Trial record 9 of 2152 for:
anemia
Skin and Blood Research Samples From Healthy Volunteers and Sickle Cell Anemia Patients
This study has been completed.
Sponsor:
Washington University School of Medicine
Information provided by (Responsible Party):
Washington University School of Medicine
ClinicalTrials.gov Identifier:
NCT00840567
First received: February 9, 2009
Last updated: October 6, 2011
Last verified: October 2011
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Purpose
The investigators plan to obtain skin and blood samples from healthy volunteers and sickle cell anemia patients to use for research to use homologous recombination to correct β-globin gene mutations in therapeutically useful cells, like autologous induced pluripotent stem cells from sickle cell anemia patients.
| Condition | Intervention |
|---|---|
|
Anemia, Sickle Cell |
Procedure: Skin and blood samples |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Basic Science |
| Official Title: | Acquisition of Skin Biopsies and Blood Samples From Normal Volunteers and Sickle Cell Anemia Patients for Research Purposes |
Resource links provided by NLM:
Further study details as provided by Washington University School of Medicine:
Primary Outcome Measures:
- To obtain skin biopsy samples from normal healthy volunteers and sickle cell anemia patients to create induced pluripotent stem cells. [ Time Frame: 1 year ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- To define the efficiency of homologous recombination in induced pluripotent stem cells derived from skin biopsy samples [ Time Frame: 1 year ] [ Designated as safety issue: No ]
- To obtain blood samples to confirm genetic mutations in sickle cell anemia patients (and no mutations in healthy volunteers). [ Time Frame: 1 year ] [ Designated as safety issue: No ]
| Enrollment: | 7 |
| Study Start Date: | January 2009 |
| Study Completion Date: | February 2011 |
| Primary Completion Date: | February 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Tissue sample
To obtain a one time skin sample (4 ml skin punch biopsy) and one time blood samples (1 teaspoon) from healthy volunteers and sickle cell anemia patients to create induced pluripotent stem cells.
|
Procedure: Skin and blood samples
Obtain skin and blood samples from healthy volunteers and sickle cell anemia patients
Other Name: skin biopsy samples
|
Detailed Description:
- To obtain skin biopsy samples from normal healthy volunteers and patients with a benign, inherited hematologic disease, such as sickle cell anemia, to create induced pluripotent stem cells. Pluripotency will be confirmed by injecting potential iPS cell lines into immunodeficient mice, assessing the ability of each line to cause cystic teratomas in recipient mice.
- To define the efficiency of homologous recombination in induced pluripotent stem cells derived from skin biopsy samples.
- To define the efficiency of homologous recombination in human embryonic stem cells using NIH-approved cell lines.
- To establish the genetic consequences of the derivation of human induced pluripotent cells in normal controls or patients with benign, inherited, hematologic diseases, by genomic analysis, including whole genome sequencing.
- To establish the genetic consequences of homologous recombination in human induced pluripotent stem cells and embryonic stem cells by genomic analysis, including whole genome sequencing.
- To obtain blood samples to confirm genetic mutations in patients with an inherited hematologic disease (and to confirm no mutations in healthy volunteers).
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | Yes |
Criteria
Inclusion Criteria:
- Age ≥ 18.
- No active systemic skin infection at biopsy site.
- No allergy to lidocaine or other local anesthetics
Exclusion Criteria:
ALL PATIENTS
- History of a bleeding disorder, easy bleeding, or bruising.
- Inability or unwillingness to provide informed consent.
SICKLE CELL PATIENTS
- Platelets ≤ 50,000
- INR ≥ 1.5
- Currently bing given intravenous heparin
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00840567
Locations
| United States, Missouri | |
| Washington University School of Medicine | |
| St. Louis, Missouri, United States, 63110 | |
Sponsors and Collaborators
Washington University School of Medicine
Investigators
| Study Chair: | Timothy Ley, M.D. | Washington Univerisity School of Medicine |
More Information
Additional Information:
Publications:
Maherali, N., Ahfeldt, T., Rigamonti, A., Utikal, J., Cowan, C. and Hochedlinger, K., A high-efficiency system for the generation and study of human induced pluripotent stem cells. Cell, 2008. 3(3): p. 340-345.
| Responsible Party: | Washington University School of Medicine |
| ClinicalTrials.gov Identifier: | NCT00840567 History of Changes |
| Other Study ID Numbers: | 08-1409 |
| Study First Received: | February 9, 2009 |
| Last Updated: | October 6, 2011 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by Washington University School of Medicine:
|
Stem cells |
Additional relevant MeSH terms:
|
Anemia Anemia, Sickle Cell Hematologic Diseases Anemia, Hemolytic, Congenital |
Anemia, Hemolytic Hemoglobinopathies Genetic Diseases, Inborn |
ClinicalTrials.gov processed this record on May 21, 2013