Trial Outcomes & Findings for Pilot Study of Imatinib Mesylate to Treat Nephrogenic Systemic Fibrosis (NCT NCT00677092)

Results Overview

The modified Rodnan Skin Score is the accepted clinical measure of scleroderma skin activity. The investigator assessed the thickening of the skin using the modified Rodnan Skin Score through simple palpation on 17 different skin sites in the fingers, hands, forearms, arms, feet, legs, and thighs (bilaterally) and face, chest, and abdomen (singly). Skin thickness was assessed on a scale of 0 to 3; 0 representing normal skin and 3 being severe thickening. The sum of the individual scores can range from 0 (normal) to 51 (severe thickening in all 17 areas). Percentage change is calculated as the Month 4 Score - Baseline Score/Baseline Score \* 100. A negative percentage change indicates improvement.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Baseline and Month 4

Results posted on

2017-05-19

Participant Flow

Participant milestones

Participant milestones
Measure	Imatinib Mesylate Treatment Imatinib mesylate 400 mg orally once daily for 4 months. Dosage was reduced to 200 mg if participant developed gastrointestinal intolerance or alopecia.
Overall Study STARTED	12
Overall Study Received Study Treatment	11
Overall Study COMPLETED	6
Overall Study NOT COMPLETED	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Imatinib Mesylate Treatment Imatinib mesylate 400 mg orally once daily for 4 months. Dosage was reduced to 200 mg if participant developed gastrointestinal intolerance or alopecia.
Overall Study Adverse Event	1
Overall Study Lost to Follow-up	1
Overall Study Withdrawal by Subject	3
Overall Study Death Prior to Receiving Treatment	1

Baseline Characteristics

Pilot Study of Imatinib Mesylate to Treat Nephrogenic Systemic Fibrosis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Imatinib Mesylate Treatment n=10 Participants Imatinib mesylate 400 mg orally once daily for 4 months. Dosage was reduced to 200 mg if the participant developed gastrointestinal intolerance or alopecia.
Age, Continuous	61.7 years STANDARD_DEVIATION 14.2 • n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	7 Participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline and Month 4

Population: All enrolled participants with Baseline and Month 4 data available for analysis.

The modified Rodnan Skin Score is the accepted clinical measure of scleroderma skin activity. The investigator assessed the thickening of the skin using the modified Rodnan Skin Score through simple palpation on 17 different skin sites in the fingers, hands, forearms, arms, feet, legs, and thighs (bilaterally) and face, chest, and abdomen (singly). Skin thickness was assessed on a scale of 0 to 3; 0 representing normal skin and 3 being severe thickening. The sum of the individual scores can range from 0 (normal) to 51 (severe thickening in all 17 areas). Percentage change is calculated as the Month 4 Score - Baseline Score/Baseline Score \* 100. A negative percentage change indicates improvement.

Outcome measures

Outcome measures
Measure	Imatinib Mesylate Treatment n=6 Participants Imatinib mesylate 400 milligrams (mg) orally once daily for 4 months. Dosage was reduced to 200 mg if the participant developed gastrointestinal intolerance or alopecia.
Percentage Change From Baseline in the Modified Rodnan Skin Score (mRSS) to Assess Skin Tethering	-24 percentage change in mRSS score Standard Deviation 14

Adverse Events

Imatinib Mesylate Treatment

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Jonathan Kay, MD

University of Massachusetts Medical School

Phone: 508-793-6936

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place