Trial Outcomes & Findings for Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders (NCT NCT00744692)

Last Updated: 2014-08-13

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE1

Target enrollment

22 participants

Primary outcome timeframe

180 days post transplant

Results posted on

2014-08-13

Participant Flow

Participant milestones

Participant milestones
Measure	RIC Cord Blood Transplant Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
Overall Study STARTED	22
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Reduced Intensity Conditioning for Umbilical Cord Blood Transplant in Pediatric Patients With Non-Malignant Disorders

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
Age, Continuous	2.83 years n=5 Participants
Age, Categorical <=18 years	22 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	13 Participants n=5 Participants

PRIMARY outcome

Timeframe: 180 days post transplant

Population: Of the 22 patients enrolled, 18 patients were alive at 180 days, the time-point for primary end point

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=18 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
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 % of participants

SECONDARY outcome

Timeframe: 42 days post transplant

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

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Describe the Pace of Neutrophil Recovery	20 days Interval 12.0 to 28.0

SECONDARY outcome

Timeframe: 1 year post transplant

Population: Of the 22 patients at baseline, 5 patients died early before a year; and 2 additional patients had early graft failure. Thus 7 patients were not available for analysis of Immune reconstitution endpoint. CD4 count is reported here.

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.

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=15 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Evaluate the Pace of Immune Reconstitution.	805 cells/uL Interval 6.0 to 2013.0

SECONDARY outcome

Timeframe: 180 days

To determine the overall survival at day180 post-transplant: determined by Kaplan Meier survival analysis

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Determine the Overall Survival at day180 Post-transplant	81.8 percentage of participants Interval 58.5 to 92.8

SECONDARY outcome

Timeframe: 100 days post transplant

To describe incidence of acute Graft Versus Host Disease (GVHD) (II - IV) : measured by cumulative incidence analysis

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Describe Incidence of Acute Graft Versus Host Disease (GVHD) (II - IV)	22.7 percentage of participants Interval 5.3 to 40.1

SECONDARY outcome

Timeframe: 2 years post transplant

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Describe the Incidence of Grade 3-4 Organ Toxicity	0 participants

SECONDARY outcome

Timeframe: at least 2 years post transplant

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=15 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Evaluate Long-term Complications, Such as Sterility, Endocrinopathy, and Growth Failure	0 percentage of patients

SECONDARY outcome

Timeframe: 2 years post transplant

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=15 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Evaluate the Incidence of Late Graft Failures at 2 Years Post-transplant	0 participants

SECONDARY outcome

Timeframe: 180 days post transplant

Platelet engraftment was defined as the first day of platelet counts more than 50,000/uL for 7 consecutive days without transfusions

Outcome measures

Outcome measures
Measure	RIC Cord Blood Transplant n=22 Participants Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
To Describe the Pace of Platelet Recovery	48 days Interval 33.0 to 144.0

Adverse Events

RIC Cord Blood Transplant

Serious events: 18 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	RIC Cord Blood Transplant n=22 participants at risk Reduced Intensity Conditioning for Umbilical Cord Blood Transplant Unrelated Umbilical Cord Blood Transplant: Reduced Intensity Conditioning for unrelated umbilical cord blood transplant Reduced Intensity Conditioning
Respiratory, thoracic and mediastinal disorders Respiratory Distress	9.1% 2/22 Adverse events that were expected and non-serious were not collected in this study.
Investigations Fever	22.7% 5/22 Adverse events that were expected and non-serious were not collected in this study.
Metabolism and nutrition disorders Dehydration	9.1% 2/22 Adverse events that were expected and non-serious were not collected in this study.
Blood and lymphatic system disorders Auto Immune Hemolytic Anemia	9.1% 2/22 Adverse events that were expected and non-serious were not collected in this study.
Infections and infestations Infection	54.5% 12/22 Adverse events that were expected and non-serious were not collected in this study.
Blood and lymphatic system disorders Hemolytic Transfusion Reaction	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Blood and lymphatic system disorders Graft Failure Post Transplant	9.1% 2/22 Adverse events that were expected and non-serious were not collected in this study.
Infections and infestations Sepsis	13.6% 3/22 Adverse events that were expected and non-serious were not collected in this study.
Gastrointestinal disorders Abdominal Pain	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Renal and urinary disorders Nephrotic Syndrome	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Nervous system disorders VP Shunt Malfunction	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Gastrointestinal disorders GI Distress	13.6% 3/22 Adverse events that were expected and non-serious were not collected in this study.
General disorders Multi System Organ Failure	9.1% 2/22 Adverse events that were expected and non-serious were not collected in this study.
Nervous system disorders Seizure	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Respiratory, thoracic and mediastinal disorders Respiratory Failure	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
General disorders Graft Versus Host Disease	18.2% 4/22 Adverse events that were expected and non-serious were not collected in this study.
Surgical and medical procedures Central Line Removal	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Blood and lymphatic system disorders Deep Vein Thrombosis	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Endocrine disorders Hyperglycemia	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Gastrointestinal disorders Pnematosis	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Nervous system disorders Posterior Reversible Encephalopathy Syndrome	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Skin and subcutaneous tissue disorders Necrotic Ulcer (skin)	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.
Surgical and medical procedures Dental Rehabilitation	4.5% 1/22 Adverse events that were expected and non-serious were not collected in this study.

Other adverse events

Adverse event data not reported

Additional Information

Dr. Suhag Parikh

Duke University Medical Center

Phone: 919-668-1100

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place