An interventional study of Stem Cell Transplantation and Miltenyi Biotec CliniMACS in Osteopetrosis, sponsored by St. Jude Children's Research Hospital. Terminated at 1 site in United States. Per ClinicalTrials.gov, last updated 2017-05-30.
Sponsored by St. Jude Children's Research Hospital · Not applicable, Interventional, and Treatment
Malignant infantile osteopetrosis (MIOP) is a rare fatal genetic disorder that is characterized by the bone's inability to regulate remodeling. The only curative therapy is hematopoietic stem cell transplantation. Stem cells provided from an HLA identical matched sibling donor is the standard of care, but not feasible for the majority of patients. In addition, due to the potentially rapid progression of this disease, the time to identify a suitable HLA matched unrelated donor is not optimal. Therefore this study is designed to test the hypothesis that children with osteopetrosis can properly engraft hematopoietic stem cells that are donated from a partially matched parental donor, or "haploidentical" stem cell donor that are processed on the investigational device, CliniMACS selection system.
The primary objective of this trial will be answered strictly by those patients enrolled who receive a haploidentical stem cell donor graft.
Patients with a matched sibling donor will be offered participation in this clinical trial and will receive a standard myeloablative conditioning regimen followed by the infusion of an unmanipulated bone marrow graft. However, data from these transplant recipients will be reported in a descriptive manner only.
Secondary Objectives in this trial include the following:
22 studies on the registry are indexed under Osteopetrosis; 4 are open to participants now.
This study's enrollment of 15 is close to the median of 15 across 17 interventional studies indexed under Osteopetrosis.
Browse Osteopetrosis studies →St. Jude Children's Research Hospital is the lead sponsor of 440 studies on the registry; 102 are open to participants now.
Of its 61 completed or terminated interventional studies of FDA-regulated products, 37 (61%) 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.
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Biopsy and archival tissue
A biopsy takes a small piece of tissue to examine. “Archival tissue” is a sample already taken, for example at diagnosis, and kept by the hospital.
Whether a stored sample will do depends on when it was taken, how much there is and what the study needs. Ask whether the study accepts your stored sample or needs a new biopsy.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Karnofsky performance status (KPS)
A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.
Studies often ask for 60 or 70 and above. Your doctor gives the score.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Procedure: Stem Cell Transplantation · Device: Miltenyi Biotec CliniMACS · Drug: Systemic chemotherapy and antibodies
An infusion of HLA partially matched family member donor stem cells processed through the use of the investigational Miltenyi Biotec CliniMACS device.
Also known as: Haploidentical stem cell transplant, Allogeneic stem cell transplant
Stem cell selection device
Also known as: T-cell depletion
Drug page: Antibodies
Haploidentical stem cell transplant recipients will receive a reduced intensity conditioning regimen consisting of OKT-3, Fludarabine, Thiotepa , and Melphalan followed by an infusion of a T-cell depleted donor stem cell product. Rituximab will be administered within 24 hours of the infusion in an effort to prevent post transplantation lymphoproliferative disorders (PTLPD). In addition to T-cell depletion of the donor product, cyclosporine will be provided as prophylaxis for (GVHD)Graft versus Host Disease Recipients of a matched sibling donor product will receive a myeloablative conditioning regimen consisting of busulfan and cyclophosphamide. Cyclosporine will be administered for GVHD prophylaxis.
Also known as: Transplantation for Osteopetrosis
Engraftment
To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient's bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.
Time frame: 100 days post-transplant
OPBMT2 was activated, July 2004. From September, 2004 through February, 2008, six transplant participants, five donors, and four genetic testing participants were recruited and enrolled on the study.
| Milestone | Haplo | Sibling |
|---|---|---|
| Started | 5 | 1 |
| Completed | 5 | 1 |
| Not completed | 0 | 0 |
To determine the need for blood or platelet transfusions and the presence of donor cells being present in the transplant recipient's bone marrow or peripheral blood by 100 day after transplantation for children with malignant infantile osteopetrosis who have received a haploidentical stem cell graft.
| Participants | Haplo | Sibling |
|---|---|---|
| Favorable engraftment | 5 | 1 |
| Un-favorable engraftment | 0 | 0 |
Collected over Adverse events were collected from September, 2004 through February, 2008.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Haplo | — | 5/5 (100%) | 5/5 (100%) |
| Sibling | — | 1/1 (100%) | 1/1 (100%) |
| Event | Haplo | Sibling |
|---|---|---|
| Delayed gastric emptyingGastrointestinal disorders | 0/5 | 1/1 |
| Hemorrhage, Diffuse AlveolarRespiratory, thoracic and mediastinal disorders | 0/5 | 1/1 |
| HypoxiaRespiratory, thoracic and mediastinal disorders | 2/5 | 1/1 |
| Fever without NeutropeniaGeneral disorders | 2/5 | 0/1 |
| Infection, CMV PneumonitisInfections and infestations | 1/5 | 0/1 |
| Infection, Moraxella Catarrhalis, Upper RespiratoryInfections and infestations | 1/5 | 0/1 |
| Infection, Respiratory Syncytial VirusInfections and infestations | 1/5 | 0/1 |
| Infection, Rotavirus, StoolInfections and infestations | 1/5 | 0/1 |
| Infection, Staph Aureus, Tracheostomy SiteInfections and infestations | 1/5 | 0/1 |
| Adult Respiratory Distress Syndrome Related To Aspiration PneumoniaRespiratory, thoracic and mediastinal disorders | 1/5 | 0/1 |
| Event | Haplo | Sibling |
|---|---|---|
| Prolonged QT intervalCardiac disorders | 0/5 | 1/1 |
| HypertensionCardiac disorders | 4/5 | 1/1 |
| HypotensionCardiac disorders | 2/5 | 1/1 |
| Fever without NeutropeniaGeneral disorders | 3/5 | 1/1 |
| Transient Erythematous RashSkin and subcutaneous tissue disorders | 0/5 | 1/1 |
| DiarrheaGastrointestinal disorders | 1/5 | 1/1 |
| MucositisGastrointestinal disorders | 3/5 | 1/1 |
| VomitingGastrointestinal disorders | 4/5 | 1/1 |
| HematemesisBlood and lymphatic system disorders | 1/5 | 1/1 |
| Elevated GGTHepatobiliary disorders | 1/5 | 1/1 |
| Age, Categorical(Participants) | Haplo | Sibling | Total |
|---|---|---|---|
| <=18 years | 5 | 1 | 6 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Sex: Female, Male(Participants) | Haplo | Sibling | Total |
|---|---|---|---|
| Female | 1 | 1 | 2 |
| Male | 4 | 0 | 4 |
The record gives a start of Jul 2004, a primary completion of Feb 2009 and a completion of Feb 2009. It does not say whether these dates are actual or estimated.
Results were first posted in Aug 2011, 2 years 6 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is terminated, as verified in Jan 2011. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Antibodies
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