Trial Outcomes & Findings for Nilotinib in the Treatment of Systemic Sclerosis (NCT NCT01166139)

NCT ID: NCT01166139

Last Updated: 2017-10-04

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE2
• Target enrollment: 10 participants
• Primary outcome timeframe: 6 Months and 12 months treatment
• Results posted on: 2017-10-04

Participant Flow

Participant milestones

Measure	Nilotinib 400 mg Twice Daily Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Overall Study STARTED	10
Overall Study COMPLETED	7
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Nilotinib in the Treatment of Systemic Sclerosis

Baseline characteristics by cohort

Measure	Nilotinib 400 mg Twice Daily n=10 Participants Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Age, Categorical <=18 years	1 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	8 Participants n=5 Participants
Age, Categorical >=65 years	1 Participants n=5 Participants
Age, Continuous	46 years n=5 Participants
Sex: Female, Male Female	8 Participants n=5 Participants
Sex: Female, Male Male	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	3 Participants n=5 Participants
Race (NIH/OMB) White	5 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants
Disease Duration, median (range)	0.7 years n=5 Participants
ANA-positive, n (%)	9 Participants n=5 Participants
Anti-Scl 70-positive, n (%)	3 Participants n=5 Participants
RNA polymerase III-positive, n (%)	5 Participants n=5 Participants
Modified Rodnan Skin Score (MRSS) at baseline	30.1 units on a scale STANDARD_DEVIATION 8.2 • n=5 Participants
Mean change in MRSS in 1 month prior to baseline	2.9 units on a scale STANDARD_DEVIATION 3.4 • n=5 Participants
Tendon friction rubs, n (%)	4 Participants n=5 Participants
Previous treatment, n (%)	0 Participants n=5 Participants
Methotrexate	2 Participants n=5 Participants
No immunosuppression	8 Participants n=5 Participants

PRIMARY outcome

Timeframe: 6 Months and 12 months treatment

Outcome measures

Measure	Nilotinib 400 mg Twice Daily n=7 Participants Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Number of Participants With Adverse Events as a Measure of Safety and Tolerability	71 Adverse Events

SECONDARY outcome

Timeframe: 6 Months of treatment

Efficacy of Nilotinib in patients with systemic sclerosis, as defined by an improvement in the Modified Rodnan Skin Score (MRSS) The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. The skin score is evaluated by manual palpation in each of these areas. The skin score is 0 for uninvolved skin, 1 for mild thickening, 2 for moderate thickening, and 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas where the minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease.

Outcome measures

Measure	Nilotinib 400 mg Twice Daily n=7 Participants Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Improvement of Modified Rodnan Skin Score Reported as a Mean (Units Equals Number of Points)	4.2 units on a scale

SECONDARY outcome

Timeframe: 12 months treatment

Improvement in Modified Rodnan skin score reported as a mean (units equals number of points).

The Modified Rodnan Skin Score (MRSS) measures dermal skin thickness through the examination of 17 body areas: fingers, hands, forearms, arms, feet, legs, and thighs (in pairs), and face, chest, and abdomen. In each of these areas, the skin score is evaluated by manual palpation. The skin score is 0 for uninvolved skin, 1 for mild thickening, 2 for moderate thickening, and 3 for severe thickening (hidebound skin). The total skin score is the sum of the skin scores of the individual areas. The minimum score is 0 and the maximum score is 51. A higher score indicates greater severity of disease.

Outcome measures

Measure	Nilotinib 400 mg Twice Daily n=7 Participants Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Efficacy of Nilotinib in Patients With Systemic Sclerosis, as Defined by an Improvement in the Modified Rodnan Skin Score	6.3 units on a scale

Adverse Events

Nilotinib 400 mg Twice Daily

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

Serious adverse events

Measure	Nilotinib 400 mg Twice Daily n=7 participants at risk Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Cardiac disorders Syncopal Episode	14.3% 1/7 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Vascular disorders Coronary Artery Bypass Graft (CABG) Surgery	14.3% 1/7 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.

Other adverse events

Measure	Nilotinib 400 mg Twice Daily n=7 participants at risk Nilotinib (Tasigna): Patients will be treated with Nilotinib 400 mg two times a day for 6 months and could continue treatment for at least 12 months.
Cardiac disorders Prolonged QTC	85.7% 6/7 • Number of events 9 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Investigations Increased AST	71.4% 5/7 • Number of events 6 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Investigations Elevated Total Bilirubin	71.4% 5/7 • Number of events 6 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Investigations Increased ALT	57.1% 4/7 • Number of events 5 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Blood and lymphatic system disorders Anemia	42.9% 3/7 • Number of events 3 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Metabolism and nutrition disorders Hyperglycemia	42.9% 3/7 • Number of events 3 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Nervous system disorders Headache	42.9% 3/7 • Number of events 3 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Ear and labyrinth disorders Nausea	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Gastrointestinal disorders Reflux	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Blood and lymphatic system disorders Elevated ESR	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Investigations Increased Lipase	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Investigations Increased Amylase	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Respiratory, thoracic and mediastinal disorders Upper Respiratory Infection	28.6% 2/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Metabolism and nutrition disorders Vomiting	14.3% 1/7 • Number of events 2 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Blood and lymphatic system disorders Decreased White Blood Cell Count	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Blood and lymphatic system disorders Increased White Blood Cell Count	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Cardiac disorders Coronary Artery Disease	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Nervous system disorders Syncope	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Cardiac disorders Hospitalization for CABG	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Infections and infestations Allergic Rhinitis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Eye disorders Cataracts	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Eye disorders Conjuctivitis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Eye disorders Eye Disorder	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Gastrointestinal disorders Diarrhea	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
General disorders Edema	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Infections and infestations Interim Bacterial Sinusitis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Infections and infestations Skin Infection	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Metabolism and nutrition disorders Low Inorganic Phosphorus	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Metabolism and nutrition disorders Vitamin D Deficiency	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Musculoskeletal and connective tissue disorders Ankle Arthiritis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Skin and subcutaneous tissue disorders Plantar Fascitis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Skin and subcutaneous tissue disorders Calcinosis	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Nervous system disorders Dizziness	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Nervous system disorders Concentration Impairment	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Psychiatric disorders Anxiety	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Reproductive system and breast disorders Erectile Dysfunction	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Skin and subcutaneous tissue disorders Rash	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.
Skin and subcutaneous tissue disorders Alopecia	14.3% 1/7 • Number of events 1 • Adverse Event events including Serious Adverse Events were observed during the 12-month treatment period.

Additional Information

Annel Fernandez

Hospital For Special Surgery

Phone: 212 774 2123

Email: fernandeza@hss.edu

Results disclosure agreements

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