Sickle Cell AnemiaNCT01350232
Treatment of Sickle Cell Anemia With Stem Cell Transplant
A study testing Fludarabine, Cytarabine and Cellular Infusions in people with Sickle Cell Anemia and Sickle Cell-hemoglobin C Disease, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University at 1 site in United States.
Trial summary
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. Read more about this study
- Who is it for?
- Ages 18 to 45 years
- Conditions the study is for
- Sickle Cell Anemia, Sickle Cell-hemoglobin C Disease, Sickle Cell-β0-thalassemia
- What is being tested?
- Fludarabine, Cytarabine and Cellular Infusions
- Type of treatment
- Vaccines, Injection or infusion, Medical device, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2009to Aug 2011
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Patient Selection:
- Patients with sickle cell disease (sickle cell anemia, sickle cell-hemoglobin C disease, or sickle cell-β0-thalassemia) confirmed by hemoglobin electrophoresis.
- Patients should have one or more of the following:
- History of acute chest syndrome requiring recurrent hospitalization or exchange transfusion (Acute chest syndrome is defined as pulmonary infiltrate involving at least one complete lung segment, consistent with alveolar consolidation but not atelectasis, accompanied by chest pain, fever, cough, tachypnea or wheezing)
- History of nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours
- Recurrent vaso-occlusive pain (≥5 episodes during the past two years) or recurrent priapism requiring hospitalization or visits to the emergency room or sickle cell day unit
Show all 10Show fewer
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50% of normal predicted value) with progression on ACE inhibitor therapy iii) Patient must have failed therapy with hydroxyurea, as HU as evidenced by at least 6 months of maximum HU dosage for sickle cell disease, i.e. dose escalation to a level which caused some minimal hematologic toxicity in terms of CBC values. Failure to respond must also be documented by no significant increase in subjects HbF levels at this maximally tolerated dosage.AND development/ persistence of items listed in (ii) Patients who are deemed not eligible for hydroxyurea by the primary hematologist will be considered eligible without having failed hydroxyurea. Non-eligibility for hydroxyurea therapy is based on:
- the diagnoses of SC disease and sickle cell-β0-thalassemia in which no clear evidence supports the use of hydroxyurea therapy and thus treatment with hydroxyurea is not considered the standard of care in these entities (2) the presence of high hemoglobin F levels in patients with sickle cell anemia and documented Hereditary Persistence of Fetal Hemoglobin (HPFH) in which hydroxyurea is not considered the standard of care (3) severe adverse reactions to hydroxyurea in patients with sickle cell anemia based on, but not limited to, count suppression, GI intolerance, and dermatomyositis Patient unwillingness to be compliant with hydroxyurea therapy is not an acceptable reason for non-eligibility iv) Patients must have an acceptable related donor
- who is matched at the HLA-A;B; C; DR loci (8 of 8 match) or mismatched for at most one locus (7 of 8 match) (well matched related donor
- who is mismatched at 2-4 alleles (haplo-identical) v) Patient age greater than 18 - 45 years vi) ECOG performance status 0-2/ Karnofsky 70-100% vii) Written informed consent obtained from the patient. viii) Transaminases <3X ULN; patients with transaminases greater than the ULN but less than 3XULN will be evaluated by the hepatology service and will undergo further imaging and biopsy as deemed necessary by hepatology. They will not be considered eligible unless cleared by hepatology.
Who cannot join
- Patient Selection:
- Pregnancy/ unwillingness to use adequate contraception during study period ii) Liver disease including
- Acute hepatitis (transaminases >3x normal value)
- Chronic hepatitis C
- Chronic hepatitis B or history of exposure to hepatitis B iii) Cardiac ejection fraction < 50% iv) Pulmonary hypertension - as evidenced by findings on resting echocardiogram of pulmonary artery systolic pressure ≥ 40 mmHg or any evidence of right ventricular dysfunction (hypokinesis or RV dilation) v) Severe renal impairment (GFR <30% of predicted normal value) vi) Severe residual functional neurologic impairment (other than hemiplegia alone) vii) DLCO ≤50 viii) Any evidence of infection by the human immunodeficiency virus ix) Psychiatric disorder that would preclude patients from signing an informed consent x) Severe neuro-cognitive or executive function making informed consent possible
Trial locations and contacts
1 site in 1 country.
United States1 site
Thomas Jefferson University
Philadelphia, Pennsylvania, 19107
About this study
Official title: Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Anemia From HLA Matched or Partially-Matched Related Donors
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU).
The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia.
Read the detailed description
Hemoglobinopathies, such as sickle cell disease and thalassemia major, constitute a group of genetic diseases associated with significant morbidity and premature death. In the 1970s, the mean survival of patients with sickle cell disease was 14.3 years. With improvements in medical practice, this has improved such that estimates are now into the third decade of life.
In patients with sickle cell disease, a single amino acid substitution in beta-hemoglobin causes erythrocytes to sickle in response to oxidative stress. The sequelae of this defect are vaso-occlusive crises, resulting in episodes of bony pain and infarction, acute chest syndrome, and strokes. Life long need for transfusion leads to complications including alloimmunization and iron overload. The latter condition is frequently associated with significant end-organ damage.
In recent years, new strategies in supportive care, such as the use of hydroxyurea to stimulate fetal hemoglobin production in patients with sickle cell anemia, have resulted in the amelioration of some of the devastating manifestations of this disease. However, this therapy does not benefit all patients, and there have been concerns about the possible risk of latent transformation to leukemia with prolonged use of this drug. Clearly, better treatment strategies are needed for this devastating group of diseases.
Patients with sickle cell anemia will be offered enrollment on a clinical trial of reduced intensity stem cell transplant. The transplant donors will be either HLA matched siblings or family members who are 50% matched for HLA. Patients will receive therapy in 2 steps.
For patients who are allo-immunized against the donor (patients who have made an immune response already against the donor's HLA type), there will be a desensitization process. This will be outpatient therapy and will include therapy with bortezomib on the 1st, 4th, 8th and 11th day of a 21 day cycle. This will be repeated for a second cycle, for a total of 8 doses of bortezomib over a 6 week period. In addition, they will receive rituximab on the 1st and 8th day of each cycle. These therapies are designed to decrease the subject's chance of rejecting the transplant, as it is known that patients with sickle cell anemia are likely to be heavily immunized against donors. For patients who have high levels of antibodies against the donors, a plasmapheresis procedure will be performed prior to admission as well. All patients will undergo red cell exchange prior to admission.
During the transplant admission, subjects will receive a "Two Step" chemotherapy and transplant regimen. The chemotherapy "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells.
- Being tested
- Fludarabine, Cytarabine, Cellular Infusions, Total Body Irradiation, Cyclophosphamide
- Keywords
- sickle cell, SS disease, SC disease, S-thalassemia, transplant, bone marrow transplant, stem cell transplant, matched sibling, haploidentical, hemoglobinopathy, anemia, desensitization
- Medical subject headings
- Anemia, Sickle Cell, Hemoglobin SC Disease, Hemoglobinopathies, Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FludarabineDrug
Also called Fludara
Subjects will receive fludarabine at a dose of 30 mg/m2 daily for 4 days as part of the preparative regimen
CytarabineDrug
Also called Ara-C, Cytosar, Cytosine arabinoside
Subjects will receive cytarabine at a dose of 2 g/m2 daily for 4 days, approximately 4 hours after the fludarabine
Cellular InfusionsDevice
Also called Stem Cells, DLI, HPCT
Subjects will receive the cellular product in 2 steps. The first is a lymphocyte infusion of 2 X 10e8 lymphocytes/kg on the day they receive total body irradiation. The second is a CD34 enriched stem cell product approximately 48 hours after the cyclophosphamide.
The CliniMACS® Plus Instrument will be used for the selection of human CD34+ hematopoietic stem and progenitor cells in human allogeneic hematopoietic stem cell transplantation.
Total Body IrradiationRadiation
Also called TBI
All subjects will receive 200cGy TBI in a single fraction on the AM they receive the lymphocyte infusion
CyclophosphamideDrug
Also called Cytoxan
All subjects will receive cyclophosphamide at a dose of 60 mg/kg at approximately 72 and 96 hours after the lymphocyte infusion
BortezomibDrug
Also called Velcade
Subjects will receive bortezomib 1.3 mg/kg on Day 1,4,8, and 11 of a 21 day cycle. This will be repeated for 2 cycles.
RituximabDrug
Also called Rituxan
Subjects will receive rituximab 375 mg/m2 on day 1 and day 8 of a 21 day cycle, on days they will also be receiving rituximab. This will be repeated for 2 cycles.
PlasmapheresisProcedure or surgery
Also called Plasma exchange
Subjects who continue to have detectable anti-donor antibody will receive plasmapheresis to reduce the antibody further
| Study group | Role | What they receive |
|---|---|---|
| HSCT | Experimental | Subjects receive the preparative regimen in 2 steps. The "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells.
Only subjects with prior alloimmunization against donor will receive desensitization. Subjects who demonstrate alloimmunization against the HLA of the donor will receive bortezomib and rituximab in combination with plasmapheresis prior to the admission for transplant. Receives: Drug: Fludarabine; Drug: Cytarabine; Device: Cellular Infusions; Radiation: Total Body Irradiation; Drug: Cyclophosphamide; Drug: Bortezomib; Drug: Rituximab; Procedure: Plasmapheresis |
What is being measured
The primary outcome is the main question the study is designed to answer.
Stable Engraftment
To determine if the reduced intensity preparative regimen of fludarabine, cytarabine, cyclophosphamide and low-dose total body irradiation will generate stable engraftment with donor hematopoietic stem cells in at least 80% of patients with severe sickle cell anemia.
Measured: 180 days post-infusion
Secondary outcomes (7)
Organ Toxicity
To assess organ toxicity related to fludarabine, cytarabine, cyclophosphamide and low-dose total body irradiation in a population with severe sickle cell anemia.
Measured: 30 days post infusion
Overall Survival
To determine the overall survival at 6 months post-transplant in patients receiving a matched or partially-matched related donor transplant after reduced-intensity conditioning.
Measured: 6 months post infusion
Acute Graft Versus Host Disease
To describe the incidence and severity of acute and chronic GVHD following this reduced intensity transplant from partially matched related donors using a combination of cyclophosphamide, tacrolimus and mycophenolate mofetil (MMF) as GVHD prophylaxis.
Measured: 100 days post infusion
Correction of Hemoglobinopathy
To evaluate the extent of correction of hemoglobinopathy following this reduced intensity transplant.
Measured: 100 days post infusion through 5 years post infusion
Immune Recovery
To assess the pace of lymphoid recovery and associated risk for opportunistic infections and relapse (return to recipient erythropoiesis) in this patient population.
Measured: 100 days post infusion through 5 years post infusion
Quality of Life
To describe the quality of life and functional status following transplantation.
Measured: Through 5 years post infusion
Cytokine Profile
To characterize the profiles of cytokines released following administration of the lymphoid portion of the transplant (donor lymphocyte infusion [DLI]).
Measured: Through 5 years after infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2011 (estimated)
- Study startSep 2009
- Primary completionAug 2011
- Study completionAug 2011
Why it stopped: Poor accrual
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Sickle Cell AnemiaStudies 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University
- Sponsor type
- University, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Responsible party
- Sponsor
- Investigators
- Joanne Filicko-O'Hara, MD, Principal investigator, Thomas Jefferson University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01350232
- Sponsor's protocol ID
- 09F.327
- Other IDs
- 1-RC2HL101496-0, JT 1526, 1RC2HL101496
- Status last verified
- May 2025
- First submitted
- 4 May 2011
- Last updated
- 16 May 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 16 May 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reduced Intensity Allogeneic Hematopoietic Stem Cell Transplantation for Sickle Cell Anemia From HLA Matched or Partially-Matched Related Donors
- ClinicalTrials.gov ID
- NCT01350232
- Acronym / sponsor ID
- 09F.327
- Keywords
- sickle cell, SS disease, SC disease, S-thalassemia, transplant, bone marrow transplant, stem cell transplant, matched sibling
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Poor accrual
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson UniversityUniversity, hospital or other
- Collaborators
- National Heart, Lung, and Blood Institute (NHLBI)
- Study start
- Sep 2009
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 9 May 2011 (estimated)
- Results
- Posted 20 Nov 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a clinical research trial in which a novel preparatory regimen was developed for bone marrow transplant (BMT) which eliminates the primary obstacle to transplant, the lack of a matched sibling donor. It is believed this regimen is sufficiently efficacious and sufficiently gentle to apply to patients with sickle cell anemia and related disorders. It is proposed to characterize the efficacy and toxicity of this regimen in high risk patients with sickle cell anemia using criteria for patient selection that have been accepted in prior BMT trials in patients with sickle cell disease, specifically only the subset of patients whose prior clinical behavior indicates that they are at high risk for serious morbidity and early mortality. In addition, it is proposed to characterize the pathophysiology of a consistent febrile response seen in the haploidentical BMT regimen the investigators have developed at Thomas Jefferson University (TJU).
The primary goal of this study is to determine the response rate to a reduced intensity conditioning regimen which consists of fludarabine, cytarabine, low dose total body irradiation and cyclophosphamide in patients with severe sickle cell anemia.
Detailed description
Hemoglobinopathies, such as sickle cell disease and thalassemia major, constitute a group of genetic diseases associated with significant morbidity and premature death. In the 1970s, the mean survival of patients with sickle cell disease was 14.3 years. With improvements in medical practice, this has improved such that estimates are now into the third decade of life.
In patients with sickle cell disease, a single amino acid substitution in beta-hemoglobin causes erythrocytes to sickle in response to oxidative stress. The sequelae of this defect are vaso-occlusive crises, resulting in episodes of bony pain and infarction, acute chest syndrome, and strokes. Life long need for transfusion leads to complications including alloimmunization and iron overload. The latter condition is frequently associated with significant end-organ damage.
In recent years, new strategies in supportive care, such as the use of hydroxyurea to stimulate fetal hemoglobin production in patients with sickle cell anemia, have resulted in the amelioration of some of the devastating manifestations of this disease. However, this therapy does not benefit all patients, and there have been concerns about the possible risk of latent transformation to leukemia with prolonged use of this drug. Clearly, better treatment strategies are needed for this devastating group of diseases.
Patients with sickle cell anemia will be offered enrollment on a clinical trial of reduced intensity stem cell transplant. The transplant donors will be either HLA matched siblings or family members who are 50% matched for HLA. Patients will receive therapy in 2 steps.
For patients who are allo-immunized against the donor (patients who have made an immune response already against the donor's HLA type), there will be a desensitization process. This will be outpatient therapy and will include therapy with bortezomib on the 1st, 4th, 8th and 11th day of a 21 day cycle. This will be repeated for a second cycle, for a total of 8 doses of bortezomib over a 6 week period. In addition, they will receive rituximab on the 1st and 8th day of each cycle. These therapies are designed to decrease the subject's chance of rejecting the transplant, as it is known that patients with sickle cell anemia are likely to be heavily immunized against donors. For patients who have high levels of antibodies against the donors, a plasmapheresis procedure will be performed prior to admission as well. All patients will undergo red cell exchange prior to admission.
During the transplant admission, subjects will receive a "Two Step" chemotherapy and transplant regimen. The chemotherapy "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HSCT | Experimental | Drug: Fludarabine, Drug: Cytarabine, Device: Cellular Infusions, Radiation: Total Body Irradiation, Drug: Cyclophosphamide, Drug: Bortezomib, Drug: Rituximab, Procedure: PlasmapheresisSubjects receive the preparative regimen in 2 steps. The "first step" will be with fludarabine and cytarabine and a low dose of total body irradiation. This will be followed by the "first step" of the transplant graft - the donor lymphocytes. The "second step" of the chemotherapy will be two doses of cyclophosphamide. This will then be followed by the "second step" of the transplant graft - the stem cells. Only subjects with prior alloimmunization against donor will receive desensitization. Subjects who demonstrate alloimmunization against the HLA of the donor will receive bortezomib and rituximab in combination with plasmapheresis prior to the admission for transplant. |
Who may qualify
- Ages
- 18 to 45 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (10 listed)
- Patients with sickle cell disease (sickle cell anemia, sickle cell-hemoglobin C disease, or sickle cell-β0-thalassemia) confirmed by hemoglobin electrophoresis.
- Patients should have one or more of the following:
- History of acute chest syndrome requiring recurrent hospitalization or exchange transfusion (Acute chest syndrome is defined as pulmonary infiltrate involving at least one complete lung segment, consistent with alveolar consolidation but not atelectasis, accompanied by chest pain, fever, cough, tachypnea or wheezing)
- History of nonhemorrhagic stroke or central nervous system event lasting longer than 24 hours
- Recurrent vaso-occlusive pain (≥5 episodes during the past two years) or recurrent priapism requiring hospitalization or visits to the emergency room or sickle cell day unit
Key exclusion criteria (5 listed)
- Pregnancy/ unwillingness to use adequate contraception during study period ii) Liver disease including
- Acute hepatitis (transaminases >3x normal value)
- Chronic hepatitis C
- Chronic hepatitis B or history of exposure to hepatitis B iii) Cardiac ejection fraction < 50% iv) Pulmonary hypertension - as evidenced by findings on resting echocardiogram of pulmonary artery systolic pressure ≥ 40 mmHg or any evidence of right ventricular dysfunction (hypokinesis or RV dilation) v) Severe renal impairment (GFR <30% of predicted normal value) vi) Severe residual functional neurologic impairment (other than hemiplegia alone) vii) DLCO ≤50 viii) Any evidence of infection by the human immunodeficiency virus ix) Psychiatric disorder that would preclude patients from signing an informed consent x) Severe neuro-cognitive or executive function making informed consent possible
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 |
|---|---|---|
| FludarabineAlso: FludaraSubjects will receive fludarabine at a dose of 30 mg/m2 daily for 4 days as part of the preparative regimen | Drug | HSCT |
| CytarabineAlso: Ara-C, Cytosar, Cytosine arabinosideSubjects will receive cytarabine at a dose of 2 g/m2 daily for 4 days, approximately 4 hours after the fludarabine | Drug | HSCT |
| Cellular InfusionsAlso: Stem Cells, DLI, HPCTSubjects will receive the cellular product in 2 steps. The first is a lymphocyte infusion of 2 X 10e8 lymphocytes/kg on the day they receive total body irradiation. The second is a CD34 enriched stem cell product approximately 48 hours after the cyclophosphamide. The CliniMACS® Plus Instrument will be used for the selection of human CD34+ hematopoietic stem and progenitor cells in human allogeneic hematopoietic stem cell transplantation. | Device | HSCT |
| Total Body IrradiationAlso: TBIAll subjects will receive 200cGy TBI in a single fraction on the AM they receive the lymphocyte infusion | Radiation | HSCT |
| CyclophosphamideAlso: CytoxanAll subjects will receive cyclophosphamide at a dose of 60 mg/kg at approximately 72 and 96 hours after the lymphocyte infusion | Drug | HSCT |
| BortezomibAlso: VelcadeSubjects will receive bortezomib 1.3 mg/kg on Day 1,4,8, and 11 of a 21 day cycle. This will be repeated for 2 cycles. | Drug | HSCT |
| RituximabAlso: RituxanSubjects will receive rituximab 375 mg/m2 on day 1 and day 8 of a 21 day cycle, on days they will also be receiving rituximab. This will be repeated for 2 cycles. | Drug | HSCT |
| PlasmapheresisAlso: Plasma exchangeSubjects who continue to have detectable anti-donor antibody will receive plasmapheresis to reduce the antibody further | Procedure or surgery | HSCT |
Primary outcome
Stable Engraftment
Time frame: 180 days post-infusion
To determine if the reduced intensity preparative regimen of fludarabine, cytarabine, cyclophosphamide and low-dose total body irradiation will generate stable engraftment with donor hematopoietic stem cells in at least 80% of patients with severe sickle cell anemia.
7 secondary outcomes
Organ Toxicity
Time frame: 30 days post infusion
Overall Survival
Time frame: 6 months post infusion
Acute Graft Versus Host Disease
Time frame: 100 days post infusion
Correction of Hemoglobinopathy
Time frame: 100 days post infusion through 5 years post infusion
Immune Recovery
Time frame: 100 days post infusion through 5 years post infusion
Quality of Life
Time frame: Through 5 years post infusion
Cytokine Profile
Time frame: Through 5 years after infusion
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 May 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Thomas Jefferson University HospitalsLink provided by the sponsor.
- Kimmel Cancer Center at Thomas Jefferson University, an NCI-Designated Cancer CenterLink 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.
Joanne Filicko-O'Hara, MD principal investigator
Thomas Jefferson University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Fludarabine, Cytarabine and Cellular Infusions in people with Sickle Cell Anemia and Sickle Cell-hemoglobin C Disease, run by Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University 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 10 inclusion and 5 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: stopped early, as of the sponsor's last update on 16 May 2025. 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Nov 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Stable Engraftment (time frame: 180 days post-infusion). 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 16 May 2025.