Trial Outcomes & Findings for High Dose Cyclophosphamide for Treatment of Scleroderma (NCT NCT00501995)

NCT ID: NCT00501995

Last Updated: 2017-06-14

Results Overview

The modified Rodnan skin score is the accepted clinical measure of scleroderma skin activity. The investigator will assess the thickening of the skin using the modified Rodnan skin score through simple palpation on 17 different body areas: fingers, hands, forearms, arms, feet, legs, and thighs (bilaterally) and face, chest, and abdomen (singly). Skin thickness is assessed on a scale of 0-3; 0 representing normal skin and 3 being severe thickening. The sum of the individual scores can range from 0-51; 0 (normal) to 51 (severe thickening in all 17 areas) A 25% improvement in the modified Rodnan Skin score will be considered significant at any time point in the study. Modified Rodnan Skin Score was evaluated at months 0,1,3,6,12 and 24 months.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

6 participants

Primary outcome timeframe

0 to 24 months

Results posted on

2017-06-14

Participant Flow

Participant milestones

Participant milestones
Measure	IV Cyclophosphamide (50 mg/kg) This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously over 1 hour daily for four consecutive days (200 mg/kg total) through a Hickman catheter . IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Overall Study STARTED	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

High Dose Cyclophosphamide for Treatment of Scleroderma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	IV Cyclophosphamide (50 mg/kg) n=6 Participants This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Age, Continuous	39 years n=5 Participants
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	6 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants
Number of Participants with diagnosis of scleroderma	6 participants n=5 Participants

PRIMARY outcome

Timeframe: 0 to 24 months

Population: Patient 4 died during the early phase of the study and longitudinal assessment of his skin score was not determined.

Outcome measures

Outcome measures
Measure	IV Cyclophosphamide (50 mg/kg) n=5 Participants This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously over 1 hour daily for four consecutive days (200 mg/kg total) through a Hickman catheter . IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Improvement in the Modified Rodnan Skin Score.	46.75 percent improvement from baseline Interval 31.0 to 60.0

SECONDARY outcome

Timeframe: 0-24 months

Population: The study group consisted of 4 men and 2 women aged 19-60 years of old.

The Health Assessment Questionnaire-Disability Index (HAQ-DI) a 48 item questionnaire assessing ability to perform activities of daily living, use of assistive devises and a 6 item analog scale of pain severity from 0 cm (no pain) to 14.3 cm (very severe pain). The lower the HAQ-DI score the less the disability. The physician global assessment (PGA) which is a visual analogue scale from 0 to 100 on which the physician rates the patient's disease severity based on their observations. A score of 0 is no disease activity and 100 is the worst possible disease activity. The Forced Vital Capacity (FVC) measure of lung capacity and Diffusing Capacity (DLCO) measures of oxygen exchange in the alveoli ( pulmonary function testing). The predicted lung volumes were referenced from NHANES/Hanikson et al and for DLCO predicts were from Knudson. Pre and post study percent predicted values were compared.

Outcome measures

Outcome measures
Measure	IV Cyclophosphamide (50 mg/kg) n=6 Participants This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously over 1 hour daily for four consecutive days (200 mg/kg total) through a Hickman catheter . IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Change in the HAQ-DI, PGA, FVC and DLCO HAQ-DI score	79 percentage change Interval 0.0 to 93.0
Change in the HAQ-DI, PGA, FVC and DLCO PGA	71 percentage change Interval 62.0 to 80.0
Change in the HAQ-DI, PGA, FVC and DLCO FVC	0 percentage change Interval -7.0 to 5.0
Change in the HAQ-DI, PGA, FVC and DLCO DLCO	14 percentage change Interval 2.0 to 29.0

Adverse Events

IV Cyclophosphamide (50 mg/kg)

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	IV Cyclophosphamide (50 mg/kg) n=6 participants at risk This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously over 1 hour daily for four consecutive days (200 mg/kg total) through a Hickman catheter . IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Infections and infestations Death	16.7% 1/6 • Number of events 1 • 12 months

Other adverse events

Other adverse events
Measure	IV Cyclophosphamide (50 mg/kg) n=6 participants at risk This is an open-labeled single arm study of Cyclophosphamide (50 mg/kg) administered intravenously over 1 hour daily for four consecutive days (200 mg/kg total) through a Hickman catheter . IV Cyclophosphamide: Cyclophosphamide (50 mg/kg) intravenously daily for 4 consecutive days (total 200 mg/kg) followed by granulocyte colony-stimulating factor (5 µg/kg/day)
Immune system disorders neutropenic fever	83.3% 5/6 • Number of events 5 • 12 months
Gastrointestinal disorders nausea/vomitting	50.0% 3/6 • Number of events 3 • 12 months
Gastrointestinal disorders diarrhea	50.0% 3/6 • Number of events 3 • 12 months
Vascular disorders oedema	50.0% 3/6 • Number of events 3 • 12 months
Respiratory, thoracic and mediastinal disorders pneumonia	16.7% 1/6 • Number of events 1 • 12 months
Musculoskeletal and connective tissue disorders septic bursitis	16.7% 1/6 • Number of events 1 • 12 months
Skin and subcutaneous tissue disorders rash	33.3% 2/6 • Number of events 2 • 12 months
Reproductive system and breast disorders sexual dysfunction	50.0% 3/6 • Number of events 3 • 12 months
Reproductive system and breast disorders amenorrhea	16.7% 1/6 • Number of events 1 • 12 months
Skin and subcutaneous tissue disorders itch	33.3% 2/6 • Number of events 2 • 12 months
Musculoskeletal and connective tissue disorders myalgias	16.7% 1/6 • Number of events 1 • 12 months
Respiratory, thoracic and mediastinal disorders cough	16.7% 1/6 • Number of events 1 • 12 months
Eye disorders blurred vision	16.7% 1/6 • Number of events 1 • 12 months
Skin and subcutaneous tissue disorders alopecia	16.7% 1/6 • Number of events 1 • 12 months
Blood and lymphatic system disorders anaemia	16.7% 1/6 • Number of events 1 • 12 months

Additional Information

Gwen Leatherman,R.N.,M.S.

Johns Hopkins University

Phone: 410-550-8582

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place