ThalassemiaNCT01319851
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation
A study testing Alefacept in people with Thalassemia and Sickle Cell Disease, run by Emory University at 1 site in United States.
Trial summary
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Read more about this study
- Who is it for?
- Ages Up to 21 years
- Conditions the study is for
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia
- What is being tested?
- Alefacept
- Type of treatment
- 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
- Sep 2010to Sep 2013
- 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 Up to 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Must be between the ages of 0-21 years at the time of admission for transplant
- Must have been transfused with at least five platelet, erythrocyte or granulocyte units (partial or full)
- Must have one of the following diseases:
- hemoglobin SS or hemoglobin SB Sβ0 thalassemia and meet one of the criteria below for having severe sickle cell disease (i) Previous central nervous system event lasting longer than 24 hours, plus objective imaging evidence of CNS vasculopathy, with or without residual neurologic findings (ii) Frequent (≥ 3 per year for 2 years) painful vaso-occlusive episodes (defined as episode lasting ≥ 4 hours and requiring hospitalization or outpatient treatment with parenteral opioids/opiates). Must have also (iii) Recurrent (≥ 3 in lifetime) acute chest syndrome events which have necessitated exchange transfusion or chronic transfusion therapy. (iv) Any combination of ≥ 3 acute chest syndrome episodes and vasoocclusive pain episodes (defined as above) yearly for 3 years. (v) Stage I or II sickle lung disease (see appendix 1) (vi) Pulmonary hypertension, measured by tricuspid regurgitant jet velocity (TRV) of greater than 2.5m/s (vii) Osteonecrosis involving multiple joints. (viii) Sickle Cell nephropathy with moderately severe renal insufficiency estimated GFR ≥30 ml/min, but ≤60 ml/min/1.73 m2 (Requires evaluation by a nephrologist). (b) Thalassemia major (c) Glanzmann thrombasthenia (d) Wiskott-Aldrich syndrome (e) Chronic-granulomatous disease (f) Severe congenital neutropenia (g) Leukocyte adhesion deficiency (h) Shwachman-Diamond syndrome (i) Diamond-Blackfan anemia (j) Fanconi anemia (k) Dyskeratosis-congenita (l) Chediak-Higashi syndrome (m) Acquired (immune; non-inherited, non-congenital) severe aplastic anemia (only patients whose best graft source is a mismatched related donor, unrelated marrow donor or cord blood unit) (n) Other inherited or congenital marrow failure syndromes complicated by severe aplastic anemia (o) Other inherited or congenital red blood cell disorders requiring monthly chronic transfusion therapy. (p) Other inherited or congenital platelet disorders resulting in at least three inpatient hospitalizations in the past two years for bleeding. (q) Other inherited or congenital granulocyte disorders resulting in at least three inpatient hospitalizations in the past two years for infection.
- Must have an available HLA identical sibling (HLA matched related), a non-HLA identical parent or sibling who is matched at least seven of eight loci (mismatch can be at an allele or antigen level), an unrelated adult donor who is matched at least seven of eight loci (mismatch can be at an allele or antigen level) or an unrelated cord blood unit that is matched at five of six loci (A (antigen level), B (antigen level), DRB1 (allele level)) and provides a minimum pre-cryopreservation TNC dose of 5.0 x 107 TNC/kg recipient weight.
Who cannot join
- Hemophagocytic lymphohistiocytosis or other disorder characterized by NK cell dysfunction, since alefacept's effect is mediated by NK cells.
- Biopsy proven cirrhosis (score IV).
- SCD chronic lung disease ≥ stage III (see appendix 1)
- Severe renal dysfunction defined as estimated GFR of <30 ml/min.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
- Severe neurologic impairment other than hemiplegia alone, defined as full scale IQ ≤ 70, quadriplegia or paraplegia, inability to ambulate, inability to communicate without assistive device, or any impairment resulting in decline of Lansky performance score to <50%.
Show all 18Show fewer
- Karnofsky or Lansky functional performance score < 50%
- Confirmed HIV seropositivity.
- Patient with unspecified chronic toxicity serious enough to detrimentally affect the patient's capacity to tolerate bone marrow transplantation.
- Patient or patient's guardian(s) unable to understand the nature and risks inherent in the BMT process.
- History of lack of compliance with medical care that would jeopardize transplant course.
- Patient is pregnant or lactating
- Donor who for psychological, physiologic, or medical reasons is unable to tolerate a bone marrow harvest or receive general anesthesia.
- Donor is HIV infected.
- Donor is pregnant
- Hemoglobin SS, or hemoglobin Sβ0 thalassemia patient who is eligible for one of the two trials of myeloablative conditioning currently being conducted by the Aflac Center (SALT: Alternate-Donor Bone Marrow and Cord Blood Transplantation for Children with High-Risk Sickle Cell Disease Busulfan, fludarabine, ATG and Reduced-Dose Cyclophosphamide Conditioning for Allogeneic Hematopoietic Stem Cell Transplantation in Patients with Severe Sickle Cell Disease: a pilot study
- Patients with thalassemia major who are eligible for any multicenter study we are participating in.
- Patients whose best graft source is a related or unrelated donor/cord blood unit that is mismatched and the patient's HLA antibody testing (see below) demonstrates an antibody directed against the disparate HLA molecule.
Trial locations and contacts
1 site in 1 country.
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
About this study
Official title: Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection.
In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases.
Read the detailed description
There are a large number of serious non-malignant diseases of childhood, most of them congenital and rare, which can be corrected by HSCT. These diseases are all characterized by deficiencies, either in number or in function, of marrow derived cells. These diseases usually affect immune or blood cells and frequently involve transfusion therapy with erythrocytes, platelets or granulocytes. Examples of such diseases include sickle cell disease, thalassemia major, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency, Shwachman-Diamond syndrome, Diamond-Blackfan anemia, Fanconi anemia, dyskeratosis-congenita, Chediak-Higashi syndrome, and severe aplastic anemia.
Allogeneic blood HSCT, whether performed for a malignant or a non-malignant condition, relies on the use of a pre-transplant conditioning regimen. Traditionally, very high doses of chemotherapy or total body irradiation have been utilized as conditioning. The use of intensive conditioning, which, practically speaking eliminates the host marrow and immune system, however, can produce serious and sometimes fatal infections and injuries to vital organs, such as the liver and lung. In children, the use of intensive conditioning can also produce serious late effects, including hypogonadism, stunted growth, impaired cognitive development and secondary malignancies.
Over the past decade, there has been a move to minimize the risk for such complications by reducing the intensity of conditioning regimens. Added impetus for reducing conditioning intensity arose from the observation in transplantation for thalassemia and sickle cell disease that sustained mixed chimerism, that is partial donor engraftment, is usually sufficient to cure non-malignant diseases. This observation suggested that sustained engraftment could be achieved without "ablation" or elimination of the host marrow. Pre-clinical studies demonstrated in small and large animals that sustained mixed chimerism can be achieved with preparative regimens consisting of TBI doses as low as 100-300 cGy (by comparison, standard intensity regimens typically employ 1000 cGy or more in combination with chemotherapy).
This approach was first translated in a clinical trial involving 45 adults with hematological malignancies who were not candidates for standard conditioning because of older age or serious co-morbidities. Using a single 200 cGy dose of TBI, sustained engraftment was achieved in 80% of cases and, remarkably, transplant related mortality was only 6.7% in this frail group of patients at 14 months. It is also notable that these transplants were performed primarily in the outpatient setting-the median length of hospitalization was 1 day. Low-dose TBI based conditioning has also been safely and effectively utilized for infants and children with severe combine immune deficiency and other severe immune deficiencies, undergoing related and unrelated donor transplantation. This clinical experience strongly suggests that if an effective low-dose TBI conditioning regimen can be developed for children with non-malignant diseases it could transform BMT from a costly, highly morbid, and sometimes life-taking procedure to a relatively inexpensive, safe and well-tolerated one.
Thousands and thousands of children around the world suffer from sickle cell disease and thalassemia major. There is a myriad of other less common serious non-malignant hematological diseases, which have even more devastating effects, for which HSCT remains the only viable cure. Low-dose TBI based conditioning represents a minimally toxic approach to transplantation for these children-a way to overcome alloimmunization, however, is needed to make this approach more effective. Alefacept, the only currently FDA approved agent that specifically targets memory T cells, the investigators believe, holds the key to making low-dose TBI based conditioning more effective and could, thereby, dramatically alter the field of transplantation for non-malignant diseases. sustained donor engraftment needs to be developed.
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia
- Being tested
- Alefacept
- Keywords
- bone marrow transplant, sickle cell disease, thalassemia, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency, Schwachman-Diamond syndrome, Diamond-Blackfan anemia, Fanconi anemia, dyskeratosis-congenita, Chediak-Higashi syndrome, severe aplastic anemia
- Medical subject headings
- Thalassemia, Anemia, Sickle Cell, Thrombasthenia, Wiskott-Aldrich Syndrome, Granulomatous Disease, Chronic, Neutropenia, Severe Congenital, Autosomal Recessive 3, Leukocyte adhesion deficiency type 1, Shwachman-Diamond Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AlefaceptDrug
Also called Amevive
0.25 mg/kg IV on day -40 and day -39 0.5 mg/kg IV on days -33, -26, -19 and -12 Alefacept was diluted in sterile water (2 ml total volume) and administered via i.v. push followed by a normal saline flush per package insert.
| Study group | Role | What they receive |
|---|---|---|
| Alefacept | Experimental | Pediatric subjects with non-malignant diseases (NMD) will receive pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses). Receives: Drug: Alefacept |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
All subjects received alefacept prior to hematopoietic stem cell transplantation and were followed up to at least two years after transplantation to ensure successful engraftment.
Measured: Two years post-transplant
Secondary outcomes (6)
Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity
Regimen-related toxicity was measured using the Bearman criteria. The Bearman criteria grades toxicity levels at Grade 1, Grade 2, Grade 3, and Grade 4. In this system, grade I toxicity is reversible without treatment and grade 2 is not life threatening, but requires treatment. Grade 3 requires life-support intervention and grade 4 is fatal. All regimen-related toxicities were determined to be unlikely attributable to the study drug.
Measured: Day 42 post-transplant
Number of Participants That Expressed Successful Neutrophil Engraftment
Neutrophil engraftment was assessed with absolute neutrophils >500*10^8/kg by 100 days post transplant. Neutrophils were counted by performing a complete blood cell count (CBC).
Measured: Day 100 post-transplant
Incidence of Greater Than or Equal to 85% CD3 Donor Chimerism
CD3 chimerism was measured from peripheral blood lymphocytes 30 days post transplant. DNA chimerism analysis was performed by amplified fragment length polymorphism.
Measured: Day 30 post-transplant
Incidence of 100% CD33 Donor Chimerism
CD33 chimerism was measured from peripheral blood lymphocytes 30 days post transplant. DNA chimerism analysis was performed by amplified fragment length polymorphism.
Measured: Day 30 post-transplant
Number of Participants Who Experienced Acute Graft-versus-host Disease (aGVHD), Measured by NIH Consensus Criteria (NCC) Score: Grade II-IV
Cumulative Incidence of Grade II-IV aGVHD Score at 30 Days. The NIH Consensus grading and severity criteria includes physical assessments of skin, oral cavity, eyes, gynecological and laboratory data and patient reports. Each domain is scored from Grade 0 (no involvement) to Grade IV (severe involvement).
Measured: Day 30 post-transplant
Number of Participants Who Experienced Chronic Graft-versus-host Disease (cGVHD), Measured by the NIH Criteria Consensus (NCC)
The severity criteria of chronic graft-versus-host disease (cGVHD) recommended by the NIH Criteria Consensus (NCC) was employed. The number of organs involved and the severity of the disease in these organs dictated the global summary score used to define the disease as mild, moderate, or severe. Mild disease indicates one or two organs involved each with a maximal score of 1. Moderate disease indicates three or more organs involved with a score of 2 in any individual organ, or lung involvement with a score of 1. Severe global GVHD is defined by a score of 3 in any organ, or a lung score of 2.
Measured: Day 100 post-transplant
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2011 (estimated)
- Study startSep 2010
- Primary completionSep 2013
- Study completionSep 2013
Why it stopped: drug company is no longer making the drug
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for ThalassemiaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe 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 N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 3 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Children's Healthcare of Atlanta
- Responsible party
- Principal investigator, John Horan, Emory University
- Investigators
- John Horan, MD, Principal investigator, Emory University/Children's Healthcare of Atlanta
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01319851
- Sponsor's protocol ID
- IRB00039680
- Other IDs
- BMT Alefacept
- Status last verified
- Jul 2017
- First submitted
- 15 Sep 2010
- Last updated
- 27 Jul 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Stenger EO, Chiang KY, Haight A, Qayed M, Kean L, Horan J. Use of Alefacept for Preconditioning in Multiply Transfused Pediatric Patients with Nonmalignant Diseases. Biol Blood Marrow Transplant. 2015 Oct;21(10):1845-52. doi: 10.1016/j.bbmt.2015.06.005. Epub 2015 Jun 19.
PubMed 26095669
Study at a glance
From the registry record, last updated by the sponsor on 27 Jul 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation for Children With Non-Malignant Diseases Who Have Been Multiply Transfused: a Pilot Study
- ClinicalTrials.gov ID
- NCT01319851
- Acronym / sponsor ID
- IRB00039680
- Conditions
- Thalassemia, Sickle Cell Disease, Glanzmann Thrombasthenia, Wiskott-Aldrich Syndrome, Chronic-granulomatous Disease, Severe Congenital Neutropenia
- Keywords
- bone marrow transplant, sickle cell disease, thalassemia, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: drug company is no longer making the drug
- Enrolment
- 3 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Children's Healthcare of Atlanta
- Study start
- Sep 2010 (actual)
- Primary completion
- Sep 2013 (actual)
- Study completion
- Sep 2013 (actual)
- First posted
- 22 Mar 2011 (estimated)
- Results
- Posted 9 Mar 2015
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Allogeneic blood and marrow transplantation remains the only viable cure for children who suffer from many serious non-malignant hematological diseases. Transplantation, however, carries a high risk of fatal complications. Much of the risk stems from the use of high dose radiation and chemotherapy for conditioning, the treatment administered just prior to transplant that eliminates the patients' marrow and immune system, effectively preventing rejection of the donors' cells. Attempts to make blood and marrow transplantation safer for children with non-malignant diseases by using lower doses of radiation and chemotherapy have largely failed because of a high rate of graft rejection.
In many such cases, it is likely that the graft is rejected because the recipient is sensitized to proteins on donor cells, including bone marrow cells, by blood transfusions. The formation of memory immune cells is a hallmark of sensitization, and these memory cells are relatively insensitive to chemotherapy and radiation. Alefacept, a drug used to treat psoriasis, on the other hand, selectively depletes these cells. The investigators are conducting a pilot study to begin to determine whether incorporating alefacept into a low dose conditioning regimen can effectively mitigate sensitization and, thereby, prevent rejection of allogeneic blood and marrow transplants for multiply transfused children with non-malignant hematological diseases.
Detailed description
There are a large number of serious non-malignant diseases of childhood, most of them congenital and rare, which can be corrected by HSCT. These diseases are all characterized by deficiencies, either in number or in function, of marrow derived cells. These diseases usually affect immune or blood cells and frequently involve transfusion therapy with erythrocytes, platelets or granulocytes. Examples of such diseases include sickle cell disease, thalassemia major, Glanzmann thrombasthenia, Wiskott-Aldrich syndrome, chronic-granulomatous disease, severe congenital neutropenia, leukocyte adhesion deficiency, Shwachman-Diamond syndrome, Diamond-Blackfan anemia, Fanconi anemia, dyskeratosis-congenita, Chediak-Higashi syndrome, and severe aplastic anemia.
Allogeneic blood HSCT, whether performed for a malignant or a non-malignant condition, relies on the use of a pre-transplant conditioning regimen. Traditionally, very high doses of chemotherapy or total body irradiation have been utilized as conditioning. The use of intensive conditioning, which, practically speaking eliminates the host marrow and immune system, however, can produce serious and sometimes fatal infections and injuries to vital organs, such as the liver and lung. In children, the use of intensive conditioning can also produce serious late effects, including hypogonadism, stunted growth, impaired cognitive development and secondary malignancies.
Over the past decade, there has been a move to minimize the risk for such complications by reducing the intensity of conditioning regimens. Added impetus for reducing conditioning intensity arose from the observation in transplantation for thalassemia and sickle cell disease that sustained mixed chimerism, that is partial donor engraftment, is usually sufficient to cure non-malignant diseases. This observation suggested that sustained engraftment could be achieved without "ablation" or elimination of the host marrow. Pre-clinical studies demonstrated in small and large animals that sustained mixed chimerism can be achieved with preparative regimens consisting of TBI doses as low as 100-300 cGy (by comparison, standard intensity regimens typically employ 1000 cGy or more in combination with chemotherapy).
This approach was first translated in a clinical trial involving 45 adults with hematological malignancies who were not candidates for standard conditioning because of older age or serious co-morbidities. Using a single 200 cGy dose of TBI, sustained engraftment was achieved in 80% of cases and, remarkably, transplant related mortality was only 6.7% in this frail group of patients at 14 months. It is also notable that these transplants were performed primarily in the outpatient setting-the median length of hospitalization was 1 day. Low-dose TBI based conditioning has also been safely and effectively utilized for infants and children with severe combine immune deficiency and other severe immune deficiencies, undergoing related and unrelated donor transplantation. This clinical experience strongly suggests that if an effective low-dose TBI conditioning regimen can be developed for children with non-malignant diseases it could transform BMT from a costly, highly morbid, and sometimes life-taking procedure to a relatively inexpensive, safe and well-tolerated one.
Thousands and thousands of children around the world suffer from sickle cell disease and thalassemia major. There is a myriad of other less common serious non-malignant hematological diseases, which have even more devastating effects, for which HSCT remains the only viable cure. Low-dose TBI based conditioning represents a minimally toxic approach to transplantation for these children-a way to overcome alloimmunization, however, is needed to make this approach more effective. Alefacept, the only currently FDA approved agent that specifically targets memory T cells, the investigators believe, holds the key to making low-dose TBI based conditioning more effective and could, thereby, dramatically alter the field of transplantation for non-malignant diseases. sustained donor engraftment needs to be developed.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Alefacept | Experimental | Drug: AlefaceptPediatric subjects with non-malignant diseases (NMD) will receive pre-conditioning with alefacept 0.5 mg/kg/dose i.v. with the first dose split on days -40 and -39 and the remaining doses given on days -33, -26, -19, and -12 (e.g. weekly for 5 doses). |
Who may qualify
- Ages
- Up to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- Must be between the ages of 0-21 years at the time of admission for transplant
- Must have been transfused with at least five platelet, erythrocyte or granulocyte units (partial or full)
- Must have one of the following diseases:
- Must have an available HLA identical sibling (HLA matched related), a non-HLA identical parent or sibling who is matched at least seven of eight loci (mismatch can be at an allele or antigen level), an unrelated adult donor who is matched at least seven of eight loci (mismatch can be at an allele or antigen level) or an unrelated cord blood unit that is matched at five of six loci (A (antigen level), B (antigen level), DRB1 (allele level)) and provides a minimum pre-cryopreservation TNC dose of 5.0 x 107 TNC/kg recipient weight.
Key exclusion criteria (18 listed)
- Hemophagocytic lymphohistiocytosis or other disorder characterized by NK cell dysfunction, since alefacept's effect is mediated by NK cells.
- Biopsy proven cirrhosis (score IV).
- SCD chronic lung disease ≥ stage III (see appendix 1)
- Severe renal dysfunction defined as estimated GFR of <30 ml/min.
- Severe cardiac dysfunction defined as shortening fraction < 25%.
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 |
|---|---|---|
| AlefaceptAlso: Amevive0.25 mg/kg IV on day -40 and day -39 0.5 mg/kg IV on days -33, -26, -19 and -12 Alefacept was diluted in sterile water (2 ml total volume) and administered via i.v. push followed by a normal saline flush per package insert. | Drug | Alefacept |
Primary outcome
Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
Time frame: Two years post-transplant
All subjects received alefacept prior to hematopoietic stem cell transplantation and were followed up to at least two years after transplantation to ensure successful engraftment.
6 secondary outcomes
Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity
Time frame: Day 42 post-transplant
Number of Participants That Expressed Successful Neutrophil Engraftment
Time frame: Day 100 post-transplant
Incidence of Greater Than or Equal to 85% CD3 Donor Chimerism
Time frame: Day 30 post-transplant
Incidence of 100% CD33 Donor Chimerism
Time frame: Day 30 post-transplant
Number of Participants Who Experienced Acute Graft-versus-host Disease (aGVHD), Measured by NIH Consensus Criteria (NCC) Score: Grade II-IV
Time frame: Day 30 post-transplant
Number of Participants Who Experienced Chronic Graft-versus-host Disease (cGVHD), Measured by the NIH Criteria Consensus (NCC)
Time frame: Day 100 post-transplant
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jul 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 26095669Stenger EO, Chiang KY, Haight A, Qayed M, Kean L, Horan J. Use of Alefacept for Preconditioning in Multiply Transfused Pediatric Patients with Nonmalignant Diseases. Biol Blood Marrow Transplant. 2015 Oct;21(10):1845-52. doi: 10.1016/j.bbmt.2015.06.005. Epub 2015 Jun 19.
Related trials for Thalassemia
Recruiting
A Long-term Follow-up Study in Participants Who Received CS-101
Beta-Thalassemia
RecruitingPhase 1/2
Repeat Peripheral Blood Stem Cell Transplantation for Patients With Sickle Cell Disease and Falling Donor Myeloid Chimerism Levels
Myeloid Chimerism
RecruitingPhase 2
AlloSCT for Malignant and Non-malignant Hematologic Diseases Utilizing Alpha/Beta T Cell and CD19+ B Cell Depletion
Acute Leukemia, Severe Aplastic Anemia
RecruitingPhase 3
Combination Therapy With Danazol, Prednisolone, and Thalidomide for Thalidomide-Refractory Transfusion-Dependent β-Thalassemia
Beta-Thalassemia, Thalassemia
Not yet recruitingObservational study
Native T1 Mapping and ECV in Chronic Hemolytic Anemia
Hemolytic Anemia, Thalassemia
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.
John Horan, MD principal investigator
Emory University/Children's Healthcare of Atlanta
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Alefacept in people with Thalassemia and Sickle Cell Disease, run by Emory 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 5 inclusion and 18 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 27 Jul 2017. 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 9 Mar 2015). 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?
Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment (time frame: Two years post-transplant). 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 NCT01319851, which also lists 1 linked publication.
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 27 Jul 2017.