Trial Outcomes & Findings for HD Melphalan and SCT in Patients With IGDD or LCDD (NCT NCT00681044)
NCT ID: NCT00681044
Last Updated: 2017-04-28
Results Overview
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
one year
Results posted on
2017-04-28
Participant Flow
Patients were enrolled from the Boston Medical Center Stem Cell Transplant Program between 2006 and 2009.
A patient enrolled on this study but was withdrawn due to insufficient stem cell yield. His clinical situation changed 5 months later and the investigators believed he would have a better yield so he was enrolled for a second time. Therefore, there were 5 enrollments, but 4 patients.
Participant milestones
| Measure |
SCT With Melphalan Conditioning
Mobilization with Filgrastim Stem Cell collection (SCC) Melphalan Conditioning Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC
melphalan: 70-100 mg/m2/day will be administered intravenously on Days -3 and -2
Stem Cell Infusion: infusion of previously collected stem cells on Day 0
|
|---|---|
|
Overall Study
STARTED
|
4
|
|
Overall Study
COMPLETED
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
HD Melphalan and SCT in Patients With IGDD or LCDD
Baseline characteristics by cohort
| Measure |
SCT With Melphalan Conditioning
n=4 Participants
Mobilization with Filgrastim Stem Cell collection Melphalan Conditioning Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC
melphalan: 70-100 mg/m2/day will be administered intravenously on Days -3 and -2
Stem Cell Infusion: infusion of previously collected stem cells on Day 0
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
4 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
4 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
4 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: one yearPopulation: No data were collected or analyzed due to study termination
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: One monthPopulation: No data were collected or analyzed due to study termination
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: One yearPopulation: No data were collected or analyzed due to study termination
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: lifePopulation: No data were collected or analyzed due to study termination
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: 100 daysPopulation: No data were collected or analyzed due to study termination
Outcome measures
Outcome data not reported
Adverse Events
SCT With Melphalan Conditioning
Serious events: 4 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
SCT With Melphalan Conditioning
n=4 participants at risk
Mobilization with Filgrastim Stem Cell collection Melphalan Conditioning Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC
melphalan: 70-100 mg/m2/day will be administered intravenously on Days -3 and -2
Stem Cell Infusion: infusion of previously collected stem cells on Day 0
|
|---|---|
|
Immune system disorders
drug reaction
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
vomiting
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Gastrointestinal disorders
diarrhea
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Gastrointestinal disorders
dysphagia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
dehydration
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
General disorders
fatigue
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Cardiac disorders
hypertension
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Skin and subcutaneous tissue disorders
Toxic Epidermal Necrosis (TEN)
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
dyspnea
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
General disorders
joint pain
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
mucositis
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Gastrointestinal disorders
voice changes
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
pulmonary edema
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Hepatobiliary disorders
hypoalbuminemia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Renal and urinary disorders
renal failure
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Renal and urinary disorders
renal insufficiency
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Renal and urinary disorders
increased creatinine
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Metabolism and nutrition disorders
hypophosphatemia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Metabolism and nutrition disorders
hyperuricemia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Metabolism and nutrition disorders
hyperkalemia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Infections and infestations
febrile neutropenia
|
50.0%
2/4 • Number of events 2 • 100 days
|
Other adverse events
| Measure |
SCT With Melphalan Conditioning
n=4 participants at risk
Mobilization with Filgrastim Stem Cell collection Melphalan Conditioning Stem Cell infusion
filgrastim: 16 mcg/kg daily beginning 3 days prior to SCC through day before final SCC
melphalan: 70-100 mg/m2/day will be administered intravenously on Days -3 and -2
Stem Cell Infusion: infusion of previously collected stem cells on Day 0
|
|---|---|
|
General disorders
fatigue
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
General disorders
insomnia
|
75.0%
3/4 • Number of events 3 • 100 days
|
|
General disorders
rigors
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
nausea
|
100.0%
4/4 • Number of events 4 • 100 days
|
|
Gastrointestinal disorders
vomiting
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Gastrointestinal disorders
diarrhea
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
General disorders
Dehydration
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
anorexia
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Cardiac disorders
hypertension
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Skin and subcutaneous tissue disorders
alopecia
|
75.0%
3/4 • Number of events 3 • 100 days
|
|
Skin and subcutaneous tissue disorders
rash
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Cardiac disorders
sinus tacchycardia
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Blood and lymphatic system disorders
peripheral edema
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Musculoskeletal and connective tissue disorders
muscle weakness
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Musculoskeletal and connective tissue disorders
back pain
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Nervous system disorders
dizziness
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Nervous system disorders
seizure
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Musculoskeletal and connective tissue disorders
joint pain
|
50.0%
2/4 • Number of events 2 • 100 days
|
|
Respiratory, thoracic and mediastinal disorders
bilateral pleural effusions
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Gastrointestinal disorders
mucositis
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Hepatobiliary disorders
elevated ALT
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Hepatobiliary disorders
hypoalbuminemia
|
75.0%
3/4 • Number of events 3 • 100 days
|
|
Hepatobiliary disorders
elevated alk phos
|
25.0%
1/4 • Number of events 1 • 100 days
|
|
Blood and lymphatic system disorders
line bleed
|
25.0%
1/4 • Number of events 1 • 100 days
|
Additional Information
Dr. Vaishali Sanchorawala, Director of Stem Cell Transplant Program
Boston Medical Center
Phone: 617-637-7017
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place