Trial Outcomes & Findings for Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement (NCT NCT00333437)

Results Overview

compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.

Recruitment status

COMPLETED

Study phase

NA

Target enrollment

7 participants

Primary outcome timeframe

Baseline, 12 months

Results posted on

2013-11-13

Participant Flow

8 patients were screened at one clinical site (UCSF) in the United States

One patient screened for enrollment did not meet eligibility criteria

Participant milestones

Participant milestones
Measure	Single-group Study Treatment Study subjects receive standard mycophenolate dosing 2 grams/day.
Overall Study STARTED	7
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pulmonary Involvement in Scleroderma: A Clinical Study of the Safety and Efficacy of Mycophenolate Mofetil in Scleroderma Patients With Lung Involvement

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Age, Categorical <=18 years	0 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	7 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	52.57 years STANDARD_DEVIATION 7.52 • n=5 Participants
Sex: Female, Male Female	5 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Region of Enrollment United States	7 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 12 months

compare pre- and post-therapy FVC (post- minus pre-). Forced vital capacity (FVC) is the volume of air (liters) that can forcibly be blown out after full inspiration.

Outcome measures

Outcome measures
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Mean Change From Baseline in Forced Vital Capacity (FVC)	0.1786 Liters Standard Deviation 0.1613

SECONDARY outcome

Timeframe: Baseline, 12 months

BAL samples were colleected from the affected lobe (as determined by lung CT scans) before beginning and after completing study therapy.

Outcome measures

Outcome measures
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils) Mean change in neutrophil count	-3 Cells/uL Standard Deviation 8.1
Mean Change in Bronchoalveolar Lavage (BAL) Components (Neutrophils, Eosinophils) Mean change in eosinophil count	-6.3 Cells/uL Standard Deviation 16.4

SECONDARY outcome

Timeframe: Baseline, 12 months

Participants reported frequency of shortness of breath experienced with exertion

Outcome measures

Outcome measures
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Change in Shortness of Breath (Self-reported) Patient-reported less shortness of breath	6 participants
Change in Shortness of Breath (Self-reported) Patient-reported no change in shortness of breath	1 participants
Change in Shortness of Breath (Self-reported) Patient-reported increased shortness of breath	0 participants

SECONDARY outcome

Timeframe: 12 months

Comparison of 6-minute walk distance before beginning and after completing study therapy

Outcome measures

Outcome measures
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Mean Change in Six Minute Walk Distance	264.3 Feet Standard Deviation 194.7

SECONDARY outcome

Timeframe: 12 months

DLCO was measured before beginning and after completion of study therapy

Outcome measures

Outcome measures
Measure	Treatment n=7 Participants Study subjects receive standard mycophenolate dosing.
Mean Change in Diffusion Capacity of the Lung for Carbon Monoxide (DLCO)	1.86 Liters Standard Deviation 1.50

Adverse Events

Treatment

Serious events: 1 serious events

Other events: 1 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	Treatment n=7 participants at risk Study subjects receive standard mycophenolate dosing.
Blood and lymphatic system disorders Leukopenia	14.3% 1/7 • Number of events 7 • 12 months Appropriate safety and serological studies were obtained prior to starting study medication and on a regular basis throughout the study. Patients were asked to keep a diary of significant medical events occurring between visits.

Other adverse events

Other adverse events
Measure	Treatment n=7 participants at risk Study subjects receive standard mycophenolate dosing.
Gastrointestinal disorders Gastrointestinal distress	14.3% 1/7 • Number of events 1 • 12 months Appropriate safety and serological studies were obtained prior to starting study medication and on a regular basis throughout the study. Patients were asked to keep a diary of significant medical events occurring between visits.

Additional Information

Jeffrey A. Golden, MD

University of California San Francisco

Phone: 415-353-2935

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place