Clinical Programs
Ceregene has two clinical-stage programs—one in Parkinson’s disease and one in Alzheimer’s disease.
We are currently preparing to initiate a new Phase 2 clinical trial with CERE-120 for Parkinson’s disease. For information on participation, see www.pdtrials.org or www.clinicaltrials.gov.
In Alzheimer's Disease (AD), we have initiated a Phase 2 clinical trial in collaboration with the Alzheimer's Disease Cooperative Study (ADCS) in patients with mild to moderate AD. For information on participation, see www.clinicaltrials.gov or www.adcs.org.
Clinical Programs at Ceregene
Phase 2 Clinical Trial in Parkinson's Disease (PD)
Enrollment in this study is anticipated to begin in August, 2010.
This study will evaluate whether the symptoms of PD can be improved by treating the brain cells, called neurons, that degenerate as PD progresses, using a neurotrophic factor called neurturin. Neurotrophic or growth factors can restore function and protect degenerating neurons from further damage. Neurturin does this for the neurons that are most impaired in PD, dopamine-containing neurons that control movement.
This study uses an approach called gene therapy to deliver neurturin, directly into the brain cells most affected by Parkinson's Disease. A single CERE-120 treatment is designed to express neurturin in the brain for many years. It is hoped that neurturin will provide long term improvement in the function of the neurons damaged by PD and thus improve movement and gait symptoms. It is also hoped that neurturin will repair some of the damaged cells and protect them from further injury, therefore reducing the rate of PD progression.
This study will help determine whether CERE-120 is effective in improving the symptoms of PD, as well as whether it continues to be safe when injected in the two areas of the brain most affected by PD (one where the dying brain cells originate, called the substantia nigra, and the other where the cell's axons terminate, called the putamen). If this study is successful, it will be instrumental in helping CERE-120 eventually gain approval as a treatment for PD patients.
This study is being conducted in 10 leading medical centers across the USA. Each center has strong expertise in Parkinson's Disease and neurosurgery. Fifty-two patients will be randomly assigned (like the flip of a coin) into one of two groups. One group will receive CERE-120 during a neurosurgical procedure and the other group will undergo a "sham" or mock surgery. Once patients have recovered from surgery, they will be asked to return to the clinic every three to six months for the next three years, to evaluate their condition. Except for the surgical team, no one will know who received CERE-120 until after the study results are available in late 2012. Those subjects who receive the sham surgery will be offered CERE-120 treatment after the conclusion of the clinical trial, provided the treatment is shown to be safe and effective. Patients will be allowed to continue taking their regular PD medicines during the study.
This is the fourth CERE-120 study in PD patients and it incorporates changes based on prior study results The earlier studies provided initial evidence supporting the safety of CERE-120, as well as important information that has helped us refine our approach to delivering CERE-120 (including injections into both the putamen and the substantia nigra, as well as a higher dose of CERE-120 than previously used). Dosing in the Phase 1 portion of this study, which involved 6 subjects, was completed in June 2010.
To be eligible for this study, subjects must:
- Have moderately advanced PD
- Be between 35-70 years of age
- Be in good general health and fit to undergo neurosurgery
- Have motor complications despite adequate PD medications (such as dyskinesia or wearing off)
Any of the following will exclude participants from study participation:
- Significant neurological disease other than PD
- Prior treatment with DBS or gene therapy
- Serious, chronic medical problems such as heart disease or uncontrolled diabetes.
- Major psychiatric disorders, or behavioral problems
- Alcohol or substance abuse within the last two years
- Systemic or brain cancer within the last three years
To learn how to participate, see www.pdtrials.org or www.clinicaltrials.gov
Phase 2 Clinical Trial in Alzheimer's Disease (AD)
This study being conducted in collaboration with the Alzheimer's Disease Cooperative Study (ADCS) and is currently enrolling participants.
This study will evaluate whether Ceregene's CERE-110 is a safe and effective treatment for Alzheimer's Disease (AD). CERE-110 employs gene therapy to deliver nerve growth factor (NGF) directly into the brain. NGF is known to promote survival of certain neurons, called cholinergic neurons, which degenerate in AD, and may therefore support sustained functioning of these neurons. Direct delivery of CERE-110 into the brain aims to selectively target the Nucleus Basalis of Meynert (NBM), where cholinergic neuronal degeneration occurs in AD.
A Phase 1 clinical trial conducted at Rush University in Chicago and the University of California San Diego indicated that a single administration of CERE-110 was generally safe and well tolerated. The Phase 1 participants underwent cognitive testing, measures of activities of daily living, MRI scans and PET (positron emission tomography) scans. Increases in brain metabolism were observed in several cortical regions of the brain at six months and 12 months after CERE-110 treatment in some of the subjects, as compared to other severity-matched individuals with AD, suggesting a potential reversal of patterns typically observed in AD.
The current Phase 2 clinical trial will examine the safety and effectiveness of NGF on Alzheimer's Disease in 50 patients at 11 research sites throughout the United States. All eligible participants will be randomized equally to one of two treatment groups. Half of the subjects will receive placebo surgery. In the other half, CERE-110 will be injected into the NBM, a region of the brain that contains the cells bodies for cholinergic neurons. At the completion of the clinical trial, subjects in the placebo arm will be given the opportunity to receive CERE-110 if the efficacy and safety data are supportive.
To be eligible for this study, subjects must:
- Have mild to moderate AD
- Be in good general health
- Be on standard of care medications (acetylcholinesterase inhibitors and/or memantine) for AD at least three months prior to entry.
- Have a reliable study partner to accompany all study visits
Any of the following will exclude participants from study participation:
- Significant neurological disease other than AD
- Major psychiatric disorders, or behavioral problems
- Alcohol or substance abuse within the last two years
- Systemic cancer within the last three yearss\
- Any significant systemic illness or serious unstable medical condition
Participants will be screened at the research sites to determine if they are eligible for the study
To learn how to participate, see www.clinicaltrials.gov or www.adcs.org.