Patients will be randomized (by block randomization) to Desferal (DFO) treatment.

Patients will be randomized (by block randomization) to isotonic saline treatment.

Diabetes Mellitus, Type 1

It is a blinded randomized cross-over study that investigates the efficacy of DFO (50mg/kg) given i.v before intermittent hypoxia (IH) to improve the cardiorespiratory and angiogenetic response in patients with diabetes.

desferal

It is a blinded randomized cross-over study that investigates the efficacy of isotonic saline given i.v before intermittent hypoxia (IH) to improve the cardiorespiratory and angiogenetic response in patients with diabetes.

Isotonic saline

Karolinska University Hospital

Desferal Administration to Improve the Impaired Reaction to Hypoxia in Diabetes

The absolute amount of endothelial precursor cells in 10 ml of blood

Sergiu Catrina

Senior consultant / associated professor

This is a randomised, double-blind, placebo-controlled, cross-over, single center study of patients with diabetes mellitus type 1 without chronic complications.

Perform in utero hematopoietic stem cell transplantation at the time of intrauterine transplantation in fetuses with alpha-thalassemia major. The cellular product is: Semi-allogeneic, Related, Maternal Bone Marrow-Derived, Miltenyi CliniMACS Plus enriched CD34+ hematopoietic stem cells administered in utero at a dose of 1 x 10^7-10^9 cells/kg fetal weight with equal to or less than 1% CD3+ T cells (equivalent to 10^5-10^7 T cells/kg fetal weight) in a final volume of 2-5ml suspended in 5% human serum albumin in Normosol buffer (Hospira, Inc.).
Stem cells will be administered immediately before the red blood cells intravenously via the umbilical vein during the clinically indicated IUT. All participants will receive one dose of stem cells but may receive additional transfusions as clinically indicated.

Alpha Thalassemia Major

Hemoglobinopathy; With Thalassemia

Hemoglobinopathies

Fetal Anemia

Fetal Hydrops

Alpha; Thalassemia

Thalassemia Major

Thalassemia Alpha

A-Thalassemia

This is a phase 1 safety study to demonstrate it is safe for both the mother and fetus to perform In utero hematopoietic stem cell transplantation of maternal derived stem cells at the time of intrauterine transplantation of red blood cells to treat fetuses affected with alpha-thalassemia major.

in utero hematopoietic stem cell transplantation

University of California

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

A Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses With Alpha Thalassemia Major

Maternal participant tolerance of bone marrow harvest defined as not requiring interventions for preterm labor, bleeding, infection or prolonged hospitalization.

safety for fetal participant defined by survival 24 hours after procedure, fetal survival till birth, neonatal survival through discharge of hospitalization and no evidence of graft versus host disease

This is defined as approximately 200-300 cc of bone marrow from which 10^7-10^9 CD34+ cells/kg fetal weight with 10^5-10^7 CD3+ cells/kg fetal weight will be isolated.

The primary efficacy endpoint is successful engraftment of maternal bone marrow- derived CD34+ hematopoietic stem cells measured by establishment of maternal participant donor cell chimerism equal to or greater than 1% donor cells in the circulation of the fetal recipient. Chimerism will be determined in cord blood at birth, or at a corrected gestational age of 40 weeks, if there is preterm delivery, followed weekly for the first 4 weeks of life, and monthly for one year in the infant to monitor the stability of engraftment.

Tippi Mackenzie

Professor of Surgery

University of California, San Francisco

California Institute for Regenerative Medicine (CIRM)

10 patients with hemochromatosis treated with Deferasirox

10 patients with hemochromatosis treated with venesection

20 patients with myelodysplastic syndrome treated with Deferasirox

10 healthy control persons to assess the normal level of investigational blood tests.

Hemochromatosis

Myelodysplastic Syndromes

Deferasirox tablets ( 250 mg or 500 mg) dispersed in a drinkable solution, 10 mg/kg/day, once daily for 12 months

Deferasirox

Treated with venesection every 8-10 day for 12 months, or until serum-ferritin has been reduced to about 50 µg/L.

Venesection

Deferasirox tablets ( 250 mg or 500 mg) dispersed in a drinkable solution starting with 10 mg/kg/day, once daily for 2 weeks and thereafter 20 mg/kg/day for 11,5 months.

Haukeland University Hospital, Clinical Trial Unit

Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic Syndrome

Deferasirox Versus Venesection in Patients With Hemochromatosis and for Treatment of Transfusional Siderosis in Myelodysplastic Syndrome: Diagnostics and New Biomarkers.

thalassemia/ödem

Desferal Administration to Improve the Impaired Reaction to Hypoxia in Diabetes

In Utero Hematopoietic Stem Cell Transplantation for Alpha-thalassemia Major (ATM)

Treatment of Iron Overload With Deferasirox (Exjade) in Hereditary Hemochromatosis and Myelodysplastic Syndrome

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