Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor

A Phase II Pilot Study of Nonmyeloablative Conditioning Hematopoietic Stem Cell Transplantation in Children With Sickle Cell Disease Who Have a Matched Related Major ABO-Incompatible Donor (Sickle-AID)

Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor (NCT03214354) is a Phase 2 interventional studying Sickle Cell Disease and Stem Cell Transplant Complications, sponsored by University of Calgary. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

About This Trial

The aim of this study to evaluate the safety and efficacy of a nonmyeloablative conditioning regimen for allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric patients with sickle cell disease (SCD) who have a matched related major ABO-incompatible donor. The nonmyeloablative regimen will use alemtuzumab, total body irradiation (TBI) and sirolimus for immune suppression. This study will expand the access of HSCT for patients with SCD who are currently not eligible because of donor restrictions.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Sickle Cell Disease and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

With a target enrollment of 12 participants, this is a small study — typical of early-phase research, rare-disease trials, or pilot studies designed to generate preliminary signal before a larger study is launched.

Who May Be Eligible (Plain English)

Who May Qualify: - Patients must be ≥ 12 months and \< 19 years of age at the time of study enrollment. - Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows: - homozygous Hb S disease (HbSS), - sickle-Hb C disease (HbSC), - sickle beta-plus-thalassemia (HbS/β+), or - sickle beta-null-thalassemia (HbS/βo) - Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following: - history of repeated (more than 1) bony (vaso-occlusive) crisis - history of stroke - elevated transcranial Doppler velocity not eligible for hydroxyurea, as per TWiTCH trial (ie. severe vasculopathy) - history of acute chest crisis or splenic sequestration crisis - history of priapism in males - history of osteonecrosis - pulmonary hypertension as documented by tricuspid regurgitation jet velocity (TRV) \> 2.5 m/s on echocardiogram - red cell allo-immunization (≥ 2 antibodies) during long term transfusion therapy - Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes. Who Should NOT Join This Trial: - Patients who are unable to comply with or follow the study protocol. - Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components. Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language

Inclusion Criteria: * Patients must be ≥ 12 months and \< 19 years of age at the time of study enrollment. * Patients must have sickle cell disease as defined by hemoglobin electropheresis, as follows: * homozygous Hb S disease (HbSS), * sickle-Hb C disease (HbSC), * sickle beta-plus-thalassemia (HbS/β+), or * sickle beta-null-thalassemia (HbS/βo) * Patients must meet standard eligibility criteria to undergo HSCT, including but not limited to one or more of the following: * history of repeated (more than 1) bony (vaso-occlusive) crisis * history of stroke * elevated transcranial Doppler velocity not eligible for hydroxyurea, as per TWiTCH trial (ie. severe vasculopathy) * history of acute chest crisis or splenic sequestration crisis * history of priapism in males * history of osteonecrosis * pulmonary hypertension as documented by tricuspid regurgitation jet velocity (TRV) \> 2.5 m/s on echocardiogram * red cell allo-immunization (≥ 2 antibodies) during long term transfusion therapy * Sickle complications should be present despite the use of hydroxyurea, but this is not an absolute requirement, if the treating team considers the patient to be at high risk for further crisis episodes. Exclusion Criteria: * Patients who are unable to comply with or follow the study protocol. * Patients with known hypersensitivity to sirolimus, its derivatives or to any of its components.

Treatments Being Tested

DRUG

Alemtuzumab

Alemtuzumab, Day -7 to -3. Dose: 0.2mg/kg/dose SC once daily x 5 days

RADIATION

Total Body Irradiation

TBI 300 cGy on Day -2

DRUG

Sirolimus

Sirolimus is used for GVHD prophylaxis

Locations (1)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

Alberta Children's Hospital

Calgary, Alberta, Canada

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT03214354), the sponsor (University of Calgary), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT03214354 clinical trial studying?

Who can participate in NCT03214354?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT03214354?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT03214354. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT03214354. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-05-08 · Data from ClinicalTrials.gov.