Chronic Inflammatory Demyelinating Polyradiculoneuropathy
Conditions
Keywords
Peripheral neuropathy, Disimmune disease, Neurology chronic disease, Rare disease
Brief summary
Primary objective: To assess the efficacy of I10E administered at a reduced maintenance dose in sustaining CIDP response after an initial 6-month treatment in PRISM study. (I10E-1302). Secondary objective: To assess the safety of I10E in this patient population.
Interventions
DRUGI10E
Patients who met all eligibility criteria will receive 0.5 g/kg of IMP every 3 weeks during 45 weeks.
Sponsors
Laboratoire français de Fractionnement et de Biotechnologies
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Male or female patient aged 18 years or more. 2. Responder patient who have completed the last visit of PRISM I10E-1302 study defined as a patient with a decrease ≥1 point in the adjusted INCAT disability score between baseline and the end-of-study (EOS) visit of PRISM I10E-1302 study. 3. Covered by national healthcare insurance system as required by local regulations. 4. Written informed consent obtained prior to any study-related procedures.
Exclusion criteria
1. History of severe allergic reaction or serious adverse reaction to any Ig. 2. Known hypersensitivity to human Ig or to any of the excipients of I10E (glycine and polysorbate 80). 3. History of cardiac insufficiency (New York Heart Association (NYHA) III/IV), uncontrolled cardiac arrhythmia, unstable ischemic heart disease, or uncontrolled hypertension. 4. History of venous thromboembolic disease, myocardial infarction or cerebrovascular accident. 5. Risk factor for blood hyperviscosity such as cryoglobulinemia or haematological malignancy with monoclonal gammopathy. 6. Body mass index (BMI) ≥40 kg/m². 7. Glomerular filtration rate \<80 mL/min/1.73m² measured according to the Modified Diet Renal Disease (MDRD) calculation. 8. Any other ongoing disease that may cause chronic peripheral neuropathy, such as toxin exposure, dietary deficiency, uncontrolled diabetes, hyperthyroidism, cancer, systemic lupus erythematosus or other connective tissue diseases, infection with HIV, Hepatitis B Virus (HBV) or Hepatitis C Virus (HCV), Lyme disease, multiple myeloma, Waldenström's macroglobulinaemia, amyloidosis, and hereditary neuropathy. 9. Woman with positive results on a urine pregnancy test or breastfeeding woman or woman of childbearing potential without an effective contraception. 10. Any other serious medical condition that would interfere with the clinical assessment of CIDP or use of I10E or prevent the patient from complying with the protocol requirements. 11. Increasing dosage or introduction of a systemic corticosteroids therapy within the last 3 months prior to screening, at a dose higher than 10 mg daily prednisolone or equivalent. Topical corticosteroids are permitted. 12. Treatment within 12 months prior to screening with immunomodulatory or immunosuppressant agents (including but not limited to cyclophosphamide, cyclosporine, interferon-α, interferon-β1a, anti-CD20, alemtuzumab, aziathioprine, etanercept, mycophenolate mofetil and methotrexate) or haemopoetic stem cell transplantation. 13. Plasma exchange, blood products or derivatives administered within the last 3 months prior to screening. 14. Anticipated poor compliance of patient with study procedures.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Efficacy Endpoint : Responder Rate at End of Study (EOS) Visit | week 48 (End-of-Study) | 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. |
Countries
France, Italy, Spain, Tunisia, Turkey (Türkiye), United Kingdom
Participant flow
Recruitment details
Between 09 November 2015 and 23 June 2017, 20 subjects from 14 sites signed an informed consent.
Pre-assignment details
20 subjects signed an informed consent but only 19 subjects enrolled (1 screening failure).
Participants by arm
| Arm | Count |
|---|---|
| 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. | 19 |
| Total | 19 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | early termination of the study | 8 |
| Overall Study | Lack of Efficacy | 4 |
| Overall Study | Physician Decision | 1 |
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | I10E Arm |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 4 Participants |
| Age, Categorical Between 18 and 65 years | 15 Participants |
| Age, Continuous | 50.0 years |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 8 Participants |
| Race (NIH/OMB) White | 11 Participants |
| Region of Enrollment France | 2 participants |
| Region of Enrollment Italy | 4 participants |
| Region of Enrollment Poland | 3 participants |
| Region of Enrollment Spain | 2 participants |
| Region of Enrollment Tunisia | 6 participants |
| Region of Enrollment Turkey | 1 participants |
| Region of Enrollment United Kingdom | 1 participants |
| Sex: Female, Male Female | 7 Participants |
| Sex: Female, Male Male | 12 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 19 |
| other Total, other adverse events | 12 / 19 |
| serious Total, serious adverse events | 1 / 19 |
Outcome results
Primary
Efficacy Endpoint : Responder Rate at End of Study (EOS) Visit
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.
Time frame: week 48 (End-of-Study)
Population: Study prematurely stopped due to sponsor decision based on the relapse rate.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| I10E Arm | Efficacy Endpoint : Responder Rate at End of Study (EOS) Visit | 15 Participants |