The main purpose of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 to participants with Parkinson’s disease.
The General University Hospital of Catalonia is the first center in Spain that has been activated for a new and particular phase I trial in Parkinson’s (REASON), which seeks to cure the disease. The trial, which has already begun in the USA, involves the administration of an intrathecal injection – fluid-filled space between the thin layers of tissue covering the brain and spinal cord – with a dose of an antisense oligonucleotide, which avoids the signal that activates a gene called LRRK2, known as “the Basque mutation” or Parkina 8, with the idea of inhibiting the formation of the alpha-synuclein protein.
Parkinson’s disease, a neurodegenerative disorder of unknown cause, which causes resting tremor, slowness of movement and stiffness, is caused by a dopamine deficiency at the basal ganglia and is characterized by the presence of this anomalous protein that is the alpha-synuclein.
This is a brilliant experiment to try to modify the natural evolution of parkinson’s.
The main purpose of this study is to evaluate the safety and tolerability of single and multiple doses of BIIB094 to participants with Parkinson’s disease. The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB094. The study is open to patients with Parkinson’s disease with verified presence or absence of mutations in the leucine-rich repeat kinase 2 gene (LRRK2), but also for patients without any verified genetic mutations related to the disease as it is postulated that this gene will also be over-expressed in all patients with Parkinson’s and not only the LRRK2 mutants. In the latter patients the progression of the disease would be avoided while in the former it could be avoided.
For Ernest Balaguer, director of research at the HUGC and head of the Clinical Research Unit (UDIC), this is a “brilliant experiment to try to modify the natural evolution of parkinson’s, both for patients who have this” basque mutation “and those who they do not have it, and it could prevent the progression of the disease. “The clinical trial appears on www.clinicaltrials.gov and will soon appear the General University Hospital of Catalonia, as well as on the Michel J Fox Foundation Trial Finder.
The Hospital develops several lines of research through the UDIC, led by Dr. Ernest Balaguer. It is noteworthy that of the last seven anti-Parkinson’s drugs that have been marketed in Spain, all of them have been evaluated at the General Hospital, because they have a great experience in these drugs
We have reached the first position, in December 2019, in the international inclusion of patients in the trial with sublingual apomorphine and motor blocks in Parkinson.
We are also leading the national inclusion of patients in the Clarity AD trial (monoclonal anti-amyloid antibody in early Alzheimer)
Finally, in the Graduate trial (monoclonal subcutaneous in Alzheimer) we are the second recruitment site in our country.
On October 22, 2019, Biogen announced the positive result of its EMERGE clinical trial (in which the UDIC has participated) that has evaluated Aducanumab (an anti-alpha synuclein monoclonal antibody) in early Alzheimer’s disease. Efficacy in slowing cognitive impairment was demonstrated (measured with the CDR-SB, MMSE, ADAS-cog and ADCS-ADL scales) and a reduction in amyloid load detected in PET (almost 80%). The most frequent side effects were cerebral edema (asymptomatic and transient, in general, detected in brain MRI) and headache.These data suport the amyloid hypothesis in this disease and a change in its diagnostic and therapeutic paradigm.
We are developping a clinical trial that evaluates the efficacy and safety of an oral medication that inhibits an enzyme released by a dental germ in patients with moderate Alzheimer’s disease.
The GAIN trial (code COR388-010) is a phase III clinical trial and we are carrying out in Spain, in the General University Hospital of Catalonia, with the collaboration between the services of neurology, neuropsychology, neuroradiology and odontology, coordinated by Dr. Balaguer, neurologist and director of research.
– Dr. Balaguer, scientific director of the UDIC, has directed the course of Coordination and Practical Management of clinical trials in Neurology (module from 5:30 hours). Completed during the LXX Annual Meeting of the Spanish Society of Neurology (Seville, 24 November 2018).
– Continue the development of our projects in Parkinson’s disease: anti-alfasinuclein monoclonal antibodies, gastric-retention levodopa, antidiscinetic drug in advanced Parkinson’s, new drug against hallucinations and delusions in Parkinson’s disease dementia.
– UDIC is performing several clinical trials in Alzheimer’s disease: oral drug acting like inhibitor of amyloid, anti-amyloid monoclonal antibodies (intravenously and subcutaneously), neuromodulator drug in moderated Alzheimer’s disease.
– Many patients still are taking part in a study in Progressive Supranuclear Paralysis with an anti-tau monoclonal antibodies.
The Hospital Universitari General de Catalunya appoints Dr. Balaguer as the new Research Director on June 12, 2013.
Drugs authorized in our country or approved by the FDA, which were evaluated by the UDIC: Rivastigmine (Exelon), Entacapone (Comtan), Rotigotine (Neupro), Pramipexol ER (Mirapexin LP), Ropinirol ER (Requip LP) Safinamide (Xadago) , Opicapona (Ongentys), Pimavanserin (Nuplazid), CVT 301 (..)
Visit of Dr. Chris Kenney: The Medical Director of ACORDA, holder of CVT 301 (Levodopa inhaled) and Tozadenant came to our facilities during his visit to our country. The UDIC was a pioneer and main recruiter of patients in both clinical trials.
Audits and Inspections:
Random inspection FDA: In 2009 our Unit got over unqualified a random inspection of Spanish centers that had participated in a clinical trial in Parkinson’s disease.
The UDIC has passed frequent national and international audits, carried out in general due to its high rate of patient inclusion. During the year 2017, international audits of one trial in Parkinson’s disease and another in Alzheimer’s disease were developed, with satisfactory results.
– Our Unit has been the first center in our country to start two new trials: antidiscinetic in phase II in Parkinson’s, and Alzheimer’s (muscarinic agonist, phase Ib).
– We are the first center, with the largest number of patients included, in the new trial in Progressive Supranuclear Paralysis
– First Spanish recruiting center in a trial with a new drug in Alzheimer’s.
The prestigious scientific journal NATURE has published a Phase II trial with a monoclonal anti-amyloid antibody (aducanumab) in Alzheimer’s disease, with a positive result. The UDIC participates in the development of Phase III of this project, in the EMERGE trial.
-Trial of Solanezumab for Mild Dementia Due to Alzheimer’s Disease.
Lawrence S.H onig, Bruno Vellas, Michael Woodward, Mercè Boada et al.
N Engl J Med 2018; 378:321-330. January 25, 2018
–Dr Balaguer en La Vanguardia.
The scientific community continues to work in the search for solutions to Alzheimer’s. Researchers shed a light of hope at the negative result of the large clinical trial with solanezumab