A Phase 2/3 interventional study of umbilical cord blood transplantation and Campath-1H in Severe Osteopetrosis, sponsored by Masonic Cancer Center, University of Minnesota. Completed at 1 site in United States. Open to participants aged 1 Day to 45 Years. Per ClinicalTrials.gov, last updated 2019-07-31.
Sponsored by Masonic Cancer Center, University of Minnesota · Phase 2/3, Interventional, and Treatment
The purpose of this research is to explore what we believe may be 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. Prior multi-institutional data in past studies found that approximately 30% of Osteopetrosis patients do not engraft. Therefore, in this study, we utilize a reduced intensity design 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.
This revised transplant protocol will test the following: 1) the ability to achieve engraftment with the reduced intensity protocol and a second infusion of stem cells on day 42, 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, and evaluation of blood parameters and genes that may be important in the disease process. 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 7 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.
Patients eligible for transplantation under this protocol will be \< or = 45 years of age, and will be diagnosed with severe osteopetrosis. This will be defined as having the following manifestations of the disease.
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.
Pre-transplant conditioning using Campath-1H, Busulfan, Fludarabine monophosphate, and total lymphoid irradiation followed by unrelated or matched related donor marrow graft transplantation (both peripheral blood and marrow) and a second CD34 cell infusion on Day 42.
Drug: Campath-1H · Radiation: Total Lymphoid Irradiation · Drug: Busulfan · Drug: Fludarabine monophosphate · Procedure: marrow graft transplantation
Pre-transplant conditioning using Campath-1H, Busulfan and Cyclophosphamide followed by unrelated umbilical cord blood transplantation and a second smaller portion cord blood graft infusion on Day 42.
Procedure: umbilical cord blood transplantation · Drug: Campath-1H · Drug: Cyclophosphamide · Drug: Busulfan
Umbilical cord blood will be collected, processed and shipped according to existing protocols. 2 cord blood units will be utilized if available. The choice of units will be based on the HLA typing standards of the University of Minnesota Blood and Marrow Program. If 2 units are not available, a single unit may be used. If a single unit is used, the unit should provide at least 10 x 107 nucleated cells/kg recipient body weight.
Also known as: UCBT
Campath-1H will be administered 0.3 mg/kg subcutaneously per day for three days starting on Day -21 through Day -19.
Also known as: Alemtuzumab
Dose 500 cGy via anteroposterior (AP) and posteroanterior(PA) fields (250 cGy AP and 250 cGy PA).
Also known as: TLI
Cyclophosphamide (50 mg/kg/dose) will be given IV on day -4, -3, -2 and -1 over 2 hours. The total dose to be given over 4 days is 200 mg/kg for cord blood grafts-receiving patients only.
Also known as: Cytoxan
patients\<12 kg: 1.1 mg/kg/dose IV every 6 hours for 8 doses total; patients \>12 kg: 0.8 mg/kg/dose IV every 6 hours for 8 doses. on Day -8 to -7 for donor grafts-receiving patients, and on Day -9 to -6 for cord blood grafts-receiving patients.
Also known as: Busulfex
Fludarabine (35 mg/m2 daily for 5 days, 175 mg/m2 total) will be administered IV over 30 minutes on days -6, -5, -4, -3, and -2 for donor grafts-receiving patients only.
Also known as: Fludara
Related donor marrow will be collected, processed and shipped according to existing protocols of the National Marrow Donor Program or other URD registry, with the goal of achieving a cell dose of ≥ 6.0 x 108 nucleated cells/kg. The proportion of cells that are CD34+ will be determined prior to the administration of the graft. This will allow a portion of the graft (2 x 106 CD34+ cells) to be frozen for a subsequent infusion on day +42.
Also known as: HSCT
Number of Patients Who Achieved Donor Cell Engraftment
Time frame: Day 100
Transplant Related Mortality at 100 Days
Time frame: day 100
Transplant Related Toxicity
Time frame: Day 100 post transplant
Incidence of Grade II - IV Acute Graft-versus-host Disease
Time frame: by Day 100 after transplant
| Milestone | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Started | 6 | 1 |
| Completed | 6 | 1 |
| Not completed | 0 | 0 |
| Participants | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Number of Patients Who Achieved Donor Cell Engraftment | 5 | 0 |
| Participants | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Transplant Related Mortality at 100 Days | 0 | 0 |
| Participants | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Transplant Related Toxicity | 3 | 1 |
| Participants | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Incidence of Grade II - IV Acute Graft-versus-host Disease | 2 | 0 |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Marrow Graft Transplant Conditioning | — | 3/6 (50%) | 6/6 (100%) |
| Cord Blood Transplant Conditioning | — | 0/1 (0%) | 1/1 (100%) |
| Event | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Multi-Organ FailureGeneral disorders | 1/6 | 0/1 |
| MyocarditisCardiac disorders | 1/6 | 0/1 |
| Acute Graft versus Host DiseaseGeneral disorders | 1/6 | 0/1 |
| Event | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning |
|---|---|---|
| Acute SinusitisInfections and infestations | 0/6 | 1/1 |
| AtelectasisRespiratory, thoracic and mediastinal disorders | 0/6 | 1/1 |
| Bacterial Infection, RespiratoryInfections and infestations | 1/6 | 1/1 |
| Bacterial Infection, FecesInfections and infestations | 1/6 | 1/1 |
| Blood Clot in Bladder WallBlood and lymphatic system disorders | 0/6 | 1/1 |
| Fungal Infection, RespiratoryInfections and infestations | 3/6 | 1/1 |
| Fungal Infection, FecesInfections and infestations | 0/6 | 1/1 |
| Requires Hemodialysis, NOSRenal and urinary disorders | 1/6 | 1/1 |
| Hyperechogenic Renomegaly with AscitesRenal and urinary disorders | 0/6 | 1/1 |
| Requires Intubation, NOSRespiratory, thoracic and mediastinal disorders | 5/6 | 1/1 |
| Age, Categorical(Participants) | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning | Total |
|---|---|---|---|
| <=18 years | 4 | 0 | 4 |
| Between 18 and 65 years | 2 | 1 | 3 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Marrow Graft Transplant Conditioning | Cord Blood Transplant Conditioning | Total |
|---|---|---|---|
| Female | 2 | 0 | 2 |
| Male | 4 | 1 | 5 |
The record gives a start of Aug 2008, a primary completion of Jun 2013 and a completion of Jun 2013. It does not say whether these dates are actual or estimated.
Results were first posted in Jul 2019, 6 years 1 month after primary completion.
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Studies of the drugs tested here: Cyclophosphamide · Fludarabine phosphate
Masonic Cancer Center, University of Minnesota →