Trial Outcomes & Findings for Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia (NCT NCT00578903)

NCT ID: NCT00578903

Last Updated: 2016-04-22

Results Overview

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 22 participants
• Primary outcome timeframe: 100 days
• Results posted on: 2016-04-22

Participant Flow

Diagnosis SAA, failure to respond to immunosuppressive therapy, lack of HLA identical family member, A 6/6 or 5/6 HLA matched unrelated donor or a 5/6 matched related donor available after high resolution typing, age<60 years

Pirour to initicating the SCT preparatory regimen, patients require a double lumen Hickman catheter and completion of the pretransplant evaluation.

Participant milestones

Measure	Patients Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Overall Study STARTED	22
Overall Study COMPLETED	22
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Allogeneic Stem Cell Transplant for Patients With Severe Aplastic Anemia

Baseline characteristics by cohort

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Age, Customized 0-10 years	9 participants n=5 Participants
Age, Customized 11-20 years	12 participants n=5 Participants
Age, Customized 21-30 years	0 participants n=5 Participants
Age, Customized 31-40 years	0 participants n=5 Participants
Age, Customized 41-50 years	1 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: 100 days

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Subjects Alive at 100 Days Post Transplant	21 participants

SECONDARY outcome

Timeframe: 100 days post transplant

Absolute neutrophil count greater than 0.5 X 10^9/ml for at least 3 days

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Patients With Engraftment Rate at 100 Days Post Transplant	19 participants

SECONDARY outcome

Timeframe: 100 days

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Patients With Acute GVHD at 100 Days Post Transplant	4 participants

SECONDARY outcome

Timeframe: 2 years

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Patients With Chronic GVHD at 2 Years Post Transplant	1 participants

SECONDARY outcome

Timeframe: 1 year

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Subjects Alive at 1 Year Post Transplant	20 participants

SECONDARY outcome

Timeframe: 2 years

Outcome measures

Measure	Patients n=22 Participants Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Number of Subjects Alive at 2 Years Post Transplant	20 participants

Adverse Events

Patients

Serious events: 10 serious events

Other events: 20 other events

Deaths: 0 deaths

Serious adverse events

Measure	Patients n=22 participants at risk Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Cardiac disorders Hypertension	4.5% 1/22 • Number of events 1
Blood and lymphatic system disorders Hematuria (in the absence of vaginal bleeding)	4.5% 1/22 • Number of events 2
Infections and infestations Catheter-Related Infection	9.1% 2/22 • Number of events 3
Infections and infestations Infection (documented clinically or microbiologically) with grade 3 or 4 neutropenia (ANC <1.0 x 1	4.5% 1/22 • Number of events 1
Infections and infestations Infection without neutropenia	27.3% 6/22 • Number of events 10
Metabolism and nutrition disorders Hyperglycemia	9.1% 2/22 • Number of events 2
Metabolism and nutrition disorders Hypocalcemia	4.5% 1/22 • Number of events 1
Metabolism and nutrition disorders Hypokalemia	9.1% 2/22 • Number of events 3
Metabolism and nutrition disorders Hypophosphatemia	4.5% 1/22 • Number of events 1
Nervous system disorders Neurology-Other	4.5% 1/22 • Number of events 1
Nervous system disorders Seizure(s)	9.1% 1/11 • Number of events 2

Other adverse events

Measure	Patients n=22 participants at risk Patients with a diagnosis of severe aplastic anemia who require an allogeneic stem cell transplant but lack an Human Leukocyte Antigen (HLA) identical family member.
Skin and subcutaneous tissue disorders Rash/desquamation	27.3% 6/22 • Number of events 8
Skin and subcutaneous tissue disorders Rash/desquamation associated with graft versus host disease (GVHD) for BMT studies, if specifi	13.6% 3/22 • Number of events 3
Gastrointestinal disorders Anorexia	27.3% 6/22 • Number of events 11
Gastrointestinal disorders Diarrhea patients without colostomy	27.3% 6/22 • Number of events 8
Gastrointestinal disorders Nausea	45.5% 10/22 • Number of events 19
Gastrointestinal disorders Stomatitis/Pharyngitis (oral/pharyngeal mucositis)	18.2% 4/22 • Number of events 4
Gastrointestinal disorders Vomiting	36.4% 8/22 • Number of events 29
Hepatobiliary disorders Alkaline Phosphatase	13.6% 3/22 • Number of events 3
Hepatobiliary disorders Bilirubin	13.6% 3/22 • Number of events 6
Hepatobiliary disorders GGT (Gamma-Glutamyl transpeptidase)	36.4% 8/22 • Number of events 26
Hepatobiliary disorders Hypoalbuminemia	45.5% 10/22 • Number of events 28
Hepatobiliary disorders SGOT (AST) (serum glutamic oxaloacetic transaminase)	45.5% 10/22 • Number of events 29
Hepatobiliary disorders SGPT (ALT) (serum glutamic pyruvic transaminase)	59.1% 13/22 • Number of events 38
Metabolism and nutrition disorders Bicarbonate	18.2% 4/22 • Number of events 6
Metabolism and nutrition disorders Hypercalcemia	22.7% 5/22 • Number of events 8
Metabolism and nutrition disorders Hypermagnesemia	22.7% 5/22 • Number of events 10
Metabolism and nutrition disorders Hypernatremia	9.1% 2/22 • Number of events 6
Metabolism and nutrition disorders Hypertriglyceridemia	22.7% 5/22 • Number of events 9
Metabolism and nutrition disorders Hypokalemia	54.5% 12/22 • Number of events 56
Metabolism and nutrition disorders Hypomagnesemia	59.1% 13/22 • Number of events 47
Metabolism and nutrition disorders Hypomagnesmia	27.3% 6/22 • Number of events 24
Metabolism and nutrition disorders Hyponatremia	68.2% 15/22 • Number of events 34
General disorders Abdominal pain or cramping	13.6% 3/22 • Number of events 3
General disorders Headache	18.2% 4/22 • Number of events 9
General disorders Pain-Other	27.3% 6/22 • Number of events 9
Respiratory, thoracic and mediastinal disorders Cough	9.1% 2/22 • Number of events 2
Respiratory, thoracic and mediastinal disorders Hypoxia	13.6% 3/22 • Number of events 3
Renal and urinary disorders Creatinine	9.1% 2/22 • Number of events 8
Renal and urinary disorders Proteinuria	9.1% 2/22 • Number of events 2
Renal and urinary disorders Renal/Genitourinary-Other	13.6% 3/22 • Number of events 4

Additional Information

Kathryn Leung

Baylor College of Medicine

Phone: 832-824-4269

Email: kleung@bcm.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place