Trial Outcomes & Findings for Reduced Intensity Hematopoietic Cell Transplantation for Patients With Resistant Langerhans Cell Histiocytosis (NCT NCT00618540)
NCT ID: NCT00618540
Last Updated: 2017-12-28
Results Overview
Count of patients alive at 1 and 3 years. Deaths from any cause are events. Surviving patients are censored at the date of last contact.
Recruitment status
TERMINATED
Study phase
PHASE2
Target enrollment
1 participants
Primary outcome timeframe
Year 1, Year 3
Results posted on
2017-12-28
Participant Flow
Participant milestones
| Measure |
Alemtuzumab Conditioning
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Overall Study
STARTED
|
1
|
|
Overall Study
COMPLETED
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced Intensity Hematopoietic Cell Transplantation for Patients With Resistant Langerhans Cell Histiocytosis
Baseline characteristics by cohort
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Age, Categorical
<=18 years
|
1 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
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
1 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Year 1, Year 3Count of patients alive at 1 and 3 years. Deaths from any cause are events. Surviving patients are censored at the date of last contact.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Overall Survival
|
0 participants
|
PRIMARY outcome
Timeframe: Year 1This outcome is defined as survival with resolution of LCH at 12 months post transplant. Unresolved disease for over 12 months post-transplant, progressive disease after this time period, recurrence of disease and death from any cause are considered events. Those who survive with resolution of disease are censored at the date of last contact.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Disease-free Survival at 12 Months Post Transplantation
|
0 participants
|
SECONDARY outcome
Timeframe: Day 100Count of patients who died by day 100 related to the transplantation.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Transplantation-related Death
|
0 participants
|
SECONDARY outcome
Timeframe: Day 100Incidence of neutrophil recovery and donor chimerism at Day 100.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Neutrophil Engraftment
|
1 participants
|
SECONDARY outcome
Timeframe: Day 100 and Month 6The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive \<21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Incidence of Grade II-IV Acute Graft-versus-host-disease (GVHD)
|
1 participants
|
SECONDARY outcome
Timeframe: Day 100 and Month 6Occurrence of symptoms in any organ system fulfilling the criteria of limited or extensive chronic GvHD (Appendix III), among patients surviving \> 90 days with evidence of engraftment. Patients without chronic GvHD will be censored at time of death or last follow-up.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Incidence of Chronic GVHD
|
0 participants
|
SECONDARY outcome
Timeframe: Day 100Incidence of platelet recovery and donor chimerism at Day 100.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Platelet Engraftment
|
0 participants
|
SECONDARY outcome
Timeframe: Day 100 and Month 6The occurrence of skin, gastrointestinal or liver abnormalities fulfilling the criteria of Grades II, III and/or IV acute GVHD are considered events (Appendix II). Patients without acute GvHD will be censored at the time of death or last follow-up. Patients that survive \<21 days and listed as not evaluable will be excluded. Patients receiving a second transplant will be censored at the time of second transplant.
Outcome measures
| Measure |
Alemtuzumab Conditioning
n=1 Participants
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Incidence of Grade III-IV Acute Graft-versus-host-disease (GVHD)
|
0 participants
|
Adverse Events
Alemtuzumab Conditioning
Serious events: 1 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Alemtuzumab Conditioning
n=1 participants at risk
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Respiratory syncytial virus (RSV) infection
|
100.0%
1/1 • Number of events 1 • The adverse event data were collected at 100 days post-transplantation.
|
Other adverse events
| Measure |
Alemtuzumab Conditioning
n=1 participants at risk
Patients administered with alemtuzumab, fludarabine phosphate, melphalan and donor stem cell transplantation in children with resistant Langerhans cell histiocytosis.
alemtuzumab: Administered intravenously (IV) 0.2 mg/kg on Days -8 through -4.
fludarabine phosphate: Administered 30 mg/m2 intravenously (IV) over 30-60 min on Days -7 through -3.
(dose adjust if age \<12 months)
melphalan: Administered 140 mg/m2 intravenously (IV) over 30 min on Day -2 (dose adjust if age \<12 months)
stem cell transplantation: Administered as allogeneic hematopoietic, peripheral blood or umbilical cord blood transplantation
|
|---|---|
|
Infections and infestations
Multiple systemic infections
|
100.0%
1/1 • The adverse event data were collected at 100 days post-transplantation.
|
Additional Information
Dr. Angela Smith, MD
University of Minnesota, Pediatric BMT
Phone: 612-626-2778
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place