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

• Register: ClinicalTrials.gov
• Last refreshed on: 19 February 2015
• Main ID: NCT01824121
• Date of registration: 31/03/2013
• Prospective Registration: No
• Primary sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Public title: Clinical Trial to Evaluate Bone Marrow Stem Cell Therapy for PSP, a Rare Form of Parkinsonism
• Scientific title: Autologous Mesenchymal Stem Cell Therapy in Progressive Supranuclear Palsy: a Randomized, Double-blind, Controlled Clinical Trial
• Date of first enrolment: December 2012
• Target sample size: 25
• Recruitment status: Recruiting
• URL: http://clinicaltrials.gov/show/NCT01824121
• Study type: Interventional
• Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Outcomes Assessor), Primary Purpose: Treatment
• Phase: Phase 1/Phase 2

Countries of recruitment

Italy

Contacts

• Name: Rosaria Giordano, MD
• Affiliation: IRCCS Ca' Granda Ospedale Maggiore Policlinico

• Name: Margherita Canesi, MD

Key inclusion & exclusion criteria

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



Age minimum: 40 Years
Age maximum: N/A
Gender: Both

Health Condition(s) or Problem(s) studied

Progressive Supranuclear Palsy

Intervention(s)

Biological: stem cell therapy

Primary Outcome(s)

incidence of adverse events [Time Frame: one year]

Secondary Outcome(s)

changes in brain images [Time Frame: one year]

Secondary ID(s)

2011-004051-39

032011

Source(s) of Monetary Support

Please refer to primary and secondary sponsors