRO-051.
Prosensa recently completed a phase I/II clinical trial for PRO-051, its lead compound
for the treatment of Duchenne Muscular Dystrophy (DMD), and the company
anticipates starting a phase II/III clinical study early next year. PRO-051 is directed to a
specific mutation in the dystrophin gene that occurs in approximately 13% of the DMD
patient population. In order to set up the pivotal study, patients with a genetic mutation
amenable to exon skipping by PRO-051 need to be selected.
TREAT-NMD has developed a global database that contains precise genetic and
clinical information from patients with DMD, including age, ambulation status and
medication use. Using the TREAT-NMD Global Database for DMD, Prosensa has
identified around 300 patients from 21 countries who meet the inclusion criteria for the
upcoming trial. The TREAT-NMD database holds up-to-date information about all these
patients that will allow them to be contacted for trial recruitment purposes through the
national registries. These patients were matched to 50 potential trial sites and selected
patients and sites will be contacted for participation in the upcoming clinical trial.
“Patient recruitment for clinical studies is a very expensive and time-consuming
process, in particular for rare diseases such as DMD” comments Dr. Giles Campion,
CMO and VP R&D of Prosensa. “Therefore, this successful collaboration with TREATNMD
is extremely valuable for us as it facilitates the acceleration of the recruitment
process and hopefully allows us to bring this treatment faster to patients.”
“We are delighted that our global patient database is providing such useful results for
pharmaceutical companies like Prosensa,” said Professor Hanns Lochmüller, leader of
the TREAT-NMD patient registries initiative. “Our approach is unique because the data
we are giving companies isn’t just generic statistical information. It is up-to-date
information about real patients who can be recruited into clinical trials through the
registry or contacted when a treatment is available. At the same time, it’s safe for
patients, since we don’t give out identifiable information to companies but act as a
trusted intermediary and all our registries have full ethical approval and comply with
data protection laws.”
Prosensa and TREAT-NMD will both be attending and presenting at the World Muscle
Society Conference in Geneva from September 9th till 12th.
Replies to This Discussion
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Permalink Reply by cheryl cliff on
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Beautiful!!!
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Permalink Reply by JUAN PEDRO ARBULU on
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from heaven!!
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Permalink Reply by Terry Porcaro on
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To be part of the TREAT-NMD global database, do you just have to register with Duchenne Connect, or is there another registration needed ????
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Hi Terry,
If you go to Treatnmd.org you will see they have their own patient registry. I don't know if they are using Duchenne Connect, wouldn't be surprised if they will, but best to be safe and register at treatnmd.org in case they don't.
Terry Porcaro said:To be part of the TREAT-NMD global database, do you just have to register with Duchenne Connect, or is there another registration needed ????
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Permalink Reply by Laurie Paschal on
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If I remember correctly from the conference, TREAT-NMD does pull from Duchene Connect.
Terry Porcaro said:To be part of the TREAT-NMD global database, do you just have to register with Duchenne Connect, or is there another registration needed ????
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Permalink Reply by Jerry Dallapè on
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Sounds like there will likely be some trial sites in the U.S.
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Gawd, I hope so!
Jerry Dallapè said:Sounds like there will likely be some trial sites in the U.S.
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Permalink Reply by jenn on
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Ofelia,Should we wait to hear from someone, or is there someone we should call to get our boys into this? I thought part of traveling to "the best doctors" meant our sons had an advantage when these trials arrived, is this true? ( I totally trust your opinions, you do your homework!)
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Permalink Reply by Ofelia Marin on
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Hi Jenn,
I would definitely ask your neuro (Dr. Wong ?) about it. I think the plan is for Prosensa to select trial sites (using Treat-NMD's help) and then trial sites contact patients (similar to what was done with PTC124). Question is how many sites will be in the US and where. Since Columbus will work with AVI I assume they will not be one of those sites...maybe Cincinnati is. I would email Dr. Wong to ask.
Ofelia
jenn said:Ofelia,Should we wait to hear from someone, or is there someone we should call to get our boys into this? I thought part of traveling to "the best doctors" meant our sons had an advantage when these trials arrived, is this true? ( I totally trust your opinions, you do your homework!)
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You did remember correctly Laurie, I just received an email from Prosensa confirming they will use DuchenneConnect for sites selected in USA.
Laurie Paschal said:If I remember correctly from the conference, TREAT-NMD does pull from Duchene Connect.
Terry Porcaro said:To be part of the TREAT-NMD global database, do you just have to register with Duchenne Connect, or is there another registration needed ????
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Permalink Reply by irishgirl on
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Make sure you child is on each and every DMD register. They begin by looking at possible candidates via DMD registries. Keep you information updated on a regular basis. BUT don't just sit back and expect someone to contact you. If and when you see a trial you'd like to be apart of, make your presence known - call, email - do what you need to do. Start with the Duchenne Connect registry if you like, but if you are not registered - do so. Everything helps!
jenn said:Ofelia,Should we wait to hear from someone, or is there someone we should call to get our boys into this? I thought part of traveling to "the best doctors" meant our sons had an advantage when these trials arrived, is this true? ( I totally trust your opinions, you do your homework!)
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Also register on this site as well...
https://dystrophy.genetics.utah.edu/
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