Trial Outcomes & Findings for Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease (NCT NCT01559129)

Last Updated: 2023-12-01

Results Overview

An adverse event (AE) is any noxious, unintended, or untoward medical occurrence that may appear or worsen during the course of a study. A TEAE is any AE that began or worsened on or after the start of study drug through 28 days after the last dose. A treatment-related TEAE is a TEAE which was considered by the investigator to be related to study drug. The severity/intensity of AEs was assessed by the investigator as Mild (asymptomatic or mild symptoms; intervention not indicated), Moderate (symptoms cause moderate discomfort, intervention may be required), or Severe (symptoms cause severe discomfort/pain, requiring medical intervention, inability to perform daily activities). A serious AE is any AE that: - Resulted in death; - Was life-threatening; - Required inpatient hospitalization or prolongation of existing hospitalization - Resulted in persistent or significant disability/incapacity; - Was a congenital anomaly/birth defect; - Constituted an important medical event.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

23 participants

Primary outcome timeframe

From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.

Results posted on

2023-12-01

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.	Pomalidomide Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase.
Treatment Phase STARTED	12	11
Treatment Phase Received Treatment	12	10
Treatment Phase COMPLETED	7	4
Treatment Phase NOT COMPLETED	5	7
Open-label Extension Period STARTED	6	2
Open-label Extension Period COMPLETED	0	0
Open-label Extension Period NOT COMPLETED	6	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.	Pomalidomide Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase.
Treatment Phase Adverse Event	0	2
Treatment Phase Progressive Disease	1	1
Treatment Phase Lack of Efficacy	0	1
Treatment Phase Study Terminated by Sponsor	3	2
Treatment Phase Protocol Exclusion Criteria	1	0
Treatment Phase Did Not Receive Study Drug	0	1
Open-label Extension Period Adverse Event	1	0
Open-label Extension Period Study Terminated by Sponsor	5	2

Baseline Characteristics

Study of Pomalidomide (CC-4047) to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Effectiveness for Patients With Systemic Sclerosis With Interstitial Lung Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase. In the open-label extension phase participants transitioned to receive 1 mg pomalidomide orally once a day for up to 2 years.	Pomalidomide n=11 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase and continued to receive the same treatment for up to 2 years during the open-label extension phase.	Total n=23 Participants Total of all reporting groups
Age, Continuous	44.8 years STANDARD_DEVIATION 13.77 • n=5 Participants	48.9 years STANDARD_DEVIATION 9.84 • n=7 Participants	46.7 years STANDARD_DEVIATION 11.97 • n=5 Participants
Age, Customized < 65 years	10 Participants n=5 Participants	10 Participants n=7 Participants	20 Participants n=5 Participants
Age, Customized ≥ 65 years	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Sex: Female, Male Female	10 Participants n=5 Participants	10 Participants n=7 Participants	20 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants	1 Participants n=7 Participants	3 Participants n=5 Participants
Race/Ethnicity, Customized Asian	0 Participants n=5 Participants	2 Participants n=7 Participants	2 Participants n=5 Participants
Race/Ethnicity, Customized Black or African American	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islanders	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race/Ethnicity, Customized White	10 Participants n=5 Participants	8 Participants n=7 Participants	18 Participants n=5 Participants
Race/Ethnicity, Customized Other	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Body Mass Index (BMI)	30.64 kg/m^2 STANDARD_DEVIATION 5.392 • n=5 Participants	27.61 kg/m^2 STANDARD_DEVIATION 9.4 • n=7 Participants	29.19 kg/m^2 STANDARD_DEVIATION 7.556 • n=5 Participants

PRIMARY outcome

Timeframe: From the start of study drug to 28 days after last dose; Treatment Phase median duration of treatment was 358 and 320 days for Placebo and Pomalidomide; Extension phase median duration of treatment was 161 days and 194 days for Placebo and pomalidomide.

Population: All randomized participants who received at least one dose of the study treatment (either pomalidomide or placebo).

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide n=6 Participants In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide n=2 Participants Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Serious TEAE	1 participants	4 participants	0 participants	1 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Any TEAE	12 participants	9 participants	5 participants	2 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Drug-related TEAE	8 participants	6 participants	3 participants	1 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Severe TEAE	1 participants	4 participants	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Serious Drug-related TEAE	0 participants	1 participants	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE Leading to Drug Interruption	2 participants	1 participants	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE Leading to Drug Withdrawal	0 participants	4 participants	0 participants	0 participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) TEAE Leading to Death	0 participants	0 participants	0 participants	0 participants

PRIMARY outcome

Timeframe: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 48 and 52

Population: The Full Analysis Set (FAS) consisted of all randomized participants who received at least one dose of study drug. Participants with a Baseline value and a Week 52 were included in the analysis.

Forced vital capacity (FVC) is a pulmonary function test and is the volume of air in the lungs that can forcibly be blown out after a full inhalation. Percent predicted values are based comparison between the participant's measured value with expected FVC for someone of the same sex, age and height (reference value). For the analysis of FVC, the baseline value was defined as the average of all values between Screening and Baseline (inclusive), and the average of Weeks 48 and 52 was treated as the Week 52 value, to reduce the total data variability at the key time points.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=11 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=8 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in Percent Predicted Forced Vital Capacity (FVC) at Week 52	-2.8 percent predicted Standard Deviation 4.02	-5.2 percent predicted Standard Deviation 5.29	—	—

PRIMARY outcome

Timeframe: Baseline and Week 52 (or the Treatment Phase Early Termination visit)

Population: FAS participants with a Baseline value and a post-baseline value at Week 52 or Early Termination

Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\]), or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=11 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in the Modified Rodnan Skin Score (mRSS) at Week 52/Early Termination	-3.7 units on a scale Standard Deviation 6.99	-2.7 units on a scale Standard Deviation 5.66	—	—

PRIMARY outcome

Timeframe: Baseline and Week 52 (or Treatment Phase Early Termination visit)

Population: FAS participants with a Baseline value and a Week 52 value

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets gastrointestinal (GI) activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in University of California, Los Angeles, Scleroderma Clinical Trial Consortium Gastrointestinal Tract (UCLA SCTC GIT 2.0) Total Score at Week 52/Early Termination	0.00 units on a scale Standard Deviation 0.18	0.1 units on a scale Standard Deviation 0.29	—	—

SECONDARY outcome

Timeframe: Baseline (defined as the average of all values between Screening and Baseline) and Weeks 12, 24, 36, 64, 76, and 156

Population: FAS participants with a Baseline value and post-baseline value at the time point.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 12	-2.4 percent predicted Standard Deviation 3.07	-1.8 percent predicted Standard Deviation 3.39	—	—
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 24	-1.3 percent predicted Standard Deviation 3.33	2.3 percent predicted Standard Deviation 12.58	—	—
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 36	-2.6 percent predicted Standard Deviation 2.48	-4.4 percent predicted Standard Deviation 3.97	—	—
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 64	-7.0 percent predicted Standard Deviation 4.29	-6.4 percent predicted Standard Deviation 4.49	—	—
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 76	-8.7 percent predicted Standard Deviation 10.34	-5.2 percent predicted Standard Deviation NA Could not be calculated for one participant	—	—
Change From Baseline in Percent Predicted Forced Vital Capacity Over Time Week 156	-6.7 percent predicted Standard Deviation 6.19	-5.9 percent predicted Standard Deviation 2.31	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and a post-baseline value at each time point.

Improvement in skin thickening is associated with improved survival and may be useful as a surrogate measurement in clinical studies. The mRSS is an assessment tool which is used to evaluate the extent and severity of the skin thickening associated with systemic sclerosis (SSc). Seventeen body areas were evaluated on a 4-point scale (0 \[normal\], 1 \[mild\], 2 \[moderate\], or 3 \[severe\]). The total score, which is the sum of the 17 individual body assessments, can range from 0 to 51.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=11 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in Modified Rodnan Skin Score Over Time Week 12	-1.8 units on a scale Standard Deviation 3.94	-0.3 units on a scale Standard Deviation 1.83	—	—
Change From Baseline in Modified Rodnan Skin Score Over Time Week 24	-3.6 units on a scale Standard Deviation 5.68	-2.0 units on a scale Standard Deviation 3.38	—	—
Change From Baseline in Modified Rodnan Skin Score Over Time Week 64	-4.3 units on a scale Standard Deviation 8.36	-4.0 units on a scale Standard Deviation 2.83	—	—
Change From Baseline in Modified Rodnan Skin Score Over Time Week 76	-8.5 units on a scale Standard Deviation 7.78	-7.0 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in Modified Rodnan Skin Score Over Time Week 156/Early Termination	-3.7 units on a scale Standard Deviation 8.52	-4.5 units on a scale Standard Deviation 3.54	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health (except for Questions 15 and 31 which are scored as 0 (better health) or 1 (worse health). The total score is calculated as the average of the first 6 scale scores (excluding constipation) which captures overall burden (severity) of SSc-associated GIT. The overall score ranges from 0 to 3, where higher scores indicate more severe symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time Week 156/Early Termination	0.0 units on a scale Standard Deviation 0.14	0.0 units on a scale Standard Deviation 0.14	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time Week 12	0.2 units on a scale Standard Deviation 0.36	-0.1 units on a scale Standard Deviation 0.30	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time Week 24	0.1 units on a scale Standard Deviation 0.23	-0.1 units on a scale Standard Deviation 0.20	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time Week 64	0.0 units on a scale Standard Deviation 0.20	0.4 units on a scale Standard Deviation 0.01	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Total Score Over Time Week 76	-0.1 units on a scale Standard Deviation 0.01	0.5 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The reflux subscale score is calculated as the average of eight reflux-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 52/Early Termination	-0.1 units on a scale Standard Deviation 0.54	0.1 units on a scale Standard Deviation 0.38	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 64	-0.2 units on a scale Standard Deviation 0.14	0.2 units on a scale Standard Deviation 0.46	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 76	-0.5 units on a scale Standard Deviation 0.18	-0.2 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 12	0.1 units on a scale Standard Deviation 0.36	-0.1 units on a scale Standard Deviation 0.51	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 24	-0.1 units on a scale Standard Deviation 0.27	0.0 units on a scale Standard Deviation 0.47	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Reflux Subscale Score Over Time Week 156/Early Termination	0.0 units on a scale Standard Deviation 0.44	0.1 units on a scale Standard Deviation 0.28	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The distension/bloating subscale score is calculated as the average of four distension/bloating-related questions; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 12	0.4 units on a scale Standard Deviation 0.56	0.2 units on a scale Standard Deviation 1.21	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 24	0.0 units on a scale Standard Deviation 0.53	0.3 units on a scale Standard Deviation 0.80	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 52/Early Termination	0.0 units on a scale Standard Deviation 0.55	0.2 units on a scale Standard Deviation 1.07	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 64	-0.2 units on a scale Standard Deviation 0.70	1.0 units on a scale Standard Deviation 1.41	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 76	0.0 units on a scale Standard Deviation 0.00	3.0 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Distension/Bloating Subscale Score Over Time Week 156/Early Termination	-0.3 units on a scale Standard Deviation 0.30	0.9 units on a scale Standard Deviation 1.24	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The fecal soilage subscale score is calculated from one soilage question; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 12	0.0 units on a scale Standard Deviation 0.00	0.0 units on a scale Standard Deviation 0.00	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 156/Early Termination	0.2 units on a scale Standard Deviation 0.41	0.0 units on a scale Standard Deviation 0.00	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 24	0.2 units on a scale Standard Deviation 0.42	0.0 units on a scale Standard Deviation 0.00	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 52/Early Termination	0.0 units on a scale Standard Deviation 0.00	0.1 units on a scale Standard Deviation 0.33	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 64	0.2 units on a scale Standard Deviation 0.41	0.0 units on a scale Standard Deviation 0.00	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Fecal Soilage Subscale Score Over Time Week 76	0.0 units on a scale Standard Deviation 0.00	0.0 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The diarrhea subscale score is calculated as the average of one diarrhea question about the frequency of loose stools (on a scale from 0 \[none\] to 3 \[5-7 days/week\] and one question about the presence of watery stools (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2, where a higher score indicates more frequent symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 12	0.4 units on a scale Standard Deviation 0.57	-0.2 units on a scale Standard Deviation 0.65	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 24	0.1 units on a scale Standard Deviation 0.21	-0.3 units on a scale Standard Deviation 0.46	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 64	0.1 units on a scale Standard Deviation 0.38	0.5 units on a scale Standard Deviation 0.71	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 52/Early Termination	0.3 units on a scale Standard Deviation 0.34	0.0 units on a scale Standard Deviation 0.75	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 76	0.0 units on a scale Standard Deviation 0.00	-0.5 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Diarrhea Subscale Score Over Time Week 156/Early Termination	0.3 units on a scale Standard Deviation 0.42	-0.8 units on a scale Standard Deviation 0.35	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The social functioning subscale score is calculated as the average of six questions about how often symptoms interfered with social activities; the score ranges from 0 to 3, where higher scores indicate more frequent symptoms.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=11 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 24	0.1 units on a scale Standard Deviation 0.30	-0.1 units on a scale Standard Deviation 0.20	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 52/Early termination	0.0 units on a scale Standard Deviation 0.25	0.2 units on a scale Standard Deviation 0.45	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 64	0.0 units on a scale Standard Deviation 0.18	0.3 units on a scale Standard Deviation 0.35	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 76	0.2 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	0.0 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 156/Early Termination	0.0 units on a scale Standard Deviation 0.25	-0.2 units on a scale Standard Deviation 0.23	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Social Functioning Subscale Score Over Time Week 12	0.3 units on a scale Standard Deviation 0.49	0.0 units on a scale Standard Deviation 0.28	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The emotional well-being subscale score is calculated as the average of nine questions regarding the impact of bowel problems on emotional status; the score ranges from 0 to 3, where higher scores indicate more frequent problems.

Outcome measures

Outcome measures
Measure	Treatment Phase: Placebo n=12 Participants Participants received placebo orally once a day for 52 weeks during the treatment phase.	Treatment Phase: Pomalidomide n=10 Participants Participants received 1 mg pomalidomide orally once a day for 52 weeks during the treatment phase.	Extension Phase: Placebo/Pomalidomide In the open-label extension phase participants received 1 mg pomalidomide orally once a day for up to 2 years.	Extension Phase: Pomalidomide/Pomalidomide Participants received 1 mg pomalidomide orally once a day for up to 2 years during the open-label extension phase.
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 24	0.1 units on a scale Standard Deviation 0.16	-0.3 units on a scale Standard Deviation 0.56	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 52/Early Termination	0.1 units on a scale Standard Deviation 0.25	-0.1 units on a scale Standard Deviation 0.42	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 76	0.0 units on a scale Standard Deviation 0.00	0.3 units on a scale Standard Deviation NA NA = Could not be calculated for one participant	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 156/Early Termination	-0.1 units on a scale Standard Deviation 0.15	0.0 units on a scale Standard Deviation 0.00	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 12	0.2 units on a scale Standard Deviation 0.40	-0.4 units on a scale Standard Deviation 0.80	—	—
Change From Baseline in UCLA SCTC GIT 2.0 Emotional Well Being Subscale Score Over Time Week 64	0.0 units on a scale Standard Deviation 0.36	0.2 units on a scale Standard Deviation 0.00	—	—

SECONDARY outcome

Timeframe: Baseline and Weeks 12, 24, 52 (or at the Treatment Phase Early Termination visit), 64, 76, and 156 (or the Extension Phase Early Termination visit).

Population: FAS participants with a Baseline value and post-baseline value at each time point.

The UCLA SCTC GIT 2.0 is a 34-item, health-related quality of life self-administered evaluation tool, which targets GI activity and severity in patients with SSc. Individual scales include reflux, distention/bloating, fecal soilage, diarrhea, social functioning, emotional well-being and constipation. The items are scored on a scale from 0 to 3, where 0 indicates better health and 3 indicates worse health. The constipation subscale score is calculated as the average of three questions regarding the frequency of constipation (scored from 0 \[no days\] to 3 \[5-7 days/week\] and one question about the presence of stools becoming harder (scored as 0 \[No\] or 1 \[Yes\]); the score ranges from 0 to 2.5, where higher scores indicate more frequent symptoms.