Allo HSCT for High Risk Hemoglobinopathies

Part of paid clinical trials in Minneapolis, Minnesota.

Sponsor
Masonic Cancer Center, University of Minnesota
Study ID
NCT06872333
Phase
PHASE2
Status
Recruiting
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Conditions

Eligibility Criteria

Sex
ALL
Age
N/A - 55 Years
Healthy Volunteers
Accepted

Interventions

  • Alemtuzumab — DRUG
    Alemtuzumab (Campath) will be administered IV over 2 hours on day -8 to day -4.
  • Total Body Irradiation — RADIATION
    400 cGy in 2 split fractions will be administered per Department of RadiationOncology SOPs.
  • Cell Infusion — BIOLOGICAL
    On day 0 the cells will be infused per cell source specific institutional guidelines
  • Thymoglobulin — DRUG
    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.
  • Fludarabine — DRUG
    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).
  • Busulfan — DRUG
    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.
  • Cyclophosphamide — DRUG
    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.
  • Sirolimus — DRUG
    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.
  • Tacrolimus — DRUG
    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.
  • Mycophenolate Mofetil — DRUG
    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.
  • Plerixafor (mozobil) — DRUG
    Plerixafor will beused to significantly increase stem cell yields on a second collection day compared to donors who continued mobilization on G-CSF only.

Study Details

A single center, open label, interventional, phase II trial for donor transplant for high risk hemoglobinopathies and other red cell transfusion dependent disorders utilizing allogeneic hematopoietic stem cell transplantation (HSCT) regimens.

Key Dates

Start date
Nov 19, 2024
Status verified
Jun 2026
Primary completion
Jun 1, 2030
Completion
Jun 1, 2032

Study Design

Enrollment
62 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER

Arms

  • Experimental: Arm A - Closed
    Arm A Matched sib regimen - Age 6 -55 (per physician preference for patients over 6) Campath/TBI
  • Experimental: Arm B
    Arm B Matched sib regimen - 0-55 (per physician preference for patients over 6) ATG/Flu/Bu
  • Experimental: Arm C
    Arm C Fully Matched unrelated donor (MUD)- - 0-55 years; ATG/Flu/Bu
  • Experimental: Arm D
    Arm D: Haploindentical or mismatched unrelated donors (MMUD) - 0-55 years; ATG/Thiotepa/Cyclophosphamide/MESNA/Flu/TBI
  • Experimental: Arm E
    Matched sib regimen - Age 6-55 (per physician preference for patients over 6) Campath/TBI with peripheral blood stem cell graft

Primary Outcome Measure

Incidence of Graft versus Host Disease (GvHD) [ Time Frame: 1 year ]

Central Contacts

Locations (1)

FacilityCityStateZIPSite coordinators
Masonic Cancer CenterMinneapolisMinnesota55455
Ashish Gupta, MBBS, MPH

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