Blood CancerPhase 2NCT00185692
Allogeneic Transplantation From Related Haploidentical Donors
A Phase 2 study testing non-myeloablative hematopoietic cell transplantation, Anti-Thymocyte Globulin and Cyclosporine in people with Blood Cancer and Leukemia, run by Stanford University at 1 site in United States.
Trial summary
The purpose of the study is to evaluate the feasibility and safety of transplanting CD34+ selected hematopoietic cells from a haploidentical related donor following a nonmyeloablative regimen of total lymphoid irradiation (TLI) and antithymocyte globulin (ATG). Read more about this study
- Who is it for?
- Ages 12 months and older
- Conditions the study is for
- Blood Cancer, Leukemia, Graft Versus Host Disease
- What is being tested?
- non-myeloablative hematopoietic cell transplantation, Anti-Thymocyte Globulin and Cyclosporine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 16 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2000to Dec 2010
- Phase
- Phase 2
- 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 12 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Age ≥ 50 years with hematologic malignancies treatable by a mixed chimera allogeneic HCT.
- For patients ≤ 50 years of age with hematologic malignacies treatable with mixed chimera HCT who because of pre-exisiting medical conditions or prior therapy are considered to be at high risk for regimen-related toxicity associated with conventional transplants.
- Indolent advanced stage NHL, CLL, HD - Must have received and failed front-line therapy.
- Multiple myeloma (Stage II or III) - Must have received prior chemotherapy. Consolidation after prior autografting is permitted.
- AML/ALL - Must be in complete hematologic remission and have received cytotoxic chemotherapy at some stage before transplant. Patients with molecular or cytogenetic relapse will be accepted providing a donor is available. Patients with persistent or refractory disease will be considered on a case by case basis and transplants must be approved by the principal investigator.
- CML - Patients will be accepted in chronic or accelerated phase. Patients who have received prior autografts after high dose therapy or have undergone intensive chemotherapy for either peripheral blood stem cell mobilization or treatment of advanced CML may be enrolled provided they are in CR, chronic phase or accelerated phase.
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- MDS - All patients with MDS will be eligible for this protocol, however, those patients with >10% blasts will require chemotherapy to reduce the blast % to < 10%.
- SAA - Patients with severe aplastic anemia who have failed front line therapy.
- A fully HLA-identical sibling donor is not available.
- A matched unrelated donor has not been identified.
- A haploidentical related donor is available who is in good health and does not have contraindications to donation.
Who cannot join
- Patients with rapidly progressive intermediate or high grade NHL
- Uncontrolled CNS involvement with disease
- Fertile men
- Women unwilling to use contraceptive techniques during and for 12 months following treatment
- Females who are pregnant
- Cardiac function: ejection fraction < 30% or cardiac failure requiring therapy
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- Pulmonary: DLCO < 40% predicted and/or receiving supplementary continuous oxygen
- Liver function abnormalities: elevation of bilirubin to > 4 mg/dl and/or transaminases > 3x the upper limit of normal. If hyperbilirubinemai is due to a known cause that will not increase the risks of transplant, than this upper limit may be exceeded.
- Renal: creatinine clearance < 50 cc/min (24 hour urine collection)
- Karnofsky performance score < 60%
- Patients with poorly controlled hypertension.
- Documented fungal disease that persists despite treatment
- HIV positive patients.
- Hepatitis B and C positive patients will be evaluated on a case by case basis
- Psychiatric disorders or psychosocial problems which in the opinion of the primary physician or principal investigator would place the patient at unacceptable risk from this regimen.
Trial locations and contacts
1 site in 1 country.
United States1 site
Stanford University School of Medicine
Stanford, California, 94305
About this study
Official title: Allogeneic Hematopoietic Cell Transplantation of Positively Selected CD34+ Cells and Defined Inoculum of T Cells From Related Haploidentical Donors for Older Patients With Indolent Hematologic Malignancies
The purpose of the study is to evaluate the feasibility and safety of transplanting CD34+ selected hematopoietic cells from a haploidentical related donor following a nonmyeloablative regimen of total lymphoid irradiation (TLI) and antithymocyte globulin (ATG).
Read the detailed description
An alternative to conventional allogeneic bone marrow transplantation is by using a non-myeloablative conditioning regimen. This regime would consist of both; total lymphoid irradiation (TLI) and anti-thymocyte globulin (ATG). Used in combination to achieve engraftment of haploidentical CD34+ selected peripheral blood stem cells in older patients or patients with underlying medical conditions that preclude standard allogeneic treatment. The expected results of this transplant regime will be expected to result in hematopoietic and immunologic reconstitution, decreased deaths related to the treatment regimen and decreased gravft-vs-host disease (GVHD).
- Conditions
- Blood Cancer, Leukemia, Graft Versus Host Disease, Malignancy, CLL, NHL
- Being tested
- non-myeloablative hematopoietic cell transplantation, Anti-Thymocyte Globulin, Cyclosporine, Mycophenolate Mofetil, G-CSF
- Medical subject headings
- Hematologic Neoplasms, Leukemia, Graft vs Host Disease, Neoplasms, Hodgkin Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
non-myeloablative hematopoietic cell transplantationProcedure or surgery
Also called Peripheral-blood stem-cell transplantation
TLI and ATG infusion of the donor graft Post-transplant immunosuppression with cyclosporine and mycophenolate mofetil.
Anti-Thymocyte GlobulinDrug
Also called ATG
1.5 mg/kg QD x 5, IV. Dosage will be based on body weight. Purified, sterile IgG fraction of immune serum of rabbits immumixied with human thymus lymphocyte. This drug acts to modify the number and function of lymphocytes.
CyclosporineDrug
Also called INN/BAN, USAN, CSA
6.25 mg/kg BID, PO.Mechanism of action is inhibition of T-cell activation by binding to a cytoplasmic protein (cyclophillin).
Also called MMF, CellCept
15 mg/kg Q 8 hours, PO. Inhibtis the enzme inosine monophsophate dehydrogenase (MPDII) noncompetitively which blocks the de nobo synthesis of guanosine required for DNA synthesis and has an effect on T and B cells.
G-CSFDrug
Also called Granulocyte colony-stimulating factor, CSF 3
16 mg/kg, SQ Growth factor used to make bone marrow produce more blood cells
SolumedrolDrug
Also called 6-Methylprednisolone, Methylprednisolone Acetate, Methylprednisolone Sodium Succinate
1.0 mg/kg IV 2 hours prior to ATG Used to treat severe inflamation
AcetaminophenDrug
Also called Tylenol
650 mg PO, 30 minutes prior to infusion Pain reliever
DiphenydramineDrug
Also called Benadryl, Allermax, Q-Dryl, Diphen Cough
50 mg IV, 30 minutes prior to infusion Used to relieve allergy symptoms
HydrocortisoneDrug
Also called Hydrocortisone Sodium Phosphate
100 mg IV, 1 hour prior to infusion Used to relieve itching, redness and swelling of the skin
| Study group | Role | What they receive |
|---|---|---|
| Transplantation of CD34+ cells | Experimental | Week #1: Total Lymphoid Inrradiation (TLI) 120 cGy + Anti-thymocyte Globulin (ATG) 1.5 mg/kg + Solumedrol 1.0 mg/kg Daily for 5 days.
Week #2: TLI 120 cGy (3 days a week, double on the 4th day) 5 days of CSP (oraly) one day after TLI was started. 3 days of MMF 4 days after TLI was started. Receives: Procedure: non-myeloablative hematopoietic cell transplantation; Drug: Anti-Thymocyte Globulin; Drug: Cyclosporine; Drug: Mycophenolate Mofetil; Drug: G-CSF; Drug: Solumedrol; Drug: Acetaminophen; Drug: Diphenydramine; Drug: Hydrocortisone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning
number achieving donor cell engraftment (>95%) by day 90 after transplant.
Measured: 100 days
Secondary outcomes (1)
Acute Graft-versus-Host Disease (GVHD) Grade 2-4 Risk From Time of Transplant Until Day 90 Post-transplant
GVHD grading system goes from 0-4 where grade 4 is the most severe. Grade 0 and 1 do not require systemic treatment, Grade 2-4 require treatment. This trial evaluated the risk of developing acute GVHD grades 2-4 within 90 days of transplant.
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Sep 2005 (estimated)
- Study startAug 2000
- Primary completionDec 2010
- Study completionDec 2010
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 Blood CancerStudies of the same condition, open and under way.
- More trials run by Stanford 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 16 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stanford University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Robert Lowsky, Stanford University
- Investigators
- Robert Lowsky, Principal investigator, Stanford University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00185692
- Sponsor's protocol ID
- IRB-13371
- Other IDs
- 75117, BMT124
- Status last verified
- Jan 2015
- First submitted
- 12 Sep 2005
- Last updated
- 4 Dec 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Dec 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Allogeneic Hematopoietic Cell Transplantation of Positively Selected CD34+ Cells and Defined Inoculum of T Cells From Related Haploidentical Donors for Older Patients With Indolent Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT00185692
- Acronym / sponsor ID
- IRB-13371
- Conditions
- Blood Cancer, Leukemia, Graft Versus Host Disease, Malignancy, CLL, NHL
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 16 participants (actual)
- Lead sponsor
- Stanford UniversityUniversity, hospital or other
- Study start
- Aug 2000
- Primary completion
- Dec 2010 (actual)
- Study completion
- Dec 2010 (actual)
- First posted
- 16 Sep 2005 (estimated)
- Results
- Posted 26 Jan 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the feasibility and safety of transplanting CD34+ selected hematopoietic cells from a haploidentical related donor following a nonmyeloablative regimen of total lymphoid irradiation (TLI) and antithymocyte globulin (ATG).
Detailed description
An alternative to conventional allogeneic bone marrow transplantation is by using a non-myeloablative conditioning regimen. This regime would consist of both; total lymphoid irradiation (TLI) and anti-thymocyte globulin (ATG). Used in combination to achieve engraftment of haploidentical CD34+ selected peripheral blood stem cells in older patients or patients with underlying medical conditions that preclude standard allogeneic treatment. The expected results of this transplant regime will be expected to result in hematopoietic and immunologic reconstitution, decreased deaths related to the treatment regimen and decreased gravft-vs-host disease (GVHD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Transplantation of CD34+ cells | Experimental | Procedure: non-myeloablative hematopoietic cell transplantation, Drug: Anti-Thymocyte Globulin, Drug: Cyclosporine, Drug: Mycophenolate Mofetil, Drug: G-CSF, Drug: Solumedrol, Drug: Acetaminophen, Drug: Diphenydramine, Drug: HydrocortisoneWeek #1: Total Lymphoid Inrradiation (TLI) 120 cGy + Anti-thymocyte Globulin (ATG) 1.5 mg/kg + Solumedrol 1.0 mg/kg Daily for 5 days. Week #2: TLI 120 cGy (3 days a week, double on the 4th day) 5 days of CSP (oraly) one day after TLI was started. 3 days of MMF 4 days after TLI was started. |
Who may qualify
- Ages
- 12 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Age ≥ 50 years with hematologic malignancies treatable by a mixed chimera allogeneic HCT.
- For patients ≤ 50 years of age with hematologic malignacies treatable with mixed chimera HCT who because of pre-exisiting medical conditions or prior therapy are considered to be at high risk for regimen-related toxicity associated with conventional transplants.
- Indolent advanced stage NHL, CLL, HD - Must have received and failed front-line therapy.
- Multiple myeloma (Stage II or III) - Must have received prior chemotherapy. Consolidation after prior autografting is permitted.
- AML/ALL - Must be in complete hematologic remission and have received cytotoxic chemotherapy at some stage before transplant. Patients with molecular or cytogenetic relapse will be accepted providing a donor is available. Patients with persistent or refractory disease will be considered on a case by case basis and transplants must be approved by the principal investigator.
Key exclusion criteria (15 listed)
- Patients with rapidly progressive intermediate or high grade NHL
- Uncontrolled CNS involvement with disease
- Fertile men
- Women unwilling to use contraceptive techniques during and for 12 months following treatment
- Females who are pregnant
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 |
|---|---|---|
| non-myeloablative hematopoietic cell transplantationAlso: Peripheral-blood stem-cell transplantationTLI and ATG infusion of the donor graft Post-transplant immunosuppression with cyclosporine and mycophenolate mofetil. | Procedure or surgery | Transplantation of CD34+ cells |
| Anti-Thymocyte GlobulinAlso: ATG1.5 mg/kg QD x 5, IV. Dosage will be based on body weight. Purified, sterile IgG fraction of immune serum of rabbits immumixied with human thymus lymphocyte. This drug acts to modify the number and function of lymphocytes. | Drug | Transplantation of CD34+ cells |
| CyclosporineAlso: INN/BAN, USAN, CSA6.25 mg/kg BID, PO.Mechanism of action is inhibition of T-cell activation by binding to a cytoplasmic protein (cyclophillin). | Drug | Transplantation of CD34+ cells |
| Mycophenolate MofetilAlso: MMF, CellCept15 mg/kg Q 8 hours, PO. Inhibtis the enzme inosine monophsophate dehydrogenase (MPDII) noncompetitively which blocks the de nobo synthesis of guanosine required for DNA synthesis and has an effect on T and B cells. | Drug | Transplantation of CD34+ cells |
| G-CSFAlso: Granulocyte colony-stimulating factor, CSF 316 mg/kg, SQ Growth factor used to make bone marrow produce more blood cells | Drug | Transplantation of CD34+ cells |
| SolumedrolAlso: 6-Methylprednisolone, Methylprednisolone Acetate, Methylprednisolone Sodium Succinate1.0 mg/kg IV 2 hours prior to ATG Used to treat severe inflamation | Drug | Transplantation of CD34+ cells |
| AcetaminophenAlso: Tylenol650 mg PO, 30 minutes prior to infusion Pain reliever | Drug | Transplantation of CD34+ cells |
| DiphenydramineAlso: Benadryl, Allermax, Q-Dryl, Diphen Cough50 mg IV, 30 minutes prior to infusion Used to relieve allergy symptoms | Drug | Transplantation of CD34+ cells |
| HydrocortisoneAlso: Hydrocortisone Sodium Phosphate100 mg IV, 1 hour prior to infusion Used to relieve itching, redness and swelling of the skin | Drug | Transplantation of CD34+ cells |
Primary outcome
Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning
Time frame: 100 days
number achieving donor cell engraftment (>95%) by day 90 after transplant.
1 secondary outcome
Acute Graft-versus-Host Disease (GVHD) Grade 2-4 Risk From Time of Transplant Until Day 90 Post-transplant
Time frame: 90 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Dec 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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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.
Robert Lowsky principal investigator
Stanford University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing non-myeloablative hematopoietic cell transplantation, Anti-Thymocyte Globulin and Cyclosporine in people with Blood Cancer and Leukemia, run by Stanford 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 11 inclusion and 15 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: finished, as of the sponsor's last update on 4 Dec 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Jan 2017). 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?
Engraftment of Haploidentical CD34+ Selected Blood Stem Cells in Older Patients or Those With Medical Co-morbidities Following Total Lymphoid Irradiation and Antithymocyte Globulin Transplant Conditioning (time frame: 100 days). 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 4 Dec 2019.