CClinicalTrials.gg
Sign In
CompletedNCT00997386ALBUMUpdated Sep 9, 2019Results posted

Reduced Intensity Allogeneic PBSCT to Treat Hematologic Malignancies and Hematopoietic Failure States

A Phase 2 interventional study of busulfan, and melphalan, and alemtuzumab in Hematologic Neoplasms, Multiple Myeloma and Anemia, Aplastic, sponsored by University of Arizona. Completed at 1 site in United States. Open to participants aged 18 Years to 75 Years. Per ClinicalTrials.gov, last updated 2019-09-09.

Sponsored by University of Arizona · Phase 2, Interventional, and Treatment

Phase
Phase 2
Study type
Interventional
Enrollment
16
Allocation
Not applicable
Ages
18 Years to 75 Years
Sex
All
01

Study summary

The purpose of this study is to look at whether the combination of lower-dose chemotherapy with two chemotherapy (anti-cancer) drugs, called busulfan and melphalan, and an antibody medication called alemtuzumab (Campath®), can prevent rejection of donor blood stem cells so that those cells take hold and build a healthy new blood cell factory after transplant. The study will also look at the safety of the combination of drugs and of the transplant of peripheral blood stem cells from a healthy relative or an unrelated donor.

Read the detailed description

Transplantation of related or unrelated allogeneic peripheral blood stem cells (PBSCs) after administration of a reduced-intensity regimen of busulfan, melphalan and alemtuzumab will be associated with satisfactory engraftment and acceptable post-transplant non-relapse mortality.

02

Conditions studied

  • Hematologic Neoplasms
  • Multiple Myeloma
  • Anemia, Aplastic
  • Hemoglobinuria, Paroxysmal
  • Myelofibrosis

Keywords

  • Hematologic malignancies
  • lymphoma, leukemia, MDS (myelodysplastic syndrome)
  • reduced-intensity preparative regimen
  • allogeneic peripheral blood stem cell transplant
  • myelofibrosis or other myeloproliferative syndromes
03

In context

Multiple Myeloma

3,632 studies on the registry are indexed under Multiple Myeloma; 745 are open to participants now.

This study's enrollment of 16 is below the median of 41 across 2,907 interventional studies indexed under Multiple Myeloma.

Browse Multiple Myeloma studies →

Lead sponsor

University of Arizona is the lead sponsor of 466 studies on the registry; 87 are open to participants now.

Of its 47 completed or terminated interventional studies of FDA-regulated products, 29 (62%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 75 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Age 50 to 75 years or age 18 to 49 with one or more of these risk factors: prior autologous, allogeneic or syngeneic HCT (Hematopoietic cell transplantation); not in first complete remission or first chronic phase; and/or presence of one or more medical conditions that would place the subject at high risk such as heart and kidney disease.
  • Subjects with hematologic cancers must have received at least one previous course of chemotherapy or biological therapy. In other words, the subject cannot enroll in this trial for initial treatment of the disease.
  • Availability of a healthy related or unrelated volunteer allogeneic donor.

Exclusion criteria

Exclusion Criteria:

  • Eligible for another study or standard of care treatment that offers higher probability of cure or long-term control of subject's disease.
  • Severe abnormal function of organs such as heart, kidneys, liver.
  • Untreated or progressive central nervous system involvement by the disease.
  • Subject is pregnant or breast-feeding.
  • Performance score is below 50: at the least, requires considerable assistance and frequent medical care.
  • Positive for the HIV [AIDS] virus
  • Life expectancy less than 12 weeks with conventional treatments.
  • For subjects capable of having children, refusal to practice birth control while on this study and for at least 12 months after PBSCT or after stopping post-transplant immunosuppressive treatments, whichever occurs later.
05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
16 participants (actual)

Study arms

  • Experimental
    busulfan, and melphalan, and alemtuzumab

    Three drug regimen using busulfan, and melphalan, and alemtuzumab.

    Drug: busulfan, and melphalan, and alemtuzumab

Interventions

  • Drugbusulfan, and melphalan, and alemtuzumab

    intravenous busulfan 3.2 mg/kg/dose daily for 2 days, on days -5 and -4 (i.e., 5 and 4 days, respectively, before PBSCT). intravenous melphalan 100 mg/m2 on day -3. intravenous alemtuzumab 30 mg/dose for 2 days, on days -2 and -1.

    Also known as: Busulfan (Busulfex®),, Melphalan (Alkeran®), Alemtuzumab (Campath®)

06

What researchers measure

Primary outcomes

  1. Number of Participants With Presence of Donor Lymphohematopoietic Chimerism (Defined as at Least 50% Donor Cells in the Peripheral Blood) in Peripheral Blood by Day +100 (i.e., 100 Days After Allogeneic PBSCT).

    To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT).

    Time frame: Day +100

Secondary outcomes

  1. Number of Participants With Relapse-free Survival.

    To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

    Time frame: Day +100

  2. Number of Participants With Event-free Survival.

    To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

    Time frame: Day +100

  3. Number of Participants With Overall Survival.

    To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

    Time frame: Day +100

07

Results

Posted Dec 24, 2018

Participant flow

Participant flow — Overall Study
MilestoneBusulfan, and Melphalan, and Alemtuzumab
Started16
Completed9
Not completed7
Withdrew: Death7

Outcome measures

PrimaryNumber of Participants With Presence of Donor Lymphohematopoietic Chimerism (Defined as at Least 50% Donor Cells in the Peripheral Blood) in Peripheral Blood by Day +100 (i.e., 100 Days After Allogeneic PBSCT).

To determine the efficacy of related or unrelated allogeneic PBSC transplantation (PBSCT) using a preparative regimen of busulfan, melphalan and alemtuzumab, as measured by durable donor lymphohematopoietic cell engraftment. The primary efficacy endpoint is the presence of donor lymphohematopoietic chimerism (defined as at least 50% donor cells in the peripheral blood) in peripheral blood by day +100 (i.e., 100 days after allogeneic PBSCT).

Time frame:
Day +100

No measurements were reported for this outcome.

SecondaryNumber of Participants With Relapse-free Survival.

To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

Time frame:
Day +100

No measurements were reported for this outcome.

SecondaryNumber of Participants With Event-free Survival.

To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

Time frame:
Day +100

No measurements were reported for this outcome.

SecondaryNumber of Participants With Overall Survival.

To determine the safety of related or unrelated allogeneic PBSCT using a preparative regimen of busulfan, melphalan and alemtuzumab. The primary safety endpoint is non-relapse mortality at day +100.

Time frame:
Day +100

No measurements were reported for this outcome.

Adverse events

Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Busulfan, and Melphalan, and Alemtuzumab7/16 (43.8%)3/16 (18.8%)16/16 (100%)
Most frequent serious events
Most frequent serious events
EventBusulfan, and Melphalan, and Alemtuzumab
Pneumatosis cystoides intestinalisGastrointestinal disorders1/16
FeverGeneral disorders1/16
Abdominal PainGastrointestinal disorders1/16
Most frequent other events
Showing 10 of 94
Most frequent other events
EventBusulfan, and Melphalan, and Alemtuzumab
Leukocytes (total WBC)Blood and lymphatic system disorders16/16
NauseaGastrointestinal disorders16/16
PainGeneral disorders16/16
Rigors/chillsGeneral disorders16/16
DiarrheaGastrointestinal disorders15/16
HypomagnesemiaBlood and lymphatic system disorders15/16
FatigueGeneral disorders14/16
Mucositis/stomatitisSkin and subcutaneous tissue disorders14/16
Neutrophils/GranulocytesBlood and lymphatic system disorders13/16
CoughRespiratory, thoracic and mediastinal disorders11/16

Baseline characteristics

Age, Categorical
Age, Categorical(Participants)Busulfan, and Melphalan, and Alemtuzumab
<=18 years0
Between 18 and 65 years7
>=65 years9
Sex: Female, Male
Sex: Female, Male(Participants)Busulfan, and Melphalan, and Alemtuzumab
Female5
Male11
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Busulfan, and Melphalan, and Alemtuzumab
Hispanic or Latino2
Not Hispanic or Latino14
Unknown or Not Reported0
Region of Enrollment
Region of Enrollment(Participants)Busulfan, and Melphalan, and Alemtuzumab
United States16
08

Study locations

1 site
  • University Medical Center and UMC-North Clinic
    Tucson, Arizona 85719, United States
09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Sep 9, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT00997386
Lead sponsor
University of Arizona
Responsible party
Sponsor
First posted
Oct 19, 2009
Start date
Sep 2009
Primary completion
Apr 2014
Completion
Jan 2016
Results posted
Dec 24, 2018
Last update
Sep 9, 2019

Study contacts

Andrew M Yeager, MD
principal investigator · University of Arizona

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Sep 2019. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Multiple Myeloma→

Other studies from this sponsor

University of Arizona

Start the discussion