Trial Outcomes & Findings for A Study Using The Experimental Drug Called Imatinib (Gleevec) in Subjects With Systemic Sclerosis (NCT NCT00512902)

NCT ID: NCT00512902

Last Updated: 2014-10-27

Results Overview

Treatment-related adverse events requiring discontinuation.

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 20 participants
• Primary outcome timeframe: Baseline vs. Endpoint (1 year)
• Results posted on: 2014-10-27

Participant Flow

University setting convenience samples.

1-year, Phase I/IIa, Open-label trial

Participant milestones

Measure	Imatinib Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Overall Study STARTED	20
Overall Study COMPLETED	12
Overall Study NOT COMPLETED	8

Reasons for withdrawal

Measure	Imatinib Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Overall Study Adverse Event	7
Overall Study Lost to Follow-up	1

Baseline Characteristics

A Study Using The Experimental Drug Called Imatinib (Gleevec) in Subjects With Systemic Sclerosis

Baseline characteristics by cohort

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	20 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age, Continuous	46.1 years STANDARD_DEVIATION 14.2 • n=5 Participants
Sex: Female, Male Female	13 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants
Region of Enrollment United States	20 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline vs. Endpoint (1 year)

Treatment-related adverse events requiring discontinuation.

Outcome measures

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Treatment-related Adverse Events	5 participants

SECONDARY outcome

Timeframe: Baseline vs. Endpoint (1 year)

Measures the amount of air breathed out as a percent of predicted.

Outcome measures

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Change in FVC (Forced Vital Capacity)	1.74 Percent predicted Standard Deviation 2.1

SECONDARY outcome

Timeframe: Baseline vs. Endpoint (1 year)

No measures of dispersion was available for TLC as data were lost. This describes the total lung capacity as a percent of predicted.

Outcome measures

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Change in TLC (Total Lung Capacity)	4.17 Percent predicted Standard Deviation NA Data was lost.

SECONDARY outcome

Timeframe: Baseline vs. Endpoint (1 year)

DLCO (diffusing capacity or transfer factor of the lung for carbon monoxide (CO)) is the extent to which oxygen passes from the air sacs of the lungs into the blood. Commonly, it refers to the test used to determine this parameter.

Outcome measures

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Change in DLco	1.46 Percent predicted Standard Deviation NA Data was lost.

SECONDARY outcome

Timeframe: Baseline vs. Endpoint

No measures of dispersion was available as data were lost. The range of this measure is 0 to 51 and measures the extent of skin thickening with higher numbers representing thickening.

Outcome measures

Measure	Imatinib n=20 Participants Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Change in Modified Rodnan Skin Score (MRSS)	3.9 units on a scale Standard Deviation NA Data was lost.

Adverse Events

Imatinib

Serious events: 3 serious events

Other events: 10 other events

Deaths: 0 deaths

Serious adverse events

Measure	Imatinib n=20 participants at risk Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
Blood and lymphatic system disorders Anemia	5.0% 1/20 • Number of events 1
Respiratory, thoracic and mediastinal disorders Dyspnea	10.0% 2/20 • Number of events 2
General disorders Generalized Edema	10.0% 2/20 • Number of events 2

Other adverse events

Measure	Imatinib n=20 participants at risk Systemic Sclerosis patients administered oral 600 mg imatinib once per day for up to one year.
General disorders Fatigue	35.0% 7/20 • Number of events 7
Renal and urinary disorders Facial/lower extremity edema	45.0% 9/20 • Number of events 16
Gastrointestinal disorders Nausea/Vomiting	45.0% 9/20 • Number of events 9
Skin and subcutaneous tissue disorders Generalized rash	20.0% 4/20 • Number of events 4
Respiratory, thoracic and mediastinal disorders Upper respiratory tract infection	20.0% 4/20 • Number of events 4
Musculoskeletal and connective tissue disorders Elevated CPK	5.0% 1/20 • Number of events 1
Respiratory, thoracic and mediastinal disorders Mild hemoptysis	5.0% 1/20 • Number of events 1
Endocrine disorders Hypothyroidism	5.0% 1/20 • Number of events 1

Additional Information

Dr. Daniel Furst, Carl M Pearson Professor of Rheumatology

University of California at Los Angeles

Phone: 310-794-9504

Email: defurst@mednet.ucla.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place