Trial Outcomes & Findings for Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Diffuse Cutaneous Systemic Sclerosis (NCT NCT04440592)

NCT ID: NCT04440592

Last Updated: 2025-12-30

Results Overview

The comparison between MT-7117 treatment group and placebo group will be performed. The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in mRSS, FVC % predicted, physician and patient global assessments, and HAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement.

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 76 participants
• Primary outcome timeframe: Week 52
• Results posted on: 2025-12-30

Participant Flow

Participant milestones

Measure	MT-7117 Oral tablet of MT-7117 once a day.	Placebo Oral tablet of placebo once a day.
Overall Study STARTED	38	38
Overall Study COMPLETED	28	36
Overall Study NOT COMPLETED	10	2

Reasons for withdrawal

Measure	MT-7117 Oral tablet of MT-7117 once a day.	Placebo Oral tablet of placebo once a day.
Overall Study Adverse Event	8	0
Overall Study Lost to Follow-up	1	0
Overall Study Withdrawal by Subject	1	1
Overall Study Other therapy needed	0	1

Baseline Characteristics

Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Diffuse Cutaneous Systemic Sclerosis

Baseline characteristics by cohort

Measure	MT-7117 n=38 Participants Oral tablet of MT-7117 once a day.	Placebo n=38 Participants Oral tablet of placebo once a day.	Total n=76 Participants Total of all reporting groups
Age, Continuous	49.2 years STANDARD_DEVIATION 12.5 • n=174 Participants	54.2 years STANDARD_DEVIATION 12.4 • n=166 Participants	51.7 years STANDARD_DEVIATION 12.6 • n=167 Participants
Sex: Female, Male Female	26 Participants n=174 Participants	30 Participants n=166 Participants	56 Participants n=167 Participants
Sex: Female, Male Male	12 Participants n=174 Participants	8 Participants n=166 Participants	20 Participants n=167 Participants
Race/Ethnicity, Customized Hispanic or Latino	7 Participants n=174 Participants	7 Participants n=166 Participants	14 Participants n=167 Participants
Race/Ethnicity, Customized Non-hispanic or Latino	31 Participants n=174 Participants	29 Participants n=166 Participants	60 Participants n=167 Participants
Race/Ethnicity, Customized Other	0 Participants n=174 Participants	2 Participants n=166 Participants	2 Participants n=167 Participants

PRIMARY outcome

Timeframe: Week 52

Population: Reason of discrepancy between Number analyzed" and "Overall Number of Participants Analyzed: The analysis was performed only in patients who have data at week 52.

The comparison between MT-7117 treatment group and placebo group will be performed. The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in mRSS, FVC % predicted, physician and patient global assessments, and HAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement.

Outcome measures

Measure	MT-7117 n=25 Participants Oral tablet of MT-7117 once a day.	Placebo n=33 Participants Oral tablet of placebo once a day.
The ACR CRISS Composite Score (0-1) at Week 52	0.8537 scores on ascale Interval 0.0 to 1.0	0.8502 scores on ascale Interval 0.0 to 1.0

SECONDARY outcome

Timeframe: 52 weeks

Population: The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT 7117 treatment for up to 52 weeks using the Health Assessment Questionnaire Disability Index (HAQ DI). The Health Assessment Questionnaire Disability Index (HAQ-DI) is a self-administered instrument consists of 20 questions referring to eight component sets consisting of dressing/grooming, arising, eating, walking, hygiene, reach, grip, and activities. Each item is scored on a 4-point scale from 0 to 3: 0 = Without any difficulty; 1 = With some difficulty; 2 = With much difficulty; 3 = Unable to do. Overall score was computed as the sum of component set scores and divided by the number of component sets answered. This outcome measure represents the change in mean score from baseline. A negative change from baseline indicates improvement.

Outcome measures

Measure	MT-7117 n=33 Participants Oral tablet of MT-7117 once a day.	Placebo n=38 Participants Oral tablet of placebo once a day.
Change in Health Assessment Questionnaire Disability Index (HAQ-DI) From Baseline up to Week 52 Change from baseline (BL) at Week 16	-0.1439 score on a scale Standard Deviation 0.3803	-0.0395 score on a scale Standard Deviation 0.3900
Change in Health Assessment Questionnaire Disability Index (HAQ-DI) From Baseline up to Week 52 Change from BL at Week 26	-0.0847 score on a scale Standard Deviation 0.3360	-0.0691 score on a scale Standard Deviation 0.4481
Change in Health Assessment Questionnaire Disability Index (HAQ-DI) From Baseline up to Week 52 Change from BL at Week 39	-0.0927 score on a scale Standard Deviation 0.3178	-0.0439 score on a scale Standard Deviation 0.5147
Change in Health Assessment Questionnaire Disability Index (HAQ-DI) From Baseline up to Week 52 Change from BL at Week 52	-0.1111 score on a scale Standard Deviation 0.4265	-0.0208 score on a scale Standard Deviation 0.5102

SECONDARY outcome

Timeframe: 52 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT-7117 treatment for up to 52 weeks on pulmonary function as measured by percent predicted forced vital capacity (%pFVC)

Outcome measures

Measure	MT-7117 n=30 Participants Oral tablet of MT-7117 once a day.	Placebo n=35 Participants Oral tablet of placebo once a day.
Change in Percent Predicted Forced Vital Capacity (%pFVC) From Baseline up to Week 52 Change from BL at Week 16	0.754 %pFVC Standard Deviation 4.938	-1.058 %pFVC Standard Deviation 5.749
Change in Percent Predicted Forced Vital Capacity (%pFVC) From Baseline up to Week 52 Change from BL at Week 26	0.904 %pFVC Standard Deviation 5.669	-0.691 %pFVC Standard Deviation 5.847
Change in Percent Predicted Forced Vital Capacity (%pFVC) From Baseline up to Week 52 Change from BL at Week 39	-1.201 %pFVC Standard Deviation 6.293	-1.457 %pFVC Standard Deviation 5.600
Change in Percent Predicted Forced Vital Capacity (%pFVC) From Baseline up to Week 52 Change from BL at Week 52	0.048 %pFVC Standard Deviation 6.917	-2.906 %pFVC Standard Deviation 5.101

SECONDARY outcome

Timeframe: 52 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using the Patient Global Assessment

Outcome measures

Measure	MT-7117 n=34 Participants Oral tablet of MT-7117 once a day.	Placebo n=38 Participants Oral tablet of placebo once a day.
Change in Patient Global Assessment From Baseline up to Week 52 Change from BL at Week 16	-0.8 score on a scale Standard Deviation 1.8	-0.3 score on a scale Standard Deviation 1.9
Change in Patient Global Assessment From Baseline up to Week 52 Change from BL at Week 26	0.0 score on a scale Standard Deviation 2.5	-0.4 score on a scale Standard Deviation 2.6
Change in Patient Global Assessment From Baseline up to Week 52 Change from BL at Week 39	-0.7 score on a scale Standard Deviation 2.2	-0.5 score on a scale Standard Deviation 2.3
Change in Patient Global Assessment From Baseline up to Week 52 Change from BL at Week 52	-0.5 score on a scale Standard Deviation 2.3	-0.3 score on a scale Standard Deviation 2.5

SECONDARY outcome

Timeframe: 52 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using the Physician Global Assessment. The Physician Global Assessment is used to assess the physician's rating of overall health of the subject. Physicians rate the perceived health of the subject on an 11-point scale from 0 (excellent) to 10 (extremely poor).

Outcome measures

Measure	MT-7117 n=34 Participants Oral tablet of MT-7117 once a day.	Placebo n=38 Participants Oral tablet of placebo once a day.
Change in Physician Global Assessment From Baseline up to Week 52 Change from BL at Week 52	-2.0 score on a scale Standard Deviation 2.9	-1.7 score on a scale Standard Deviation 2.2
Change in Physician Global Assessment From Baseline up to Week 52 Change from BL at Week 26	-1.6 score on a scale Standard Deviation 2.1	-1.7 score on a scale Standard Deviation 2.1
Change in Physician Global Assessment From Baseline up to Week 52 Change from BL at Week 16	-1.6 score on a scale Standard Deviation 1.9	-1.7 score on a scale Standard Deviation 1.8
Change in Physician Global Assessment From Baseline up to Week 52 Change from BL at Week 39	-2.1 score on a scale Standard Deviation 2.5	-2.2 score on a scale Standard Deviation 2.3

SECONDARY outcome

Timeframe: 52 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using the modified Rodnan Skin Score (mRSS). mRSS evaluates a subject's skin thickness which will be assessed by palpation and rated using an mRSS that ranges from 0 (normal) to 3 (severe skin thickening) across 17 different body sites. The total score is the sum of the individual skin scores from all of these sites and ranges from 0 to 51 units.

Outcome measures

Measure	MT-7117 n=34 Participants Oral tablet of MT-7117 once a day.	Placebo n=38 Participants Oral tablet of placebo once a day.
Change in Modified Rodnan Skin Score (mRSS) From Baseline From Baseline up to Week 52 Change from BL at Week 16	-3.2 score on a scale Standard Deviation 4.7	-3.6 score on a scale Standard Deviation 3.9
Change in Modified Rodnan Skin Score (mRSS) From Baseline From Baseline up to Week 52 Change from BL at Week 26	-4.9 score on a scale Standard Deviation 5.5	-5.3 score on a scale Standard Deviation 6.9
Change in Modified Rodnan Skin Score (mRSS) From Baseline From Baseline up to Week 52 Change from BL at Week 39	-6.4 score on a scale Standard Deviation 7.0	-6.2 score on a scale Standard Deviation 7.5
Change in Modified Rodnan Skin Score (mRSS) From Baseline From Baseline up to Week 52 Change from BL at Week 52	-6.6 score on a scale Standard Deviation 8.2	-6.5 score on a scale Standard Deviation 7.9

SECONDARY outcome

Timeframe: 39 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT 7117 treatment for up to 39 weeks using the ACR CRISS Score. The ACR CRISS exponential algorithm determines the predicted probability of improvement from baseline, incorporating change in mRSS, FVC % predicted, physician and patient global assessments, and HAQ-DI. The outcome is a continuous variable between 0.0 and 1.0 (0 - 100%). A higher score indicates greater improvement.

Outcome measures

Measure	MT-7117 n=29 Participants Oral tablet of MT-7117 once a day.	Placebo n=35 Participants Oral tablet of placebo once a day.
ACR CRISS Score up to Week 39 Week 16	0.0349 scores on ascale Interval 0.0 to 1.0	0.1236 scores on ascale Interval 0.0 to 1.0
ACR CRISS Score up to Week 39 Week 26	0.2626 scores on ascale Interval 0.0 to 1.0	0.6232 scores on ascale Interval 0.0 to 1.0
ACR CRISS Score up to Week 39 Week 39	0.3890 scores on ascale Interval 0.0 to 1.0	0.5289 scores on ascale Interval 0.0 to 1.0

SECONDARY outcome

Timeframe: 52 weeks

Population: Reason of discrepancy between Number analyzed" of each row and "Overall Number of Participants Analyzed": The analysis was performed only in patients who have data at each week.

To evaluate the efficacy of MT-7117 treatment for up to 52 weeks using CRISS Score. Subjects with CRISS score is >=0.60 are considered improved, while subjects with CRISS score < 0.60 are considered not improved.

Outcome measures

Measure	MT-7117 n=29 Participants Oral tablet of MT-7117 once a day.	Placebo n=35 Participants Oral tablet of placebo once a day.
ACR CRISS Score Responder (CRISS>=0.6) From Baseline up to Week 52 Week 52	14 Participants	18 Participants
ACR CRISS Score Responder (CRISS>=0.6) From Baseline up to Week 52 Week 16	10 Participants	8 Participants
ACR CRISS Score Responder (CRISS>=0.6) From Baseline up to Week 52 Week 26	10 Participants	18 Participants
ACR CRISS Score Responder (CRISS>=0.6) From Baseline up to Week 52 Week 39	11 Participants	15 Participants

Adverse Events

MT-7117

Serious events: 4 serious events

Other events: 35 other events

Deaths: 0 deaths

Placebo

Serious events: 2 serious events

Other events: 28 other events

Deaths: 0 deaths

Serious adverse events

Measure	MT-7117 n=38 participants at risk Oral tablet of MT-7117 once a day.	Placebo n=38 participants at risk Oral tablet of placebo once a day.
Infections and infestations Pneumonia	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks
Investigations Alanine aminotransferase increased	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks
Investigations Aspartate aminotransferase increased	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks
Musculoskeletal and connective tissue disorders Systemic scleroderma	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Malignant melanoma in situ	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks
Reproductive system and breast disorders Uterine polyp	0.00% 0/38 • 60 weeks	2.6% 1/38 • 60 weeks
Respiratory, thoracic and mediastinal disorders Organising pneumonia	0.00% 0/38 • 60 weeks	2.6% 1/38 • 60 weeks
Skin and subcutaneous tissue disorders Skin ulcer	2.6% 1/38 • 60 weeks	0.00% 0/38 • 60 weeks

Other adverse events

Measure	MT-7117 n=38 participants at risk Oral tablet of MT-7117 once a day.	Placebo n=38 participants at risk Oral tablet of placebo once a day.
Skin and subcutaneous tissue disorders Skin hyperpigmentation	42.1% 16/38 • 60 weeks	2.6% 1/38 • 60 weeks
Skin and subcutaneous tissue disorders Skin ulcer	2.6% 1/38 • 60 weeks	5.3% 2/38 • 60 weeks
Skin and subcutaneous tissue disorders Solar lentigo	5.3% 2/38 • 60 weeks	2.6% 1/38 • 60 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Melanocytic naevus	31.6% 12/38 • 60 weeks	5.3% 2/38 • 60 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Seborrhoeic keratosis	5.3% 2/38 • 60 weeks	0.00% 0/38 • 60 weeks
Nervous system disorders Headache	5.3% 2/38 • 60 weeks	5.3% 2/38 • 60 weeks
Respiratory, thoracic and mediastinal disorders Cough	0.00% 0/38 • 60 weeks	7.9% 3/38 • 60 weeks
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Skin and subcutaneous tissue disorders Erythema	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Skin and subcutaneous tissue disorders Nail pigmentation	5.3% 2/38 • 60 weeks	0.00% 0/38 • 60 weeks
Skin and subcutaneous tissue disorders Pruritus	7.9% 3/38 • 60 weeks	5.3% 2/38 • 60 weeks
Skin and subcutaneous tissue disorders Skin discolouration	21.1% 8/38 • 60 weeks	0.00% 0/38 • 60 weeks
Eye disorders Cataract	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Gastrointestinal disorders Diarrhoea	26.3% 10/38 • 60 weeks	13.2% 5/38 • 60 weeks
Gastrointestinal disorders Dysphagia	5.3% 2/38 • 60 weeks	2.6% 1/38 • 60 weeks
Gastrointestinal disorders Nausea	18.4% 7/38 • 60 weeks	5.3% 2/38 • 60 weeks
Gastrointestinal disorders Vomiting	15.8% 6/38 • 60 weeks	5.3% 2/38 • 60 weeks
General disorders Fatigue	10.5% 4/38 • 60 weeks	7.9% 3/38 • 60 weeks
Infections and infestations Bronchitis	5.3% 2/38 • 60 weeks	2.6% 1/38 • 60 weeks
Infections and infestations COVID-19	10.5% 4/38 • 60 weeks	13.2% 5/38 • 60 weeks
Infections and infestations Gastroenteritis	2.6% 1/38 • 60 weeks	5.3% 2/38 • 60 weeks
Infections and infestations Lower respiratory tract infection	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Infections and infestations Nasopharyngitis	7.9% 3/38 • 60 weeks	13.2% 5/38 • 60 weeks
Infections and infestations Upper respiratory tract infection	5.3% 2/38 • 60 weeks	5.3% 2/38 • 60 weeks
Injury, poisoning and procedural complications Accidental overdose	0.00% 0/38 • 60 weeks	10.5% 4/38 • 60 weeks
Investigations Gamma-glutamyltransferase increased	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Metabolism and nutrition disorders Decreased appetite	5.3% 2/38 • 60 weeks	2.6% 1/38 • 60 weeks
Metabolism and nutrition disorders Hypercholesterolaemia	5.3% 2/38 • 60 weeks	0.00% 0/38 • 60 weeks
Musculoskeletal and connective tissue disorders Muscle spasms	5.3% 2/38 • 60 weeks	0.00% 0/38 • 60 weeks
Musculoskeletal and connective tissue disorders Systemic scleroderma	0.00% 0/38 • 60 weeks	5.3% 2/38 • 60 weeks
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Dysplastic naevus	5.3% 2/38 • 60 weeks	2.6% 1/38 • 60 weeks

Additional Information

Clinical Trials, Information Desk

Tanabe Pharma America, Inc.

Phone: Please email

Email: information.US@mb.tanabe-pharma.com

Results disclosure agreements

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

Restriction type: OTHER