A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

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Brief Title

A Study to Assess the Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod in Adults With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, an Autoimmune Disorder That Affects the Peripheral Nerves)

Official Title

A Phase 2 Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Brief Summary

      This is a Phase 2 study to evaluate the safety and efficacy of the subcutaneous formulation
      of efgartigimod in adults with CIDP.
    


Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Stage A: Percentage of patients with confirmed evidence of clinical improvement(ECI)

Secondary Outcome

 Stage A: Time to initial confirmed ECI

Condition

Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)

Intervention

efgartigimod PH20 SC in stage B

Study Arms / Comparison Groups

 efgartigimod PH20 SC
Description:  patients receiving efgartigimod PH20 SC in both stage A as stage B

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Biological

Estimated Enrollment

400

Start Date

April 15, 2020

Completion Date

August 15, 2023

Primary Completion Date

August 15, 2023

Eligibility Criteria

        Inclusion Criteria:

          1. Ability to understand the requirements of the trial, provide written informed consent
             (include consent for the use and disclosure of research-related health information),
             willingness and ability to comply with the trial protocol procedures (including
             required trial visits)

          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),
             progressing or relapsing forms.

          4. CIDP Disease Activity Status (CDAS) score ≥2 at screening.

          5. INCAT score ≥2 at the first run-in visit (for patients entering run-in) or stage A
             baseline (for treatment-naïve patients with documented evidence for worsening on the
             total adjusted INCAT disability score within 3 months prior to screening). Patients
             with an INCAT score of 2 at trial entry must have this score exclusively from the leg
             disability score; for patients with an INCAT score of ≥3 at trial entry, there are no
             specific requirements for arm or leg scores.

          6. Fulfilling any of the following treatment conditions:

               -  Currently treated with pulsed corticosteroids, oral corticosteroids equivalent to
                  prednisolone/prednisone ≤10mg/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; or

               -  Without previous treatment (treatment-naive); 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 pregnancy test at screening and a
             negative urine pregnancy test up to Stage A baseline.

          8. Women of childbearing potential must use a highly effective or acceptable method of
             contraception from screening to 90 days after the last administration of IMP

          9. Male patients agree not to donate sperm during the trial period and 90 days
             thereafter.

        Exclusion Criteria:

          1. Pure sensory atypical CIDP (EFNS/PNS definition).

          2. Polyneuropathy of other causes, including the following: Multifocal motor neuropathy;
             Monoclonal gammopathy of uncertain significance with anti-myelin associated,
             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 (such as severe depression, psychosis, bipolar 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): serology positive for HCV-Ab; 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
             Fc-containing 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.

         10. Pregnant and lactating women and those intending to become pregnant during the trial
             or within 90 days after last IMP administration.

         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 (TNM [tumor,
             nodes, and metastases classification] stage T1a or T1b).

         14. Patients who previously participated in a trial with efgartigimod and have received at
             least one administration of IMP.

         15. Patients with known medical history of hypersensitivity to any of the ingredients of
             IMP.

         16. Patients with clinical evidence of other significant serious disease or patients who
             underwent a recent or have a planned major surgery, or any other reason which could
             confound the results of the trial or put the patient at undue risk.
      

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

, +1 857-350-4834, [email protected]

Location Countries

Austria

Location Countries

Austria

Administrative Informations


NCT ID

NCT04281472

Organization ID

ARGX-113-1802

Secondary IDs

2019-003076-39

Responsible Party

Sponsor

Study Sponsor

argenx


Study Sponsor

, , 


Verification Date

May 2022