Trial Outcomes & Findings for Efficacy and Safety Study of I10E in the Maintenance Treatment of Patients With CIDP: Extension of PRISM Study I10E-1302 (NCT NCT02317562)

Last Updated: 2021-04-20

Results Overview

Since the study was prematurely terminated and an important number of subjects early withdrawn, the responder rate is biased and consequently not interpretable. Responders were defined as subjects with either: No change or decrease in the adjusted INCAT disability score and without any change in CIDP treatment between baseline and EOS visit. OR An increase by 1 point in the adjusted INCAT disability score without requirement of any change in CIDP treatment between baseline and EOS visit.

Recruitment status

TERMINATED

Study phase

PHASE3

Target enrollment

19 participants

Primary outcome timeframe

week 48 (End-of-Study)

Results posted on

2021-04-20

Participant Flow

Between 09 November 2015 and 23 June 2017, 20 subjects from 14 sites signed an informed consent.

20 subjects signed an informed consent but only 19 subjects enrolled (1 screening failure).

Participant milestones

Participant milestones
Measure	I10E Arm Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Overall Study STARTED	19
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	14

Reasons for withdrawal

Reasons for withdrawal
Measure	I10E Arm Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Overall Study Physician Decision	1
Overall Study early termination of the study	8
Overall Study Lack of Efficacy	4
Overall Study Withdrawal by Subject	1

Baseline Characteristics

Efficacy and Safety Study of I10E in the Maintenance Treatment of Patients With CIDP: Extension of PRISM Study I10E-1302

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	I10E Arm n=19 Participants Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Age, Categorical <=18 years	0 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	15 Participants n=93 Participants
Age, Categorical >=65 years	4 Participants n=93 Participants
Age, Continuous	50.0 years n=93 Participants
Sex: Female, Male Female	7 Participants n=93 Participants
Sex: Female, Male Male	12 Participants n=93 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=93 Participants
Race (NIH/OMB) Asian	0 Participants n=93 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=93 Participants
Race (NIH/OMB) Black or African American	0 Participants n=93 Participants
Race (NIH/OMB) White	11 Participants n=93 Participants
Race (NIH/OMB) More than one race	0 Participants n=93 Participants
Race (NIH/OMB) Unknown or Not Reported	8 Participants n=93 Participants
Region of Enrollment Turkey	1 participants n=93 Participants
Region of Enrollment Italy	4 participants n=93 Participants
Region of Enrollment United Kingdom	1 participants n=93 Participants
Region of Enrollment France	2 participants n=93 Participants
Region of Enrollment Tunisia	6 participants n=93 Participants
Region of Enrollment Spain	2 participants n=93 Participants
Region of Enrollment Poland	3 participants n=93 Participants

PRIMARY outcome

Timeframe: week 48 (End-of-Study)

Population: Study prematurely stopped due to sponsor decision based on the relapse rate.

Outcome measures

Outcome measures
Measure	I10E Arm n=19 Participants Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Efficacy Endpoint : Responder Rate at End of Study (EOS) Visit	15 Participants

Adverse Events

I10E Arm

Serious events: 1 serious events

Other events: 12 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	I10E Arm n=19 participants at risk Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Renal and urinary disorders Urinary retention	5.3% 1/19 • 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. (up to 48 weeks after inclusion).

Other adverse events

Other adverse events
Measure	I10E Arm n=19 participants at risk Participation in the study was proposed to all subjects who completed and responded to treatment in PRISM study, satisfying in eligibility criteria and were willing to continue I10E at a reduced maintenance dose. Each subject was expected to receive 16 doses of I10E (study drug) at 0.5 g/kg over 1 to 2 days, every 3 weeks.
Vascular disorders Hypertension	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Vascular disorders Hypotension	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
General disorders Fatigue	10.5% 2/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
General disorders Chills	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
General disorders Injection site erythema	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
General disorders Injection site oedema	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
General disorders Pain	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
General disorders Pyrexia	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Psychiatric disorders Conversion disorder	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Psychiatric disorders Insomnia	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Injury, poisoning and procedural complications Ligament sprain	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Injury, poisoning and procedural complications Post traumatic pain	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Investigations Blood pressure increased	10.5% 2/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Investigations Blood lactate dehydrogenase	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Cardiac disorders Palpitations	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Blood and lymphatic system disorders Anaemia	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Respiratory, thoracic and mediastinal disorders Nasal congestion	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Nervous system disorders Headache	15.8% 3/19 • 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. (up to 48 weeks after inclusion).
Nervous system disorders Migraine	5.3% 1/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Nervous system disorders Neuralgia	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Nervous system disorders Sciatica	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Ear and labyrinth disorders Tinnitus	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Abdominal pain	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Diarrhoea	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Duodenal ulcer	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Gastritis	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Hiatus hernia	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Gastrointestinal disorders Vomiting	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Skin and subcutaneous tissue disorders Dry skin	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Skin and subcutaneous tissue disorders Eczema nummular	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Skin and subcutaneous tissue disorders Erythema	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Skin and subcutaneous tissue disorders Rash pruritic	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Pain in extremity	15.8% 3/19 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Arthralgia	10.5% 2/19 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Back pain	10.5% 2/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Neck pain	10.5% 2/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Muscle spasms	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Musculoskeletal pain	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Musculoskeletal and connective tissue disorders Tendonitis	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Conjunctivitis	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Gastroenteritis viral	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Infected bite	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Influenza	5.3% 1/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Infections and infestations Nasopharyngitis	5.3% 1/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).
Infections and infestations Tooth abscess	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Upper respiratory tract infection	5.3% 1/19 • 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. (up to 48 weeks after inclusion).
Infections and infestations Urinary tract infection	5.3% 1/19 • Number of events 2 • 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. (up to 48 weeks after inclusion).

Additional Information

Clinical Information Desk

LFB

Phone: 0033169827010

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place