Plain English Summary
Background and study aims
Parkinson’s disease (PD) is a chronic condition where nerve cells in a small part of the brain called the substantia nigra become damaged and die. The nerve cells in this region send signals that controls the muscles of the body. Dopamine is the main neurotransmitter produced by these nerve cells. As more of these cells die, the amount of dopamine produced also falls. Over time, the lack of nerve cells and low levels of dopamine affects how well the person affected can control their muscles. The most common symptoms of the condition are slowness of movement, muscle stiffness and shaking (tremors). Although temporarily effective, current treatments fail to control symptoms and stop the disease progressing. The aim of this study was to test the safety and possible benefits of a novel strategy based on grafting human fetal brain stem cells (hfSCs) I the hope that they help address both dopamine and non-dopamine aspects of the disease.
Who can participate?
Patients with moderate to severe Parkinson’s disease.
What does the study involve?
Participants are first temporarily immunosuppressed with the drug cyclosporine. This is to make sure that they don’t reject the stem cells. The cells are then injected into a part of the brain called the dorsal putamina. All participants are then followed up for a year to see if any side effects or complications arise and whether their symptoms improve.
What are the possible benefits and risks of participating?
Benefits may include an improvement in PD symptoms. Potential risks include bleeding in the brain, brain swelling and immune rejection of the cells.
Where is the study run from?
Angeles Pedregal Hospital (Mexico)
When is the study starting and how long is it expected to run for?
February 2011 to August 2018
Who is funding the study?
Celavie Biosciences
Who is the main contact?
Professor Ignacio Madrazo
imadrazon@parkinsonmx.com
Trial website
Contact information
Type
Scientific
Primary contact
Prof Ignacio Madrazo
ORCID ID
http://orcid.org/0000-0002-1181-3029
Contact details
Camino a Santa Teresa 1055-474
Mexico City
10700
Mexico
52555684319
imadrazon@parkinsonmx.com
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
CMN2012-027
Study information
Scientific title
Undifferentiated human fetal brain-derived stem cells grafted into putamina of parkinsonian patients is safe and moderately effective: a phase I clinical trial.
Acronym
Study hypothesis
If undifferentiated human fetal brain-derived stem cells (hfSC) are transplanted in the putamina, then patients will Parkinson's Disease will not suffer harm and will decrease their disease.
Ethics approval
1. Federal Commission for Prevention of Sanitary Risks (Comisión Federal para la Prevensión de Riesgos Sanitarios), 12/02/2014, ref: CMN2012-027
2. Research Committee and Research Ethics Operadora Hospital (Comité de de Investigación y Ética en Investigación de Operadora de Hospitales Ángeles S.A. de C.V), 01/08/2014
Study design
Longitudinal, prospective, interventional, uncontrolled study
Primary study design
Interventional
Secondary study design
Randomised controlled trial
Trial setting
Hospitals
Trial type
Treatment
Patient information sheet
Not available in web format, please use the contact details below to request a patient information sheet
Condition
Parkinson's Disease
Intervention
Stereotactical transplant of stem cells into both putamina. One million million stem cells were deposited in two areas (anterior and posterior) of both putamina. All of this is by one trephine in the coronal suture.
The patients were protected 24 hours before surgery with IV antibiotics, and a day before started with cyclosporine and indometacine which lasted for one month in the first drug and two months in the second. After surgery, they were transferred to the ICU for 24 hours and finally after a post-op MRI control, sent home.
Intervention type
Procedure/Surgery
Phase
Drug names
Primary outcome measure
1. Safety of transplantation of cells, measured by recording side effects or complications arising after surgery.
2. Degree of motor improvement, measured using the unified Parkinson's disease rating scale (UPDRS) score
Secondary outcome measures
1. Cognitive performance, measured using the mini–mental state examination (MMSE) score at baseline and at 1 year follow-up
2. Uptake patterns of DTBZ, FDOPA and RAC
Overall trial start date
18/02/2011
Overall trial end date
25/08/2018
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
Healthy patient with Parkinson's Disease
Participant type
Patient
Age group
All
Gender
Both
Target number of participants
8
Total final enrolment
8
Participant exclusion criteria
1. Pregnancy
2. Secondary pathology
Recruitment start date
01/06/2011
Recruitment end date
14/11/2012
Locations
Countries of recruitment
Mexico
Trial participating centre
Angeles Pedregal Hospital
Camino a Santa Teresa 1055
Mexico City
10700
Mexico
Funders
Funder type
Industry
Funder name
Celavie Biosciences
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Planned publication in a high-impact peer-reviewed journal.
IPD sharing statement: we intend to share participant level data in Dryad, however the details are still being decided.
Intention to publish date
31/12/2018
Participant level data
Stored in repository
Basic results (scientific)
See additional files (ISRCTN39104513_BasicResults_1Oct18.pdf and http://www.isrctn.com/ISRCTN39104513)
Publication list
2019 results in: https://www.ncbi.nlm.nih.gov/pubmed/30574805 (added 08/08/2019)
Publication citations
Additional files
- ISRCTN39104513_BasicResults_1Oct18.pdf Uploaded 01/10/2018
- ISRCTN39104513_BasicResults_version 2_4Oct18.pdf Uploaded 04/10/2018