Trial Outcomes & Findings for Efficacy and Safety Study of I10E in Treatment of Patients With CIDP (NCT NCT02293460)
NCT ID: NCT02293460
Last Updated: 2021-01-27
Results Overview
Responders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline. Adjusted INCAT disability score can vary from 0 (normal) to 9 (maximal disability). If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored. If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
44 participants
Primary outcome timeframe
24 weeks after first treament injection
Results posted on
2021-01-27
Participant Flow
Between 24 February 2015 and 22 March 2017, 59 subjects from 23 sites signed an informed consent.
59 subjects signed an informed consent in the pre-assigment period. Among them, 18 subjects were considered as a screening failure including 3 subjects re-screened and finally enrolled. Of 44 enrolled subjects, on subject was discontinued (consent withdrawn by subject) from the study before the first administration of study drug.
Participant milestones
| Measure |
Single Arm
This study included patients never previously treated with IgG and patients already treated with IgG but in clinical relapse following IgG therapy discontinuation.
|
|---|---|
|
Overall Study
STARTED
|
43
|
|
Overall Study
COMPLETED
|
37
|
|
Overall Study
NOT COMPLETED
|
6
|
Reasons for withdrawal
| Measure |
Single Arm
This study included patients never previously treated with IgG and patients already treated with IgG but in clinical relapse following IgG therapy discontinuation.
|
|---|---|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
Lack of Efficacy
|
2
|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Efficacy and Safety Study of I10E in Treatment of Patients With CIDP
Baseline characteristics by cohort
| Measure |
Total Treated Set
n=43 Participants
All subjects who received at least one infusion of I10E
|
|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
32 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
11 Participants
n=5 Participants
|
|
Age, Continuous
|
50.0 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
19 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
24 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
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
24 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
19 Participants
n=5 Participants
|
|
Region of Enrollment
Turkey
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Italy
|
16 participants
n=5 Participants
|
|
Region of Enrollment
United Kingdom
|
1 participants
n=5 Participants
|
|
Region of Enrollment
France
|
2 participants
n=5 Participants
|
|
Region of Enrollment
Tunisia
|
8 participants
n=5 Participants
|
|
Region of Enrollment
Spain
|
6 participants
n=5 Participants
|
|
Region of Enrollment
Poland
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 24 weeks after first treament injectionResponders were defined as patients with a decrease ≥1 point in the adjusted INCAT disability score compared to baseline. Adjusted INCAT disability score can vary from 0 (normal) to 9 (maximal disability). If a patient was treated with a not-allowed treatment during the study period, then all adjusted INCAT disability score measured after the intake of these not-allowed treatments were censored. If the score at EoS visit was missing, then the Last Observation Carried Forward (LOCF) approach was applied to replace this missing value.
Outcome measures
| Measure |
Full Analysis Set
n=42 Participants
All TTS subjects having an available assessment of the primary efficacy criteria.
|
|---|---|
|
Efficacy Endpoint: Responder Rate at End of Study
|
32 Participants
|
Adverse Events
Total Treated Set
Serious events: 7 serious events
Other events: 39 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Total Treated Set
n=43 participants at risk
All subjects who received at least one infusion of I10E
|
|---|---|
|
Injury, poisoning and procedural complications
Femur fracture
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Injury, poisoning and procedural complications
Synovial rupture
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Investigations
Fibrin D dimer increased
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Colorectal adenocarcinoma
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Blood and lymphatic system disorders
Neutropenia
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Immune system disorders
Anaphylactic reaction
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Nervous system disorders
Headache
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Nervous system disorders
Transient ischaemic attack
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Gastrointestinal disorders
Pancreatitis
|
2.3%
1/43 • Number of events 1 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
Other adverse events
| Measure |
Total Treated Set
n=43 participants at risk
All subjects who received at least one infusion of I10E
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
7.0%
3/43 • Number of events 3 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Blood and lymphatic system disorders
Anaemia
|
7.0%
3/43 • Number of events 3 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Nervous system disorders
Headache
|
46.5%
20/43 • Number of events 79 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Ear and labyrinth disorders
Vertigo
|
7.0%
3/43 • Number of events 5 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
General disorders
Pyrexia
|
18.6%
8/43 • Number of events 11 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
General disorders
Asthenia
|
9.3%
4/43 • Number of events 4 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
General disorders
Chills
|
7.0%
3/43 • Number of events 10 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
General disorders
Malaise
|
7.0%
3/43 • Number of events 5 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Gastrointestinal disorders
Nausea
|
11.6%
5/43 • Number of events 6 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.0%
3/43 • Number of events 4 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Skin and subcutaneous tissue disorders
Rash
|
7.0%
3/43 • Number of events 3 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
11.6%
5/43 • Number of events 5 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
9.3%
4/43 • Number of events 6 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Infections and infestations
Influenza
|
18.6%
8/43 • Number of events 10 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
|
Infections and infestations
Urinary tract infection
|
11.6%
5/43 • Number of events 5 • The safety was assessed by recording all AEs occurring during the study, i.e. after signature of the informed consent and until the end of the study recorded (week 24 after inclusion).
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place