Clinical trial NCT01010217
Conditions: Blood Stem Cell Transplant Failure Leukemia Hematologic Malignancies
Keywords: Hematologic Neoplasms Blood Disorders Blood Cancer Transplantation, Blood And Marrow Acute myelogenous leukemia Myelodysplastic syndrome Acute lymphocytic leukemia Aplastic anemia Chronic myelogenous leukemia Chronic lymphocytic leukemia Myeloproliferative disease Hodgkin's disease Non-Hodgkin's lymphoma Multiple myeloma Cyclophosphamide Fludarabine Melphalan Mesna Rituximab Tacrolimus Prograf Mycofenolate mofetil MMF CellCept G-CSF Filgrastim Neupogen
Sponsor: M.D. Anderson Cancer Center
Start date: 2009-11-05; Completion date: 2017-10-05
Enrollment: 176 participants; Allocation: NON_RANDOMIZED; Intervention model: SINGLE_GROUP

Brief summary:
The goal of this clinical research study is to learn about the safety of giving a stem cell transplant from a tissue-mismatched donor, followed by cyclophosphamide, to patients with certain types of blood disorders or blood cancers. Melphalan, thiotepa, and fludarabine will also be given before the transplant.

Researchers will study the health status of these patients at 3 months after the transplant.

Detailed description:
The Study Treatment:

Cyclophosphamide is designed to interfere with the multiplication of cancer cells, which may slow or stop their growth and spread throughout the body. This may cause the cancer cells to die.

In this study, researchers want to learn if cyclophosphamide can help to prevent graft-versus-host disease (GVHD -- when transplanted immune tissue, such as white blood cells, attacks the tissues of the recipient's body).

Melphalan, thiotepa, and fludarabine are commonly used in combination with a stem cell transplant.

Study Treatment Administration:

If you are found to be eligible to take part in this study, you will receive chemotherapy for 6 days:

* You will receive melphalan by vein over 30 minutes on Day -8 (8 days before the transplant).
* You will receive thiotepa by vein over 4 hours on Day -7.
* You will receive fludarabine by vein over 1 hour on Days -6, -5, -4, and -3.

If thiotepa is not available, you will receive melphalan by vein over 30 minutes on Day -6 and fludarabine by vein over 1 hour on Days -5, -4, -3, and -2. You will receive total body irradiation (TBI) on Day -1.

On Day 0, you will receive the donor's stem cells by vein. This may last anywhere from 15 minutes to several hours.

On Days 3 and 4, you will receive cyclophosphamide by vein over 3 hours. You will also receive mesna by vein over 30 minutes every 4 hours for a total of 10 mesna doses on Days 3 and 4. Mesna is given to lower the risk of side effects to the bladder.

Starting on Day 5, you will receive tacrolimus and mycophenolate mofetil (MMF) to help lower the risk of GVHD. Tacrolimus will be given by vein as a continuous infusion for about 2 weeks. After the 2 weeks of taking tacrolimus by vein, you will take tacrolimus by mouth as a pill for at least 3 months. MMF will be given by mouth, 3 times a day, usually until Day 35.

Starting on Day 7, you will receive filgrastim (G-CSF) once a day as an injection under the skin, until your blood cell counts reach a high enough level.

Depending on the type of disease that you have, your doctor may decide to give you rituximab by vein over several hours on Days -13, -6, 1, and 8. Rituximab is given to help the body get rid of abnormal white blood cells.

Length of Study Participation:

You will be in the hospital for about 4 weeks after the transplant. You will be taken off study if the disease gets worse. The study drugs will be stopped if intolerable side effects occur.

Follow-Up Visits:

You will be asked to stay close enough to Houston to be able to come back for any visits for at least 100 days after the transplant.

At 1, 3, 6, and 12 months after the transplant, the following tests and procedures will be performed:

* You will have a physical exam.
* Blood (about 4 tablespoons) will be drawn for routine tests.
* You will have a bone marrow biopsy/aspirate to check the status of the disease. If you have lymphoma or aplastic anemia, you may have a bone marrow biopsy/aspirate at 3, 6, and 12 months, if your doctor thinks it is needed.
* Blood (about 4 tablespoons) will be drawn to measure tumor cells and to predict graft failure and/or relapse.
* You may have urine collected and/or scans performed such as x-rays, CT scans, and/or a positron emission tomography (PET) scan. These scans and urine tests would only be done if the study doctor thinks they are needed to check the status of the disease.
* Blood (about 4 tablespoons) will be drawn to check your immune system.

If you have AML, at 2 months after the transplant, blood (about 4 tablespoons) will be drawn to check your immune system.

If you have MM, you will have a bone survey once a year.

If the study doctor thinks it is needed based on side effects you may be having, additional follow-up tests will be performed.

You may be contacted by phone 1-2 times a year to ask about the status of the disease. These calls will take about 10 minutes to complete.

This is an investigational study. All of the drugs used in this study are commercially available and FDA approved. However, it is investigational to give high-dose cyclophosphamide for preventing GVHD that may occur after a stem cell transplant from a tissue-mismatched donor.

Up to 337 patients will take part in this study. All will be enrolled at MD Anderson.

Arms: arm 1: Arm 1 - Stem Cell Transplantation (SCT), Melphalan 140 mg/m\^2 , Thiotepa 5 mg/kg, Fludarabine 40 mg/m\^2 + high-dose post-transplant cyclophosphamide 50 mg/kg/day arm 2: Arm 2 - SCT, Melphalan, Thiotepa, Fludarabine + high-dose post-transplant cyclophosphamide. arm 3: Arm 3 - SCT, Melphalan, Thiotepa, Fludarabine + high-dose post-transplant cyclophosphamide
Interventions: intervention 1: Cyclophosphamide intervention 2: Fludarabine intervention 3: Melphalan intervention 4: Mesna intervention 5: Rituximab intervention 6: Stem Cell Transplantation intervention 7: Thiotepa intervention 8: Tacrolimus intervention 9: Mycofenolate mofetil intervention 10: G-CSF
Intervention descriptions: intervention 1: 50 mg/kg/day intravenous (IV) over 3 hours on Days 3 and 4. intervention 2: 40 mg/m\^2 IV over 1 hour on Days -6, -5, -4, and -3. intervention 3: 140 mg/m\^2 IV (or 100 mg/m\^2 with reduced intensity Regimen 2) over 30 minutes on Day -8. intervention 4: 10 mg/kg IV every 4 hours for a total of 10 doses starting just prior to first dose of Cyclophosphamide on Days 3 and 4. intervention 5: CD20+ lymphoid malignancies: 375 mg/m2 on Day -13 followed by 1000 mg/m2 on Day -6, +1, and +8. intervention 6: Infusion of donor's stem cells by vein on Day 0, may last anywhere from 15 minutes to several hours. intervention 7: 5 mg/kg Regimen 1 (or 5 mg/kg with reduced intensity Regimen 2) IV over 4 hours on Day -7. intervention 8: 0.015 mg/kg by vein or orally daily starting on Day +5 for 3 months intervention 9: 15 mg/kg/dose orally three times a day starting on Day +5 to Day +100 or otherwise indicated intervention 10: 5 mcg/kg/day subcutaneously starting Day 7 once a day daily until neutrophil recovery \> 1000/mcl.

Protocol documents:
Prot_SAP_000.pdf: 
2009-0266
March 26, 2015
Abstract - Page 1
Protocol Abstract Page
Three-arm Clinical Trial for Patients with Hematologic Malignancies
and Mismatched Donors - Haploidentical, 1 Antigen Mismatch Related or 
Unrelated, and Matched unrelated donor (MUD)- Using a T-cell Replete 
Allograft and High-dose Post-transplant Cyclophosphamide
2009-0266
Core Protocol Information
Study Chairman: 
Stefan Ciurea
Department:
Stem Cell Transplantation and Cellular Therapy
Phone:
713-794-5780
Unit:
423
Full Title: 
Three-arm Clinical Trial for Patients with Hematologic Malignancies
and Mismatched Donors - Haploidentical, 1 Antigen Mismatch Related or Unrelated, and 
Matched unrelated donor (MUD)- Using a T-cell Replete Allograft and High-dose 
Post-transplant Cyclophosphamide
Protocol Phase:
Phase II
Version Status:
Terminated 10/05/2017
Version:
33
Document Status:
Final
Abstract
Objectives:
To determine the safety and 100-day non-relapse mortality (NRM) of T-cell replete allogeneic stem cell 
transplantation using melphalan, thiotepa, and fludarabine conditioning followed by high-dose 
post-transplant cyclophosphamide for patients with hematologic malignancies without a matched donor, 
treated on 5 arms: 1) Haploidentical related, 2) One antigen mismatched related or unrelated, 3) Matched 
unrelated donor (MUD), 4) Second transplant, and 5) Myelofibrosis. 
Secondary objectives
1.
To determine the NRM at 6 months.
2.
To estimate the proportion of patients with engraftment/graft failure. 
3.
To estimate the cumulative incidence of grade III-IV Acute Graft vs. Host Disease (aGVHD).
4.
To assess immune reconstitution and the incidence of infectious episodes
5.
To assess the rate of chronic GVHD within the first year post transplantation
6.
To assess disease response, disease-free survival (DFS) and overall survival (OS) after 
transplantation. 


2009-0266
March 26, 2015
Abstract - Page 2
Rationale: (Be as concise as possible)
Haploidentical stem cell transplantation has been associated traditionally with a high rate of 
graft-versus-host disease. Recently, a novel immunosupression regimen using high-dose post transplant 
cyclophosphamide has been reported for patients receiving a haploidentical graft. Using this approach 
the rates of GVHD are similar with those seen in the matched sibling donor setting. We are proposing to 
use this approach for patients with hematologic malignancies who lack a matched sibling or unrelated 
donor.
Eligibility: (List All Criteria)
Inclusion:
1)
Patients < 55 years (Myeloablative regimen #1) or > 55 and </= 75 years or significant 
comorbidities (Reduced intensity regimen #2) old lacking a matched related volunteer donor identified in 
time for transplant for which a related haploidentical donor (</= 7/8 allele match at the A, B, C, DR loci), a 
7/8 allele matched related or unrelated donor is identified, or a matched unrelated donor (MUD). The 
patients must be diagnosed with a high-risk disease defined as following:
2)
Acute lymphocytic leukemia (ALL) in CR1 with high-risk features including adverse cytogenetics 
such as t(9;22), t(1;19), t(4;11), or MLL generearrangements; ALL in second or greater remission or ALL 
with relapsed disease, peripheral blood blasts < 1000/microliter, ALL patients must show response to 
most recent recieved chemotherapy;
3)
Acute myeloid leukemia (AML) in CR1 with intermediate-risk disease or high-risk features defined 
as: Greater than 1 cycle of induction therapy required to achieve remission; Preceding myelodysplastic 
syndrome (MDS) or myeloproliferative disease; Presence of FLT3 mutations or internal tandem 
duplications; FAB M6 or M7 classification; Adverse cytogenetics, -5, del 5q, -7, del7q, abnormalities 
involving 3q, 9q, 11q, 20q, 21q, 17, +8 [> 3 abnormalities], peripheral blood blasts <1000/microliter, AML 
patients must show response to most recent recieved chemotherapy;
4)
AML in second or greater remission, primary induction failure and patients with relapsed disease, 
peripheral blood blasts <1000/microliter; patients > 55 years and </= 75 years need to be in morphologic 
remission at transplant (< 5% blasts).
5)
Myelodysplastic syndrome (MDS) with International Prognostic Scoring System (IPSS) 
intermediate-2 or higher; or therapy-related MDS
6)
Aplastic anemia with Absolute neutrophil count (ANC)<1000 and transfusion dependent after they 
failed immunospresssion therapy
7)
Chronic myeloid leukemia (CML) >/=1st chronic phase, after failed >/=2 lines of tyrosine kinase 
inhibitors; in accelerated or blast phase with > 30% bone marrow blasts;
8)
Prior allogeneic stem cell transplant more than 6 months from the first transplant, in remission.
9)
Chemotherapy-sensitive relapsed lymphoma (Complete or partial response), Hodgkin’s or 
non-Hodgkin’s lymphoma, no evidence of "bulky" disease (> 10 cm in diameter);
10)
Patients with chemo-sensitive CLL with persistent or recurrent disease after fludarabine-based 
regimens, no evidence of "bulky" disease (> 10 cm in diameter)
11)
Patients with poor prognosis multiple myeloma by cytogenetics (del13, del 17p, t(1;14) or t(14;16) 
or hypodiploidy, with advanced disease (stage>/=2) and /or relapsed after autologous stem cell 


2009-0266
March 26, 2015
Abstract - Page 3
transplant.
12)
Patients with myelofibrosis (Lille >0, transfusion dependency, progression to blast phase; however, 
in remission from AML) or CMML. These patients will be treated with the reduced-intensity conditioning 
regimen #2 and will be subject to the same stopping rule as the group >/= 55 years or with comorbidities.
13)
Zubrod performance status 0-1 or Lansky PS greater or equal to 70%.
14)
Patients above >/=65 years old should have an age-adjusted co-morbidity index of </= 3.
15)
Available donor able to undergo a bone marrow harvest. For matched unrelated donor transplants 
only: Peripheral blood stem cells may be collected if donor is unavailable for bone marrow harvest or if 
adequate bone marrow cannot be collected.
16)
Bilirubin </= 1.5 mg/dl (unless Gilbert's syndrome), ALT or AST </= 200 IU/ml.
17)
Serum creatinine clearance >/=50 ml/min (calculated with Cockroft-Gault formula); Creatinine for 
children </=1.5 mg/dl or </=2 times upper limit of normal for age (whichever is less);
18)
Diffusing capacity for carbon monoxide (DLCO) >/= 45% predicted corrected for hemoglobin. For 
pediatric patients, if unable to perform pulmonary function, >/= 92% oxygen saturation with pulse 
oximetry.
19)
LVEF >/= 40%.
20)
Patient or patient’s legal representative, parent(s) or guardian should provide written informed 
consent. Assent of a minor if participant’s age is at least seven and less than eighteen years.
Exclusion:
1)
HIV positive; active hepatitis B or C
2)
Patients with active infections. The PI is the final arbiter of the eligibility.
3)
Liver cirrhosis with greater than grade 1 stage 1 inflammation/fibrosis
4)
Uncontrolled CNS involvement by tumor cells
5)
Patients with AML must have less than 30% bone marrow blasts and no peripheral blood blasts.
6)
History of another primary malignancy that has not been in remission for at least 3 years. (The 
following are exempt from the 3-year limit: non-invasive nonmelanoma skin cancer, fully excised 
melanoma in situ [Stage 0], curatively treated localized prostate cancer, and cervical carcinoma in situ on 
biopsy or a squamous intraepithelial lesion on PAP smear.)
7)
Positive Beta HCG test in a woman with child bearing potential defined as not post-menopausal for 
12 months or no previous surgical sterilization.
8)
Inability to comply with medical therapy or follow-up
Is there an age limit?  Yes
Why?  Provide scientific justification:
Above age 75 the expected outcomes are significantly worse.


2009-0266
March 26, 2015
Abstract - Page 4
Disease Group:
Blood And Marrow Transplantation
Treatment Agents/Devices/Interventions:
Cyclophosphamide, Fludarabine, Melphalan, MESNA, Rituximab
Proposed Treatment/Study Plan:
Patients will be treated on 4 groups: Group 1 – Haploidentical transplant patients; Group 2–
One antigen mismatched related or unrelated; Group 3 - Matched unrelated donor (MUD) 
and Group 4 Second transplant. 
Patients with lymphoma will be treated with the reduced intensity regimen (#2).
Patients  < 55 years will receive the myeloablative regimen (#1). 
Patients age > 55 and < 65 years old or that in the opinion of the investigator(s) would 
preclude myeloablative therapy may receive the reduced intensity regimen (#2). 
Patients with CLL or low-grade lymphoma may be treated with the reduced-intensity 
conditioning regimen even if less than 55 years at the discretion of the treating physician. 
For matched unrelated donor transplants only: Peripheral blood stem cells may be used if donor is 
unavailable for bone marrow harvest or if adequate bone marrow cannot be collected. 
Overweight patients may receive peripheral blood instead of bone marrow stem cells due to low 
CD34+ cell numbers per kilogram that can be collected from the donor. Peripheral blood collection is 
recommended for obese individuals (if patient’s weight is >/= 1.5x the donor weight). 
Myeloablative Regimen #1 with Rituximab
Day -13
**Rituximab 375mg/m
2
Day -9
Admit / Hydration IV (Sunday - Wednesday)
Day -8
Melphalan 140 mg/m2 (Patients <12 kg receive 4.6 mg/kg)
Day -7
Thiotepa  5 mg/kg
Day -6
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day) 
**Rituximab 1000mg/m
2
Day -5
Fludarabine 40 mg/m
2  (Patients <12 kg receive 1.3 mg/kg/day)
Day -4
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -3
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -2
Rest
Day -1
Rest


2009-0266
March 26, 2015
Abstract - Page 5
Day 0
Stem Cell Infusion
Day +1
**Rituximab 1000mg/m
2
Day +3
Cyclophosphamine 50 mg/kg/day
Day +4
Cyclophosphamine 50 mg/kg/day
Day +5
Tacrolimus start 0.015 mg/kg/day IV or po for 3 months
MMF 15 mg/kg/dose po TID to Day +100 or otherwise indicated
Day +7
                Start G-CSF 5mcg/kg/day (rounded up to the nearest vial) 
+8
               **Rituximab 1000mg/m
2
**CD20+ lymphoid malignancies: Rituximab 375mg/m2 on day -13 followed by 1000mg/m2 on day -6, +1, and 
+8.  Prophylaxis according to BMT standard practice
Myeloablative Regimen #1 without Rituximab
Day -9
Admit / Hydration IV (Sunday - Wednesday)
Day -8
Melphalan 140 mg/m2 (Patients <12 kg receive 4.6 mg/kg)
Day -7
Thiotepa  5 mg/kg
Day -6
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day) 
Day -5
Fludarabine 40 mg/m
2  (Patients <12 kg receive 1.3 mg/kg/day)
Day -4
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -3
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -2
Rest
Day -1
Rest
Day 0
Stem Cell Infusion
Day +3
Cyclophosphamine 50 mg/kg/day
Day +4
Cyclophosphamine 50 mg/kg/day
Day +5
Tacrolimus start 0.015 mg/kg/day IV or po for 3 months
MMF 15 mg/kg/dose po TID to Day +100 or otherwise indicated
Day +7
                Start G-CSF 5mcg/kg/day (rounded up to the nearest vial) 


2009-0266
March 26, 2015
Abstract - Page 6
Reduced Intensity Regimen #2 with Rituximab
Day -13
**Rituximab 375mg/m
2
Day -9
Admit / Hydration IV (Sunday - Wednesday)
Day -8
Melphalan 100 mg/m2 (Patients <12 kg receive 4.6 mg/kg)
Day -7
Thiotepa  5 mg/kg
Day -6
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day) 
**Rituximab 1000mg/m
2
Day -5
Fludarabine 40 mg/m
2  (Patients <12 kg receive 1.3 mg/kg/day)
Day -4
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -3
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -2
Rest
Day -1
Rest
Day 0
Stem Cell Infusion
Day +1
**Rituximab 1000mg/m
2
Day +3
Cyclophosphamine 50 mg/kg/day
Day +4
Cyclophosphamine 50 mg/kg/day
Day +5
Tacrolimus start 0.015 mg/kg/day IV or po for 3 months
MMF 15 mg/kg/dose po TID to Day +100 or otherwise indicated
Day +7
                Start G-CSF 5mcg/kg/day (rounded up to the nearest vial) 
Day +8
               **Rituximab 1000mg/m
2
**CD20+ lymphoid malignancies: Rituximab 375mg/m2 on day -13 followed by 1000mg/m2 on day -6, +1, and 
+8.  Prophylaxis according to BMT standard practice
Reduced Intensity Regimen #2 without Rituximab
Day -9
Admit / Hydration IV (Sunday - Wednesday)
Day -8
Melphalan 100 mg/m2 (Patients <12 kg receive 4.6 mg/kg)
Day -7
Thiotepa  5 mg/kg
Day -6
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day) 
Day -5
Fludarabine 40 mg/m
2  (Patients <12 kg receive 1.3 mg/kg/day)


2009-0266
March 26, 2015
Abstract - Page 7
Day -4
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -3
Fludarabine 40 mg/m
2
  (Patients <12 kg receive 1.3 mg/kg/day)
Day -2
Rest
Day -1
Rest
Day 0
Stem Cell Infusion
Day +3
Cyclophosphamine 50 mg/kg/day
Day +4
Cyclophosphamine 50 mg/kg/day
Day +5
Tacrolimus start 0.015 mg/kg/day IV or po for 3 months
MMF 15 mg/kg/dose po TID to Day +100 or otherwise indicated
Day +7
                Start G-CSF 5mcg/kg/day (rounded up to the nearest vial) 


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Statistical Considerations:
General


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Where Will Participants Be Enrolled:
Only at MDACC
Is this an NCI-Cancer Therapy Evaluation Protocol (CTEP)?
No
Is this an NCI-Division of Cancer Prevention Protocol (DCP)?
No
Estimated Accrual:
Total Accrual at MDACC:
337
Estimated monthly accrual at MDACC:
4
Accrual Comments:
The maximum total sample size will be 337 patients, 122 in the first group, 98 in the second group, 48 in 
the third group, 24 in the fourth group, 21 in the fifth group, and 24 in the sixth group, with accrual 
estimated to take 3 years.
Do you expect your target population to include non-english 
speaking participants?
Yes
Please select expected languages of non-English speaking participants.  (Select all that apply)
Expected languages of non-English speaking participants:
Spanish
Location of Treatment:
This protocol is performed on an Inpatient AND Outpatient basis.
Length of Stay: What is the length & frequency of hospitalization?
Duration of hospitalization is the standard duration for patients receiving a stem cell transplant 
(approximately 30 days).
Return Visits: How often must participants come to MDACC?
Frequent visits initially to Ambulatory Treatment Center up to 100 days after transplant  followed by 
periodic clinic visits.
Home Care: Specify what, if any, treatment may be given at home.
N/A
Name of Person at MDACC Responsible for Data Management:  Patricia Cole
Prior protocol at M. D. Anderson:
Has the Principal Investigator ever had a clinical or behavioral protocol at MDACC that accrued patients?
No


2009-0266
March 26, 2015
Abstract - Page 20
Data Monitoring Committee:
Is treatment assignment randomized? No
Is this a blinded or double-blinded study? No
Does this protocol have a schedule for interim and final analysis?
Yes
Please describe:
We will monitor the data continuously (using a Bayesian method); there is a final analysis of this study.
Radiation Safety:
Does this study involve the administration of radioisotopes or a radioisotope 
labeled agent?
No
Is the radioactive compound (or drug) FDA approved and/or commercially 
available?
No
Investigational New Drugs:
Does this protocol require an IND?
No
Please confirm that the protocol meets all criteria for exemption according to 21CFR 312.2(b), 
noted below:
(b) Exemptions. (1) The clinical investigation of a drug product that is lawfully marketed in the United 
States is exempt from the requirements of this part if all the following apply:
(i)
The investigation is not intended to be reported to FDA as a well-controlled study in support of a 
new indication for use nor intended to be used to support any other significant change in the 
labeling for the drug;
(ii) If the drug that is undergoing investigation is lawfully marketed as a prescription drug product, the 
investigation is not intended to support a significant change in the advertising for the product;
(iii) The investigation does not involve a route of administration or dosage level or use in a patient 
population or other factor that significantly increases the risks (or decreases the acceptability of 
the risks) associated with the use of the drug product;
(iv) The investigation is conducted in compliance with the requirements for institutional review set 
forth in part 56 and with the requirements for informed consent set forth in part 50; and
(v) The investigation is conducted in compliance with the requirements of 312.7.
Rationale for Exemption:
Please include a detailed rationale as to why this drug should be considered exempt from FDA IND 
regulations, including any available references to the prior use of the regimen or drug combination in 
human subjects.
Drugs are comercially available and have been used in stem cell transplantation conditioning.
If this protocol includes an FDA Approved Therapy, please list the disease, dose and route of 
administration:


2009-0266
March 26, 2015
Abstract - Page 21
Approved Use
Proposed in this Protocol
Disease:
Dose:
Route of Administration:
Investigational Device:
Is the Investigational Device approved by the FDA?
N/A
Is the Investigational Device being used in the manner approved by 
the FDA?
N/A
Has the Investigational Device been modified in a manner not 
approved by the FDA?
N/A
Name of Device:
Manufacturer:
What is the FDA Status of the Investigational Device?
Is the study being conducted under an Investigational Device Exemption (IDE)? No
IDE Holder:
IDE Number:
Risk Assessment:
Please answer the following questions regarding the Investigational Device.
Intended as an implant?
No
Purported or represented to be for use supporting or sustaining human life?
No
For use of substantial importance in diagnosing, curing, mitigating, or treating 
disease, or otherwise preventing impairment of human health?
No
You may attach sponsor documentation of the risk assessment:
Will participant be charged for the Investigational Device? No
Sponsorship and Support Information:
Does the Study have a Sponsor or Supporter?
No
Is this Protocol listed on any Federal Grant or Foundation Funding Application?No


2009-0266
March 26, 2015
Abstract - Page 22
Biosafety:
Does this study involve the use of Recombinant DNA Technology? 
No
Does this study involve the use of organisms that are infectious to humans? 
No
Does this study involve stem cells?
Yes
Please Explain: The study involves infusion of hematopoietic stem cells/progenitor cells after high dose 
chemotherapy.
Technology Commercialization:
Does this study include any agents or devices manufactured or produced at 
MD Anderson Cancer Center?
No
Laboratory Tests:
Where will laboratory tests be performed on patient materials?  (Please select all that apply)
Division of Pathology & Laboratory Medicine CLIA Certified Laboratory
Manufacturing:
Will you manufacture in full or in part (split manufacturing) a drug or biological 
product at the M. D. Anderson Cancer Center for the proposed clinical study?
No