Beta Thalassemia MajorEarly Phase 1NCT04776850
Pre-transplant Immunosuppression and Donor Stem Cell Transplant for the Treatment of Severe Hemoglobinopathies
A Early Phase 1 study testing Bortezomib, Busulfan and Cyclophosphamide in people with Beta Thalassemia Major and Sickle Beta 0 Thalassemia, run by M.D. Anderson Cancer Center.
Trial summary
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Read more about this study
- Who is it for?
- Ages 2 to 30 years
- Conditions the study is for
- Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia
- What is being tested?
- Bortezomib, Busulfan and Cyclophosphamide
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 29 Dec 2020to 5 Dec 2022
- Phase
- Early Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients 2-30 years-of-age with confirmed sickle cell disease (SCD) (SS \& sickle beta [SB]-thalassemia, both sickle beta 0 [SB0] and sickle beta plus [SB+]) or severe B-thalassemia major are potentially eligible
- Patients with SCD should also meet the following eligibility criteria as outlined by the Center for Medicaid and Medicare Services: sickle cell disease and at least one of the following:
- Stroke or neurological deficit lasting > 24 hours
- Recurrent acute chest syndrome (ACS): 2 or more episodes of ACS in 2-year period preceding enrollment
- Recurrent vaso-occlusive pain crises: 3 or more episodes per year in 2-year period preceding enrollment or recurrent priapism (3 or more episodes in the 2 years preceding enrollment)
- Chronic transfusion program defined as 8 or more packed red blood cells (PRBC) transfusions per year to prevent central nervous system and/or vaso-occlusive complications in 1-year period preceding enrollment
Show all 19Show fewer
- Impaired neuropsychological function and abnormal cerebral magnetic resonance imaging (MRI) scan (silent strokes)
- Stage I or II sickle lung disease
- Sickle nephropathy (moderate or severe proteinuria or a glomerular filtration rate 30-50% of predicted normal value)
- Bilateral proliferative retinopathy and major visual impairment in at least one eye
- Osteonecrosis of multiple joints
- Echocardiographic finding of tricuspid valve regurgitant jet velocity (TRJV) >= 2.7 m/sec
- Patients with B-thalassemia are considered as severe if they are/have any of the following:
- Transfusion-dependent
- Evidence of extra-medullary hematopoiesis
- Pesaro Class III
- Patients shall not proceed to HCT without confirmation of primary diagnosis by review of available newborn screening results or hemoglobin electrophoresis and/or genetic testing
- DONOR: High resolution HLA typing will be performed on all willing and available biologic parents and siblings without clinically significant hemoglobinopathy. Preference will be given to donors with the lowest number of HLA-allele mismatches
- DONOR: Donor-specific anti-HLA antibodies will be obtained and analyzed from all patients. Preference will be given to donors with absent or low titer anti HLA-donor specific antibody levels when possible
Who cannot join
- Uncontrolled infection
- Females who are pregnant and/or unwilling to cease breastfeeding
- Seropositivity for human immunodeficiency virus (HIV)
- Lansky or Karnofsky performance status < 70%
- Life expectancy severely limited by concomitant illness
- Uncontrolled arrhythmias or symptomatic cardiac disease
Show all 17Show fewer
- Uncontrolled symptomatic pulmonary disease
- Evidence of chronic active hepatitis or cirrhosis
- Serum conjugated (direct) bilirubin > 2 x upper limit of normal for age. Participants are not excluded if the serum conjugated (direct) bilirubin is > 2 x the upper limit of normal for age as per local laboratory and:
- There is evidence of a hyperhemolytic reaction after a recent red blood cell (RBC) transfusion, OR
- There is evidence of moderate direct hyperbilirubinemia defined as direct serum bilirubin < 5 times upper limit of normal (ULN) and not caused by underlying hepatic disease
- Alanine aminotransferase (ALT) and aspartate aminotransferase (AST) > 5 x upper limit of normal for age
- Serum creatinine > 1.5 x upper limit of normal for age AND estimated or measure creatinine clearance < 70 mL/min/1.72 m^2
- Patient, parent or guardian unable/unwilling to provide consent and when indicated, assent
- Patients with available HLA-matched related donor
- Prior receipt of gene therapy
- DONOR: All potential donors shall be tested by hemoglobin electrophoresis. Any potential donor with a clinically significant hemoglobinopathy will be deemed ineligible. Donors with sickle cell trait and beta thalassemia trait are eligible to donate
About this study
Official title: Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.
Read the detailed description
PRIMARY OBJECTIVE:
- To estimate event-free survival (EFS) at 2-years post HCT.
SECONDARY OBJECTIVES:
- Assess event-free survival (EFS) at 100 days and 1 year post HCT. II. Assess overall survival (OS) at 100 days and 1 year post HCT. III. 30-day transplant-related mortality (TRM). IV. Time to platelet and neutrophil engraftment. V. Rate of graft failure (primary and secondary) through day 100. VI. Incidence of acute graft-versus-host disease (aGVHD) by day 100. VII. Incidence of chronic graft-versus-host disease (cGVHD) by 1 year and 2 years.
VIII. Rate of grade II or greater organ toxicity through day 100. IX. Incidence of hepatic sinusoidal obstruction syndrome (SOS) by day 100. X. Incidence of central nervous system (CNS) toxicities including posterior reversible encephalopathy syndrome (PRES) by day 100.
XI. Incidence of infectious complications through day 100.
EXPLORATORY OBJECTIVES:
- Effect of HCT on clinical and laboratory manifestations of hemoglobinopathies by 1 and 2 years after HCT.
II. Assess the pharmacokinetic profile of busulfan and thymoglobulin to better understand post-transplant outcomes.
III. Assess immune reconstitution kinetics post HCT.
OUTLINE:
DONOR-SPECIFIC ANTI-HLA DESENSITIZATION: Patients with donor-specific anti-HLA antibody titers >= 1:3,000 at the start of PTIS receive rituximab intravenously (IV) on days -69, -41, -28, and -13 and bortezomib IV over less than 1 minute on days -68, -65, -40, and -37 in the absence of unacceptable toxicity. Patients with donor-specific anti-HLA antibody titers > 1:15,000 at the start of PTIS receive receive rituximab IV on days -69, -41, -28, and -13 and bortezomib IV over less than 1 minute on days -68, -65, -62, -58, -40, -37, -34, and -31 in the absence of unacceptable toxicity. Patients with donor-specific anti-HLA antibody titers > 1:5,000 at the start of conditioning may also undergo plasmapheresis on day -12.
PTIS: Patients receive fludarabine phosphate (fludarabine) IV over 1 hour and dexamethasone IV over less than 1 minute on days -68 to -64 and days -40 to -36 in the absence of disease progression or unacceptable toxicity.
CONDITIONING: Patients receive lapine T-lymphocyte immune globulin (rATG) IV over 4-6 hours on days -12 to -10, fludarabine phosphate IV over 1 hour on days -8 to -4, and busulfan IV over 2 hours on days -7 to -4 in the absence of disease progression or unacceptable toxicity.
TRANSPLANT: Patients undergo HCT on day 0.
GVHD PROPHYLAXIS: Patients receive cyclophosphamide IV over 2-3 hours on days 3 and 4 in the absence of unacceptable toxicity. Beginning on day 5, patients also receive tacrolimus IV continuously daily and then orally (PO) twice daily (BID) at the discretion of the treating physician for up to 12 months, and mycophenolate mofetil IV or PO BID up to day 60 in the absence of unacceptable toxicity.
After completion of HCT, patients are followed up periodically for up to 2 years.
- Conditions
- Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia, Sickle Beta Thalassemia, Sickle Cell Disease, Sickle Cell-SS Disease
- Being tested
- Bortezomib, Busulfan, Cyclophosphamide, Dexamethasone, Fludarabine Phosphate
- Medical subject headings
- beta-Thalassemia, Anemia, Sickle Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BortezomibDrug
Also called [(1R)-3-Methyl-1-[[(2S)-1-oxo-3-phenyl-2-[(pyrazinylcarbonyl)amino]propyl]amino]butyl]boronic Acid, LDP 341, MLN341, PS-341
Given IV
BusulfanDrug
Also called 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, Busulfanum
Given IV
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, Ciclofosfamida
Given IV
DexamethasoneDrug
Also called Aacidexam, Adexone, Aknichthol Dexa, Alba-Dex
Given IV
Also called 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, Fludara
Given IV
Hematopoietic Cell TransplantationProcedure or surgery
Also called HCT, Hematopoietic Stem Cell Transplantation, HSCT, Stem Cell Transplant
Undergo HCT
Lapine T-Lymphocyte Immune GlobulinBiological
Also called Anti-Thymocyte Globulin Rabbit, Grafalon, Rabbit Anti-Human Thymocyte Globulin (RATG), Rabbit Anti-Thymocyte Globulin
Given IV
Also called CellCept, MMF
Given IV or PO
PlasmapheresisProcedure or surgery
Also called Plasma Exchange, Therapeutic Plasma Exchange, Therapeutic Plasmapheresis
Undergo plasmapheresis
RituximabBiological
Also called ABP 798, BI 695500, C2B8 Monoclonal Antibody, Chimeric Anti-CD20 Antibody
Given IV
TacrolimusDrug
Also called FK 506, Fujimycin, Hecoria, Prograf
Given IV or PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (PTIS, HCT) | Experimental | See Detailed Description. Receives: Drug: Bortezomib; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Dexamethasone; Drug: Fludarabine Phosphate; Procedure: Hematopoietic Cell Transplantation; Biological: Lapine T-Lymphocyte Immune Globulin; Drug: Mycophenolate Mofetil; Procedure: Plasmapheresis; Biological: Rituximab; Drug: Tacrolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event-free survival (EFS)
EFS is defined as survival time following HCT without a qualifying event. Will be summarized by the Kaplan-Meier method with 95% confidence intervals.
Measured: At 2 years post-hematopoietic cell transplantation (HCT)
Secondary outcomes (14)
Event-free survival
EFS is defined as survival time following HCT without a qualifying event. Will be summarized by the Kaplan-Meier method with 95% confidence intervals.
Measured: Up to 100 days post-HCT
Event-free survival
EFS is defined as survival time following HCT without a qualifying event. Will be summarized by the Kaplan-Meier method with 95% confidence intervals.
Measured: Up to 1 year post-HCT
Overall survival
Will be summarized by the Kaplan-Meier method.
Measured: Up to 100 days post-HCT
Overall survival
Will be summarized by the Kaplan-Meier method.
Measured: Up to 1 year post-HCT
Transplant-related mortality
Will be summarized by the Kaplan-Meier method.
Measured: Up to 30 days post-HCT
Time to platelet and neutrophil engraftment
Will be estimated using the method of Gooley et al.
Measured: Up to 2 years post-HCT
Incidence of graft failure (primary and secondary)
Will be estimated using the method of Gooley et al.
Measured: Up to 100 days post-HCT
Incidence of acute graft-versus-host disease
Will be estimated using the method of Gooley et al.
Measured: Up to 100 days post-HCT
Incidence of chronic graft-versus-host disease
Will be estimated using the method of Gooley et al.
Measured: Up to 1 year post-HCT
Incidence of chronic graft-versus-host disease
Will be estimated using the method of Gooley et al.
Measured: Up to 2 years post-HCT
Incidence of grade II or greater organ toxicity
Will be reported as counts with percentages.
Measured: Up to 100 days post-HCT
Incidence of hepatic sinusoidal obstruction syndrome
Will be reported as counts with percentages.
Measured: Up to 100 days post-HCT
Incidence of central nervous system toxicities including posterior reversible encephalopathy syndrome
Will be reported as counts with percentages.
Measured: Up to 100 days post-HCT
Incidence of infectious complications
Will be reported as counts with percentages.
Measured: Up to 100 days post-HCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Mar 2021
- Study start29 Dec 2020
- Primary completion5 Dec 2022
- Study completion5 Dec 2022 (estimated)
Why it stopped: Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Beta Thalassemia MajorStudies of the same condition, open and under way.
- More trials run by M.D. Anderson Cancer 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- M.D. Anderson Cancer Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Kris M Mahadeo, Principal investigator, M.D. Anderson Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04776850
- Sponsor's protocol ID
- 2020-0952
- Other IDs
- NCI-2021-00365, 2020-0952
- Status last verified
- Nov 2022
- First submitted
- 8 Feb 2021
- Last updated
- 24 Oct 2024
- 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 24 Oct 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pre-Transplant Immunosuppression and Related Haploidentical Hematopoietic Cell Transplantation for Patients With Severe Hemoglobinopathies
- ClinicalTrials.gov ID
- NCT04776850
- Acronym / sponsor ID
- 2020-0952
- Conditions
- Beta Thalassemia Major, Sickle Beta 0 Thalassemia, Sickle Beta Plus Thalassemia, Sickle Beta Thalassemia, Sickle Cell Disease, Sickle Cell-SS Disease
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Competing protocol opened in Adult SCT that will include pediatric patients and is now multi-center. No patients enrolled on study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- M.D. Anderson Cancer CenterUniversity, hospital or other
- Study start
- 29 Dec 2020 (actual)
- Primary completion
- 5 Dec 2022 (actual)
- Study completion
- 5 Dec 2022 (estimated)
- First posted
- 2 Mar 2021 (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.
This clinical trial studies the effect of pre-transplant immunosuppression (PTIS) and donor stem cell transplant in treating patients with severe blood diseases (hemoglobinopathies). PTIS helps prepare the body for the transplant and lowers the risk of developing graft versus host disease (GVHD). Hematopoietic cells are found in the bone marrow and produce blood cells. Hematopoietic cell transplantation (HCT) injects healthy hematopoietic cells into the body to support blood cell production. PTIS and HCT may help to control severe hemoglobinopathies.
Detailed description
PRIMARY OBJECTIVE:
- To estimate event-free survival (EFS) at 2-years post HCT.
SECONDARY OBJECTIVES:
- Assess event-free survival (EFS) at 100 days and 1 year post HCT. II. Assess overall survival (OS) at 100 days and 1 year post HCT. III. 30-day transplant-related mortality (TRM). IV. Time to platelet and neutrophil engraftment. V. Rate of graft failure (primary and secondary) through day 100. VI. Incidence of acute graft-versus-host disease (aGVHD) by day 100. VII. Incidence of chronic graft-versus-host disease (cGVHD) by 1 year and 2 years.
VIII. Rate of grade II or greater organ toxicity through day 100. IX. Incidence of hepatic sinusoidal obstruction syndrome (SOS) by day 100. X. Incidence of central nervous system (CNS) toxicities including posterior reversible encephalopathy syndrome (PRES) by day 100.
XI. Incidence of infectious complications through day 100.
EXPLORATORY OBJECTIVES:
- Effect of HCT on clinical and laboratory manifestations of hemoglobinopathies by 1 and 2 years after HCT.
II. Assess the pharmacokinetic profile of busulfan and thymoglobulin to better understand post-transplant outcomes.
III. Assess immune reconstitution kinetics post HCT.
OUTLINE:
DONOR-SPECIFIC ANTI-HLA DESENSITIZATION: Patients with donor-specific anti-HLA antibody titers >= 1:3,000 at the start of PTIS receive rituximab intravenously (IV) on days -69, -41, -28, and -13 and bortezomib IV over less than 1 minute on days -68, -65, -40, and -37 in the absence of unacceptable toxicity. Patients with donor-specific anti-HLA antibody titers > 1:15,000 at the start of PTIS receive receive rituximab IV on days -69, -41, -28, and -13 and bortezomib IV over less than 1 minute on days -68, -65, -62, -58, -40, -37, -34, and -31 in the absence of unacceptable toxicity. Patients with donor-specific anti-HLA antibody titers > 1:5,000 at the start of conditioning may also undergo plasmapheresis on day -12.
PTIS: Patients receive fludarabine phosphate (fludarabine) IV over 1 hour and dexamethasone IV over less than 1 minute on days -68 to -64 and days -40 to -36 in the absence of disease progression or unacceptable toxicity.
CONDITIONING: Patients receive lapine T-lymphocyte immune globulin (rATG) IV over 4-6 hours on days -12 to -10, fludarabine phosphate IV over 1 hour on days -8 to -4, and busulfan IV over 2 hours on days -7 to -4 in the absence of disease progression or unacceptable toxicity.
TRANSPLANT: Patients undergo HCT on day 0.
GVHD PROPHYLAXIS: Patients receive cyclophosphamide IV over 2-3 hours on days 3 and 4 in the absence of unacceptable toxicity. Beginning on day 5, patients also receive tacrolimus IV continuously daily and then orally (PO) twice daily (BID) at the discretion of the treating physician for up to 12 months, and mycophenolate mofetil IV or PO BID up to day 60 in the absence of unacceptable toxicity.
After completion of HCT, patients are followed up periodically for up to 2 years.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (PTIS, HCT) | Experimental | Drug: Bortezomib, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Dexamethasone, Drug: Fludarabine Phosphate, Procedure: Hematopoietic Cell Transplantation, Biological: Lapine T-Lymphocyte Immune Globulin, Drug: Mycophenolate Mofetil, Procedure: Plasmapheresis, Biological: Rituximab, Drug: TacrolimusSee Detailed Description. |
Who may qualify
- Ages
- 2 to 30 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (19 listed)
- Patients 2-30 years-of-age with confirmed sickle cell disease (SCD) (SS \& sickle beta [SB]-thalassemia, both sickle beta 0 [SB0] and sickle beta plus [SB+]) or severe B-thalassemia major are potentially eligible
- Patients with SCD should also meet the following eligibility criteria as outlined by the Center for Medicaid and Medicare Services: sickle cell disease and at least one of the following:
- Patients with B-thalassemia are considered as severe if they are/have any of the following:
- Patients shall not proceed to HCT without confirmation of primary diagnosis by review of available newborn screening results or hemoglobin electrophoresis and/or genetic testing
- DONOR: High resolution HLA typing will be performed on all willing and available biologic parents and siblings without clinically significant hemoglobinopathy. Preference will be given to donors with the lowest number of HLA-allele mismatches
Key exclusion criteria (17 listed)
- Uncontrolled infection
- Females who are pregnant and/or unwilling to cease breastfeeding
- Seropositivity for human immunodeficiency virus (HIV)
- Lansky or Karnofsky performance status < 70%
- Life expectancy severely limited by concomitant illness
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 |
|---|---|---|
| BortezomibAlso: [(1R)-3-Methyl-1-[[(2S)-1-oxo-3-phenyl-2-[(pyrazinylcarbonyl)amino]propyl]amino]butyl]boronic Acid, LDP 341, MLN341, PS-341Given IV | Drug | Treatment (PTIS, HCT) |
| BusulfanAlso: 1, 4-Bis[methanesulfonoxy]butane, BUS, Bussulfam, BusulfanumGiven IV | Drug | Treatment (PTIS, HCT) |
| CyclophosphamideAlso: (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Carloxan, CiclofosfamidaGiven IV | Drug | Treatment (PTIS, HCT) |
| DexamethasoneAlso: Aacidexam, Adexone, Aknichthol Dexa, Alba-DexGiven IV | Drug | Treatment (PTIS, HCT) |
| Fludarabine PhosphateAlso: 2-F-ara-AMP, 9H-Purin-6-amine, 2-fluoro-9-(5-O-phosphono-.beta.-D-arabinofuranosyl)-, Beneflur, FludaraGiven IV | Drug | Treatment (PTIS, HCT) |
| Hematopoietic Cell TransplantationAlso: HCT, Hematopoietic Stem Cell Transplantation, HSCT, Stem Cell TransplantUndergo HCT | Procedure or surgery | Treatment (PTIS, HCT) |
| Lapine T-Lymphocyte Immune GlobulinAlso: Anti-Thymocyte Globulin Rabbit, Grafalon, Rabbit Anti-Human Thymocyte Globulin (RATG), Rabbit Anti-Thymocyte GlobulinGiven IV | Biological | Treatment (PTIS, HCT) |
| Mycophenolate MofetilAlso: CellCept, MMFGiven IV or PO | Drug | Treatment (PTIS, HCT) |
| PlasmapheresisAlso: Plasma Exchange, Therapeutic Plasma Exchange, Therapeutic PlasmapheresisUndergo plasmapheresis | Procedure or surgery | Treatment (PTIS, HCT) |
| RituximabAlso: ABP 798, BI 695500, C2B8 Monoclonal Antibody, Chimeric Anti-CD20 AntibodyGiven IV | Biological | Treatment (PTIS, HCT) |
| TacrolimusAlso: FK 506, Fujimycin, Hecoria, PrografGiven IV or PO | Drug | Treatment (PTIS, HCT) |
Primary outcome
Event-free survival (EFS)
Time frame: At 2 years post-hematopoietic cell transplantation (HCT)
EFS is defined as survival time following HCT without a qualifying event. Will be summarized by the Kaplan-Meier method with 95% confidence intervals.
14 secondary outcomes
Event-free survival
Time frame: Up to 100 days post-HCT
Event-free survival
Time frame: Up to 1 year post-HCT
Overall survival
Time frame: Up to 100 days post-HCT
Overall survival
Time frame: Up to 1 year post-HCT
Transplant-related mortality
Time frame: Up to 30 days post-HCT
Time to platelet and neutrophil engraftment
Time frame: Up to 2 years post-HCT
Incidence of graft failure (primary and secondary)
Time frame: Up to 100 days post-HCT
Incidence of acute graft-versus-host disease
Time frame: Up to 100 days post-HCT
Incidence of chronic graft-versus-host disease
Time frame: Up to 1 year post-HCT
Incidence of chronic graft-versus-host disease
Time frame: Up to 2 years post-HCT
Incidence of grade II or greater organ toxicity
Time frame: Up to 100 days post-HCT
Incidence of hepatic sinusoidal obstruction syndrome
Time frame: Up to 100 days post-HCT
Incidence of central nervous system toxicities including posterior reversible encephalopathy syndrome
Time frame: Up to 100 days post-HCT
Incidence of infectious complications
Time frame: Up to 100 days post-HCT
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Oct 2024.
- MD Anderson 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.
Kris M Mahadeo principal investigator
M.D. Anderson Cancer Center
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Bortezomib, Busulfan and Cyclophosphamide in people with Beta Thalassemia Major and Sickle Beta 0 Thalassemia, run by M.D. Anderson Cancer Center.
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 19 inclusion and 17 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: withdrawn, as of the sponsor's last update on 24 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.
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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Event-free survival (EFS) (time frame: At 2 years post-hematopoietic cell transplantation (HCT)). 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 24 Oct 2024.