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NCT Number: NCT06872333

Allo HSCT for High Risk Hemoglobinopathies

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.

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Key information

Conditions

Graft Failure Anemia Anemia, Hemolytic Anemia, Hemolytic, Congenital Anemia, Sickle Cell Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemoglobinopathies Sickle Cell Disease

Age range

Up to 55 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Masonic Cancer Center

Minneapolis, Minnesota, 55455, United States

Location status: Recruiting

Location contact

Ashish Gupta, MBBS, MPH

CONTACT

Who can participate

Healthy volunteers accepted: Yes

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Sickle Cell Disease (SCD)
  • SCD Patients with a fully matched sibling donor (MSD) irrespective of the frequency or severity of symptoms MSD transplant can be considered. Parents/patient must be counseled as to the risks and benefits and provide their voluntary informed consent
  • Transfusion Dependent Alpha- or Beta- Thalassemia
  • Diamond Blackfan Anemia
  • Other Non-Malignant Hematologic Disorders
  • Karnofsky ≥ 60%, Lansky play score ≥ 60. Patients with lower performance score can be considered based on study team's evaluation.
  • Sexually active persons of childbearing potential or persons with partners of childbearing potential must agree to use a highly effective form of contraception during study treatment and for at least 4 months after the transplant.

Exclusion criteria

  • Pregnant, breastfeeding or intending to become pregnant during the study. Persons of childbearing potential must have a negative pregnancy test (serum or urine) within 30 days of the start of treatment
  • HIV infection with a detectable viral load. All HIV+ patients must be evaluated by infectious disease (ID) and an HIV management plan established prior to transplantation.
  • Active, uncontrolled infection - infection that is stable or improving after 1 week of appropriate therapy (4 weeks for presumed or documented fungal infections) will be permitted
  • Known allergy to any of the study components
  • Psychiatric illness/social situations that, in the judgement of the enrolling Investigator, would limit compliance with study requirements
  • Other illness or a medical issue that, in the judgement of the enrolling Investigator, would exclude the patient from participating in this study

Treatment and study plan

Alemtuzumab

Drug

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

Other names: Campath

Total Body Irradiation

Radiation

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

Other names: TBI

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.

Other names: Rabbit ATG

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.

Other names: Cyclophosphamide with MESNA

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.

Other names: (MMF)

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.

Primary outcomes

  1. Incidence of Graft versus Host Disease (GvHD)

    Time frame: 1 year

Secondary outcomes

  1. Overall Survival

    Time frame: 1 and 2 years

    Overall survival post HCT

  2. Grade 3-4 Acute GvHD

    Time frame: 2 years

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

  3. Chronic Graft versus Host Disease (GvHD) Free

    Time frame: 2 years

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

  4. Failure Free Survival

    Time frame: 2 years

    Failure Free Survival 2 years post HCT.

Study contacts

Contact information is provided by the study sponsor or research team.

Ashish Gupta, MBBS, MPH

CONTACT

[email protected]

612-626-2961

Sponsors and collaborators

Lead sponsor

Masonic Cancer Center, University of Minnesota

Other

Registry information

Official study title

Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathies and Other Red Cell Transfusion Dependent Disorders

Important dates

Study start
2024
Primary completion
2030
Study completion
2032
First posted
Mar 12, 2025
Registry last updated
Jun 4, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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