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Clinical Trial to Evaluate Bone Marrow Stem Cell Therapy for PSP, a Rare Form of Parkinsonism

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ClinicalTrials.gov Identifier: NCT01824121
Recruitment Status : Unknown
Verified March 2013 by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico.
Recruitment status was:  Recruiting
First Posted : April 4, 2013
Last Update Posted : April 4, 2013
Sponsor:
Information provided by (Responsible Party):
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Tracking Information
First Submitted Date  ICMJE March 31, 2013
First Posted Date  ICMJE April 4, 2013
Last Update Posted Date April 4, 2013
Study Start Date  ICMJE December 2012
Estimated Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 31, 2013)
incidence of adverse events [ Time Frame: one year ]
incidence of adverse events collected by clinical monitoring and performing routine laboratory tests
Original Primary Outcome Measures  ICMJE Same as current
Change History No Changes Posted
Current Secondary Outcome Measures  ICMJE
 (submitted: March 31, 2013)
changes in brain images [ Time Frame: one year ]
change vs baseline in the striatal density of dopamine transporters in SPECT brain images and in the normalized regional cerebral flow / glucose metabolism in the gray matter in PET brain images after one year
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures
 (submitted: March 31, 2013)
  • changes in motor function [ Time Frame: one year ]
    changes vs baseline in total and motor UPDRS scores, Hoehn & Yahr staging, SEADL score, CGI and multifactorial movement analysis
  • changes in cognitive functions [ Time Frame: one year ]
    changes vs baseline in MMSE score and in a series of neuropsychological measures (verbal comprehension, perceptual organization, immediate memory, delayed memory, word list recognition, language attention / concentration, visuospatial ability, processing speed, executive function)
Original Other Pre-specified Outcome Measures Same as current
 
Descriptive Information
Brief Title  ICMJE Clinical Trial to Evaluate Bone Marrow Stem Cell Therapy for PSP, a Rare Form of Parkinsonism
Official Title  ICMJE Autologous Mesenchymal Stem Cell Therapy in Progressive Supranuclear Palsy: a Randomized, Double-blind, Controlled Clinical Trial
Brief Summary

There is evidence suggesting that stem cells harvested from the bone marrow and transplanted into the brain may be effective in slowing down the progression of parkinsonism. Mesenchymal stem cells are able to produce growth factors that provide support to diseased nervous cells.

In this study mesenchymal stem cells will be harvested from the bone marrow, cultivated in a test tube so that they multiply and then infused into the arteries that supply blood to the brain in 20 patients suffering from a rare form of parkinsonism, Progressive Supranuclear Palsy. Each patient will undergo two infusions, one with the stem cells and one without, at an interval of 6 months. The sequence of the two infusions will be assigned randomly; patients and assessors will not know the sequence (double-blind). Patients will be followed-up for up to 1 year after the last infusion, with regular assessments to assess safety, efficacy on motor and cognitive functions, and effects on the brain by neuroimaging techniques.

The study has a preliminary phase with 5 patients all given stem cell therapy alone, designed to assess safety

Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Progressive Supranuclear Palsy
Intervention  ICMJE Biological: stem cell therapy
Bone marrow will be collected from the iliac crest under local anesthesia. Mesenchymal Stem Cells (MSCs) will be isolated and cultivated in vitro. Patients will be catheterized and the MSCs will then be administered by intra-arterial route via the internal carotid artery and the vertebral artery that is largest in caliber, injecting small boluses manually through a microcatheter.
Study Arms  ICMJE
  • Active Comparator: immediate stem cell therapy
    patients will undergo active intervention i.e. they will be given stem cell therapy immediately. After 6 months they will undergo a sham procedure.
    Intervention: Biological: stem cell therapy
  • Sham Comparator: delayed stem cell therapy
    patients allocated to delayed stem cell therapy will undergo a sham procedure (incannulation of the femoral vein and infusion of saline solution). They will receive stem cell therapy after 6 months
    Intervention: Biological: stem cell therapy
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Unknown status
Estimated Enrollment  ICMJE
 (submitted: March 31, 2013)
25
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 2014
Estimated Primary Completion Date December 2014   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

diagnosis of 'probable Progressive Supranuclear Palsy - Richardson's disease subtype' according to current diagnostic criteria (Litvan et al. 1996 and 2003)

  • age at onset at least 40 years;
  • disease duration 12 months to 8 years;
  • supranuclear ophthalmoplegia;
  • postural instability or falls within 3 years from disease onset
  • positive MRI for PSP criteria (Quattrone et al, 2008)
  • Stable pharmacological treatment for at least 90 days
  • Lack of response to chronic levodopa (at least 12-month treatment).
  • Able to stand in upright posture without assistance for at least 30 seconds
  • Written informed consent (including video taping)

Exclusion Criteria:

  • Idiopathic Parkinson's disease;
  • Cerebellar ataxia
  • Symptomatic autonomic dysfunction
  • Evidence of any other neurological disease that could explain signs;
  • History of repeated strokes with stepwise progression of parkinsonian features;
  • History of major stroke;
  • Any history of severe or repeated head injury;
  • A history of encephalitis;
  • A history of neuroleptic use for a prolonged period of time or within the past 6 months;
  • Street-drug related parkinsonism;
  • Significant other neurological disease on CT-scan/MRI;
  • Oculogyric crises;
  • Major signs of corticobasal degeneration;
  • Signs of Lewy body disease;
  • Other life-threatening disease likely to interfere with the main outcome measure;
  • Any clinically significant laboratory abnormality, with the exception of cholesterol, triglycerides and glucose;
  • Renal failure (serum creatinine more than 300 mM/l);
  • Transaminase elevation more than twice the upper limit of normal;
  • Any concomitant disorder associated with bone marrow function impairment
  • Any concomitant disorder that requires chronic treatment with immunosuppressors, anti-inflammatory agents, and/or growth factors
  • dementia (MMSE < 24 according to Folstein 1975 or defined according to DSM-IV TR criteria)
  • any other disorder that could interfere with the evaluation of treatment or that could make intra-arterial infusion inadvisable
  • any other features that, according to the investigator, could reduce adherence to protocol procedures or prevent rapid access in case of emergency;
  • women of child-bearing age
  • participation in another clinical trial with experimental treatment in the last 30 days
  • brain MRI evidence of severe vascular abnormalities, space-occupying lesions or normal pressure hydrocephalus
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 40 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Italy
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT01824121
Other Study ID Numbers  ICMJE 032011
2011-004051-39 ( EudraCT Number )
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Study Sponsor  ICMJE Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Rosaria Giordano, MD IRCCS Ca' Granda Ospedale Maggiore Policlinico
PRS Account Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Verification Date March 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP