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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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24 August 2021 |
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Main ID: |
NCT00881556 |
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Date of registration:
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14/04/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
RDEB |
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Scientific title:
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A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB) |
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Date of first enrolment:
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August 20, 2009 |
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Target sample size:
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3 |
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Recruitment status: |
Terminated |
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URL:
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https://clinicaltrials.gov/show/NCT00881556 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Early Phase 1
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Countries of recruitment
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United States
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Contacts
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Name:
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Angela Christiano, PhD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Columbia University |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Recessive Dystrophic Epidermolysis Bullosa (RDEB)
- Diagnosis of RDEB using molecular diagnosis and sequencing of mutations
- Skin biopsy to determine status of type VII collagen
- Age =21 years
- Patient must have adequate organ function as below:
1. Adequate renal function defined as:
- Serum creatinine less than or equal to 1.5 x normal, or
- Creatinine clearance or radioisotope glomerular filtration rate (GFR) =40
ml/min/m2 or > 60 ml/min/1.73 m2 or an equivalent GFR as determined by the
institutional normal range
2. Adequate liver function defined as:
- Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase
(AST)) or serum glutamic-pyruvic transaminase (SGPT) (alanine
aminotransferase (ALT))< 5.0 x normal
3. Adequate cardiac function defined as:
- Shortening fraction of =28% by echocardiogram, or
- Ejection fraction of =48% by radionuclide angiogram or echocardiogram
4. Adequate pulmonary function defined as:
- Uncorrected diffusing capacity of the lungs for carbon monoxide (DLCO) =35%
by pulmonary function test
- For children who are uncooperative, no evidence of dyspnea at rest
Exclusion Criteria:
- Karnofsky/Lansky Performance Score <50%
- Pregnant or nursing
- Uncontrolled bacterial, viral or mold infection
- History or presence of skin squamous cell carcinoma
Age minimum:
N/A
Age maximum:
21 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Epidermolysis Bullosa
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Intervention(s)
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Drug: Tacrolimus
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Drug: Alemtuzumab
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Drug: Palifermin
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Drug: Busulfan
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Drug: Fludarabine
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Drug: Lorazepam
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Primary Outcome(s)
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Event-free survival (EFS)
[Time Frame: Up to 2 years]
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Overall Survival (OS)
[Time Frame: Up to 2 years]
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Secondary Outcome(s)
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Percentage of donor skin dermal chimerism following RIC and AlloSCT in selected patients with RDEB.
[Time Frame: Up to Day +730]
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Percentage of whole blood (CD45), T-cell (CD3), and NK cell (CD56) chimerism following RIC and AlloSCT in selected patients with RDEB
[Time Frame: Up to Day +730]
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Secondary ID(s)
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AAAD5420
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CHNY-08-536
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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