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

NCT01824121 | Unknown Phase 1

• 2 other identifiers: 0320112011-004051-39
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

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

Conditions

Progressive Supranuclear Palsy

Keywords

Progressive Supranuclear palsy; stem cell therapy

Outcome Measures

Primary Outcomes (1)

• incidence of adverse events

  incidence of adverse events collected by clinical monitoring and performing routine laboratory tests

  one year

Secondary Outcomes (1)

• changes in brain images

  one year

Other Outcomes (2)

• changes in motor function

  one year

• changes in cognitive functions

  one year

Study Arms (2)

immediate stem cell therapy

ACTIVE COMPARATOR

patients will undergo active intervention i.e. they will be given stem cell therapy immediately. After 6 months they will undergo a sham procedure.

Biological: stem cell therapy

delayed stem cell therapy

SHAM COMPARATOR

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

Biological: stem cell therapy

Interventions

stem cell therapy BIOLOGICAL

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.

delayed stem cell therapy; immediate stem cell therapy

Eligibility Criteria

• Age: 40 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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;

Sponsors & Collaborators

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico: lead

Study Sites (1)

ICP Parkinson Institute

Milan, 20126, Italy

RECRUITING

Related Publications (3)

• Giordano R, Canesi M, Isalberti M, Marfia G, Campanella R, Vincenti D, Cereda V, Ranghetti A, Palmisano C, Isaias IU, Benti R, Marotta G, Lazzari L, Montemurro T, Vigano M, Budelli S, Montelatici E, Lavazza C, Rivera-Ordaz A, Pezzoli G. Safety and Effectiveness of Cell Therapy in Neurodegenerative Diseases: Take-Home Messages From a Pilot Feasibility Phase I Study of Progressive Supranuclear Palsy. Front Neurosci. 2021 Oct 12;15:723227. doi: 10.3389/fnins.2021.723227. eCollection 2021.

  PMID: 34712113 DERIVED

• Canesi M, Giordano R, Lazzari L, Isalberti M, Isaias IU, Benti R, Rampini P, Marotta G, Colombo A, Cereda E, Dipaola M, Montemurro T, Vigano M, Budelli S, Montelatici E, Lavazza C, Cortelezzi A, Pezzoli G. Finding a new therapeutic approach for no-option Parkinsonisms: mesenchymal stromal cells for progressive supranuclear palsy. J Transl Med. 2016 May 10;14(1):127. doi: 10.1186/s12967-016-0880-2.

  PMID: 27160012 DERIVED

• Giordano R, Canesi M, Isalberti M, Isaias IU, Montemurro T, Vigano M, Montelatici E, Boldrin V, Benti R, Cortelezzi A, Fracchiolla N, Lazzari L, Pezzoli G. Autologous mesenchymal stem cell therapy for progressive supranuclear palsy: translation into a phase I controlled, randomized clinical study. J Transl Med. 2014 Jan 17;12:14. doi: 10.1186/1479-5876-12-14.

  PMID: 24438512 DERIVED

MeSH Terms

Conditions

Supranuclear Palsy, Progressive

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Ophthalmoplegia; Ocular Motility Disorders; Cranial Nerve Diseases; Tauopathies; Neurodegenerative Diseases; Paralysis; Neurologic Manifestations; Eye Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Rosaria Giordano, MD

  IRCCS Ca' Granda Ospedale Maggiore Policlinico

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Margherita Canesi, MD

CONTACT

0039 02 5799; canesi@parkinson.it

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 31, 2013
• First Posted: April 4, 2013
• Study Start: December 1, 2012
• Primary Completion: December 1, 2014
• Study Completion: December 1, 2014
• Last Updated: April 4, 2013

Record last verified: 2013-03

Locations

IT (1)