Efgartigimod alfa for Primary immune thrombocytopenia

Inclusion criteria

• {"criterion_text":"- Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits)."}
• {"criterion_text":"- Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period. Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator. The decision of enrolling the participant will be evaluated case by case."}
• {"criterion_text":"- Agree to use contraceptive measures consistent with local regulations and the following: Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP"}
• {"criterion_text":"- In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP or available through another program for patients with primary ITP), the following criteria apply:"}
• {"criterion_text":"- Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits)."}
• {"criterion_text":"- Participant has completed a 52-week treatment period."}

Exclusion criteria

• {"criterion_text":"- Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines)"}
• {"criterion_text":"- Use of any other investigational drug or participation in any other investigational trial"}
• {"criterion_text":"- Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients"}
• {"criterion_text":"- Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC"}

Primary endpoints

• {"endpoint_text":"- Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)","definition_or_measurement_approach":"Safety assessed by recording incidence, frequency and severity of AEs, AESIs and SAEs as reported during the study visits."}
• {"endpoint_text":"- Vital signs, electrocardiogram (ECG), and laboratory safety evaluations","definition_or_measurement_approach":"Measured by routine vital sign assessments, ECG recordings and laboratory safety tests performed at scheduled visits."}

Secondary endpoints

• {"endpoint_text":"- First 52-week treatment period only: Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10^9/L","definition_or_measurement_approach":"Calculated as percentage of weeks during treatment in which platelet count ≥50×10^9/L."}
• {"endpoint_text":"- First 52-week treatment period only: Proportion of participants with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period","definition_or_measurement_approach":"Proportion achieving platelet count ≥50×10^9/L on ≥4 occasions during the 52-week period."}
• {"endpoint_text":"- First 52-week treatment period only: Mean change from baseline in platelet count at each visit","definition_or_measurement_approach":"Mean change from baseline platelet count calculated at each scheduled visit."}
• {"endpoint_text":"- First 52-week treatment period only: For participants rolling over from the ARGX-113-2004 trial with a platelet count of <30×10^9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L","definition_or_measurement_approach":"Time from baseline to achieving two consecutive platelet counts ≥50×10^9/L for these participants."}
• {"endpoint_text":"- First 52-week treatment period only: The percentage of weeks in the trial with platelet counts of ≥ 30×10^9/L and ≥20×10^9/L above baseline","definition_or_measurement_approach":"Calculated percentage of weeks with platelet counts ≥30×10^9/L and ≥20×10^9/L above baseline."}
• {"endpoint_text":"- First 52-week treatment period only: In patients with a baseline platelet count of <15×10^9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10^9/L and ≥20×10^9/L above baseline","definition_or_measurement_approach":"Calculated percentage of weeks with platelet counts ≥30×10^9/L and ≥20×10^9/L above baseline for patients with baseline <15×10^9/L."}
• {"endpoint_text":"- First 52-week treatment period only: In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10^9/L for at least 4 of the 6 visits between week 19 and week 24","definition_or_measurement_approach":"Proportion achieving platelet counts ≥50×10^9/L in ≥4 of the 6 visits between weeks 19–24."}
• {"endpoint_text":"- First 52-week treatment period only: In participants with the first exposure to efgartigimod PH20 SC, the proportion of participants achieving platelet counts of ≥50×10^9/L for at least 6 of the 8 visits between week 17 and week 24","definition_or_measurement_approach":"Proportion achieving platelet counts ≥50×10^9/L in ≥6 of the 8 visits between weeks 17–24."}
• {"endpoint_text":"- First 52-week treatment period only: Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline","definition_or_measurement_approach":"Proportion with reduction in dose and/or frequency of concurrent ITP therapies versus baseline."}
• {"endpoint_text":"- First 52-week treatment period only: Rate of receipt of rescue therapy (rescue per participant per month)","definition_or_measurement_approach":"Rate calculated as number of rescue therapies per participant per month."}
• {"endpoint_text":"- First 52-week treatment period only: Incidence and severity of the World Health Organization (WHO)-classified bleeding events","definition_or_measurement_approach":"Incidence and severity classified according to WHO bleeding event categories."}
• {"endpoint_text":"- First 52-week treatment period only: Serum efgartigimod concentration observed predose (Ctrough)","definition_or_measurement_approach":"Predose serum concentration (Ctrough) measured at scheduled PK sampling times."}
• {"endpoint_text":"- First 52-week treatment period only: Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue], Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]), QoL (Short Form-36 [SF-36]), and ITP-Patient Assessment Questionnaire [ITP-PAQ] at planned visits","definition_or_measurement_approach":"Change from baseline in PRO and QoL instruments (FACIT-fatigue, FACT-Th6, SF-36, ITP-PAQ) at planned visits."}
• {"endpoint_text":"- First 52-week treatment period only: PD markers: total IgG","definition_or_measurement_approach":"Measurement of total IgG as pharmacodynamic marker at scheduled time points."}
• {"endpoint_text":"- First 52-week treatment period only: Number and percentage of participants who performed self-administration at home over time","definition_or_measurement_approach":"Count and percentage of participants performing self-administration at home over time."}
• {"endpoint_text":"- First 52-week treatment period only: Number and percentage of caregivers who administered the injection to the participant at home over time","definition_or_measurement_approach":"Count and percentage of caregivers administering injections at home over time."}
• {"endpoint_text":"- First 52-week treatment period only: Number of training visits needed for the participant or caregiver to become competent in the (self-)administration of efgartigimod PH20 SC","definition_or_measurement_approach":"Number of training visits required until participant or caregiver competency in (self-)administration achieved."}
• {"endpoint_text":"- First 52-week treatment period only: Number and percentage of self-administrations or caregiver-supported administrations at home First 52-week treatment period and additional 52-week treatment periods","definition_or_measurement_approach":"Number and percentage of self-administered or caregiver-supported administrations at home across treatment periods."}
• {"endpoint_text":"- First 52-week treatment period and additional 52-week treatment periods: Incidence and prevalence of antibodies to efgartigimod","definition_or_measurement_approach":"Incidence and prevalence of anti-drug antibodies measured across treatment periods."}
• {"endpoint_text":"- First 52-week treatment period and additional 52-week treatment periods: Titers of antibodies to efgartigimod","definition_or_measurement_approach":"Antibody titers to efgartigimod measured at scheduled immunogenicity assessments."}
• {"endpoint_text":"- First 52-week treatment period and additional 52-week treatment periods: Presence of neutralizing antibodies (NAb) against efgartigimod","definition_or_measurement_approach":"Assessment of presence of neutralizing antibodies against efgartigimod during immunogenicity testing."}

Bulgaria

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

12-01-2026

Processing Time Days

564

Number Of Sites

1

Number Of Participants

4

Greece

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

24-07-2024

Processing Time Days

27

Number Of Sites

1

Number Of Participants

4

Italy

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

25-08-2025

Processing Time Days

424

Number Of Sites

2

Number Of Participants

4

Portugal

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

19-07-2024

Processing Time Days

22

Number Of Sites

2

Number Of Participants

2

Romania

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

12-08-2024

Processing Time Days

46

Number Of Sites

2

Number Of Participants

5

Norway

Earliest CTIS Part Ii Submission Date

27-06-2024

Latest Decision Or Authorization Date

19-07-2024

Processing Time Days

22

Number Of Sites

1

Number Of Participants

1

Primary sponsor

Full Name

Argenx

Organisation Type

Pharmaceutical company

Country Of Registered Address

Belgium

Contract research organisations

Name

IQVIA Limited

Responsibilities

Pharmacovigilance

Name

Icon Public Limited Company

Name

PRA Hellas CRO A.E.

Responsibilities

Study start up, contract negotiation and monitoring activities in Greece

Third parties

• {"country":"United Kingdom","full_name":"Drug Development Solutions Limited","duties_or_roles":"Analysis of Pharmacokinetics and Anti-Drug Antibodies (ADA)","organisation_type":"Pharmaceutical company"}
• {"country":"Germany","full_name":"Azenta Germany GmbH","duties_or_roles":"Long-Term Storage of Pharmacokinetics- Pharmacodynamics (PK-PD), Anti-Drug Antibodies (ADA) Samples","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"IQVIA Limited","duties_or_roles":"Pharmacovigilance","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"SGS Belgium","duties_or_roles":"Medical Data Review, Secure Data Office","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"rHuPH20 immunogenicity testing","organisation_type":"Pharmaceutical company"}
• {"country":"Belgium","full_name":"Cerba Research","duties_or_roles":"Clinical chemistry, Clinical haematology, Serology/ endocrinology","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"rHuPH20 immunogenicity testing","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Icon Public Limited Company","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"Greece","full_name":"PRA Hellas CRO A.E.","duties_or_roles":"Study start up, contract negotiation and monitoring activities in Greece","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Pharmaceutical Product Development LLC","duties_or_roles":"Analysis of Total IgG","organisation_type":"Pharmaceutical company"}