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High Dose Cyclophosphamide for Treatment of Scleroderma

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ClinicalTrials.gov Identifier: NCT00501995
Recruitment Status : Completed
First Posted : July 17, 2007
Results First Posted : July 2, 2014
Last Update Posted : June 14, 2017
Sponsor:
Information provided by (Responsible Party):
Fredrick M. Wigley, Johns Hopkins University

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Scleroderma
Intervention Drug: IV Cyclophosphamide
Enrollment 6
Recruitment Details  
Pre-assignment Details  
Arm/Group Title IV Cyclophosphamide (50 mg/kg)
Hide Arm/Group Description

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)

Period Title: Overall Study
Started 6
Completed 5
Not Completed 1
Arm/Group Title IV Cyclophosphamide (50 mg/kg)
Hide Arm/Group Description 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)
Overall Number of Baseline Participants 6
Hide Baseline Analysis Population Description
Patients with scleroderma were considered eligible for this open-label trial if they had a diagnosis of diffuse cutaneous scleroderma and evidence of clinically active disease.
Age, Continuous  
Mean (Full Range)
Unit of measure:  Years
Number Analyzed 6 participants
39
(19 to 60)
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
<=18 years
0
   0.0%
Between 18 and 65 years
6
 100.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 6 participants
Female
2
  33.3%
Male
4
  66.7%
Region of Enrollment   [1] 
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 6 participants
6
[1]
Measure Description: Number of participants
Number of Participants with diagnosis of scleroderma  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 6 participants
6
1.Primary Outcome
Title Improvement in the Modified Rodnan Skin Score.
Hide Description 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.
Time Frame 0 to 24 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Patient 4 died during the early phase of the study and longitudinal assessment of his skin score was not determined.
Arm/Group Title IV Cyclophosphamide (50 mg/kg)
Hide Arm/Group Description:

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 Number of Participants Analyzed 5
Mean (Full Range)
Unit of Measure: percent improvement from baseline
46.75
(31 to 60)
2.Secondary Outcome
Title Change in the HAQ-DI, PGA, FVC and DLCO
Hide Description 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.
Time Frame 0-24 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
The study group consisted of 4 men and 2 women aged 19-60 years of old.
Arm/Group Title IV Cyclophosphamide (50 mg/kg)
Hide Arm/Group Description:

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 Number of Participants Analyzed 6
Mean (Full Range)
Unit of Measure: percentage change
HAQ-DI score
79
(0 to 93)
PGA
71
(62 to 80)
FVC
0
(-7 to 5)
DLCO
14
(2 to 29)
Time Frame 12 months
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title IV Cyclophosphamide (50 mg/kg)
Hide Arm/Group Description

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)

All-Cause Mortality
IV Cyclophosphamide (50 mg/kg)
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
IV Cyclophosphamide (50 mg/kg)
Affected / at Risk (%) # Events
Total   1/6 (16.67%)    
Infections and infestations   
Death  [1]  1/6 (16.67%)  1
Indicates events were collected by systematic assessment
[1]
An infection leading to death within 8 weeks after cyclophosphamide therapy.
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
IV Cyclophosphamide (50 mg/kg)
Affected / at Risk (%) # Events
Total   6/6 (100.00%)    
Blood and lymphatic system disorders   
anaemia   1/6 (16.67%)  1
Eye disorders   
blurred vision   1/6 (16.67%)  1
Gastrointestinal disorders   
nausea/vomitting   3/6 (50.00%)  3
diarrhea   3/6 (50.00%)  3
Immune system disorders   
neutropenic fever   5/6 (83.33%)  5
Musculoskeletal and connective tissue disorders   
septic bursitis   1/6 (16.67%)  1
myalgias   1/6 (16.67%)  1
Reproductive system and breast disorders   
sexual dysfunction   3/6 (50.00%)  3
amenorrhea   1/6 (16.67%)  1
Respiratory, thoracic and mediastinal disorders   
pneumonia   1/6 (16.67%)  1
cough   1/6 (16.67%)  1
Skin and subcutaneous tissue disorders   
rash   2/6 (33.33%)  2
itch   2/6 (33.33%)  2
alopecia   1/6 (16.67%)  1
Vascular disorders   
oedema   3/6 (50.00%)  3
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Name/Title: Gwen Leatherman,R.N.,M.S.
Organization: Johns Hopkins University
Phone: 410-550-8582
Responsible Party: Fredrick M. Wigley, Johns Hopkins University
ClinicalTrials.gov Identifier: NCT00501995     History of Changes
Other Study ID Numbers: 00-11-17-02
First Submitted: July 13, 2007
First Posted: July 17, 2007
Results First Submitted: March 25, 2014
Results First Posted: July 2, 2014
Last Update Posted: June 14, 2017