An Early Phase 1 interventional study of Alemtuzumab and Total Body Irradiation in Sickle Cell Disease, sponsored by Kathleen Dorritie. Terminated at 1 site in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2022-06-28.
Sponsored by Kathleen Dorritie · Early Phase 1, Interventional, and Treatment
Among 22 studies testing alemtuzumab in Anemia, Sickle Cell — 6 open to participants now, 14 under way, none of them phase 3. Alemtuzumab · Anemia, Sickle Cell.
Among the 22 studies testing alemtuzumab in Anemia, Sickle Cell, this one is the 21st largest by enrollment, is one of 3 that started in 2018, is one of 3 in phase 1, and was the 15th registered.
1,134 studies on the registry are indexed under Anemia, Sickle Cell; 238 are open to participants now.
This study's enrollment of 1 is below the median of 40 across 769 interventional studies indexed under Anemia, Sickle Cell.
Browse Anemia, Sickle Cell studies →6 of the 22 studies testing alemtuzumab in Anemia, Sickle Cell are open now, in 2 countries. Each count lists the studies.
Alemtuzumab: first FDA approval 2001 (LEMTRADA), first EMA approval 2001. What it is approved for, and how studies give it, is on its page →
Of the 177 Anemia, Sickle Cell studies recruiting now, 64 (36%) set no upper age limit and 97 (55%) stop at 75 or younger; 127 take children under 18; 29 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →22 studies; the count open now beside each. This study's row is marked.
First in 1999; this study’s year is marked. 3 started in 2018.
Median enrollment 44 across the 22 interventional studies. Of the 8 that completed or were terminated, 6 (75%) have results posted.
None of the 22 has an industry lead sponsor. The 10 most frequent:
Kathleen Dorritie is the lead sponsor of 2 studies on the registry; none are open to participants now.
Kathleen Dorritie has 1 study filed under Anemia, Sickle Cell — fewer than ten, so there is no comparison to draw.
3 of the studies testing alemtuzumab in Anemia, Sickle Cell that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04018937Emory UniversityEarly Human Leukocyte Antigen (HLA) Matched Sibling Hematopoietic Stem Cell Transplantation | 2026-11 |
| NCT01962415Paul SzabolcsReduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT | 2026-11 |
| NCT05384756City of Hope Medical CenterTMLI and Alemtuzumab for Treatment of Sickle Cell Disease | 2026-12-01 |
Among the 22 studies testing alemtuzumab in Anemia, Sickle Cell, from their registry updates:
Nothing recorded among them since 2026-09-25.
This is a prospective pilot study of matched-related donor allogeneic stem cell transplantation in adults with severe sickle cell disease using a matched-sibling PBSC graft with a non-myeloablative conditioning regimen (Alemtuzumab).
Stem cell transplantation recipients will be given Alemtuzumab, which is a non-myeloablative pre-transplant conditioning regimen. This non-myeloablative therapy uses doses of chemotherapy and radiation to weaken (but not destroy) the patients bone marrow and immune system, while still allowing their body to accept the donor's stem cells. Alemtuzumab will be given 7 days prior to stem cell infusion at 0.03 mg/kg IV, 6 days prior to stem cell infusion at 0.1 mg/kg IV, and 5 thru 3 days prior to stem cell infusion at 0.3 mg/kg IV.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Patient selection
Presence of at least 1 of the following manifestations:
Three or more pain crises per year in the 2-year period preceding referral (required intravenous pain management in the outpatient or inpatient hospital setting).
This may include painful episodes related to priapism, osteonecrosis or any sickle-related complication.
h. Sickle nephropathy defined as Cr ≥ 1.5 times the or biopsy proven i.Reversible SCD complication not ameliorated by hydroxyurea: i.Two or more vaso-occlusive crises requiring hospitalizations ii. Any episode of ACS while on hydroxyurea
Adequate physical function as measured by all of the following:
i. Serum conjugated (direct) \< 2x upper limit of normal for age as per local laboratory; ii. and AST \< 5 times upper limit of normal. iii. Patients with hyperbilirubinemia because of hyper hemolysis, or who experience a sudden, profound change in the serum hemoglobin after a RBC transfusion are not excluded.
Absence of clinical or radiographic evidence of neurologic event within 6 months prior to proceeding with transplantation.
Donor selection
Exclusion Criteria:
Patient selection
Donor selection A. Inclusion Criteria
B. Exclusion Criteria
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
TIA (transient ischaemic attack)
A “mini-stroke”: temporary stroke-like symptoms caused by reduced blood flow to part of the brain, spinal cord or eye, without signs of a new stroke on a scan.
Stroke symptoms need emergency care at once, even if they go away.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Immunosuppression
A weakened immune system. It can come from an illness, such as HIV or some blood cancers, or from medicines that turn the immune system down, for example after an organ transplant or for an autoimmune disease; steroids at higher doses count.
Read whether the line is about a condition or about medicines, and which medicines and doses it means.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis B tests
Hepatitis B is a virus that infects the liver. These are separate tests: HBsAg positive usually means a current infection (a recent vaccination can briefly cause it too); total anti-HBc (HBcAb) positive means an infection now or in the past; HBV DNA measures how much virus is in the blood.
Some studies accept a past infection if HBV DNA is undetectable, sometimes with antiviral medicine during the study. Check what this study allows.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hepatitis C tests
Hepatitis C is a virus that infects the liver. An HCV antibody test can stay positive after an infection has cleared (and is occasionally positive without one); an HCV RNA test shows whether the virus is there now.
After treatment, hepatitis C counts as cured when HCV RNA is still undetectable 12 weeks or more after the treatment ended. Studies often accept people cured this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Sickle cell patient receives sibling donor peripheral blood stem cell transplant with non-myeloablative pre-transplant conditioning.
Drug: Alemtuzumab · Radiation: Total Body Irradiation · Drug: Sirolimus
Alemtuzumab is a non-myeloablative pre-transplant conditioning regimen. Non-myeloablative therapy uses doses of chemotherapy and radiation to weaken (but not destroy) the patients bone marrow and immune system, while still allowing their body will accept the donor's stem cells. Alemtuzumab will be given 7 days prior to stem cell infusion at 0.03 mg/kg IV, 6 days prior to stem cell infusion at 0.1 mg/kg IV, and 5 thru 3 days prior to stem cell infusion at 0.3 mg/kg IV.
300 cGy will be administered in a single fraction on Day - 2. TBI is used commonly as part of pre-transplant conditioning in a variety of settings.
Also known as: TBI
Sirolimus will be used for the prevention of graft-verus-host disease and will begin on Day - 1.
Also known as: Rapammune
Treatment Success
Evaluating reversal of Hb S % to that of the donor's phenotype, in recipients of HLA matched-sibling peripheral blood - hematopoietic stem cell transplantation (HSCT) with NMA conditioning regimen. Testing for treatment success will include Hb Electrophoresis. Recipients with donors AA should have nearly 0% Hb S. Recipients with donors AS should have similar Hb S % (approximately \< 60%) as the donor. The proportion of patients experiencing treatment success, will be calculated with a 90% exact confidence interval.
Time frame: up to 1 year after HSCT
Engraftment
To estimate the probability of engraftment, using absolute neutrophil count and platelets along with chimerism analysis after HSCT. This will by evaluated by: 1) Time to ANC of \>500/uL on the first of three consecutive days. 2) Time to platelet count \>50,000/µL for 3 days without transfusion. 3) Peripheral blood chimerism analysis of whole blood and CD3-lineage will be performed on or around day +30, +90, +180, and +360. 4) Additional peripheral blood chimerism studies may be performed as clinically indicated. The statistical analysis plan will be calculated with a 90% exact confidence interval.
Time frame: up to 1 year after HSCT
Probability of developing acute GVHD after HSCT.
Acute GVHD will be diagnosed and graded using the clinical and laboratory criteria in Appendix A of the protocol. During the inpatient stay, clinical evaluations will occur daily. During the outpatient stay, each patient should be evaluated at least every other week until they are discharged from the transplant clinic. Subsequent evaluations will occur at roughly Days +30, +60, +90 and until +100 days. Clinical evaluations will be performed and grade will be assigned at the time of diagnosis by an attending physician. The decision to initiate GVHD therapy will be made by the attending physician. The statistical analysis plan will be calculated with a 90% exact confidence interval.
Time frame: up to 100 days after HSCT
Probability of developing chronic GVHD after HSCT.
Chronic GVHD will be diagnosed using NIH criteria outlined in Appendix B of the protocol. b. Evaluation will be conducted by BMT team at day +100, +360 and yearly up to 2 years after HCT. The patient's sickle cell physician will conduct clinical assessments between these time points as required per institutional guidelines. This will be evaluate by: 1) The occurrence of chronic GHVD meeting NIH criteria and requiring systemic pharmacological immunosuppression 2) The use of additional immune suppressive agents other than first line therapy 3) Time to completion of prednisone, if required for treatment of acute GVHD 4) Time to completion of all immunosuppression 5) Requirement for immunosuppression at 1 year and 2 years after transplant. The statistical analysis plan will be calculated with a 90% exact confidence interval.
Time frame: up to 2 years after HSCT
Graft failure or Relapse
Relapse is defined by the presence of recipient's Hb SS on electrophoresis. Testing for recurrent disease will include Hb electrophoresis to determine recipient vs. donor Hb-type and peripheral blood chimerism to document loss of donor cell engraftment. Endpoints will include: time to relapse and status of compliance with immunosuppressive therapy. Primary graft failure defined as the absence of donor cells assessed by peripheral blood chimerism assays, by any lineage, on or after day +30. Secondary graft failure defined as the presence of \< 20% donor cells by peripheral blood chimerism assays in a patient with prior evidence of ≥ 20% of donor cells. The analysis plan for graft failure or relapse will be calculated with a 90% exact confidence interval.
Time frame: up to 2 years after HSCT
Discontinuation of Immunosuppressive therapy
The time (in days) it takes to taper immunosuppressive therapy will be measured. Tapering will begin once 50% donor CD3 chimerism is achieved. To estimate the time to discontinuation of immunosuppressive therapy. The product-limit (Kaplan-Meier) estimate of the distribution of time to discontinuation will be calculated with a 90% confidence region. In an exploratory analysis, proportional hazards (Cox) regression will be applied to identify potential predictors of time to discontinuation.
Time frame: up to 2 years after HSCT
Sickle cell disease related organ damage
Cardiac function, pulmonary function, renal function, cerebrovascular function will be measured. We will measure and record BNP, left atrial diameter and the tricuspid regurgitant velocity, noted on transthoracic echocardiogram, (TTE) as a marker of improvement at an outpatient cardiology follow-up visit. We will measure and record a 6-minute walking distance test and pulmonary function test variables to include: FEV1, FVC, TLC, RV, DLCO, and O2 saturation at an outpatient pulmonology follow-up visit.We will measure and record the urine-albumin creatinine ratio as a measure of micro-/macro-albuminuria and 24-hr urine proteinat an outpatient nephrology follow-up visit.We will obtain a brain MRI at an outpatient neurology follow-up visit. All evaluations will take place at baseline, Day +365, and Day +720.
Time frame: up to 2 years after HSCT
Lymphocyte subsets
To evaluate lymphocyte subsets. This analysis will be descriptive.
Time frame: up to 2 years after HSCT
Changes in monthly transfusions after HSCT
We will monitor the number of transfusions required on a monthly basis pre- and post-HSCT Patient's monthly transfusions will be recorded starting 3 months pre-HSCT. Patient's monthly transfusions will be recorded post-HSCT at the following time points: Days +30, +60, +90, +180, +365, +720. To evaluate changes in monthly transfusion dependence after HSCT in patients, will be modeled using mixed effects regression.
Time frame: up to 2 years after HSCT
Changes in the annual frequency of SCD-related hospitalization after
The patients frequency of hospital stays will be measured compared to prior HSCT. Number of hospitalizations per year post-HSCT will be recorded at Days 0, +365 and +720. A mixed effects Poisson regression model will compare the patients' frequency of hospitalization three years pre-transplant to post-transplant.
Time frame: up to 2 years after HSCT
Quality of life measures
Quality of life will be measured utilizing validated tools as well as new tools specific for sickle cell disease including the questionnaires: SF-36, Adult Sickle Cell Quality of life Measurement Information System (ASCQ-Me), Pittsburgh Sleep Quality Index, Generalized Anxiety Disorder-7, Brief Pain Inventory, and Painimation. To evaluate improvement in quality of life measures after HSCT. The recommended summary indices for the quality of life instruments will be calculated and their change over time by mixed effects linear models.
Time frame: up to 2 years after HSCT
Transplant-related mortality
TRM is defined as mortality in any patient for whom there has not been a diagnosis of relapse.To estimate transplant-related mortality: The product-limit (Kaplan-Meier) estimate of the distribution of time to discontinuation will be calculated with a 90% confidence region. In an exploratory analysis, proportional hazards (Cox) regression will be applied to identify potential predictors of time to discontinuation.
Time frame: up to 2 years after HSCT
Opioid independence
We will monitor the narcotic use in all recipients. At our institution, opioid adjustments are made by our SCD physicians who will be caring for these patients both pre- and post-transplant. Information on weekly narcotic use will be obtained and will be converted to the oral morphine equivalent (OME) dose for data collection. This will be done for the one week duration immediately prior to the following time points: Days 0, +30, +60, +90, +180, +365, +720.
Time frame: up to 2 years after HSCT
MDC, whole blood and CD3 Lineage
To estimate the probability of MDC, whole blood and CD3-lineage at 1 year.The statistical analysis plan will be calculated with a 90% exact confidence interval.
Time frame: up to 1 year after HSCT
Plan to share: No
No publications or documents are linked to this record.
Ran from Mar 2018 to primary completion in Sep 2018, 5 months.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jun 2022. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Alemtuzumab · Sirolimus
Kathleen Dorritie