Recruiting
Phase 1

Haploidentical Transplantation

Sponsor:

City of Hope Medical Center

Code:

NCT03279094

Conditions

Sickle Cell Disease

Eligibility Criteria

Sex: All

Age: 1 - 30

Healthy Volunteers: Not accepted

Interventions

Hematopoietic stem cell transplantation

Study Details

Brief summary:

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

Sickle Cell Disease

Study ID

NCT03279094

Start date

Feb 2, 2018

Status verified date

May, 2026

Completion date

Jan 25, 2027

Anticipated

Primary completion date

Jan 25, 2027

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 1 - 30

Healthy Volunteers: Not accepted

Inclusion criteria

  • Diagnosis: Patients with sickle cell anemia (Hgb SS or SB° Thalassemia) with baseline Hgb S more than 60%.
  • Disease status:
  • Significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; or increased transcranial Doppler velocity (>200 m/s).
  • History of one or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea).
  • History of one or more severe vaso-occlusive pain crises per year in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea).
  • Recurrent priapism requiring medical therapy.
  • Osteonecrosis of two or more joints despite the institution of supportive care measures.
  • Prior treatment with regular RBC transfusion therapy, defined as receiving 8 or more transfusions per year for > 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
  • Echocardiograph finding of tricuspid valve regurgitation jet (TRJ) velocity ≥ 2.5 m/sec.
  • Ages 1 to 30.
  • Child Bearing Potential- Transplantation could be teratogenic and/or lethal to the developing fetus. For this reason, women of child-bearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control or abstinence) prior to study entry and for six months following duration of study participation. Should a woman become pregnant or suspect that she is pregnant while participating on the trial, she should inform her treating physician immediately.
  • Informed Consent/Assent: All subjects must have the ability to understand and the willingness to sign a written informed consent.
  • The recipient must have a related donor who is genotypically haploidentical on HLA-A, B, C and DRB1 loci.
  • No HLA matched sibling or 10/10 matched unrelated donor is available.

Exclusion criteria:

  • Any uncontrolled illness including ongoing or active bacterial, viral or fungal infection.
  • Patients may not be receiving any other investigational agents, or concurrent biological, chemotherapy, or radiation therapy.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen.
  • Pregnant women are excluded from this study.
  • Patients with any active malignancy are ineligible for this study, other than non-melanoma skin cancers.
  • Medical problem or neurologic/psychiatric dysfunction which would impair patient ability to be compliant with the medical regimen and to tolerate transplantation or would prolong hematologic recovery which in the opinion of the principal investigator would place the recipient at unacceptable risk.
  • Prior autologous or allogeneic transplant.
  • Fully HLA-matched related or unrelated donor is available to donate.
  • Non-Compliance: Subjects, who in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study.

Study Design

Enrollment

11 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Haploidentical stem cell transplantation

Interventions

Hematopoietic stem cell transplantation

Haploidentical stem cell transplantation with pre-transplant immunosuppressive therapy

Primary outcome measure

  • Rate of unacceptable adverse events that are defined as any of the following events that occur from start of pre-transplant immunosuppressive therapy to the first 100 days post HCT: [ Time Frame: 190 days ]

Central Contacts and Locations

Central contacts

Anna B. Pawlowska, MD

626-218-8442apawlowska@coh.org

Locations

City of Hope Medical Center

Recruiting

Duarte, California, United States, 91010

Contacts

Mary Suarez, CRC

626-218-5795masuarez@coh.org

Principal Investigator:

Anna B. Pawlowska, MD

More Information

Sponsor

City of Hope Medical Center

Last update posted

May 12, 2026

Last verified

May, 2026

Keywords

  • Sickle Cell Disease
  • Hematopoietic stem cell transplantation
  • Haploidentical stem cell transplantation
  • Post-transplant Cytoxan

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by City of Hope Medical Center on 2026-05-12.