A Phase 1 interventional study of Unrelated Umbilical Cord Blood Transplant and Reduced Intensity Conditioning in Non Malignant Disorders, Immunodeficiencies and Congenital Marrow Failures, sponsored by Duke University. Completed at 1 site in United States. Open to participants aged Up to 21 Years. Per ClinicalTrials.gov, last updated 2014-08-13.
Sponsored by Duke University · Phase 1, Interventional, and Treatment
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
Myeloablative doses of chemotherapy and/or radiation therapy are employed with the primary purpose of eradicating malignant cells. Additionally, these regimens exert varying degree of immunosuppression/immunoablation that aids in reducing the likelihood of rejection by host hematopoietic cells. However, myeloablative /immunoablative regimens have also been associated with significant regimen related toxicity (RRT) and regimen related mortality (RRM) that may cause death in up to 20% of patients and significantly higher rate of severe organ dysfunction or failure. While most of these RRT occur typically in the first 100 days [ e.g. VOD (veno occlusive disease), pulmonary or intracranial hemorrhage, multiorgan failure (MOF)], there are significant long term toxicities of TBI and/or chemotherapy including growth impairment, gonadal dysfunction/failure, hypothyroidism, cataracts, neurocognitive impairment, and second malignancies.
The primary objective is to determine the feasibility of attaining acceptable rates of donor cell engraftment (>25% donor chimerism at 180 days) following reduced intensity conditioning (RIC) regimens in pediatric patients \< 21 years receiving cord blood transplantation for non-malignant disorders.
The secondary objectives are:
416 studies on the registry are indexed under Thalassemia; 67 are open to participants now.
This study's enrollment of 22 is below the median of 37 across 277 interventional studies indexed under Thalassemia.
Browse Thalassemia studies →Duke University is the lead sponsor of 2,025 studies on the registry; 275 are open to participants now.
Of its 194 completed or terminated interventional studies of FDA-regulated products, 159 (82%) have results posted.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
Reduced Intensity Conditioning for Umbilical Cord Blood Transplant
Biological: Unrelated Umbilical Cord Blood Transplant · Drug: Reduced Intensity Conditioning
Reduced Intensity Conditioning for unrelated umbilical cord blood transplant
Also known as: Campath, Hydroxyurea, Fludarabine, Melphalan, Thiotepa
Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders.
Determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \< 21 years receiving cord blood transplant for non-malignant disorders.
Time frame: 180 days post transplant
To Describe the Pace of Neutrophil Recovery
Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions
Time frame: 42 days post transplant
To Evaluate the Pace of Immune Reconstitution.
Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.
Time frame: 1 year post transplant
To Determine the Overall Survival at day180 Post-transplant
To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis
Time frame: 180 days
To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)
To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis
Time frame: 100 days post transplant
To Describe the Incidence of Grade 3-4 Organ Toxicity
Time frame: 2 years post transplant
To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure
Time frame: at least 2 years post transplant
To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant
Time frame: 2 years post transplant
To Describe the Pace of Platelet Recovery
Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions
Time frame: 180 days post transplant
| Milestone | RIC Cord Blood Transplant |
|---|---|
| Started | 22 |
| Completed | 22 |
| Not completed | 0 |
Determine the feasibility of attaining acceptable rates of donor cell engraftment (\>25% donor cells at 180 days) following reduced intensity conditioning regimens in children \< 21 years receiving cord blood transplant for non-malignant disorders.
| % of participants | RIC Cord Blood Transplant |
|---|---|
| Determine the Feasibility of Attaining Acceptable Rates of Donor Cell Engraftment (>25% Donor Cells at 180 Days) Following RIC Regimens in Children < 21 Years Receiving UCBT for Non-malignant Disorders. | 88 |
Neutrophil recovery was defined as the first day of an absolute neutrophil count (ANC) more than 500/uL for 3 consecutive days not secondary to granulocyte infusions
| days | RIC Cord Blood Transplant |
|---|---|
| To Describe the Pace of Neutrophil Recovery | 20 (12 to 28) |
Immune reconstitution after RIC in UCBT was described. CD4 count is a standard measure of immune reconstitution and is described here. Additional data is available upon request.
| cells/uL | RIC Cord Blood Transplant |
|---|---|
| To Evaluate the Pace of Immune Reconstitution. | 805 (6 to 2013) |
To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis
| percentage of participants | RIC Cord Blood Transplant |
|---|---|
| To Determine the Overall Survival at day180 Post-transplant | 81.8 (58.5 to 92.8) |
To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis
| percentage of participants | RIC Cord Blood Transplant |
|---|---|
| To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV) | 22.7 (5.3 to 40.1) |
| participants | RIC Cord Blood Transplant |
|---|---|
| To Describe the Incidence of Grade 3-4 Organ Toxicity | 0 |
| percentage of patients | RIC Cord Blood Transplant |
|---|---|
| To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure | 0 |
| participants | RIC Cord Blood Transplant |
|---|---|
| To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant | 0 |
Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions
| days | RIC Cord Blood Transplant |
|---|---|
| To Describe the Pace of Platelet Recovery | 48 (33 to 144) |
Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| RIC Cord Blood Transplant | — | 18/22 (81.8%) | 0/22 (0%) |
| Event | RIC Cord Blood Transplant |
|---|---|
| InfectionInfections and infestations | 12/22 |
| FeverInvestigations | 5/22 |
| Graft Versus Host DiseaseGeneral disorders | 4/22 |
| SepsisInfections and infestations | 3/22 |
| GI DistressGastrointestinal disorders | 3/22 |
| Respiratory DistressRespiratory, thoracic and mediastinal disorders | 2/22 |
| DehydrationMetabolism and nutrition disorders | 2/22 |
| Auto Immune Hemolytic AnemiaBlood and lymphatic system disorders | 2/22 |
| Graft Failure Post TransplantBlood and lymphatic system disorders | 2/22 |
| Multi System Organ FailureGeneral disorders | 2/22 |
22 subjects undergoing umbilical cord blood transplant
| Age, Continuous(years) | RIC Cord Blood Transplant |
|---|---|
| Median | 2.83 (0.3 to 8) |
| Age, Categorical(Participants) | RIC Cord Blood Transplant |
|---|---|
| <=18 years | 22 |
| Between 18 and 65 years | 0 |
| >=65 years | 0 |
| Sex: Female, Male(Participants) | RIC Cord Blood Transplant |
|---|---|
| Female | 9 |
| Male | 13 |
This study is completed, as verified in Jul 2014. 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.
Duke University