Laboratory Blood Test For the development of the new biomarkers using the technique of Mass-spectrometry 7,5 ml EDTA blood and a dry blood spot filter card are taken. To proof the correct Gilbert diagnosis in those patients where up to the enrollment in the study no genetic testing has been done, sequencing of Gilbert disease will be done. The analyses will be done at: Centogene AG Am Strande 7 18055 Rostock Germany

Meulengracht Syndrome

Hyperbilirubinemia

Unconjugated Benign Bilirubinemia

Centogene is one of the leading laboratories focusing on genetic testing for rare hereditary disorders. We now offer more than 2200 routine genetic and biochemical tests.

Children Hospital, Faculty of Medicine, Cairo University

Amrita Institute of Medical Sciences & Research Centre

Navi Mumbai Institute of Research In Mental And Neurological Handicap (NIRMAN)

Lady Ridgeway Hospital for Children

Biomarker for Gilbert Disease (BioGilbert)

Biomarker for Gilbert Disease - An International, Multi- Center, Epidemiological Protocol

Next-Generation Sequencing (NGS) of the UGT1A1 gene will be performed. The mutation will be confirmed by Sanger sequencing.

The quantitative determination of small molecules (molecular weight 150-700 kD, given as ng/μl) within a dried blood spot sample will be validated via liquid chromatography multiple reaction-monitoring mass spectrometry (LC/MRM-MS) and compared with a merged control cohort. The statistically best validated molecule will be considered as a disease specific biomarker.

Centogene AG Rostock

Cancer

Patients will take a pill of disulfiram at a fixed dose of 250 mg with their evening meal. This dose of disulfiram used to treat alcoholism. We will start by administering 2 mg of copper as copper gluconate along with 250 mg disulfiram.
Patients must not consume beverages containing alcohol while taking disulfiram.

Disulfiram

Commercially available. Copper gluconate has long been manufactured and sold as a food supplement. Copper gluconate should be taken separately from disulfiram, and if possible with breakfast.
Copper gluconate should not be administered to individuals with Wilson's disease or a family history of Wilson's disease.
Patients will receive 2 mg, 4mg, 6mg or 8mg daily

Copper Gluconate

Huntsman Cancer Institute

Phase I Study of Disulfiram and Copper Gluconate for the Treatment of Refractory Solid Tumors Involving the Liver

University of Utah

One cord blood unit will be thawed on day -14 before transplantation and selected using the CliniMACS for primitive cells that express CD133. These cells will be expanded ex vivo for a total of 14 days, using a two-stage procedure. On Day 0 the expanded cells will be harvested, washed three times with CliniMACS buffer (Miltenyi) plus 1% HSA per standard laboratory and clinical practice and the expanded cell product will be infused to a patient who has been prepared with a standard, myeloablative preparative regimen. A second, unexpanded, cord blood product will be infused on Day +1 for safety.

Patients with a hematologic malignancy who are candidates for myeloablative allogeneic BMT but lack an available matched sibling or unrelated marrow donor are eligible for this study if they have two suitably matched cord blood products with an adequate (1 x 10e7 TNC/kg IBW) but suboptimal (<5 x 10e7 TNC/kg IBW) cell dose.
One cord blood unit is expanded beginning 14 days before graft infusion (6 or 7 days before the start of the preparative regimen). Patients receive a standard myeloablative preparative regimen. The expanded cord blood unit is infused on day 0 and the unmanipulated cord blood unit is expanded on day 1.

Single Arm

Sex: Female, Male

Region of Enrollment

Ex vivo expansion of cord blood: Day 0: the expanded cell product will be infused to patient; Day +1: A second, unexpanded, cord blood product will be infused

Receive Two Cord Blood Units

Number of Participants With Toxicities Related to Infusion of Expanded Cord Blood Products

Number of participants who reached 3 consecutive days with ANC > 500

Neutrophil Engraftment Within 21 Days

Number of participants who died without relapse in less than 100 days.

Non-relapse Mortality

Patients with a hematologic malignancy who are candidates for myeloablative allogeneic BMT but lack an available matched sibling or unrelated marrow donor are eligible for this study if they have two suitably matched cord blood products with an adequate (1 x 10e7 TNC/kg IBW) but suboptimal (<5 x 10e7 TNC/kg IBW) cell dose. Eligible diagnoses include AML, MDS, ALL after failure of at least one regimen, mixed lineage leukemia, CML beyond first chronic phase, and lymphoma ineligible for autologous transplantation.

Overall Study

Allen Chen, MD,PhD,MHS

Acute Myeloid Leukemia

Leukemia, Lymphoblastic, Acute

Leukemia, Bilineage, Acute

Leukemia, Myeloid, Chronic

Day 0: the expanded cell product will be infused to patient; Day +1: A second, unexpanded, cord blood product will be infused

hemolysis/diarrhea

Biomarker for Gilbert Disease (BioGilbert)

Phase I Study of Disulfiram and Copper Gluconate for the Treatment of Refractory Solid Tumors Involving the Liver

Transplantation of Umbilical Cord Blood Following Chemotherapy for Blood Cancers

Plasma and Abscess Fluid Pharmacokinetics of Cefpirome and Moxifloxacin After Single and Multiple Dose Administration

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