Sickle Cell DiseasePhase 1NCT03249831
A Blood Stem Cell Transplant for Sickle Cell Disease
A Phase 1 study testing Cyclophosphamide, Pentostatin and Rabbit anti-thymocyte globulin in people with Sickle Cell Disease and Sickle Cell Disorder, run by City of Hope Medical Center at 1 site in United States.
Trial summary
Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies
- What is being tested?
- Cyclophosphamide, Pentostatin and Rabbit anti-thymocyte globulin
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 3 participants
- Where
- 1 siteUnited States
- Study dates
- 4 Jan 2019to 25 Jan 2027
- Phase
- Phase 1
- 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 18 to 45 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- Confirmed diagnosis of hemoglobin SS or S-βº Thalassemia sickle cell disease
- Severe disease status as defined by presence of one or more of the following:
- Clinically significant neurologic event (stroke) or any neurological deficit lasting > 24 hours; or increased transcranial Doppler velocity (>200 m/s). A stroke is defined as a sudden neurologic change lasting more than 24 hours that is accompanied by cerebral magnetic resonance imaging (MRI) changes.
- History of ≥ 1 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 ≥ 2 severe vaso-occlusive pain crises (VOC) 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). A severe VOC is defined as an episode of pain lasting more than 2 hours severe enough to require care at a medical facility. Note that priapism that lasts more than 2 hours and requires care at a medical facility is also considered a VOC.
- Osteonecrosis of ≥ 2 joints despite the institution of supportive care measures.
Show all 14Show fewer
- Prior treatment with regular RBC transfusion therapy, defined as receiving ≥ 8 transfusions per year for > 1 year to prevent vaso-occlusive clinical complications (i.e. pain, stroke, and acute chest syndrome)
- No HLA matched sibling or 10/10 matched unrelated donor
- Related donor who:
- Is genotypically haploidentical on HLA-A, B, C and DRB1 loci AND
- Meets institutional criteria
- Failed prior hydroxyurea therapy or have intolerance to hydroxyurea
- Meets protocol specified organ function criteria
- Women of childbearing potential or sexually active male: Agreement to use adequate contraception prior to study entry and 6 months post-transplant.
Who cannot join
- Prior stem cell transplant
- Prior bone marrow transplant
- Concurrent other investigational agents, chemotherapy, biological therapy or radiation therapy
- Planned use of moderate and strong CYP3A4 inhibitors
- Active infection
- Major surgery within the last 30 days
Show all 10Show fewer
- Clinically significant liver fibrosis or cirrhosis if on chronic transfusion therapy > 6 months
- Active malignancy (other than non-melanoma skin cancers)
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to any in the pre- or post-transplant regimen.
- Women of childbearing potential: pregnant or breastfeeding
Trial locations and contacts
1 site in 1 country.
United States1 site
City of Hope Medical Center
Duarte, California, 91010
About this study
Official title: Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot).
Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism.
Read more
Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor).
Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications.
This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because:
- Half-matched related donors will be used, and
- A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and
- Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes.
It is hoped that the research transplant:
- Will reverse sickle cell disease and improve patient quality of life,
- Will reduce side effects and help the patient recover faster from the transplant,
- Help the patient keep the transplant longer and
- Reduce serious transplant-related complications.
Read the detailed description
This is a pilot study to determine the safety and feasibility of the COH-MC-17 regimen and ability of the regimen to induce a mixed chimeric status in severe sickle cell disease patients (hemoglobin SS or S-βº Thalassemia). The COH-MC-17 regimen consists of a non-myeloablative regimen (cyclophosphamide, pentostatin and rabbit-anti-thymocyte globulin (ATG)) followed by a CD4+ T-cell-depleted haploidentical hematopoietic cell transplant (HaploHCT).
- Conditions
- Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
- Being tested
- Cyclophosphamide, Pentostatin, Rabbit anti-thymocyte globulin, Tacrolimus, Mycophenolate mofetil
- Keywords
- Sickle Cell Disease, Sickle Cell Disorders, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell, Haploidentical Transplant, Nonmyeloablative Conditioning, CD4+ T cell, CD4+ T cell-depleted Hematopoietic Cell Transplant, Mixed Chimerism
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobinopathies, Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Cytoxan
Orally daily
PentostatinDrug
Also called NIPENT
Intravenous
Rabbit anti-thymocyte globulinDrug
Also called Rabbit ATG, Thymoglobulin
Intravenous
TacrolimusDrug
Also called PROGRAF®
Initially IV. If patient tolerates, convert to oral.
Also called MMF, CellCept®, Myfortic
IV or oral
CD4+ T-cell-depleted Haploidentical Hematopoietic TransplantBiological
Also called CD4+ T-cell depleted HaploHCT, CD4+ T-cell depleted hematopoietic progenitor cell (HPC) product
Infusion
| Study group | Role | What they receive |
|---|---|---|
| COH-MC-17 and immunosuppressants | Experimental | Participants receive COH-MC-17: a 21-day nonmyeloablative conditioning regimen (cyclophosphamide, pentostatin and rabbit anti-thymocyte globulin), followed by CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant on Day 0.
Immunosuppressants (tacrolimus and mycophenolate mofetil) given on Day -1 onwards until discontinuation post-transplant.
The minimally manipulated transplant product is manufactured using the CliniMACS device. Receives: Drug: Cyclophosphamide; Drug: Pentostatin; Drug: Rabbit anti-thymocyte globulin; Drug: Tacrolimus; Drug: Mycophenolate mofetil; Biological: CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Toxicity per NCI-Common Terminology Criteria for Adverse Events version 4.0
Measured: Day -22 to 2 years post-transplant
Unacceptable Toxicity at least possibly related to COH-MC-17
Measured: Day -22 to Day +60 post-transplant
Mixed Chimerism defined as 30-90% donor cells
Measured: Day +60 post-transplant
Feasibility of producing an infusion ready CD4+ T-cell-depleted hematopoietic product
Measured: From apheresis to Day 0
Secondary outcomes (20)
Adverse events of Grade 3 or higher
Measured: Up to 2 years post-transplant
Neutrophil count ≥ 500/mm3, time to recovery
Measured: Up to 2 years post-transplant
Platelet count ≥ 20,000/mm3, time to recovery
Measured: Up to 2 years post-transplant
Marrow failure
Measured: Up to 2 years post-transplant
Sickle cell disease related complications
Measured: Up to 2 years post-transplant
Non-relapse mortality
Measured: Up to 2 years post-transplant
Acute Graft versus Host Disease per 1994 Keystone Consensus Criteria
Measured: Day + 100 post-transplant
Chronic Graft versus Host Disease per 2014 National Institutes of Health Consensus Criteria
Measured: Day+ 180, + 1 year and +2 years post-transplant
Overall Survival
Measured: Up to 2 years post-transplant
Disease-Free Survival
Measured: Up to 2 years post-transplant
Event-Free Survival
Measured: Up to 2 years post-transplant
Disease Relapse
Measured: Up to 2 years post-transplant
Persistent post-immunosuppressant mixed chimerism
Between 5% and 95% donor chimerism at two years post- transplant, at least 6 months post- immunosuppressant
Measured: Up to 2 years post-transplant
Persistent immunosuppressant -dependent mixed chimerism
Between 5% and 95% donor chimerism at two years post- transplant and on immunosuppressant
Measured: +2 years post-transplant
Complete chimerism: >95% donor chimerism
Measured: +2 years post-transplant
Primary donor graft failure: Defined as < 5% donor chimerism by Day + 30 post- transplant
Measured: Day +30 post-transplant
Secondary donor graft failure: Defined as < 5% donor chimerism beyond Day +30 in patients with prior documentation of ≥ 5% donor cells by Day +30
Measured: > Day + 30 up to 2 years post-transplant
Donor chimerism in blood
Measured: Day +30, Day +60, Day +100, Day+180, and +1 yr, +1.5 yr, +2yr post-transplant
Donor chimerism in bone marrow
Measured: Day + 100, Day + 180 and + 1 yr post-transplant
Percent HbS levels
Measured: Baseline, and then Day + 30, Day + 100, Day + 180, +1 yr, +1.5, +2yr post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2017
- Study start4 Jan 2019
- Primary completion25 Jan 2027 (estimated)
- Study completion25 Jan 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
- More trials run by City of Hope Medical CenterThe sponsor's other registered studies.
- 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 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- City of Hope Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Responsible party
- Sponsor
- Investigators
- Joseph Rosenthal, MD, Principal investigator, City of Hope Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03249831
- Sponsor's protocol ID
- 16453
- Status last verified
- Mar 2026
- First submitted
- 10 Aug 2017
- Last updated
- 5 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Evaluate the Safety and Feasibility of Induction of Mixed Chimerism in Sickle Cell Disease Patients With COH-MC-17: a Non-Myeloablative, Conditioning Regimen and CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant
- ClinicalTrials.gov ID
- NCT03249831
- Acronym / sponsor ID
- 16453
- Conditions
- Sickle Cell Disease, Sickle Cell Disorder, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell
- Keywords
- Sickle Cell Disease, Sickle Cell Disorders, Hemoglobinopathies, Thalassemia, Anemia, Sickle Cell, Haploidentical Transplant, Nonmyeloablative Conditioning, CD4+ T cell
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 3 participants (actual)
- Lead sponsor
- City of Hope Medical CenterUniversity, hospital or other
- Collaborators
- California Institute for Regenerative Medicine (CIRM)
- Study start
- 4 Jan 2019 (actual)
- Primary completion
- 25 Jan 2027 (estimated)
- Study completion
- 25 Jan 2027 (estimated)
- First posted
- 15 Aug 2017 (actual)
- 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.
Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot).
Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism.
Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor).
Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications.
This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because:
- Half-matched related donors will be used, and
- A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and
- Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes.
It is hoped that the research transplant:
- Will reverse sickle cell disease and improve patient quality of life,
- Will reduce side effects and help the patient recover faster from the transplant,
- Help the patient keep the transplant longer and
- Reduce serious transplant-related complications.
Detailed description
This is a pilot study to determine the safety and feasibility of the COH-MC-17 regimen and ability of the regimen to induce a mixed chimeric status in severe sickle cell disease patients (hemoglobin SS or S-βº Thalassemia). The COH-MC-17 regimen consists of a non-myeloablative regimen (cyclophosphamide, pentostatin and rabbit-anti-thymocyte globulin (ATG)) followed by a CD4+ T-cell-depleted haploidentical hematopoietic cell transplant (HaploHCT).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| COH-MC-17 and immunosuppressants | Experimental | Drug: Cyclophosphamide, Drug: Pentostatin, Drug: Rabbit anti-thymocyte globulin, Drug: Tacrolimus, Drug: Mycophenolate mofetil, Biological: CD4+ T-cell-depleted Haploidentical Hematopoietic TransplantParticipants receive COH-MC-17: a 21-day nonmyeloablative conditioning regimen (cyclophosphamide, pentostatin and rabbit anti-thymocyte globulin), followed by CD4+ T-cell-depleted Haploidentical Hematopoietic Transplant on Day 0. Immunosuppressants (tacrolimus and mycophenolate mofetil) given on Day -1 onwards until discontinuation post-transplant. The minimally manipulated transplant product is manufactured using the CliniMACS device. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (14 listed)
- Confirmed diagnosis of hemoglobin SS or S-βº Thalassemia sickle cell disease
- Severe disease status as defined by presence of one or more of the following:
- No HLA matched sibling or 10/10 matched unrelated donor
- Related donor who:
- Failed prior hydroxyurea therapy or have intolerance to hydroxyurea
Key exclusion criteria (10 listed)
- Prior stem cell transplant
- Prior bone marrow transplant
- Concurrent other investigational agents, chemotherapy, biological therapy or radiation therapy
- Planned use of moderate and strong CYP3A4 inhibitors
- Active infection
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 |
|---|---|---|
| CyclophosphamideAlso: CytoxanOrally daily | Drug | COH-MC-17 and immunosuppressants |
| PentostatinAlso: NIPENTIntravenous | Drug | COH-MC-17 and immunosuppressants |
| Rabbit anti-thymocyte globulinAlso: Rabbit ATG, ThymoglobulinIntravenous | Drug | COH-MC-17 and immunosuppressants |
| TacrolimusAlso: PROGRAF®Initially IV. If patient tolerates, convert to oral. | Drug | COH-MC-17 and immunosuppressants |
| Mycophenolate mofetilAlso: MMF, CellCept®, MyforticIV or oral | Drug | COH-MC-17 and immunosuppressants |
| CD4+ T-cell-depleted Haploidentical Hematopoietic TransplantAlso: CD4+ T-cell depleted HaploHCT, CD4+ T-cell depleted hematopoietic progenitor cell (HPC) productInfusion | Biological | COH-MC-17 and immunosuppressants |
Primary outcome
Toxicity per NCI-Common Terminology Criteria for Adverse Events version 4.0
Time frame: Day -22 to 2 years post-transplant
Unacceptable Toxicity at least possibly related to COH-MC-17
Time frame: Day -22 to Day +60 post-transplant
Mixed Chimerism defined as 30-90% donor cells
Time frame: Day +60 post-transplant
Feasibility of producing an infusion ready CD4+ T-cell-depleted hematopoietic product
Time frame: From apheresis to Day 0
20 secondary outcomes
Adverse events of Grade 3 or higher
Time frame: Up to 2 years post-transplant
Neutrophil count ≥ 500/mm3, time to recovery
Time frame: Up to 2 years post-transplant
Platelet count ≥ 20,000/mm3, time to recovery
Time frame: Up to 2 years post-transplant
Marrow failure
Time frame: Up to 2 years post-transplant
Sickle cell disease related complications
Time frame: Up to 2 years post-transplant
Non-relapse mortality
Time frame: Up to 2 years post-transplant
Acute Graft versus Host Disease per 1994 Keystone Consensus Criteria
Time frame: Day + 100 post-transplant
Chronic Graft versus Host Disease per 2014 National Institutes of Health Consensus Criteria
Time frame: Day+ 180, + 1 year and +2 years post-transplant
Overall Survival
Time frame: Up to 2 years post-transplant
Disease-Free Survival
Time frame: Up to 2 years post-transplant
Event-Free Survival
Time frame: Up to 2 years post-transplant
Disease Relapse
Time frame: Up to 2 years post-transplant
Persistent post-immunosuppressant mixed chimerism
Time frame: Up to 2 years post-transplant
Persistent immunosuppressant -dependent mixed chimerism
Time frame: +2 years post-transplant
Complete chimerism: >95% donor chimerism
Time frame: +2 years post-transplant
Primary donor graft failure: Defined as < 5% donor chimerism by Day + 30 post- transplant
Time frame: Day +30 post-transplant
Secondary donor graft failure: Defined as < 5% donor chimerism beyond Day +30 in patients with prior documentation of ≥ 5% donor cells by Day +30
Time frame: > Day + 30 up to 2 years post-transplant
Donor chimerism in blood
Time frame: Day +30, Day +60, Day +100, Day+180, and +1 yr, +1.5 yr, +2yr post-transplant
Donor chimerism in bone marrow
Time frame: Day + 100, Day + 180 and + 1 yr post-transplant
Percent HbS levels
Time frame: Baseline, and then Day + 30, Day + 100, Day + 180, +1 yr, +1.5, +2yr post-transplant
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 5 Mar 2026.
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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.
Joseph Rosenthal, MD principal investigator
City of Hope Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Cyclophosphamide, Pentostatin and Rabbit anti-thymocyte globulin in people with Sickle Cell Disease and Sickle Cell Disorder, run by City of Hope Medical Center at 1 site 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 14 inclusion and 10 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 5 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
Toxicity per NCI-Common Terminology Criteria for Adverse Events version 4.0 (time frame: Day -22 to 2 years post-transplant). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 5 Mar 2026.