A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)

Conditions

• Sickle Cell Disease
• Thalassemia
• Thalassemia, Beta

Eligibility Criteria

Sex

ALL

Age

2 Years - 50 Years

Healthy Volunteers

Not accepted

Interventions

• Fludarabine — DRUG
  PK-guided fludarabine dosing will be used for each of the 2 cycles, using the InsightRx DoseMeRx platform.
• Cyclophosphamide — DRUG
  Cyclophosphamide will be administered Post-Transplant
• Tacrolimus — DRUG
  Tacrolimus will be administered beginning on day +5
• Mycophenolate Mofetil — DRUG
  Mycophenolate mofetil (MMF) will be administered three times daily starting on day +5.
• Rabbit ATG — BIOLOGICAL
  The dose and schedule of ATG will be determined according to the nomogram in Appendix A
• Dexamethasone — DRUG
  Standard Regimen: Dexamethasone on days -68 to -64 and days -40 to -36.
• Bortezomib — DRUG
  Bortezomib on days -71, -68, -65, -61, -43, -40, -37, and -33
• Rituximab — DRUG
  Rituximab on days -71, -58, -43, and -30.

Study Details

Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD. In this study, depending on how participants' body responds to the fludarabine and dexamethasone, the study doctor may decide participants should receive another drug, called cyclophosphamide, instead of fludarabine. In addition, depending on the results of participants' routine blood tests, participants may receive the drugs bortezomib and rituximab, which also help with immune suppression.

Key Dates

Start date

Feb 9, 2023

Status verified

Jun 2026

Primary completion

Feb 9, 2027

Completion

Feb 9, 2027

Study Design

Enrollment

24 participants (estimated)

Allocation

NA

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Arms

• Experimental: Participants with Sickle Cell Disease or β-Thalassemia
  Participants will have severe sickle cell disease or transfusion-dependent β-thalassemia.

Primary Outcome Measure

Number of participants with treatment related mortality/TRM or primary graft failure [ Time Frame: 1 year ]

Central Contacts

• Maria Cancio, MD
  212-639-2446
  [email protected]
• Jaap Jan Boelens, MD, PhD
  212-639-3643
  [email protected]

Locations (6)

Find similar trials in Basking Ridge, NJ

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