A Phase 2/3 interventional study of Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) and Stem Cell Transplantation in Osteopetrosis, sponsored by Tehran University of Medical Sciences. Status unknown at 1 site in Iran, Islamic Republic of. Open to participants aged Up to 5 Years. Per ClinicalTrials.gov, last updated 2012-06-04.
Sponsored by Tehran University of Medical Sciences · Phase 2/3, Interventional, and Treatment
The purpose of this study is to evaluate the efficacy and side effects of donor hematopoietic cells using chemotherapy regimen without total-body irradiation in children undergoing a hematopoietic stem cell transplant for Malignant infantile osteopetrosis. The blood stem cells will be derived from either related donor or unrelated umbilical cord blood or haploidentical donor.
22 studies on the registry are indexed under Osteopetrosis; 4 are open to participants now.
This study's planned enrollment of 10 is below the median of 15 across 17 interventional studies indexed under Osteopetrosis.
Browse Osteopetrosis studies →Tehran University of Medical Sciences is the lead sponsor of 231 studies on the registry; 12 are open to participants now.
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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.
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.
Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen) · Procedure: Stem Cell Transplantation · Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)
For sibling full match: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv For other related full match, sibling or other related with one antigen mismatch and umbilical cord blood: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv * ATG rabbit (Thymoglobulin) 10 mg/kg or ATG horse (Atgam) 40 mg/kg For haploidentical: * Busulfan 16 mg/kg \>5year - 20 mg/kg \<5year po * Cyclophosphamide 200 mg/kg iv * Fludarabine 160 mg/m\^2
Also known as: Systemic chemotherapy
Patients undergoing Hematopoietic Stem Cell Transplantation from one of below source: 1. Sibling full match 2. Other related full match 3. Sibling or other related with 1 mismatch antigen 4. Cord Blood 5. Haploidentical
Also known as: HSCT
Drug pages: Cyclosporine · Methotrexate
* Cyclosporin A 1.5 mg/kg/day iv from -2, then 3 mg/kg/day iv (from +7 in PBSCT and +11 in BMT or UCBT) then 9 mg/kg/day po * 10 mg/m\^2 iv day +1 then 6 mg/m\^ iv day +3 and +6 (Not for UCBT)
Also known as: Graft-versus-host disease (GVHD) prophylaxis
Overall Survival and Progressive Free Survival in patient with infantile Osteopetrosis who receive allogeneic HSCT
Time frame: 1 year
One year overall survival after allogeneic HSCT
Time frame: 1 year
One year Progressive Free Survival after allogeneic HSCT
Time frame: 1 year
Transplantation Related Mortality (TRM) after allogeneic HSCT
Time frame: 1 year
Acute and chronic GVHD rate after allogeneic HSCT
Time frame: 1 year
The record gives a start of Sep 2009, a primary completion of Nov 2012 and a completion of Dec 2012. It does not say whether these dates are actual or estimated.
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This study is status unknown, as verified in May 2012. You cannot join it, but the record below documents what was studied.
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Studies of the drugs tested here: Cyclosporine · Methotrexate
Tehran University of Medical Sciences →