Trial Outcomes & Findings for Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders (NCT NCT00579137)
NCT ID: NCT00579137
Last Updated: 2013-07-02
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE1/PHASE2
Target enrollment
3 participants
Primary outcome timeframe
100 Days
Results posted on
2013-07-02
Participant Flow
Participant milestones
| Measure |
Participants With SCID or Primary Immunodeficiency Disorder
Participants received an allogeneic stem cell transplant with the following conditioning:
Day 8 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; \> 10 kg: 30 mg/m2
D7 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; \> 10 kg: 30 mg/m2
D6 Campath 1H as per CAGT SOP, Fludarabine 10 kg or less: 1 mg/kg; \> 10 kg: 30 mg/m2
D5 Anti-CD45 MAb 400ug/kg over 6 hr, Fludarabine 10 kg or less: 1 mg/kg; \> 10 kg: 30 mg/m2
D4 Anti-CD45 MAb 400ug/kg over 6 hr, Fludarabine 10 kg or less: 1 mg/kg; \> 10 kg: 30 mg/m2
D3 Anti-CD45 MAb 400ug/kg over 6 hr
D2 Anti-CD45 MAb 400ug/kg over 6 hr
D1 rest
D0 Stem Cell Infusion
Campath dose is weight based: for patients less than 15 kg administer Campath 3 mg; for patients \>15 kg to 30 kg administer Campath 5 mg; for patients \> 30 kg administer Campath 10 mg. Campath will be dosed and administered as per CAGT SOP.
Anti-CD45 infusion will be administered according to CAGT SOPs.
|
|---|---|
|
Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders
Baseline characteristics by cohort
| Measure |
Single Group
n=3 Participants
only one group
|
|---|---|
|
Age, Customized
<=1 years
|
3 participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 100 DaysOutcome measures
| Measure |
Single Group
n=3 Participants
only one group
|
|---|---|
|
Number of Patients With Donor Engraftment
|
1 participants
|
SECONDARY outcome
Timeframe: 1 YearOutcome measures
| Measure |
Single Group
n=3 Participants
only one group
|
|---|---|
|
Patients Alive at 1 Year
|
3 participants
|
SECONDARY outcome
Timeframe: 100 daysOutcome measures
| Measure |
Single Group
n=3 Participants
only one group
|
|---|---|
|
Number of Patients With Grade III or IV Toxicity
|
0 participants
|
SECONDARY outcome
Timeframe: 100 daysOutcome measures
| Measure |
Single Group
n=3 Participants
only one group
|
|---|---|
|
Number of Patients With Grade III to IV Acute GVHD
|
0 participants
|
Adverse Events
Single Group
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Single Group
n=3 participants at risk
only one group
|
|---|---|
|
Metabolism and nutrition disorders
AST, SGOT(serum glutamic oxaloacetic transaminase)
|
33.3%
1/3 • Number of events 5
|
|
Metabolism and nutrition disorders
Albumin, serum-low (hypoalbuminemia)
|
33.3%
1/3 • Number of events 5
|
|
Immune system disorders
Allergic reaction/hypersensitivity (including drug fever)
|
33.3%
1/3 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Bronchospasm, wheezing
|
33.3%
1/3 • Number of events 1
|
|
Metabolism and nutrition disorders
Calcium, serum-low (hypocalcemia)
|
33.3%
1/3 • Number of events 2
|
|
Gastrointestinal disorders
Constipation
|
33.3%
1/3 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
33.3%
1/3 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
Dermatology/Skin - Other: Livedo reticularis
|
33.3%
1/3 • Number of events 2
|
|
Gastrointestinal disorders
Diarrhea
|
33.3%
1/3 • Number of events 5
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
33.3%
1/3 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea (shortness of breath)
|
33.3%
1/3 • Number of events 2
|
|
General disorders
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0 x 10e9/L)
|
33.3%
1/3 • Number of events 1
|
|
Metabolism and nutrition disorders
Glucose, serum-low (hypoglycemia)
|
33.3%
1/3 • Number of events 1
|
|
Blood and lymphatic system disorders
Hemoglobin
|
33.3%
1/3 • Number of events 5
|
|
Hepatobiliary disorders
Hepatobiliary/Pancreas - Other: Liver GvHD
|
33.3%
1/3 • Number of events 1
|
|
Infections and infestations
Infection (documented clinically or microbiologically) with Grade 3 or 4 neutrophils (ANC <1.0 x 10e
|
33.3%
1/3 • Number of events 2
|
|
Infections and infestations
Infection with normal ANC or Grade 1 or 2 neutrophils - Catheter-related
|
33.3%
1/3 • Number of events 1
|
|
Blood and lymphatic system disorders
Leukocytes (total WBC)
|
33.3%
1/3 • Number of events 17
|
|
Blood and lymphatic system disorders
Lymphopenia
|
33.3%
1/3 • Number of events 23
|
|
Gastrointestinal disorders
Mucositis/stomatitis (clinical exam) - Oral cavity
|
33.3%
1/3 • Number of events 1
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • Number of events 3
|
|
Blood and lymphatic system disorders
Neutrophils/granulocytes (ANC/AGC)
|
33.3%
1/3 • Number of events 10
|
|
Blood and lymphatic system disorders
PTT (Partial Thromboplastin Time)
|
33.3%
1/3 • Number of events 1
|
|
General disorders
Pain - Abdomen NOS
|
33.3%
1/3 • Number of events 1
|
|
Metabolism and nutrition disorders
Phosphate, serum-low (hypophosphatemia)
|
33.3%
1/3 • Number of events 4
|
|
Blood and lymphatic system disorders
Platelets
|
33.3%
1/3 • Number of events 1
|
|
Metabolism and nutrition disorders
Potassium, serum-high (hyperkalemia)
|
33.3%
1/3 • Number of events 7
|
|
Respiratory, thoracic and mediastinal disorders
Pulmonary/Upper Respiratory - Other: Nasal congestion
|
33.3%
1/3 • Number of events 1
|
|
Skin and subcutaneous tissue disorders
Rash/desquamation
|
33.3%
1/3 • Number of events 3
|
|
Metabolism and nutrition disorders
Sodium, serum-low (hyponatremia)
|
66.7%
2/3 • Number of events 2
|
|
Metabolism and nutrition disorders
Triglyceride, serum-high (hypertriglyceridemia)
|
33.3%
1/3 • Number of events 1
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
1/3 • Number of events 5
|
|
Metabolism and nutrition disorders
ALT, SGPT (serum glutamic pyruvic transaminase)
|
33.3%
1/3 • Number of events 5
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place