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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 16 February 2021
Main ID:  NCT04281472
Date of registration: 20/02/2020
Prospective Registration: Yes
Primary sponsor: argenx
Public 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) ADHERE
Scientific 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)
Date of first enrolment: April 15, 2020
Target sample size: 400
Recruitment status: Recruiting
URL:  https://clinicaltrials.gov/show/NCT04281472
Study type:  Interventional
Study design:  Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).  
Phase:  Phase 2
Countries of recruitment
Belgium Bulgaria Denmark France Georgia Germany Hungary Israel
Japan Latvia Poland Russian Federation Serbia Spain Ukraine United States
Contacts
Name:     Antonio Guglietta, MD
Address: 
Telephone: +1 857-350-4834
Email: ClinicalTrials@argenx.com
Affiliation: 
Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Intervention(s)
Biological: efgartigimod PH20 SC in stage B
Other: placebo in stage B
Primary Outcome(s)
Stage A: Percentage of patients with confirmed evidence of clinical improvement(ECI) [Time Frame: Up to 12 weeks during the open-label stage A]
Stage B: Time to first adjusted INCAT deterioration compared to Stage B baseline [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Secondary Outcome(s)
Stage A: Change from Stage A baseline over time in adjusted INCAT score [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Change from Stage A baseline over time in I-RODS disability scores [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Change from Stage A baseline over time in mean grip strength [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Change from Stage A baseline over time in Medical Research Council (MRC) Sum score [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Change from Stage A baseline over time in TUG score [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Changes of serum IgG levels over time [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Exposure adjusted occurrence of treatment-emergent (serious) adverse events [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Incidence of clinically significant laboratory abnormalities [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20 [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Pre-dosing efgartigimod serum concentrations over time [Time Frame: Up to 12 weeks during the open-label stage A]
Stage A: Time to initial confirmed ECI [Time Frame: Up to 12 weeks during the open-label stage A]
Stage B: Change from Stage B baseline over time in 24-item I-RODS disability score [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Change from Stage B baseline over time in adjusted INCAT score [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Change from Stage B baseline over time in mean grip strength [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Change from Stage B baseline over time in MRC Sum score [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Change from Stage B baseline over time in TUG score [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Changes of serum IgG levels over time [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Exposure adjusted occurrence of treatment-emergent adverse events and serious adverse events [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Incidence of clinically significant laboratory abnormalities [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Percentage of patients with and titers of binding antibodies towards efgartigimod and/or rHuPH20 and the presence of neutralizing antibodies against efgartigimod and/or rHuPH20 [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Percentage of patients with improved functional level compared to Stage B baseline [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Pre-dosing efgartigimod serum concentrations over time [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Time to 10% decrease in the 24-item I-RODS [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Stage B: Time to CIDP disease progression [Time Frame: Up to 48 weeks during the randomized placebo-controlled stage B]
Secondary ID(s)
2019-003076-39
ARGX-113-1802
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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