Sickle Cell DiseasePhase 2HaploSCDNCT01461837
Haplo T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease
A Phase 2 study testing CD34 selected T-cell depleted allogeneic SCT in people with Sickle Cell Disease, run by New York Medical College at 6 sites in United States.
Trial summary
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease… Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- Sickle Cell Disease
- What is being tested?
- CD34 selected T-cell depleted allogeneic SCT
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 21 participants
- Where
- 6 sitesUnited States
- Study dates
- Jan 2012to Dec 2026
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
- Patients must demonstrate one or more of the following Sickle Cell Disease Complications
- Clinically significant neurologic event (stroke) or any neurologic deficit lasting >24 hours that is accompanied by an infarct on cerebral MRI
- Minimum of two episodes of acute chest syndrome.
- Recurrent painful events (at least 3 in the 2 years prior to enrollment).
- Abnormal TCD study requiring starting on chronic transfusion therapy.
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- At least one silent infarct lesion on a MRI scan of the head.
- A familial haploidentical donor without homozygous sickle cell disease
- Adequate organ function (renal, liver, cardiac and pulmonary function)
- Karnofsky or Lansky (age appropriate) Performance Score ≥50%
- Liver biopsy is optional to assess for iron overload in chronically transfused patients.
Who cannot join
- Females who are pregnant or breast-feeding
- SCD Patients with documented uncontrolled infection
- SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
- Karnofsky/Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
- Demonstrated lack of compliance with medical care.
- Clinically significant fibrosis or cirrhosis of the liver
- Previously received a HSCT
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Lurie Children's Hospital
Chicago, Illinois, 60611-2605
University of California Los Angeles (UCLA)
Los Angeles, California, 90095
Medical College of Wisconsin/Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Children's Hospital and Research Center Oakland
Oakland, California, 94609
Washington University/St. Louis Children's Hospital
St Louis, Missouri, 63110
New York Medical College
Valhalla, New York, 10595
About this study
Official title: Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.
Funding Source - FDA OOPD
Read the detailed description
The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk Sickle Cell Disease (SCD). It is hypothesized that it will be safe and well tolerated, and result in sustained donor chimerism, acceptable engraftment and immune reconstitution. Also, that it will limit SCD related organ damage resulting in improved and/or stable neurological, neurocognitive, pulmonary and pulmonary vascular function and health related quality of life (QOL).
Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.
- Conditions
- Sickle Cell Disease
- Being tested
- CD34 selected T-cell depleted allogeneic SCT
- Keywords
- sickle cell disease, stem cell transplantation, haploidentical
- Medical subject headings
- Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CD34 selected T-cell depleted allogeneic SCTDrug
Also called Familial haploidentical, T-cell depleted, allogeneic stem cell transplantation, high risk Sickle Cell Disease
Hydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0
| Study group | Role | What they receive |
|---|---|---|
| Haplo Stem Cell Transplantation | Experimental | CD34 selected T-cell depleted allogeneic SCT Receives: Drug: CD34 selected T-cell depleted allogeneic SCT |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment related events
Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease
Measured: 1 year
Secondary outcomes (3)
neurological/neurocognitive status
Change from baseline in neurological/neurocognitive status
Measured: 2 years
Pulmonary/pulmonary vascular status
Change from baseline of Pulmonary/pulmonary vascular status
Measured: 2 years
Health-related quality of life
Change from baseline of Health-related quality of life (CHRIs-HSCT/CHRIs-General)
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Oct 2011 (estimated)
- Study startJan 2012
- Primary completionDec 2025 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 21 participants (actual)
Sponsor and investigators
- Lead sponsor
- New York Medical College
- Sponsor type
- University, hospital or other
- Collaborators
- UCSF Benioff Children's Hospital Oakland, Medical College of Wisconsin, Washington University School of Medicine, Tufts Medical Center, University of California, San Francisco, University of California, Los Angeles, Miltenyi Biomedicine GmbH, Ann & Robert H Lurie Children's Hospital of Chicago
- Responsible party
- Principal investigator, Mitchell Cairo, New York Medical College
- Investigators
- Mitchell S Cairo, MD, Principal investigator, New York Medical College
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01461837
- Sponsor's protocol ID
- NYMC526-4090
- Other IDs
- FD-R-0004090
- Acronym
- HaploSCD
- Status last verified
- Aug 2025
- First submitted
- 19 Oct 2011
- Last updated
- 8 Aug 2025
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS. Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial. Front Neurol. 2024 May 22;15:1263373. doi: 10.3389/fneur.2024.1263373. eCollection 2024.
PubMed 38841694Parsons SK, Rodday AM, Weidner RA, Morris E, Braniecki S, Shenoy S, Talano JA, Moore TB, Panarella A, Flower A, Milner J, Fabricatore S, Mahanti H, van de Ven C, Shi Q, Cairo MS. Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant. Bone Marrow Transplant. 2022 Apr;57(4):586-592. doi: 10.1038/s41409-022-01584-y. Epub 2022 Feb 2.
PubMed 35110690
Study at a glance
From the registry record, last updated by the sponsor on 8 Aug 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Familial Haploidentical T-Cell Depleted Transplantation in High-Risk Sickle Cell Disease (IND 14359)
- ClinicalTrials.gov ID
- NCT01461837
- Acronym / sponsor ID
- HaploSCD / NYMC526-4090
- Conditions
- Sickle Cell Disease
- Keywords
- sickle cell disease, stem cell transplantation, haploidentical
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 21 participants (actual)
- Lead sponsor
- New York Medical CollegeUniversity, hospital or other
- Collaborators
- UCSF Benioff Children's Hospital Oakland, Medical College of Wisconsin, Washington University School of Medicine, Tufts Medical Center, University of California, San Francisco, University of California, Los Angeles, Miltenyi Biomedicine GmbH, Ann & Robert H Lurie Children's Hospital of Chicago
- Study start
- Jan 2012
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 28 Oct 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is being done to determine the safety and outcome (long-term control) of a high-dose chemotherapy regimen followed by an infusion of CD34 selected (immune cells) stem cells from a partially matched adult family member donor, called haploidentical stem cell transplantation, in high-risk sickle cell disease patients.
Funding Source - FDA OOPD
Detailed description
The purpose of this study is to investigate host myeloimmunosuppressive conditioning followed by familial haploidentical T cell depleted allogeneic stem cell transplantation in patients with high risk Sickle Cell Disease (SCD). It is hypothesized that it will be safe and well tolerated, and result in sustained donor chimerism, acceptable engraftment and immune reconstitution. Also, that it will limit SCD related organ damage resulting in improved and/or stable neurological, neurocognitive, pulmonary and pulmonary vascular function and health related quality of life (QOL).
Patients 2-20.99 years of age with a diagnosis of high-risk SCD and with an unaffected HLA partially matched family donor and meeting eligibility criteria (inclusion and exclusion criteria) are eligible.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haplo Stem Cell Transplantation | Experimental | Drug: CD34 selected T-cell depleted allogeneic SCTCD34 selected T-cell depleted allogeneic SCT |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (11 listed)
- Homozygous Hemoglobin S Disease, or Hemoglobin S Beta0/+ thalassemia
- Patients must demonstrate one or more of the following Sickle Cell Disease Complications
- A familial haploidentical donor without homozygous sickle cell disease
- Adequate organ function (renal, liver, cardiac and pulmonary function)
- Karnofsky or Lansky (age appropriate) Performance Score ≥50%
Key exclusion criteria (7 listed)
- Females who are pregnant or breast-feeding
- SCD Patients with documented uncontrolled infection
- SCD patients who have an unaffected HLA matched family donor willing to proceed to donation
- Karnofsky/Lansky (age appropriate) Performance Score <50% (hemiplegia alone secondary to a previous stroke is not an exclusion)
- Demonstrated lack of compliance with medical care.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CD34 selected T-cell depleted allogeneic SCTAlso: Familial haploidentical, T-cell depleted, allogeneic stem cell transplantation, high risk Sickle Cell DiseaseHydroxyurea (60 mg/kg/day) and azathioprine (3 mg/kg/day) day -59 to day -11; fludarabine (30 mg/m2) Days -17, -16, -15, -14, -13; busulfan (3.2 mg/kg/day) Days -12, -11, -10, -9; thiotepa (10 mg/kg IV) day -8; cyclophosphamide (50 mg/kg) Days -7, -6, -5, -4; TLI on day -3; rabbit ATG (2.0 mg/kg/day) day -5,-4,-3, and -2; Stem Cell infusion day 0 | Drug | Haplo Stem Cell Transplantation |
Primary outcome
Treatment related events
Time frame: 1 year
Death, primary or late graft rejection, or recurrence of disease and acceptable rate of hematopoietic engraftment, acute and chronic graft-versus-host disease
3 secondary outcomes
neurological/neurocognitive status
Time frame: 2 years
Pulmonary/pulmonary vascular status
Time frame: 2 years
Health-related quality of life
Time frame: 4 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Aug 2025.
- Derived from this study: PubMed 38841694Braniecki S, Vichinsky E, Walters MC, Shenoy S, Shi Q, Moore TB, Talano JA, Parsons SK, Flower A, Panarella A, Fabricatore S, Morris E, Mahanti H, Milner J, McKinstry RC, Duncan CN, van de Ven C, Cairo MS. Neurocognitive outcome in children with sickle cell disease after myeloimmunoablative conditioning and haploidentical hematopoietic stem cell transplantation: a non-randomized clinical trial. Front Neurol. 2024 May 22;15:1263373. doi: 10.3389/fneur.2024.1263373. eCollection 2024.
- Derived from this study: PubMed 35110690Parsons SK, Rodday AM, Weidner RA, Morris E, Braniecki S, Shenoy S, Talano JA, Moore TB, Panarella A, Flower A, Milner J, Fabricatore S, Mahanti H, van de Ven C, Shi Q, Cairo MS. Significant improvement of child physical and emotional functioning after familial haploidentical stem cell transplant. Bone Marrow Transplant. 2022 Apr;57(4):586-592. doi: 10.1038/s41409-022-01584-y. Epub 2022 Feb 2.
- Consortium website for Parent-to-Child (haplo) Bone Marrow Transplant for Sickle Cell Disease (SCD)Link provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mitchell S Cairo, MD principal investigator
New York Medical College
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CD34 selected T-cell depleted allogeneic SCT in people with Sickle Cell Disease, run by New York Medical College at 6 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 8 Aug 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment related events (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01461837, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Aug 2025.