Ignite Creation Date:
2025-12-24 @ 11:30 PM
Ignite Modification Date:
2026-01-02 @ 3:35 AM
Study NCT ID:
NCT00881556
Status:
TERMINATED
Last Update Posted:
2021-08-17
First Post:
2009-04-14
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D004820', 'term': 'Epidermolysis Bullosa'}, {'id': 'D016108', 'term': 'Epidermolysis Bullosa Dystrophica'}], 'ancestors': [{'id': 'D012868', 'term': 'Skin Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D012873', 'term': 'Skin Diseases, Genetic'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D012872', 'term': 'Skin Diseases, Vesiculobullous'}, {'id': 'D003095', 'term': 'Collagen Diseases'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D051523', 'term': 'Fibroblast Growth Factor 7'}, {'id': 'C024352', 'term': 'fludarabine'}, {'id': 'C042382', 'term': 'fludarabine phosphate'}, {'id': 'D002066', 'term': 'Busulfan'}, {'id': 'D008140', 'term': 'Lorazepam'}, {'id': 'D000074323', 'term': 'Alemtuzumab'}, {'id': 'D016559', 'term': 'Tacrolimus'}], 'ancestors': [{'id': 'D005346', 'term': 'Fibroblast Growth Factors'}, {'id': 'D036341', 'term': 'Intercellular Signaling Peptides and Proteins'}, {'id': 'D010455', 'term': 'Peptides'}, {'id': 'D000602', 'term': 'Amino Acids, Peptides, and Proteins'}, {'id': 'D011506', 'term': 'Proteins'}, {'id': 'D001685', 'term': 'Biological Factors'}, {'id': 'D002072', 'term': 'Butylene Glycols'}, {'id': 'D006018', 'term': 'Glycols'}, {'id': 'D000438', 'term': 'Alcohols'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D008698', 'term': 'Mesylates'}, {'id': 'D000476', 'term': 'Alkanesulfonates'}, {'id': 'D017738', 'term': 'Alkanesulfonic Acids'}, {'id': 'D000473', 'term': 'Alkanes'}, {'id': 'D006839', 'term': 'Hydrocarbons, Acyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D013451', 'term': 'Sulfonic Acids'}, {'id': 'D013456', 'term': 'Sulfur Acids'}, {'id': 'D013457', 'term': 'Sulfur Compounds'}, {'id': 'D001570', 'term': 'Benzodiazepinones'}, {'id': 'D001569', 'term': 'Benzodiazepines'}, {'id': 'D001552', 'term': 'Benzazepines'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D061067', 'term': 'Antibodies, Monoclonal, Humanized'}, {'id': 'D000911', 'term': 'Antibodies, Monoclonal'}, {'id': 'D000906', 'term': 'Antibodies'}, {'id': 'D007136', 'term': 'Immunoglobulins'}, {'id': 'D007162', 'term': 'Immunoproteins'}, {'id': 'D001798', 'term': 'Blood Proteins'}, {'id': 'D012712', 'term': 'Serum Globulins'}, {'id': 'D005916', 'term': 'Globulins'}, {'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}]}}, 'protocolSection': {'designModule': {'phases': ['EARLY_PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 3}}, 'statusModule': {'overallStatus': 'TERMINATED', 'startDateStruct': {'date': '2009-08-20', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2021-08', 'completionDateStruct': {'date': '2015-09', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2021-08-13', 'studyFirstSubmitDate': '2009-04-14', 'studyFirstSubmitQcDate': '2009-04-14', 'lastUpdatePostDateStruct': {'date': '2021-08-17', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2009-04-15', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Event-free survival (EFS)', 'timeFrame': 'Up to 2 years'}, {'measure': 'Overall Survival (OS)', 'timeFrame': 'Up to 2 years'}], 'secondaryOutcomes': [{'measure': 'Percentage of whole blood (CD45), T-cell (CD3), and NK cell (CD56) chimerism following RIC and AlloSCT in selected patients with RDEB', 'timeFrame': 'Up to Day +730'}, {'measure': 'Percentage of donor skin dermal chimerism following RIC and AlloSCT in selected patients with RDEB.', 'timeFrame': 'Up to Day +730'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Allogeneic Stem Cell Transplant', 'AlloSCT', 'RDEB', 'recessive dystrophic epidermolysis bullosa'], 'conditions': ['Epidermolysis Bullosa']}, 'descriptionModule': {'briefSummary': 'Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (AlloSCT) from family-related donors and unrelated cord blood (UCB) donors will be safe and well tolerated in selected patients with RDEB.\n\nTo determine the event-free survival (EFS) and overall survival (OS) following RIC consisting of busulfan/fludarabine/alemtuzumab (BFA) and AlloSCT in selected patients with RDEB.', 'detailedDescription': 'Epidermolysis bullosa (EB), is a diverse group of genodermatoses, which is considered a rare and orphan disease and affects approximately 1 in 20,000 people in the United States for a cumulative total of close to 20,000\\[1-4\\]. There are three major subtypes of inherited EB, including EB simplex (EBS), junctional EB (JEB), and dystrophic EB\\[1-4\\]. RDEB is among the most severe and represents approximately 10% of all forms of EB\\[1-4\\]. A rough estimate would then project that there are several thousand patients with RDEB in the U.S. at the current time. Up to 30 different clinical phenotypes and mutations in at least 10 structural genes in different sub-types of EB have been reported\\[4-8\\]. In addition to heritable subtypes of EB, there is an acquired autoimmune form in which the patients develop auto-antibodies directed against similar proteins of the inherited dystrophic forms of EB, including EB acquisita (EBA).\n\nWe have previously reported our experience with RIC with BFA \\[48\\] in pediatric AlloSCT recipients (mean age 9.5 yrs \\[1.4-21\\], 11/4 M/F, 10 non-malignant, 5 malignant disease, \\[6 sibling, 5 UCB, 5 matched unrelated donor\\]); median time to ANC ≥ 500/mm3 and platelet count ≥20K/mm3 was 22 and 30 days, respectively. Probability of day +180 and 365 donor chimerism was 90% (Figure 7), and OS was 95% (Figure 8). This conditioning regimen therefore results in a high degree of donor chimerism and survival with minimal regimen related mortality.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '21 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Recessive Dystrophic Epidermolysis Bullosa (RDEB)\n* Diagnosis of RDEB using molecular diagnosis and sequencing of mutations\n* Skin biopsy to determine status of type VII collagen\n* Age ≤21 years\n* Patient must have adequate organ function as below:\n\n 1. Adequate renal function defined as:\n\n * Serum creatinine less than or equal to 1.5 x normal, or\n * 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\n 2. Adequate liver function defined as:\n\n * Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase (AST)) or serum glutamic-pyruvic transaminase (SGPT) (alanine aminotransferase (ALT))\\< 5.0 x normal\n 3. Adequate cardiac function defined as:\n\n * Shortening fraction of ≥28% by echocardiogram, or\n * Ejection fraction of ≥48% by radionuclide angiogram or echocardiogram\n 4. Adequate pulmonary function defined as:\n\n * Uncorrected diffusing capacity of the lungs for carbon monoxide (DLCO) ≥35% by pulmonary function test\n * For children who are uncooperative, no evidence of dyspnea at rest\n\nExclusion Criteria:\n\n* Karnofsky/Lansky Performance Score \\<50%\n* Pregnant or nursing\n* Uncontrolled bacterial, viral or mold infection\n* History or presence of skin squamous cell carcinoma'}, 'identificationModule': {'nctId': 'NCT00881556', 'acronym': 'RDEB', 'briefTitle': 'Allogeneic Stem Cell Transplantation (ALLOSCT) in Recessive Dystrophic Epidermolysis Bullosa (RDEB)', 'organization': {'class': 'OTHER', 'fullName': 'Columbia University'}, 'officialTitle': 'A Pilot Study of Reduced Intensity Conditioning (RIC) and Allogeneic Stem Cell Transplantation (ALLOSCT) In Children With Recessive Dystrophic Epidermolysis Bullosa (RDEB)', 'orgStudyIdInfo': {'id': 'AAAD5420'}, 'secondaryIdInfos': [{'id': 'CHNY-08-536', 'type': 'OTHER', 'domain': 'CUMC'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RIC Group', 'description': 'Reduced Intensity Transplant Conditioning (RIC):\n\nPalifermin (Kepivance®) 60 mcg/kg/day for 6 days Fludarabine 30 mg/m2 IV x 1 for 6 days Busulfan 4 mg/kg/day IV divided BID for 4 days Lorazepam 0.02-0.05 mg/kg for 5 days Alemtuzumab 20 mg/m2 IV for 5 days Tacrolimus 0.03mg/kg/24 hours as continuous infusion for 4 days', 'interventionNames': ['Drug: Palifermin', 'Drug: Fludarabine', 'Drug: Busulfan', 'Drug: Lorazepam', 'Drug: Alemtuzumab', 'Drug: Tacrolimus']}], 'interventions': [{'name': 'Palifermin', 'type': 'DRUG', 'otherNames': ['Kepivance'], 'description': '60 mcg/kg/day for 6 days', 'armGroupLabels': ['RIC Group']}, {'name': 'Fludarabine', 'type': 'DRUG', 'otherNames': ['Fludara'], 'description': '30 mg/m2 IV x 1 for 6 days', 'armGroupLabels': ['RIC Group']}, {'name': 'Busulfan', 'type': 'DRUG', 'otherNames': ['Myleran'], 'description': '4 mg/kg/day IV divided BID for 4 days', 'armGroupLabels': ['RIC Group']}, {'name': 'Lorazepam', 'type': 'DRUG', 'otherNames': ['Ativan'], 'description': '0.02-0.05 mg/kg for 5 days', 'armGroupLabels': ['RIC Group']}, {'name': 'Alemtuzumab', 'type': 'DRUG', 'otherNames': ['Lemtrada'], 'description': '20 mg/m2 IV for 5 days', 'armGroupLabels': ['RIC Group']}, {'name': 'Tacrolimus', 'type': 'DRUG', 'otherNames': ['Prograf', 'Protopic', 'Hecoria'], 'description': '0.03mg/kg/24 hours as continuous infusion for 4 days', 'armGroupLabels': ['RIC Group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': "The Children's Hospital", 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '60614', 'city': 'Chicago', 'state': 'Illinois', 'country': 'United States', 'facility': "Children's Memorial Hospital", 'geoPoint': {'lat': 41.85003, 'lon': -87.65005}}, {'zip': '10032', 'city': 'New York', 'state': 'New York', 'country': 'United States', 'facility': "Morgan Stanley Children's Hospital of NYP", 'geoPoint': {'lat': 40.71427, 'lon': -74.00597}}], 'overallOfficials': [{'name': 'Angela Christiano, PhD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Columbia University'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Columbia University', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}