A study to assess the long-term safety and efficacy of a subcutaneous formulation of efgartigimod in adults with chronic inflammatory demyelinating polyneuropathy (an autoimmune disorder that affects the peripheral nerves)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

18 Aug 2020 → ongoing

Decision date (initial)

2024-02-05

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Argenx BV

External identifiers

EU CT number

2023-507885-21-00

EudraCT number

2019-003107-35

ClinicalTrials.gov

NCT04280718

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Pharmacokinetic, Safety, Efficacy, Pharmacodynamic

To assess the long-term safety and tolerability of efgartigimod PH20 SC
(efgartigimod co-formulated with recombinant human hyaluronidase
PH20 [rHuPH20] for subcutaneous [SC] administration)

Secondary objectives 1

1. * To determine the long-term efficacy * To evaluate the immunogenicity (anti-drug antibodies [ADA]) of efgartigimod and rHuPH20 * To evaluate the pharmacokinetics (PK) of efgartigimod PH20 SC * To evaluate the pharmacodynamic (PD) effect of efgartigimod PH20 SC (ie, total immunoglobulin G [IgG] levels) * To evaluate additional patient-reported outcomes (PROs) (including patient-reported quality of life and satisfaction with treatment) * To explore self-administration of the treatment * To explore administration of the treatment by caregivers.

Conditions and MedDRA coding

Chronic Inflammatory Demyelinating Polyneuropathy

Study design 2 periods

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 1

1. 1. Ability to understand the requirements of the trial, provide written informed consent (including 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) of this trial. 2. Male or female patient with 1 of the following options: - Have completed the Week-48 visit of Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC; or - Have deteriorated during Stage B of the ARGX-113-1802 trial and are considered to be eligible for treatment with efgartigimod PH20 SC, or - Have been offered the participation in the OLE trial due to early termination of the ARGX-113-1802 trial (because sufficient events for the primary endpoint analysis of the that trial have been reached and it is stopped) and are considered to be eligible for treatment with efgartigimod PH20 SC treatment; or - Have completed the Week-48 visit of the previous cycle of the OLE trial and are considered to be eligible to continue with efgartigimod PH20 SC treatment. 3. Women of childbearing potential who have a negative urine pregnancy test at baseline before IMP administration. 4. Women of childbearing potential must use an acceptable method of contraception from signing the ICF until the date of the last dose of IMP

Exclusion criteria 1

1. 1. Week-48/ED visit in the ARGX-113-1802 trial or the Week-48 visit of the previous OLE participation occurred more than 14 days prior to SD1 of the OLE trial or the start of a new treatment cycle in the OLE trial and more than 21 days since the last dose of IMP. 2. Pregnant and lactating women and those intending to become pregnant during the trial. 3. Patients with clinical evidence of other significant serious disease or patients who underwent a recent or have a planned major surgery, or patients who (intend to) use prohibited medications and therapies during the trial, or any other reason which could confound the results of the trial or put the patient at undue risk.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Incidence of TEAEs and SAEs by system organ class (SOC) and preferred term (PT). TEAEs and SAEs are monitored throughout the entire study duration.
2. Incidence of clinically significant laboratory abnormalities. Blood sampling for laboratory analysis is taken at every study visit.

Secondary endpoints 9

1. Efficacy; Change from baseline over time in the following scores and measurements: − Adjusted INCAT score; − MRC Sum score; − 24-item I-RODS disability scores; − Mean grip strength assessed by Martin vigorimeter; − TUG score. Percentage of patients without clinical deterioration over time, defined by adjusted INCAT increase ≥1 point compared to baseline.
2. Immunogenicity; - Percentage of patients with and titers of binding antibodies (BAb) towards efgartigimod; and the presence of neutralizing antibodies (NAb) against efgartigimod.
3. Pharmacokinetics; - Efgartigimod serum concentrations (during the first 48-week treatment cycle [followed by a safety follow-up period, if applicable]).
4. Pharmacodynamics; - Changes from baseline over time of serum IgG levels (total). Efficacy, immunogenicity, pharmacokinetic and pharmacodynamic endpoints are assessed at every study visit
5. Additional Patient-reported Outcome; Change from baseline over time in: − Health-related quality-of-life questionnaire (EQ-5D-5L); − Brief Pain Inventory – Short Form (BPI-SF); − 9-item Treatment Satisfaction Questionnaire for Medication (TSQM-9); − Rasch-transformed-Fatigue Severity Scale (RT-FSS); − Hospital Anxiety and Depression Scale (HADS). Patient report outcomes are assessed at every study visit with the exception of visit 2 (week 4)
6. Percentage of patients performing self-administration over time.
7. Percentage of patients with treatment administered by caregiver over time.
8. This is mock end point to allow the upload of the full translation of endpoint number 5.
9. This is mock end point to allow the upload of the full translation of endpoint number 1.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Chief Scientific Officer Vice President Clinical Development

Public contact point

Organisation

Argenx

Contact name

Chief Scientific Officer Vice President Clinical Development

Third parties 16

Locations

11 EU/EEA countries · 40 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 138 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications