Allo HSCT for High Risk Hemoglobinopathies

Browse trials for

• Anemia, Sickle Cell 1,103
• Hemoglobinopathies 105

Anemia, Sickle Cell

1,103 studies on the registry are indexed under Anemia, Sickle Cell; 235 are open to participants now.

This study's planned enrollment of 62 is above the median of 40 across 750 interventional studies indexed under Anemia, Sickle Cell.

Lead sponsor

Masonic Cancer Center, University of Minnesota is the lead sponsor of 284 studies on the registry; 34 are open to participants now.

Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.

Study arms

• Experimental
  Arm A - Closed

  Arm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI

  Drug: Alemtuzumab · Radiation: Total Body Irradiation · Biological: Cell Infusion · Drug: Sirolimus · Drug: Mycophenolate Mofetil

• Experimental
  Arm B

  Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu

  Biological: Cell Infusion · Drug: Thymoglobulin · Drug: Fludarabine · Drug: Busulfan · Drug: Tacrolimus · Drug: Mycophenolate Mofetil

• Experimental
  Arm C

  Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu

  Biological: Cell Infusion · Drug: Thymoglobulin · Drug: Fludarabine · Drug: Busulfan · Drug: Tacrolimus · Drug: Mycophenolate Mofetil · Drug: Plerixafor (mozobil)

• Experimental
  Arm D

  Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI

  Drug: Sirolimus · Drug: Tacrolimus · Drug: Plerixafor (mozobil)

• Experimental
  Arm E

  Matched sib regimen - Age 6-55 (per physician preference for patients over 6) Campath/TBI with peripheral blood stem cell graft

  Radiation: Total Body Irradiation · Biological: Cell Infusion · Drug: Thymoglobulin · Drug: Fludarabine · Drug: Cyclophosphamide · Drug: Sirolimus · Drug: Tacrolimus · Drug: Mycophenolate Mofetil

Interventions

• DrugAlemtuzumab

  Alemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4.

  Also known as: Campath

• RadiationTotal Body Irradiation

  400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs.

  Also known as: TBI

• BiologicalCell Infusion

  On day 0 the cells will be infused per cell source specific institutional guidelines

• DrugThymoglobulin

  ATG will be administered IV every 24 hours beginning on day -8 for all patients. Dosing will be model-based using Bayesian methodology13,14,15. Total doses and total number of doses (1-4 doses) will be determined based on absolute lymphocyte count and weight.

  Also known as: Rabbit ATG

• DrugFludarabine

  Fludarabine will be administered IV over 1 hour every 24 hours on day -5 to day - 2. The daily dose of fludarabine will be determined by model-based dosing utilizing Bayesian methodology with a cumulative area under the curve (cAUC) of 20 mg\*hr/L (range 18-22 mg\*hr/L).

• DrugBusulfan

  Busulfan dosing and administration and therapeutic drug monitoring (TDM) per institutional guidelines. Initial busulfan dosing will be determined by model-based dosing utilizing Bayesian methods with a cumulative area under the curve (cAUC) of 75 mg\*hr/L.

• DrugCyclophosphamide

  Cyclophosphamide will be administered at a dose of 14.5 mg/kg over 2 hours IV daily on days -6 and -5. Cyclophosphamide dosing is calculated based on actual body weight (ABW). For Arm D - Cyclophosphamide 50 mg/kg IV will be administered over 2 hours on days +3 and +4. Cyclophosphamide dosing for post-transplant is calculated based on ideal body weight (IBW) unless patient weighs less than IBW, in which case actual body weight (ABW) will be used.

  Also known as: Cyclophosphamide with MESNA

• DrugSirolimus

  Patients on Arm A and Arm D will receive sirolimus; beginning on day -3 and continuing until day +180 for patients on Arm A or beginning on day +5 and continuing until 1 year post transplant for patients on Arm D.

• DrugTacrolimus

  Patients on Arm B and Arm C will receive tacrolimus, beginning on day -3 and continuing until day +180. Tacrolimus dosing and monitoring will be per institutional guidelines.

• DrugMycophenolate Mofetil

  MMF will begin on day -3 (Arm A, B \& C) or day +5 (Arm D). Patients treated on adult service will receive 15 mg/kg (max 1500 mg/dose) given every 12 hours, rounded to nearest 250 mg. Patients on pediatric service will receive 15 mg/kg (max 1000 mg/dose) given every 8 hours. MMF dosing will be monitored and altered as clinically appropriate based on institutional guidelines. MMF will be stopped at day +30 (Arms A, B \& C) or day +35 (Arm D) or 7 days after engraftment, whichever day is later, if no acute GVHD.

  Also known as: (MMF)

• DrugPlerixafor (mozobil)

  Plerixafor will beused to significantly increase stem cell yields on a second collection day compared to donors who continued mobilization on G-CSF only.

Primary outcomes

1. Incidence of Graft versus Host Disease (GvHD)

   Time frame: 1 year

Secondary outcomes

1. Overall Survival

   Overall survival post HCT

   Time frame: 1 and 2 years

2. Grade 3-4 Acute GvHD

   Grade 3-4 acute Graft versus Host Disease (GvHD) at 2 years post HCT.

   Time frame: 2 years

3. Chronic Graft versus Host Disease (GvHD) Free

   Chronic Graft versus Host Disease (GvHD) Free at 2 years post HCT.

   Time frame: 2 years

4. Failure Free Survival

   Failure Free Survival 2 years post HCT.

   Time frame: 2 years