A Phase 2 interventional study of Stem Cell or Umbilical Cord Blood Transplantation and Campath, Busulfan, Clofarabine in Osteopetrosis, sponsored by Masonic Cancer Center, University of Minnesota. Terminated at 1 site in United States. Open to participants aged Up to 45 Years. Per ClinicalTrials.gov, last updated 2017-12-28.
Sponsored by Masonic Cancer Center, University of Minnesota · Phase 2, Interventional, and Treatment
We believe that hematopoietic stem cell transplantation (HSCT) will help subjects with Osteopetrosis generate functioning osteoclasts, and by so doing assist in the resolution of the abnormal bone architecture, and the anemia and bone marrow failure that is also characteristic of this disease. However, we have found in past studies that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we plan to use a different combination of pre-transplant drugs to try to make transplants safer for this disease, as well as to provide a second infusion of stem cells in patients with matched related or unrelated donors. The purpose of this research is to find a safer and more effective means of performing stem cell transplantation in patients with Osteopetrosis, using chemotherapy and radiation designed to bring about engraftment and lessen transplant mortality.
This transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol, 2) the mortality associated with transplant by day 100, 3) patient outcomes, based on differential imaging and biologic evaluations prior to transplantation and at designated points after transplantation (day 100, 6 months, 1, 2 and 5 years). Additional biologic studies will include microarray analysis, Campath levels just prior to the administration of the graft, and establishment of mesenchymal stem cell lines. In older patients, studies to evaluation osteoclast differentiation and function will also be offered.
22 studies on the registry are indexed under Osteopetrosis; 4 are open to participants now.
This study's enrollment of 3 is below the median of 15 across 17 interventional studies indexed under Osteopetrosis.
Browse Osteopetrosis studies →Masonic Cancer Center, University of Minnesota is the lead sponsor of 286 studies on the registry; 35 are open to participants now.
Of its 39 completed or terminated interventional studies of FDA-regulated products, 28 (72%) have results posted.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients enrolled and received study treatment.
Procedure: Stem Cell or Umbilical Cord Blood Transplantation · Drug: Campath, Busulfan, Clofarabine · Procedure: Total Lymphoid Irradiation
Stem Cell (unrelated or matched related donor grafts (both peripheral blood and marrow) infusion on Day 0 and 42; Umbilical Cord Blood on Day 0 and 42
Also known as: Bone Marrow Transplant
Drug pages: Alemtuzumab · Busulfan · Clofarabine
* 12 Campath-1H 0.3 mg/kg intravenously (IV) over 2 hours * 11 Campath-1H 0.3 mg/kg intravenously over 2 hours * 10 Campath-1H 0.3 mg/kg intravenously over 2 hours * 9 Busulfan \<12 kg: 2.2 mg/kg/dose IV every 12 hours \>12 kg: 1.6 mg/kg/dose IV every 12 hours * 8 Busulfan \<12 kg: 2.2 mg/kg/dose IV every 12 hours \>12 kg: 1.6 mg/kg/dose IV every 12 hours * 7 "Rest" * 6 Clofarabine 40 mg/m2 intravenously over 2 hours * 5 Clofarabine 40 mg/m2 intravenously over 2 hours * 4 Clofarabine 40 mg/m2 intravenously over 2 hours * 3 Clofarabine 40 mg/m2 intravenously over 2 hours * 2 Clofarabine 40 mg/m2 intravenously over 2 hours
Also known as: Busulfex, Clolar,Alemtuzumab
Dose 500 cGy via anteroposterior (AP) and posteroanterior (PA) fields (250 cGy AP and 250 cGy PA).
Also known as: Therapuetic radation, radiation therapy
Number of Patients Achieving Donor Cell Engraftment
Number of patients with persistent presence of donor-derived cells at Day 100
Time frame: Day 100
Number of Patients With Transplant Related Death
Number of participants died during study by Day 100 and reason for death was related to transplant.
Time frame: Day 100
Number of Patients With Transplant Related Toxicity
Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.
Time frame: Day 100
Differential Imaging and Biologic Evaluations
These outcome measures were not assessed due to early study termination.
Time frame: Day 100, 6 months, 1, 2 and 5 years
| Milestone | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Started | 3 |
| Completed | 3 |
| Not completed | 0 |
Number of patients with persistent presence of donor-derived cells at Day 100
| Participants | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Number of Patients Achieving Donor Cell Engraftment | 2 |
Number of participants died during study by Day 100 and reason for death was related to transplant.
| Participants | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Number of Patients With Transplant Related Death | 3 |
Number of patients experiencing adverse effects due to transplant categorized by body system using Common Terminology Criteria for Adverse Events coding from the National Cancer Institute, Version 3.0.
| Participants | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Neurologic | 1 |
| Renal | 1 |
| Pulmonary | 3 |
| Peri-Transplant Mortality (Death by Day 100) | 3 |
| Vascular | 1 |
These outcome measures were not assessed due to early study termination.
No measurements were reported for this outcome.
Collected over Adverse events were collected from Day 1 through Day 100 of the study.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Patients With Osteopetrosis Who Received Transplant | — | 3/3 (100%) | 3/3 (100%) |
| Event | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Death, peri-transplantGeneral disorders | 3/3 |
| Event | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| PulmonaryRespiratory, thoracic and mediastinal disorders | 3/3 |
| NeurologicNervous system disorders | 1/3 |
| RenalRenal and urinary disorders | 1/3 |
| VascularVascular disorders | 1/3 |
| Age, Categorical(Participants) | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| <=18 years | 2 |
| Between 18 and 65 years | 1 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| Female | 0 |
| Male | 3 |
| Region of Enrollment(participants) | Patients With Osteopetrosis Who Received Transplant |
|---|---|
| United States | 3 |
This study is terminated, as verified in Dec 2017. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions 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.
Studies of the drugs tested here: Alemtuzumab · Busulfan · Clofarabine
Masonic Cancer Center, University of Minnesota →