Congenital Pediatric DisordersUCBNCT00950846
Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
A study testing Busulfan, Cytoxan and Fludarabine in people with Congenital Pediatric Disorders, run by Baylor College of Medicine at 1 site in United States.
Trial summary
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence of GVHD. Read more about this study
- Who is it for?
- Ages Up to 17 years
- Conditions the study is for
- Congenital Pediatric Disorders
- What is being tested?
- Busulfan, Cytoxan and Fludarabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 1 siteUnited States
- Study dates
- Sep 2009to 4 Feb 2021
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Patients less than 18 years of age.
- Patients with a congenital or acquired immunologic, hematological, or metabolic pediatric disease (including SCID) in which stem cell transplantation has been beneficial.
- Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, 5-6 HLA- A and B (at low to intermediate resolution) and DRB1 (at high resolution).
- Total cryopreserved HSC graft cell dose must be 5 x 10^7 or greater nucleated cells per kilogram recipient body weight.
- Lansky/Karnofsky scores 60 or greater.
- Patient has DLCO > 50% predicted or FEV1 > 50%, if applicable.
- Written informed consent and/or signed assent line from patient, parent or guardian.
Who cannot join
- Patients with uncontrolled infections as assessed by the principal investigator only. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to starting conditioning. For fungal infections patients must be receiving definitive systemic antifungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Severe renal disease (creatinine > 3X normal for age).
- Severe hepatic disease (direct bilirubin > 3 mg/dL or SGOT > 500).
- Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 20%).
- HIV positive.
Trial locations and contacts
1 site in 1 country.
About this study
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence of GVHD.
This study is for patients that were born with a disease that affects their body's metabolism or immune system. The doctor plans to treat the patient for this illness with a stem cell transplant.
Read more
While improved medical care has allowed many people with these diseases to live longer, the only way to truly cure the diseases is by means of a stem cell transplant from a donor who does not have the disease. A stem cell transplant will replace sick cells with new healthy donor cells. Stem cells grow into different types of blood cells that people need, including red blood cells, white blood cells, and platelets. In a stem cell transplant, the patients own stem cells would be killed by chemotherapy drug and then replaced by stem cells from the donor. Stem cells can be collected from the bone marrow, peripheral blood or umbilical cords. In this study, umbilical cords will be the source of the stem cells.
Currently, large inventories of umbilical cord blood units are available in public banks for transplantation in those lacking bone marrow donors. UCB transplants offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- Rapid availability,
- Absence of donor risk,
- Low risk of transmissible infectious diseases,
- Low risk of acute GvHD (as compared to recipients of unrelated donor marrow and peripheral blood cells).
The two main causes of death after umbilical cord blood transplantation for disorders for these kinds of patients, are graft failure and infection.
In this study we are trying to address these two problems by using different drugs to prepare patients for the transplant.
To help improve engraftment (cells begin to grow), we will include the drug Fludarabine to the usually used Busulfan and Cytoxan that the study patients will receive before their transplant.
We will try to decrease the chance of developing graft-versus-host disease (GvHD) by using Cyclosporin A (CSA) and Mycophenolate Mofetil (MMF), instead of Anti-Thymocyte Globulin (ATG) which is normally used.
Read the detailed description
Patients will be examined to make sure that they meet the requirements of this study. There will be tests of the heart and of the lungs. X-rays will be taken of the lungs and other organs, depending on the disease. An MRI and consultations with different specialists will also be conducted.
Patients also must have a negative pregnancy test before entering this study if they are a woman of childbearing potential. The blood will be tested for viruses and to look at the functioning of the liver and kidneys. The examination also includes HIV testing. If the patient has HIV, they will not be able to be treated on this protocol.
After we have determined that the patient is eligible for treatment on this study and a suitable UCB stem donor has been found, they will have a central line placed.
After placement of the central line, the following chemotherapy will be given to after admission to the hospital:
- 9 days before the infusion through 6 days before the infusion: Busulfan every 6 hours for 16 total doses.
- 5 days before the infusion through 2 days before the infusion: Cytoxan given daily for 4 days over 2 hours. (It can be given over 1 to 4 hours if needed as decided by the physician). Mesna will be given per standards.
- 4 days before the infusion through 1 day before the infusion: Fludarabine given daily for 4 days over 1 hour.
Stem cell transplant (infusion of the UCB stem cells) - defined as Day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.
Standard Therapy: Phenytoin will be given according to the standards of the TCH formulary.
Cyclosporin A (CSA) will be given starting 2 days prior to the stem cell infusion. It will be given daily over 2 hours every 12 hours, and then tapered if no GvHD is present.
Administration of Mycophenolate Mofetil (MMF) will start on the day the stem cell infusion is completed, and will continue daily for 45 days unless the patient develops GvHD.
Intravenous Immunoglobulins (IVIG) will be given as per CAGT SOP for infections prophylaxis.
Granulocyte Colony-Stimulating Factor (GCSF) will be given daily starting at Day +7 until ANC is greater than 2,500 for three consecutive days.
Study Evaluation: Patients will have various study evaluations, including blood samples, before and after the transplant.
Follow-Up: After year 1, the patients will be asked to return to the clinic once a year for consultations. These consultations with specialists will be similar to the ones the patients had before their transplant.
- Conditions
- Congenital Pediatric Disorders
- Being tested
- Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
- Keywords
- Congenital Pediatric Disorders, Umbilical Cord Blood Transplant, Congenital Abnormalities, Cord Blood Stem Cell Transplantation, Busulfan, Cytoxan, Cyclophosphamide, Fludarabine
- Medical subject headings
- Congenital Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Busulfex
Day -9, -8, -7 and -6
Patients less than or equal to 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients >12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses.
CytoxanDrug
Also called Cyclophosphamide
(50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
FludarabineDrug
Also called Fludera
40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
Cord Blood Stem Cell InfusionProcedure or surgery
The cord blood stem cells will be infused on Day 0.
| Study group | Role | What they receive |
|---|---|---|
| Umbilical Cord Blood Transplant Treatment Plan | Experimental | Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion Receives: Drug: Busulfan; Drug: Cytoxan; Drug: Fludarabine; Procedure: Cord Blood Stem Cell Infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Measured: 100 days
Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Measured: 1 year
Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Measured: 3 years
Secondary outcomes (5)
Number of Participants With Platelet Engraftment
Achievement of untransfused platelet count > 20 x 10^9/L on three consecutive days
Measured: Day 42
Incidence of Severe Grade III-IV Acute GvHD at Day 100.
Number of participants with acute GVHD graded by the method of Przepiorka et al, which evaluates skin involvement, lower and upper GI, and liver function (bilirubin), each being graded in stages from 0 to 4, where 0 means no acute GVHD, and 4 is the highest stage of acute GVHD.
Measured: Day 100
Number of Participants With Chronic GvHD
Number of participants with chronic GVHD graded by the method of Przepiorka et al, which evaluates skin, joints, oral, ocular, hepatic, esophagus, GI, respiratory, platelet, and musculoskeletal involvement, in stages from 0 to 3.
Measured: 1 year
Number of Participants With Donor Engraftment After Transplant.
To evaluate donor engraftment at 100 days, 6 and 12 months after transplant.
Measured: 100 days, 6 months and 12 months
Number of Participants With Neutrophil Engraftment
Achievement of absolute neutrophil count > 0.5 x 10^9/L on three consecutive days
Measured: Day 42
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Aug 2009 (estimated)
- Study startSep 2009
- Primary completion21 Jul 2020
- Study completion4 Feb 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Congenital Pediatric DisordersStudies of the same condition, open and under way.
- More trials run by Baylor College of MedicineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baylor College of Medicine
- Sponsor type
- University, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Responsible party
- Principal investigator, Caridad Martinez, Baylor College of Medicine
- Investigators
- Caridad Martinez, MD, Principal investigator, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00950846
- Sponsor's protocol ID
- 25064-UCB
- Acronym
- UCB
- Status last verified
- Oct 2023
- First submitted
- 30 Jul 2009
- Last updated
- 30 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Martinez C, Aguayo-Hiraldo P, Chaimowitz N, Forbes L, Rider N, Nicholas S, Seeborg F, Chinen J, Chinn I, Davis C, Roseblatt H, Noroski L, Omer B, John T, Yassine K, Naik S, Craddock J, Bhar S, Allen C, Ahmed N, Sasa G, Steffin D, Doherty E, George A, Salem B, Friend B, Hegde M, Brenner MK, Heslop HE, Leen A, Pena A, Wu M, Hanson IC, Krance RA. Cord blood transplantation for nonmalignant disorders: early functional immunity and high survival. Blood Adv. 2023 May 9;7(9):1823-1830. doi: 10.1182/bloodadvances.2022009038.
PubMed 36453638
Study at a glance
From the registry record, last updated by the sponsor on 30 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
- ClinicalTrials.gov ID
- NCT00950846
- Acronym / sponsor ID
- UCB / 25064-UCB
- Conditions
- Congenital Pediatric Disorders
- Keywords
- Congenital Pediatric Disorders, Umbilical Cord Blood Transplant, Congenital Abnormalities, Cord Blood Stem Cell Transplantation, Busulfan, Cytoxan, Cyclophosphamide, Fludarabine
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- Baylor College of MedicineUniversity, hospital or other
- Collaborators
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Study start
- Sep 2009 (actual)
- Primary completion
- 21 Jul 2020 (actual)
- Study completion
- 4 Feb 2021 (actual)
- First posted
- 3 Aug 2009 (estimated)
- Results
- Posted 30 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence of GVHD.
This study is for patients that were born with a disease that affects their body's metabolism or immune system. The doctor plans to treat the patient for this illness with a stem cell transplant.
While improved medical care has allowed many people with these diseases to live longer, the only way to truly cure the diseases is by means of a stem cell transplant from a donor who does not have the disease. A stem cell transplant will replace sick cells with new healthy donor cells. Stem cells grow into different types of blood cells that people need, including red blood cells, white blood cells, and platelets. In a stem cell transplant, the patients own stem cells would be killed by chemotherapy drug and then replaced by stem cells from the donor. Stem cells can be collected from the bone marrow, peripheral blood or umbilical cords. In this study, umbilical cords will be the source of the stem cells.
Currently, large inventories of umbilical cord blood units are available in public banks for transplantation in those lacking bone marrow donors. UCB transplants offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- Rapid availability,
- Absence of donor risk,
- Low risk of transmissible infectious diseases,
- Low risk of acute GvHD (as compared to recipients of unrelated donor marrow and peripheral blood cells).
The two main causes of death after umbilical cord blood transplantation for disorders for these kinds of patients, are graft failure and infection.
In this study we are trying to address these two problems by using different drugs to prepare patients for the transplant.
To help improve engraftment (cells begin to grow), we will include the drug Fludarabine to the usually used Busulfan and Cytoxan that the study patients will receive before their transplant.
We will try to decrease the chance of developing graft-versus-host disease (GvHD) by using Cyclosporin A (CSA) and Mycophenolate Mofetil (MMF), instead of Anti-Thymocyte Globulin (ATG) which is normally used.
Detailed description
Patients will be examined to make sure that they meet the requirements of this study. There will be tests of the heart and of the lungs. X-rays will be taken of the lungs and other organs, depending on the disease. An MRI and consultations with different specialists will also be conducted.
Patients also must have a negative pregnancy test before entering this study if they are a woman of childbearing potential. The blood will be tested for viruses and to look at the functioning of the liver and kidneys. The examination also includes HIV testing. If the patient has HIV, they will not be able to be treated on this protocol.
After we have determined that the patient is eligible for treatment on this study and a suitable UCB stem donor has been found, they will have a central line placed.
After placement of the central line, the following chemotherapy will be given to after admission to the hospital:
- 9 days before the infusion through 6 days before the infusion: Busulfan every 6 hours for 16 total doses.
- 5 days before the infusion through 2 days before the infusion: Cytoxan given daily for 4 days over 2 hours. (It can be given over 1 to 4 hours if needed as decided by the physician). Mesna will be given per standards.
- 4 days before the infusion through 1 day before the infusion: Fludarabine given daily for 4 days over 1 hour.
Stem cell transplant (infusion of the UCB stem cells) - defined as Day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.
Standard Therapy: Phenytoin will be given according to the standards of the TCH formulary.
Cyclosporin A (CSA) will be given starting 2 days prior to the stem cell infusion. It will be given daily over 2 hours every 12 hours, and then tapered if no GvHD is present.
Administration of Mycophenolate Mofetil (MMF) will start on the day the stem cell infusion is completed, and will continue daily for 45 days unless the patient develops GvHD.
Intravenous Immunoglobulins (IVIG) will be given as per CAGT SOP for infections prophylaxis.
Granulocyte Colony-Stimulating Factor (GCSF) will be given daily starting at Day +7 until ANC is greater than 2,500 for three consecutive days.
Study Evaluation: Patients will have various study evaluations, including blood samples, before and after the transplant.
Follow-Up: After year 1, the patients will be asked to return to the clinic once a year for consultations. These consultations with specialists will be similar to the ones the patients had before their transplant.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Umbilical Cord Blood Transplant Treatment Plan | Experimental | Drug: Busulfan, Drug: Cytoxan, Drug: Fludarabine, Procedure: Cord Blood Stem Cell InfusionBusulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion |
Who may qualify
- Ages
- Up to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- Patients less than 18 years of age.
- Patients with a congenital or acquired immunologic, hematological, or metabolic pediatric disease (including SCID) in which stem cell transplantation has been beneficial.
- Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, 5-6 HLA- A and B (at low to intermediate resolution) and DRB1 (at high resolution).
- Total cryopreserved HSC graft cell dose must be 5 x 10^7 or greater nucleated cells per kilogram recipient body weight.
- Lansky/Karnofsky scores 60 or greater.
Key exclusion criteria (5 listed)
- Patients with uncontrolled infections as assessed by the principal investigator only. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to starting conditioning. For fungal infections patients must be receiving definitive systemic antifungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Severe renal disease (creatinine > 3X normal for age).
- Severe hepatic disease (direct bilirubin > 3 mg/dL or SGOT > 500).
- Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction < 20%).
- HIV positive.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BusulfanAlso: BusulfexDay -9, -8, -7 and -6 Patients less than or equal to 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients >12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses. | Drug | Umbilical Cord Blood Transplant Treatment Plan |
| CytoxanAlso: Cyclophosphamide(50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg. | Drug | Umbilical Cord Blood Transplant Treatment Plan |
| FludarabineAlso: Fludera40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg. | Drug | Umbilical Cord Blood Transplant Treatment Plan |
| Cord Blood Stem Cell InfusionThe cord blood stem cells will be infused on Day 0. | Procedure or surgery | Umbilical Cord Blood Transplant Treatment Plan |
Primary outcome
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
Time frame: 100 days
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
Time frame: 1 year
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
Time frame: 3 years
To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
5 secondary outcomes
Number of Participants With Platelet Engraftment
Time frame: Day 42
Incidence of Severe Grade III-IV Acute GvHD at Day 100.
Time frame: Day 100
Number of Participants With Chronic GvHD
Time frame: 1 year
Number of Participants With Donor Engraftment After Transplant.
Time frame: 100 days, 6 months and 12 months
Number of Participants With Neutrophil Engraftment
Time frame: Day 42
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 36453638Martinez C, Aguayo-Hiraldo P, Chaimowitz N, Forbes L, Rider N, Nicholas S, Seeborg F, Chinen J, Chinn I, Davis C, Roseblatt H, Noroski L, Omer B, John T, Yassine K, Naik S, Craddock J, Bhar S, Allen C, Ahmed N, Sasa G, Steffin D, Doherty E, George A, Salem B, Friend B, Hegde M, Brenner MK, Heslop HE, Leen A, Pena A, Wu M, Hanson IC, Krance RA. Cord blood transplantation for nonmalignant disorders: early functional immunity and high survival. Blood Adv. 2023 May 9;7(9):1823-1830. doi: 10.1182/bloodadvances.2022009038.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Caridad Martinez, MD principal investigator
Baylor College of Medicine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Busulfan, Cytoxan and Fludarabine in people with Congenital Pediatric Disorders, run by Baylor College of Medicine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients. (time frame: 100 days). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00950846, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Oct 2023.