Development of IPS from Donated Somatic Cells of Patients with Neurological Diseases

NCT00874783 | Recruiting

• 1 other identifier: 0599-08-HMO-CTIL
• Study Type: observational
• Target: 120
• Locations: 1 country
• Sites: 1

Brief Summary

Human fibroblasts and possibly other human somatic cells may be reprogrammed into induced pluripotent stem (iPS) cells by the forced expression of transcription factors (1-5). The iPS cells seem to share many properties with human embryonic stem cells. Induced pluripotent stem cells potentially may be useful in the future as an unlimited source of cells for transplantation. The major goal of the project is to develop human iPS cells from cell cultures from skin biopsies or the patient's hair. The iPS cells will be developed primarily for modeling diseases and drug discovery as well as basic research, and for developing the technology that may eventually allow the use of iPS cells for future transplantation therapy. The iPS cells developed in the course of this application are not intended for use in transplantation therapy. Future development of iPS cells for clinical transplantation therapies will be subjected to the appropriate authorization by ethical and regulatory committees.

Conditions

Neurodegenerative Disorders

Keywords

Amyotrophic Lateral Sclerosis; Familial Dysautonomia; Parkinson's Disease; Alzheimer's Disease; Age Related Macular Degeneration; Retinitis Pigmentosa; Huntington's Disease; Machado - Joseph Disease; SMA - Spinal Muscular Atrophy; Ataxia Telangiectasia

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

120 donors to cover 10 different neurodegenerative disorders (previously specified) based on 10 donors per disorder and 20 healthy control donors.

You may qualify if:

• Donors suffering from different (specified) neurodegenerative disorders scheduled to undergo surgery for medical reasons or will donate a single or a few hairs--to be removed intact from the scull or other areas in the body.
• Healthy donors scheduled to undergo surgery for medical reasons or will donate a single or a few hairs--to be removed intact from the scull or other areas in the body.

You may not qualify if:

• None

Sponsors & Collaborators

• Hadassah Medical Organization: lead

Study Sites (1)

Hadassah Ein Kerem

Jerusalem, Israel, 9112100, Israel

RECRUITING

Related Publications (1)

• Yu J, Vodyanik MA, Smuga-Otto K, Antosiewicz-Bourget J, Frane JL, Tian S, Nie J, Jonsdottir GA, Ruotti V, Stewart R, Slukvin II, Thomson JA. Induced pluripotent stem cell lines derived from human somatic cells. Science. 2007 Dec 21;318(5858):1917-20. doi: 10.1126/science.1151526. Epub 2007 Nov 20.

  PMID: 18029452 BACKGROUND

Biospecimen

Retention: SAMPLES WITH DNA

donation of a skin specimen of up to 10mm in diameter from skin which will be removed in a surgical operation from a patient who is scheduled to undergo an operation for medical reasons and with no relation to the study at hand.

MeSH Terms

Conditions

Neurodegenerative Diseases; Amyotrophic Lateral Sclerosis; Dysautonomia, Familial; Parkinson Disease; Alzheimer Disease; Macular Degeneration; Retinitis Pigmentosa; Huntington Disease; Muscular Disorders, Atrophic; Muscular Atrophy, Spinal; Ataxia Telangiectasia

Condition Hierarchy (Ancestors)

Nervous System Diseases; Spinal Cord Diseases; Central Nervous System Diseases; Motor Neuron Disease; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases; Primary Dysautonomias; Autonomic Nervous System Diseases; Hereditary Sensory and Autonomic Neuropathies; Nervous System Malformations; Heredodegenerative Disorders, Nervous System; Polyneuropathies; Peripheral Nervous System Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Movement Disorders; Synucleinopathies; Dementia; Tauopathies; Neurocognitive Disorders; Mental Disorders; Retinal Degeneration; Retinal Diseases; Eye Diseases; Eye Diseases, Hereditary; Retinal Dystrophies; Chorea; Dyskinesias; Cognition Disorders; Muscular Diseases; Musculoskeletal Diseases; Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Neurocutaneous Syndromes; Ataxia; Neurologic Manifestations; Telangiectasis; Vascular Diseases; Cardiovascular Diseases; Primary Immunodeficiency Diseases; DNA Repair-Deficiency Disorders; Immunologic Deficiency Syndromes; Immune System Diseases

Study Officials

• Benjamin E Reubinoff, MD, PhD

  Hadassah Medical Organization

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Benjamin E. Reubinoff, MD PhD

CONTACT

011-972-2-677-4569; benjaminr@ekmd.huji.ac.il

Shelly E Tannenbaum, MSQA

CONTACT

97226777947; stannenbaum@hadassah.org.il

Study Design

• Study Type: observational
• Observational Model: CASE CONTROL
• Time Perspective: PROSPECTIVE
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 2, 2009
• First Posted: April 3, 2009
• Study Start: April 1, 2009
• Primary Completion (Estimated): December 1, 2030
• Study Completion (Estimated): December 1, 2030
• Last Updated: March 4, 2025

Record last verified: 2025-03

Locations

IL (1)