A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia
- Conditions
- Primary Immune Thrombocytopenia
- Interventions
- Biological: efgartigimod PH20 SC
- Registration Number
- NCT04812925
- Lead Sponsor
- argenx
- Brief Summary
A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 173
- 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).
- 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 and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case.
3a. Agree to use contraceptives consistent with local regulations and the following:
• Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP.
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 patient program for patients with primary ITP), the following criteria apply:
-
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).
-
Participant has completed a 52-week treatment period.
Exclusion criteria:
- 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)
- Use of any other investigational drug or participation in any other investigational trial
- Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients
- 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
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SINGLE_GROUP
- Arm && Interventions
Group Intervention Description efgartigimod PH20 SC efgartigimod PH20 SC Patients receiving efgartigimod PH20 SC treatment
- Primary Outcome Measures
Name Time Method Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs) 216 weeks Vital sign measurement: blood pressure in the overall population 216 weeks ECG: PR, QT and QRS interval in the overall population 216 weeks Laboratory safety evaluations: CRP analysis in the overall population 216 weeks
- Secondary Outcome Measures
Name Time Method Severity of the World Health Organization (WHO)-classified bleeding events 52 weeks Mean change from baseline in platelet count at each visit 52 weeks Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits 52 weeks Number of patients who performed self-administration at home over time 52 weeks Percentage of caregivers who administered the injection to the patient at home over time 52 weeks In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline 52 weeks For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L 52 weeks In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24 7 weeks (week 17-24) Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L 52 weeks Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period 52 weeks The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline 52 weeks In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24 5 weeks (week 19-24) Rate of receipt of rescue therapy (rescue per patient per month) 52 weeks Percentage of patients who performed self-administration at home over time 52 weeks Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline 52 weeks Number of caregivers who administered the injection to the patient at home over time 52 weeks Number of self- or caregiver-supported administrations at home 52 weeks Presence of neutralizing antibodies (NAb) against efgartigimod 216 weeks Incidence of the World Health Organization (WHO)-classified bleeding events 52 weeks Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits 52 weeks Pharmacodynamics markers: total IgG 52 weeks Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC 52 weeks Percentage of self- or caregiver-supported administrations at home 52 weeks Incidence and prevalence of antibodies to efgartigimod 216 weeks Titers of antibodies to efgartigimod 216 weeks Serum efgartigimod concentration observed predose (Ctrough) 52 weeks Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits 52 weeks
Trial Locations
- Locations (83)
Investigator site 2160002
🇹🇳Tunis, Tunisia
Investigator site 560002
🇨🇱Santiago, Chile
Investigator Site 0610003
🇦🇺West Perth, Australia
Investigator site 860055
🇨🇳Huizhou, China
Investigator site 270003
🇿🇦Johannesburg, South Africa
Investigator Site 0810017
🇯🇵Iruma, Japan
Investigator Site 0860008
🇨🇳Bengbu, China
Investigator site 610005
🇦🇺Westmead, Australia
Investigator site 390043
🇮🇹Ferrara, Italy
Investigator site 270004
🇿🇦Observatory, South Africa
Investigator site 270002
🇿🇦Randburg, South Africa
Investigator site 640005
🇳🇿Christchurch, New Zealand
Investigator site 0470003
🇳🇴Sarpsborg, Norway
Investigator site 440005
🇬🇧Coventry, United Kingdom
Investigator site 270001
🇿🇦Pretoria, South Africa
Investigator Site 0070038
🇷🇺Nizhny Novgorod, Russian Federation
Investigator Site 0660001
🇹🇭Bangkok Noi, Thailand
Investigator Site 0900014
🇹🇷Kocaeli, Turkey
Investigator site UK0440014
🇬🇧Penzance, United Kingdom
Investigator Site 0860015
🇨🇳Shenzhen, China
Investigator Site 0860001
🇨🇳Tianjin, China
Investigator Site 0860010
🇨🇳Wuhan, China
Investigator Site 0860002
🇨🇳Wuxi, China
Investigator Site 0860011
🇨🇳Zhengzhou, China
Investigator site 860058
🇨🇳Zhenjiang, China
Investigator site 860013
🇨🇳Beijing, China
Investigator site 820004
🇰🇷Seoul, Korea, Republic of
Investigator site 0660004
🇹🇭Chiang Mai, Thailand
Investigator Site 0900008
🇹🇷Ankara, Turkey
Investigator Site 0010062
🇺🇸Fort Wayne, Indiana, United States
Investigator site US0010042
🇺🇸Iowa City, Iowa, United States
Investigator site UK044041
🇬🇧London, United Kingdom
Investigator Site 0610012
🇦🇺Garran, Australia
Investigator site 540004
🇦🇷Buenos Aires, Argentina
Investigator site 390041
🇮🇹Napoli, Italy
Investigator Site 3510007
🇵🇹Lisboa, Portugal
Investigator site 0070040
🇷🇺Kirov, Russian Federation
Investigator site TH0660009
🇹🇭Khon Kaen, Thailand
Investigator Site 0540001
🇦🇷Buenos Aires, Argentina
Investigator site 0560003
🇨🇱Reñaca, Chile
Investigator site 300008
🇬🇷Athens, Greece
Investigator site JP0810015
🇯🇵Hirakata, Japan
Investigator site 810023
🇯🇵Shimotsuke, Japan
Investigator Site 0810038
🇯🇵Tama, Japan
Investigator site 520002
🇲🇽Aguascalientes, Mexico
Investigator site 3510004
🇵🇹Porto, Portugal
Investigator Site 0400007
🇷🇴Craiova, Romania
Investigator site 660005
🇹🇭Bangkok, Thailand
Investigator site 900019
🇹🇷Trabzon, Turkey
Investigator site 9950011
🇬🇪Tbilisi, Georgia
Investigator Site 0480037
🇵🇱Skorzewo, Poland
Investigator site 3510001
🇵🇹Vila Nova De Gaia, Portugal
Investigator Site 0400005
🇷🇴Bucharest, Romania
Investigator Site 0660003
🇹🇭Bangkok, Thailand
Investigator Site 0900015
🇹🇷Ankara, Turkey
Investigator site 900004
🇹🇷İzmir, Turkey
Investigator site KO0820007
🇰🇷Seoul, Korea, Republic of
Investigator Site 3590017
🇧🇬Plovdiv, Bulgaria
Investigator site 9950009
🇬🇪Tbilisi, Georgia
Investigator Site 0300009
🇬🇷Thessaloníki, Greece
Investigator site 3530003
🇮🇪Dublin, Ireland
Investigator Site 0810051
🇯🇵Kitakyushu, Japan
Investigator site 0810016
🇯🇵Shibukawa, Japan
Investigator Site 0480026
🇵🇱Nowy Sącz, Poland
Investigator site 400012
🇷🇴Bucuresti, Romania
Investigator site 2610001
🇹🇳Sousse, Tunisia
Investigator Site 0900007
🇹🇷Adapazarı, Turkey
Investigator Site 0900003
🇹🇷Ankara, Turkey
Investigator site 900010
🇹🇷Mersin, Turkey
Investigator site 0560004
🇨🇱Temuco, Chile
Investigator Site 9950007
🇬🇪Tbilisi, Georgia
Investigator Site 0390032
🇮🇹Milan, Italy
Investigator Site 0390044
🇮🇹Napoli, Italy
Investigator Site 0810053
🇯🇵Kanagawa, Japan
Investigator site 9620001
🇯🇴Irbid, Jordan
Investigator site PL0480013
🇵🇱Katowice, Poland
Investigator Site 0070037
🇷🇺Novosibirsk, Russian Federation
Investigator Site 0660002
🇹🇭Bangkok, Thailand
Investigator site 0900017
🇹🇷Tekirdağ, Turkey
Investigator Site 0010095
🇺🇸Oklahoma City, Oklahoma, United States
Investigator Site 0070026
🇷🇺Moscow, Russian Federation
Investigator Site 0010116
🇺🇸Bentonville, Arkansas, United States
Investigator site 0010045
🇺🇸Washington, District of Columbia, United States