Trial Outcomes & Findings for A Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Diffuse Systemic Sclerosis (Scleroderma) (NCT NCT00442611)

Results Overview

Modified Rodnan Skin Score measures skin thickness and is the sum of scores from 17 surface anatomic areas rated on a 0-3 scale (0=normal skin; 1=mild thickness; 2=moderate thickness; 3=severe thickness with inability to pinch the skin into a fold). Total modified Rodnan Skin Score ranges from 0 (best possible outcome) to 51 (worst possible outcome).

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

10 participants

Primary outcome timeframe

6 months

Results posted on

2017-10-31

Participant Flow

Participant milestones

Participant milestones
Measure	Abatacept Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Overall Study STARTED	7	3
Overall Study COMPLETED	6	3
Overall Study NOT COMPLETED	1	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Study to Evaluate the Safety and Efficacy of Abatacept in Patients With Diffuse Systemic Sclerosis (Scleroderma)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Abatacept n=7 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	Total n=10 Participants Total of all reporting groups
Age, Continuous	39.8 years STANDARD_DEVIATION 11.4 • n=5 Participants	48.6 years STANDARD_DEVIATION 13.9 • n=7 Participants	44.2 years STANDARD_DEVIATION 12.5 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants	3 Participants n=7 Participants	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants	3 participants n=7 Participants	10 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 months

Modified Rodnan Skin Score measures skin thickness and is the sum of scores from 17 surface anatomic areas rated on a 0-3 scale (0=normal skin; 1=mild thickness; 2=moderate thickness; 3=severe thickness with inability to pinch the skin into a fold). Total modified Rodnan Skin Score ranges from 0 (best possible outcome) to 51 (worst possible outcome).

Outcome measures

Outcome measures
Measure	Abatacept n=7 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Change in Modified Rodnan Skin Score	-8.6 MRSS score Standard Deviation 7.5	-2.3 MRSS score Standard Deviation 15

SECONDARY outcome

Timeframe: Baseline; Month 6

Outcome measures

Outcome measures
Measure	Abatacept n=7 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Oral Aperture at Baseline and Month 6 Right side baseline	31.1 mm Standard Deviation 6.51	21.0 mm Standard Deviation 4.32
Oral Aperture at Baseline and Month 6 Right side month 6	29.4 mm Standard Deviation 4.98	21.0 mm Standard Deviation 1.41
Oral Aperture at Baseline and Month 6 Left side baseline	30.9 mm Standard Deviation 7.18	20.0 mm Standard Deviation 2.45
Oral Aperture at Baseline and Month 6 Left side month 6	29.3 mm Standard Deviation 4.65	20.0 mm Standard Deviation 4.08

SECONDARY outcome

Timeframe: Baseline; Month 6

Outcome measures

Outcome measures
Measure	Abatacept n=7 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Hand Extension at Baseline and Month 6 Right hand baseline	177.7 mm Standard Deviation 28.11	98.3 mm Standard Deviation 20.95
Hand Extension at Baseline and Month 6 Right hand month 6	178.9 mm Standard Deviation 31.38	103.3 mm Standard Deviation 24.94
Hand Extension at Baseline and Month 6 Left hand baseline	183.7 mm Standard Deviation 23.96	110.7 mm Standard Deviation 35.98
Hand Extension at Baseline and Month 6 Left hand month 6	184.3 mm Standard Deviation 25.23	116.3 mm Standard Deviation 40.58

SECONDARY outcome

Timeframe: Baseline; Month 6

Outcome measures

Outcome measures
Measure	Abatacept n=7 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Digital Ulcerations at Baseline and Month 6 Right hand baseline	0.3 ulcers Standard Deviation 0.70	0.7 ulcers Standard Deviation 0.47
Digital Ulcerations at Baseline and Month 6 Right hand month 6	0.1 ulcers Standard Deviation 0.35	1.3 ulcers Standard Deviation 0.47
Digital Ulcerations at Baseline and Month 6 Left hand baseline	0.0 ulcers Standard Deviation 0.00	0.7 ulcers Standard Deviation 0.47
Digital Ulcerations at Baseline and Month 6 Left hand month 6	0.0 ulcers Standard Deviation 0.00	1.0 ulcers Standard Deviation 0.82

SECONDARY outcome

Timeframe: 6 months

Population: Participants with available data were analyzed.

FVC (Forced Vital Capacity) is the amount of air that can be forcibly exhaled from the lungs after taking the deepest possible breath. DLCO (Diffusing capacity of the lung for carbon monoxide) is the extent to which oxygen passes from the lungs to the blood.

Outcome measures

Outcome measures
Measure	Abatacept n=6 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Change in Pulmonary Function Tests FVC % Predicted	1.3 % Predicted Standard Deviation 8.5	.3 % Predicted Standard Deviation 8.5
Change in Pulmonary Function Tests DLCO % Predicted	2.0 % Predicted Standard Deviation 6.3	-7.4 % Predicted Standard Deviation 10.7

SECONDARY outcome

Timeframe: 6 months

Population: Participants with available data were analyzed.

The HAQ Disability Index (HAQ-DI) includes 20 items in 8 functional domains (dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities) assessing the patient's usual abilities in the past seven days. Each item is scored on a 0-3 scale (0=without any difficulty; 1=with some difficulty; 2=with much difficulty; 3=unable to do). The use of assistive devices for any domain increases the domain score by 1 point to a maximum of 3. The overall score is calculated by summing the highest item score in each of the domains and dividing the sum by 8, with an overall score of 0 indicating no disability, and a score of 3 indicating severe disability. The time points compared were 6 months to baseline (6 months minus baseline).

Outcome measures

Outcome measures
Measure	Abatacept n=6 Participants Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 Participants Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Change in Scleroderma Health Assessment Questionnaire	-0.04 HAQ-DI score Standard Deviation .24	.25 HAQ-DI score Standard Deviation .25

Adverse Events

Abatacept

Serious events: 1 serious events

Other events: 6 other events

Deaths: 0 deaths

IV Fluid

Serious events: 0 serious events

Other events: 3 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Abatacept n=7 participants at risk Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 participants at risk Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Vascular disorders Infection	14.3% 1/7 • Number of events 1 • 6 months	0.00% 0/3 • 6 months

Other adverse events

Other adverse events
Measure	Abatacept n=7 participants at risk Abatacept (dosed based upon weight) administered intravenously (IV) on days 1, 15, 30 and monthly thereafter for a total of 7 doses.	IV Fluid n=3 participants at risk Placebo to match abatacept (IV fluid) administered on days 1, 15, 30 and monthly thereafter for a total of 7 doses.
Respiratory, thoracic and mediastinal disorders infection	28.6% 2/7 • Number of events 2 • 6 months	33.3% 1/3 • Number of events 1 • 6 months
Skin and subcutaneous tissue disorders pruritus	28.6% 2/7 • Number of events 2 • 6 months	0.00% 0/3 • 6 months
Vascular disorders lower extremity edema	0.00% 0/7 • 6 months	33.3% 1/3 • Number of events 1 • 6 months
General disorders headache	14.3% 1/7 • Number of events 1 • 6 months	0.00% 0/3 • 6 months
General disorders dry mouth	0.00% 0/7 • 6 months	33.3% 1/3 • Number of events 1 • 6 months
Gastrointestinal disorders nausea	0.00% 0/1 • 6 months	33.3% 1/3 • Number of events 1 • 6 months
Gastrointestinal disorders fever	14.3% 1/7 • Number of events 1 • 6 months	0.00% 0/3 • 6 months

Additional Information

Dr. Lorinda Chung

Stanford University

Phone: (650)493-5000

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place