A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients with Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
- Conditions
- damaged myelin sheath of nerve fibersInflammation of peripheral nervous system10012303
- Registration Number
- NL-OMON54028
- Lead Sponsor
- Argenx BVBA
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 4
Patients are eligible to be included in the trial only if all of the following
criteria apply:
1. Ability to understand the requirements of the trial, provide written
informed consent, willingness and ability to comply with the trial protocol
procedures.
2. Male or female patient aged 18 years or older, at the time of signing the
informed consent.
3. Diagnosed with probable or definite CIDP according to criteria of the
European Federation of Neurological Societies/Peripheral Nerve Society
(EFNS/PNS, 2010),(11) progressing or relapsing forms.
4. CIDP Disease Activity Status (CDAS)(14) score >=2 at screening.
5. An INCAT score >=2, with a score of 2 exclusively from leg disability, at the
first run-in visit (RI-V1; for patients entering run-in) or Stage A baseline
(A-V1; for treatment-naïve patients with documented evidence for worsening on
the total adjusted INCAT disability score within 3 months prior to screening).
6. Fulfilling any of the following treatment conditions:
• Currently treated with pulsed corticosteroids, oral corticosteroids
equivalent to prednisolone/prednisone <=10 mg/day, and/or IVIg or SCIg, if this
treatment has been started within the last 5 years before screening, and the
patient is willing to discontinue this treatment at the first run-in visit
(RI-V1); OR
• Without previous treatment (treatment-naïve); OR
• Treatment with corticosteroids and/or IVIg or SCIg discontinued at least 6
months prior to screening.
Note: Patients not treated with monthly or daily corticosteroids, IVIg or SCIg
for at least 6 months prior to screening are considered as equal to treatment
naïve patients.
7. Women of childbearing potential who have a negative serum pregnancy test at
screening and a negative urine pregnancy test up to Stage A baseline (D1A).
8. Women of childbearing potential must use an acceptable method of
contraception from signing the ICF until the date of the last dose of IMP.
9. Non-sterilized male patients who are sexually active with a female partner
of childbearing potential must use a condom and his partner must use a highly
effective method of contraception from screening to 90 days after the last
administration of IMP. Male patients practicing true sexual abstinence (when
this is in line with the preferred and usual life style of the participant) can
be included. Sterilized male patients who have had vasectomy with documented
aspermia post-procedure can be included. In addition, male patients are not
allowed to donate sperm from screening to 90 days after the last administration
of IMP.
1. Pure sensory atypical CIDP.
2. Polyneuropathy of other causes, including the following:
-Multifocal motor neuropathy;
-Monoclonal gammopathy of uncertain significance with anti-myelinassociated
glycoprotein immunoglobulin M (IgM) antibodies;
-Hereditary demyelinating neuropathy;
-Polyneuropathy, organomegaly, endocrinopathy, monoclonal protein
and skin change syndromes;
-Lumbosacral radiculoplexus neuropathy;
-Polyneuropathy most likely due to diabetes mellitus;
-Polyneuropathy most likely due to systemic illnesses;
-Drug- or toxin-induced polyneuropathy.
3. Any other disease that could better explain the patient's signs and symptoms.
4. Any history of myelopathy or evidence of central demyelination.
5. Current or past history (within 12 months of screening) of alcohol, drug or
medication abuse.
6. Severe psychiatric disorder, history of suicide attempt, or current suicidal
ideation that in the opinion of the investigator could create undue risk to the
patient or could affect adherence with the trial protocol.
7. Patients with clinically significant active or chronic uncontrolled
bacterial, viral, or fungal infection at screening, including patients who test
positive for an active viral infection at screening with:
* Active Hepatitis B Virus (HBV): serologic panel test results indicative of an
active (acute or chronic) infection;
* Active Hepatitis C Virus (HCV)
* Human Immunodeficiency Virus (HIV) positive serology associated with an
Acquired Immune Deficiency Syndrome (AIDS)-defining condition or with a cluster
of differentiation 4 (CD4) count <=200 cells/mm3.
8. Total IgG level <6 g/L at screening.
9. Treatment with the following:
-Within 3 months (or 5 half-lives of the drug, whichever is longer) before
screening: plasma exchange or immunoadsorption, any concomitant Fccontaining
therapeutic agents or other biological, or any other investigational product;
-Within 6 months before screening: rituximab, alemtuzumab, any other
monoclonal antibody, cyclophosphamide, interferon, tumor necrosis factor-alpha
inhibitors, fingolimod, methotrexate, azathioprine, mycophenolate, any other
immunomodulating or immunosuppressive medications, and oral daily
corticosteroids >10 mg/day.
Note: Patients using IVIg, SCIg, pulsed corticosteroids, and oral daily
corticosteroids <=10 mg/day can be included.
-Patients who (intend to) use prohibited medications and therapies during the
trial.
10. Pregnant and lactating women and those intending to become pregnant during
the trial.
11. Patients with any other known autoimmune disease that, in the opinion of
the investigator, would interfere with an accurate assessment of clinical
symptoms of CIDP.
12. Patients who received a live-attenuated vaccine fewer than 28 days before
screening. Receiving an inactivated, sub-unit, polysaccharide, or conjugate
vaccine any time before screening is not exclusionary.
13. Patients who have a history of malignancy unless deemed cured by adequate
treatment with no evidence of recurrence for >=3 years before the first IMP
administration. Patients with the following cancer can be included anytime:
-Adequately treated basal cell or squamous cell skin cancer,
-Carcinoma in situ of the cervix,
-Carcinoma in situ of the breast, or
-Incidental histological finding of Prostate cancer
14. P
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Stage A<br /><br>Primary endpoint: Percentage of patients with confirmed ECI.<br /><br><br /><br>Stage B:<br /><br>Primary Endpoint :<br /><br>• Time to first confirmed adjusted INCAT deterioration compared to Stage B<br /><br>baseline.<br /><br>Note: Time to first confirmed adjusted INCAT deterioration is defined by the<br /><br>time from first dose of double-blind IMP to the first adjusted INCAT score<br /><br>increase of >=1 point compared to Stage B baseline, if the deterioration is<br /><br>confirmed at a consecutive visit 3-7 daysone week after fthe first adjusted<br /><br>INCAT score increase of >=1 point. For patients with an increase of 2 or more<br /><br>points on the adjusted INCAT score compared to Stage B baseline, no<br /><br>confirmation is required.</p><br>
- Secondary Outcome Measures
Name Time Method