Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 26 of 215 for:    Inflammatory Myopathies

Study of Long-term Safety, Efficacy Tolerability of BYM338 in Patients With Sporadic Inclusion Body Myositis (BYM338)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02250443
Recruitment Status : Completed
First Posted : September 26, 2014
Results First Posted : April 10, 2018
Last Update Posted : April 10, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Sporadic Inclusion Body Myositis (sIBM)
Intervention Drug: BYM338 (Bimagrumab)
Enrollment 10
Recruitment Details Due to lack of efficacy in patients with sIBM, the study was terminated early.
Pre-assignment Details  
Arm/Group Title BYM338
Hide Arm/Group Description BYM338 Group
Period Title: Overall Study
Started 10
Completed 0
Not Completed 10
Reason Not Completed
Administrative problems             7
Adverse Event             3
Arm/Group Title BYM338
Hide Arm/Group Description BYM338 Group
Overall Number of Baseline Participants 10
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 10 participants
70.1  (10.39)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 10 participants
Female
4
  40.0%
Male
6
  60.0%
1.Primary Outcome
Title Number of Participants With Adverse Events as a Measure of Safety and Tolerability
Hide Description Any Adverse Event was defined as occurrence of any symptom regardless of intensity grade, Serious Adverse Event (SAEs) assessed as medical occurrences that result in death, are life threatening, require hospitalization or prolongation of hospitalization or result in persistent or significant disability/incapacity
Time Frame Up to 29 month
Hide Outcome Measure Data
Hide Analysis Population Description
safety analysis set - included all patients that received at least one dose of study drug. No statistical analysis provided for Number of Participants Who Experienced Adverse Events (AEs) and Serious Adverse Events (SAEs) During this extension study
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Measure Type: Number
Unit of Measure: Participants
Death 0
Serious adverse events (SAE) 2
Adverse Events (AE) 10
2.Secondary Outcome
Title Changes From Baseline in Lean Body Mass (LBM) by Dual-Energy X-ray Absorptiometery (DXA)
Hide Description To assess the effect of multiple doses of BYM338 on lean body mass as measured by DXA in terms of change from baseline.
Time Frame Baseline, Day 1, 57, 113, 169, 365, 533, and day 729
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamics (PD) analysis set: Patients with evaluable PD parameter data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Percentage Change in LBM
Day 1 Number Analyzed 10 participants
0  (0)
Day 57 Number Analyzed 10 participants
4.292  (2.7480)
Day 113 Number Analyzed 10 participants
5.552  (3.6066)
Day 169 Number Analyzed 10 participants
7.463  (4.5687)
Day 365 Number Analyzed 10 participants
6.919  (2.9669)
Day 533 Number Analyzed 8 participants
6.885  (3.7894)
Day 729 Number Analyzed 1 participants
0.727 [1]   (NA)
[1]
NA- Not available, standard deviation is not evaluable when n=1
3.Secondary Outcome
Title Pharmacokinetics (PK) Parameter of Cmin From Multiple i.v. Dosing
Hide Description To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins & 4 hours post-dose on Day 1. Pre-dose only on each subsequent administration
Time Frame Day 29, 85, 169, 253, 337, 421, 505, 589, 673, 757, 1177
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: ng/mL
Day 29 (n=10) Number Analyzed 10 participants
13.4  (5.01)
Day 85 (n=10) Number Analyzed 10 participants
24.1  (13.1)
Day 169 (n=10) Number Analyzed 10 participants
26.3  (15.9)
Day 253 (n=9) Number Analyzed 9 participants
28.8  (12.1)
Day 337 (n=10) Number Analyzed 10 participants
24.4  (14.6)
Day 421 (n=9) Number Analyzed 9 participants
24.5  (11.5)
Day 505 (n=8) Number Analyzed 8 participants
28.3  (10.6)
Day 589 (n=6) Number Analyzed 6 participants
39.8  (30.2)
Day 673 (n=2) Number Analyzed 2 participants
20.5  (1.98)
Day 757 (n=1) Number Analyzed 1 participants
20.3 [1]   (NA)
Day 1177 (n=1) Number Analyzed 1 participants
31.4 [1]   (NA)
[1]
NA - Not available, standard deviation is not evaluable when n=1
4.Secondary Outcome
Title Changes From Baseline in Physical Function Reported by Patients
Hide Description Self-reported physical function was assessed by a newly developed patient reported outcome named sporadic inclusion body myositis (sIBM) functional assessment (sIFA). The sIFA consists of 11 items scored on an 11 point numerical rating scale from 0 (no difficulty) to 10 (unable to do) across 3 domains: upper body functioning, lower body functioning and general functioning. Participants completed the assessment where the recall period was the past week prior to completing the patient reported outcome (PRO). The total score on the sIFA scale ranges from 0 (minimum) to 110 (maximum). Higher values represent a worse outcome. A positive change from baseline indicates deterioration. Due to the no-signal this analysis was cancelled.
Time Frame Baseline, Week 104
Hide Outcome Measure Data
Hide Analysis Population Description
Due to the early study termination and the small sample size in this open-label trial, this PRO analysis was cancelled.
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 0
No data displayed because Outcome Measure has zero total analyzed.
5.Secondary Outcome
Title Changes From Baseline in Muscle Strength.
Hide Description Quadriceps muscle strength was measured, Quadriceps Quantitative Muscle Testing (QMT) by portable fixed dynamometry (PFD). A negative change from baseline indicates deterioration
Time Frame Baseline, Day 1, 113, 169, 365, 533, 729
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Newtons
Quadriceps score left side Day 1 Number Analyzed 9 participants
0  (0)
left side Day 113 Number Analyzed 9 participants
-5.80  (28.160)
left side Day 169 Number Analyzed 8 participants
-5.95  (26.587)
left side Day 365 Number Analyzed 9 participants
-9.51  (28.149)
left side Day 533 Number Analyzed 7 participants
-25.77  (21.321)
left side Day 729 Number Analyzed 1 participants
178.26 [1]   (NA)
Quadriceps score Right side Day 1 Number Analyzed 9 participants
0  (0)
Right side Day 113 Number Analyzed 10 participants
-4.83  (29.904)
Right side Day 169 Number Analyzed 9 participants
-22.81  (18.784)
Right side Day 365 Number Analyzed 9 participants
-11.63  (47.141)
Right side Day 533 Number Analyzed 7 participants
-31.00  (29.173)
Right side Day 729 Number Analyzed 1 participants
-57.66 [2]   (NA)
[1]
NA- Not available, standard deviation is not evaluable when n=1
[2]
NA - Not available, standard deviation is not evaluable when n=1
6.Secondary Outcome
Title Changes From Baseline in Muscle Function (Hand-grip and Pinch-grip Dynamometry)
Hide Description The effect of BYM338 on additional muscle function measures (hand-grip and pinch-grip dynamometry).
Time Frame Baseline,Day 1, 113, 169, 365, 533, 729
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Newtons
Left hand-grip day 1 Number Analyzed 10 participants
0  (0)
Left hand-grip day 113 Number Analyzed 10 participants
25.58  (37.544)
Left hand-grip day 169 Number Analyzed 10 participants
21.36  (43.815)
Left hand-grip day 365 Number Analyzed 10 participants
-1.42  (47.973)
Left hand-grip day 533 Number Analyzed 7 participants
2.78  (16.934)
Left hand-grip day 729 Number Analyzed 2 participants
8.95  (24.034)
Right hand-grip day 1 Number Analyzed 10 participants
0  (0)
Right hand-grip day 113 Number Analyzed 10 participants
99.59  (184.472)
Right hand-grip day 169 Number Analyzed 10 participants
109.05  (214.248)
Right hand-grip day 365 Number Analyzed 10 participants
77.46  (160.133)
Right hand-grip day 533 Number Analyzed 8 participants
53.99  (127.776)
Right hand-grip day 729 Number Analyzed 2 participants
163.66  (237.402)
Left hand pinch grip day 1 Number Analyzed 1 participants
0  (0)
Left hand pinch grip day 113 Number Analyzed 10 participants
7.82  (27.292)
Left hand pinch grip day 169 Number Analyzed 10 participants
-4.74  (20.210)
Left hand pinch grip day 365 Number Analyzed 10 participants
-7.30  (35.224)
Left hand pinch grip day 533 Number Analyzed 8 participants
-11.40  (21.204)
Left hand pinch grip day 729 Number Analyzed 2 participants
-8.41  (46.672)
Right hand pinch grip day 1 Number Analyzed 10 participants
0  (0)
Right hand pinch grip day 113 Number Analyzed 10 participants
-7.55  (26.792)
Right hand pinch grip day 169 Number Analyzed 10 participants
-9.35  (30.942)
Right hand pinch grip day 365 Number Analyzed 10 participants
0.59  (32.094)
Right hand pinch grip day 533 Number Analyzed 8 participants
-8.65  (21.491)
Right hand pinch grip day 729 Number Analyzed 2 participants
-35.40  (44.820)
7.Secondary Outcome
Title Changes From Baseline in Muscle Function 6 Minute Walking Distance
Hide Description The effect of BYM338 on additional muscle function measures (6 minute walking distance). The 6MWD test measured the distance (in meters) that a participant walked in a 6 minute timeframe. A positive change from baseline indicates improvement.
Time Frame Baseline,Day 1, 113, 169, 365, 533, 729
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Meters
Day 1 Number Analyzed 10 participants
0  (0)
Day 113 Number Analyzed 9 participants
-1.56  (13.685)
Day 169 Number Analyzed 9 participants
-7.41  (12.463)
Day 365 Number Analyzed 7 participants
-8.97  (15.763)
Day 533 Number Analyzed 6 participants
-16.99  (22.382)
Day 729 Number Analyzed 1 participants
-17.86 [1]   (NA)
[1]
NA - Not available, standard deviation is not evaluable when n=1
8.Secondary Outcome
Title Change From Baseline of Thigh Muscle Volume (TMV) by MRI Scan
Hide Description Thigh Muscle Volume (TMV) change was evaluated by a responder analysis. Patients whose loss of muscle TMV by MRI was equal or more than 2% at Week 8 and 16 were considered responders
Time Frame Baseline, Day 1, 57, 113
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacodynamics (PD) analysis set: Patients with available PD data and no protocol deviations with relevant impact on PD data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 10
Mean (Standard Deviation)
Unit of Measure: Percentage Change
Day 1 0  (0)
Day 57 4.14  (4.250)
Day 113 4.51  (6.300)
9.Secondary Outcome
Title Pharmacokinetics (PK) Parameter of Cmax
Hide Description To obtain pharmacokinetic data from multiple i.v. dosing of BYM338 in this patient population. Pre-dose, 30 mins & 4 hours post-dose on Day 1.
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 9
Mean (Standard Deviation)
Unit of Measure: ug/mL
278  (62.6)
10.Secondary Outcome
Title Time to Reach the Maximum Concentration After Drug Administration (Tmax)
Hide Description The time to reach the maximum concentration after drug administration
Time Frame Day 1
Hide Outcome Measure Data
Hide Analysis Population Description
Pharmacokinetics (PK) Analysis set: Patients with available PK data and no protocol deviations with relevant impact on PK data
Arm/Group Title BYM338
Hide Arm/Group Description:
BYM338 Group
Overall Number of Participants Analyzed 9
Median (Full Range)
Unit of Measure: hr
0.744
(0.648 to 4.73)
Time Frame period up to 104 weeks
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title BYM338 10mg/kg i.v.
Hide Arm/Group Description BYM338 10mg/kg i.v.
All-Cause Mortality
BYM338 10mg/kg i.v.
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
BYM338 10mg/kg i.v.
Affected / at Risk (%)
Total   2/10 (20.00%) 
Blood and lymphatic system disorders   
Anaemia  1  1/10 (10.00%) 
Cardiac disorders   
Myocardial infarction  1  1/10 (10.00%) 
Tachyarrhythmia  1  1/10 (10.00%) 
Gastrointestinal disorders   
Gastrointestinal haemorrhage  1  1/10 (10.00%) 
Haemorrhoidal haemorrhage  1  1/10 (10.00%) 
Metabolism and nutrition disorders   
Dehydration  1  1/10 (10.00%) 
Iron deficiency  1  1/10 (10.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Lung adenocarcinoma  1  1/10 (10.00%) 
Oesophageal carcinoma  1  1/10 (10.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.1)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
BYM338 10mg/kg i.v.
Affected / at Risk (%)
Total   10/10 (100.00%) 
Blood and lymphatic system disorders   
Lymphadenopathy  1  1/10 (10.00%) 
Cardiac disorders   
Atrial fibrillation  1  1/10 (10.00%) 
Myocardial infarction  1  1/10 (10.00%) 
Ear and labyrinth disorders   
Tinnitus  1  1/10 (10.00%) 
Gastrointestinal disorders   
Abdominal pain  1  1/10 (10.00%) 
Diarrhoea  1  6/10 (60.00%) 
Frequent bowel movements  1  1/10 (10.00%) 
Haemorrhoidal haemorrhage  1  1/10 (10.00%) 
Inguinal hernia  1  1/10 (10.00%) 
Nausea  1  2/10 (20.00%) 
Vomiting  1  1/10 (10.00%) 
General disorders   
Oedema peripheral  1  3/10 (30.00%) 
Peripheral swelling  1  1/10 (10.00%) 
Infections and infestations   
Cellulitis  1  1/10 (10.00%) 
Fungal skin infection  1  1/10 (10.00%) 
Influenza  1  1/10 (10.00%) 
Nasopharyngitis  1  1/10 (10.00%) 
Upper respiratory tract infection  1  1/10 (10.00%) 
Urinary tract infection  1  3/10 (30.00%) 
Injury, poisoning and procedural complications   
Avulsion fracture  1  1/10 (10.00%) 
Bone contusion  1  1/10 (10.00%) 
Concussion  1  1/10 (10.00%) 
Contusion  1  1/10 (10.00%) 
Corneal abrasion  1  1/10 (10.00%) 
Fall  1  9/10 (90.00%) 
Laceration  1  1/10 (10.00%) 
Ligament sprain  1  3/10 (30.00%) 
Limb injury  1  1/10 (10.00%) 
Scratch  1  1/10 (10.00%) 
Skin abrasion  1  5/10 (50.00%) 
Tibia fracture  1  1/10 (10.00%) 
Investigations   
Mammogram abnormal  1  1/10 (10.00%) 
Natural killer cell count increased  1  1/10 (10.00%) 
Vitamin D decreased  1  1/10 (10.00%) 
Weight decreased  1  1/10 (10.00%) 
Metabolism and nutrition disorders   
Abnormal loss of weight  1  1/10 (10.00%) 
Decreased appetite  1  2/10 (20.00%) 
Glucose tolerance impaired  1  1/10 (10.00%) 
Gout  1  2/10 (20.00%) 
Iron deficiency  1  1/10 (10.00%) 
Musculoskeletal and connective tissue disorders   
Arthralgia  1  3/10 (30.00%) 
Back pain  1  1/10 (10.00%) 
Bursitis  1  1/10 (10.00%) 
Muscle spasms  1  9/10 (90.00%) 
Muscular weakness  1  1/10 (10.00%) 
Musculoskeletal pain  1  2/10 (20.00%) 
Myalgia  1  1/10 (10.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)   
Basal cell carcinoma  1  1/10 (10.00%) 
Squamous cell carcinoma of skin  1  1/10 (10.00%) 
Nervous system disorders   
Dementia  1  1/10 (10.00%) 
Dizziness  1  1/10 (10.00%) 
Dysgeusia  1  1/10 (10.00%) 
Headache  1  2/10 (20.00%) 
Hypogeusia  1  1/10 (10.00%) 
Psychiatric disorders   
Depression  1  1/10 (10.00%) 
Insomnia  1  1/10 (10.00%) 
Renal and urinary disorders   
Nephrolithiasis  1  1/10 (10.00%) 
Urinary incontinence  1  1/10 (10.00%) 
Urinary retention  1  1/10 (10.00%) 
Reproductive system and breast disorders   
Breast hyperplasia  1  1/10 (10.00%) 
Cervical polyp  1  1/10 (10.00%) 
Respiratory, thoracic and mediastinal disorders   
Cough  1  1/10 (10.00%) 
Dysphonia  1  1/10 (10.00%) 
Productive cough  1  1/10 (10.00%) 
Upper-airway cough syndrome  1  1/10 (10.00%) 
Skin and subcutaneous tissue disorders   
Acne  1  5/10 (50.00%) 
Blister  1  1/10 (10.00%) 
Night sweats  1  1/10 (10.00%) 
Papule  1  1/10 (10.00%) 
Pruritus  1  1/10 (10.00%) 
Rash  1  3/10 (30.00%) 
Skin hypertrophy  1  1/10 (10.00%) 
Vascular disorders   
Thrombophlebitis superficial  1  1/10 (10.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (19.1)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The terms and conditions of Novartis' agreements with its investigators may vary. However, Novartis does not prohibit any investigator from publishing. Any publications from a single-site are postponed until the publication of the pooled data (i.e., data from all sites) in the clinical trial or disclosure of trial results in their entirety.
Results Point of Contact
Name/Title: Study Director
Organization: Novartis Pharmaceutical
Phone: 862-778-8300
Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT02250443     History of Changes
Other Study ID Numbers: CBYM338X2205E1
First Submitted: March 20, 2014
First Posted: September 26, 2014
Results First Submitted: August 9, 2017
Results First Posted: April 10, 2018
Last Update Posted: April 10, 2018