Actively Recruiting

Phase 1
Age: 1Year - 30Years
All Genders
NCT03279094

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

Led by City of Hope Medical Center · Updated on 2026-05-12

11

Participants Needed

1

Research Sites

468 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

This is a study to evaluate the safety and toxicity of a treatment regimen consisting of 2 cycles of pre-transplant immunosuppressive therapy followed by myeloablative preparative regimen and allogeneic hematopoietic stem cell transplantation from a haploidentical donor in patients with sickle cell disease. The overall goal of this study is to expand the donor pool for hematopoietic stem cell transplantation in sickle cell disease using haploidentical donors, and to develop a non-toxic, myeloablative regimen, with the goal of achieving a consistent donor chimerism utilizing pre-transplant immunosuppressive therapy.

CONDITIONS

Official Title

Haploidentical Transplantation With Pre-Transplant Immunosuppressive Therapy for Patients With Sickle Cell Disease

Who Can Participate

Age: 1Year - 30Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of sickle cell anemia (Hgb SS or SB0 Thalassemia) with baseline Hgb S over 60%
  • History of significant neurologic event (stroke) or neurological deficit lasting more than 24 hours or elevated transcranial Doppler velocity over 200 m/s
  • History of one or more acute chest syndrome episodes in past 2 years despite supportive care
  • History of one or more severe vaso-occlusive pain crises per year in past 2 years despite supportive care
  • Recurrent priapism requiring medical therapy
  • Osteonecrosis of two or more joints despite supportive care
  • Prior treatment with regular red blood cell transfusions (8 or more per year for over 1 year) to prevent complications
  • Echocardiograph showing tricuspid valve regurgitation jet velocity of 2.5 m/sec or higher
  • Age between 1 and 30 years
  • Agreement to use effective contraception before and for six months after study participation for women of childbearing potential and men
  • Ability to understand and willingness to sign informed consent or assent
  • Availability of a related haploidentical donor matching on HLA-A, B, C, and DRB1
  • No available HLA matched sibling or 10/10 matched unrelated donor
Not Eligible

You will not qualify if you...

  • Any uncontrolled illness including active bacterial, viral, or fungal infection
  • Receiving other investigational agents or concurrent biological, chemotherapy, or radiation therapy
  • History of allergic reaction to any compound similar to those in the transplant regimen
  • Pregnant women
  • Any active malignancy except non-melanoma skin cancers
  • Medical or neurologic/psychiatric conditions impairing compliance or transplant tolerance
  • Prior autologous or allogeneic transplant
  • Availability of fully HLA-matched related or unrelated donor
  • Inability to comply with safety monitoring requirements in the investigator's opinion

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 1 location

1

City of Hope Medical Center

Duarte, California, United States, 91010

Actively Recruiting

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Research Team

A

Anna B. Pawlowska, MD

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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