Trial Outcomes & Findings for Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma (NCT NCT00278525)

Last Updated: 2014-04-30

Results Overview

-Data are reporting number of participants that were classified as treatment failures Time to Treatment Failure Definition-Treatment failure will not occur until a minimum of 12 months after enrollment at which time failure is defined as: 1. Failure of skin score (if \> 14 on enrollment) to improve or increase in skin score by a 25% above lowest post treatment value and must be documented on 2 occasion 6 months apart 2. Deterioration in diffusing capacity of the lung for carbon monoxide (DLCO), diffusing capacity divided by the alveolar volume (DLCO/VA) or forced vital capacity (FVC) by 10% below enrollment level or 10% below best post treatment value, due to systemic sclerosis, and documented on 2 occasion 6 months apart 3. Renal failure due to systemic sclerosis and defined as chronic dialysis for more than 12 months 4. Gastrointestinal failure due to systemic sclerosis and defined as initiation of total parenteral nutrition(TPN) for more than 12 months

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

19 participants

Primary outcome timeframe

12 months

Results posted on

2014-04-30

Participant Flow

Eligible (n=19) Randomized (n=19) 1. Allocated to Hematopoietic Stem Cell Transplantation (HSCT (n=10) + crossed over to HSCT(n=7) • Received HSCT (n=17); Lost to follow-up (n=0); Analysed (n=17) 2. Allocated to cyclophosphamide (n=9) • Received allocated intervention (n=9) Lost to follow-up (n=0); Crossed over (n=7); Analysed (n=9)

Participant milestones

Participant milestones
Measure	Standard of Care Cyclophosphamide will be given as approved immunosuppressive therapy	Stem Cell Trasplantation intervention as stem cell transplantation after conditioning regimen
Overall Study STARTED	9	10
Overall Study COMPLETED	9	10
Overall Study NOT COMPLETED	0	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Cyclophosphamide and rATG With Hematopoietic Stem Cell Support in Systemic Scleroderma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stem Cell Trasplantation n=10 Participants intervention as stem cell transplantation after conditioning regimen	Standard of Care n=9 Participants medication as standard of care will be given	Total n=19 Participants Total of all reporting groups
Age, Categorical <=18 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Categorical Between 18 and 65 years	10 Participants n=93 Participants	9 Participants n=4 Participants	19 Participants n=27 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants	0 Participants n=4 Participants	0 Participants n=27 Participants
Age, Continuous	45 years STANDARD_DEVIATION 13 • n=93 Participants	44 years STANDARD_DEVIATION 10 • n=4 Participants	44 years STANDARD_DEVIATION 10 • n=27 Participants
Sex: Female, Male Female	10 Participants n=93 Participants	8 Participants n=4 Participants	18 Participants n=27 Participants
Sex: Female, Male Male	0 Participants n=93 Participants	1 Participants n=4 Participants	1 Participants n=27 Participants
Region of Enrollment United States	10 participants n=93 Participants	9 participants n=4 Participants	19 participants n=27 Participants

PRIMARY outcome

Timeframe: 12 months

Population: All participants were included

Outcome measures

Outcome measures
Measure	Stem Cell Trasplantation n=10 Participants intervention as stem cell transplantation after conditioning regimen	Standard of Care n=9 Participants Intravenous (IV) will be given 1000 mg/m2 cyclophosphamide monthly for 6 months.
Time to Treatment Failure	0 participants 0.01	8 participants 0.11

PRIMARY outcome

Timeframe: 12 months

Data are reporting number of participants that were classified as disease improvement. Definition of disease improvement: Disease improvement defined by at least 25% improvement in skin score (Rodnan), or 10% improvement in pulmonary function tests \[diffusing capacity of the lung for carbon monoxide (DLCO), diffusing capacity divided by the alveolar volume (DLCO/VA), or forced vital capacity (FVC)\], or in cardiac tests \[pulmonary artery (PA) systolic pressure by right heart cath\] that persists \> 6 months or ability to wean off total parenteral nutrition (TPN)

Outcome measures

Outcome measures
Measure	Stem Cell Trasplantation n=10 Participants intervention as stem cell transplantation after conditioning regimen	Standard of Care n=9 Participants Intravenous (IV) will be given 1000 mg/m2 cyclophosphamide monthly for 6 months.
Disease Improvement	10 participants	0 participants

Adverse Events

Stem Cell Trasplantation

Serious events: 0 serious events

Other events: 7 other events

Deaths: 0 deaths

Standard of Care

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Stem Cell Trasplantation n=17 participants at risk intervention as stem cell transplantation after conditioning regimen	Standard of Care n=9 participants at risk medication as standard of care will be given
Infections and infestations Cytomegalovirus reactivation on surveillance blood test	5.9% 1/17 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Infections and infestations Cellulitis	0.00% 0/17 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	11.1% 1/9 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Gastrointestinal disorders Gastroenteritis	0.00% 0/17 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	22.2% 2/9 • Number of events 2 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Infections and infestations Positive stool culture for Clostridium difficile	5.9% 1/17 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Infections and infestations Positive blood culture for micrococcus	5.9% 1/17 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Cardiac disorders Supraventricular tachycardia	5.9% 1/17 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Cardiac disorders Atrial fibrillation	5.9% 1/17 • Number of events 1 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities
Cardiac disorders Volume overload	11.8% 2/17 • Number of events 2 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities	0.00% 0/9 • 100 days after the transplant Common Toxicity Criteria Scale used to grade all non-hematologic toxicities

Additional Information

Dr. Richard Burt

Northwestern University

Phone: 312-908-0059

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place