Clinical trial NCT04812925: A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia
Official title: A Phase 3, Multicenter, Open-Label, Long-Term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2021-03-24
Enrollment: 173
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia


Eligibility criteria: Inclusion Criteria:

1. 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).
2. 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:

4. 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).

5. Participant has completed a 52-week treatment period.

Exclusion criteria:

1. 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)
2. Use of any other investigational drug or participation in any other investigational trial
3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients
4. 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 outcomes:
measure: Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
timeFrame: 216 weeks
measure: Vital sign measurement: blood pressure in the overall population
timeFrame: 216 weeks
measure: ECG: PR, QT and QRS interval in the overall population
timeFrame: 216 weeks
measure: Laboratory safety evaluations: CRP analysis in the overall population
timeFrame: 216 weeks
Secondary outcomes:
measure: Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L
timeFrame: 52 weeks
measure: 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
timeFrame: 52 weeks
measure: Mean change from baseline in platelet count at each visit
timeFrame: 52 weeks
measure: 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
timeFrame: 52 weeks
measure: The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline
timeFrame: 52 weeks
measure: 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
timeFrame: 52 weeks
measure: 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
timeFrame: 5 weeks (week 19-24)
measure: 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
timeFrame: 7 weeks (week 17-24)
measure: Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
timeFrame: 52 weeks
measure: Rate of receipt of rescue therapy (rescue per patient per month)
timeFrame: 52 weeks
measure: Incidence of the World Health Organization (WHO)-classified bleeding events
timeFrame: 52 weeks
measure: Severity of the World Health Organization (WHO)-classified bleeding events
timeFrame: 52 weeks
measure: Serum efgartigimod concentration observed predose (Ctrough)
timeFrame: 52 weeks
measure: Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits
timeFrame: 52 weeks
measure: Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits
timeFrame: 52 weeks
measure: Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits
timeFrame: 52 weeks
measure: Pharmacodynamics markers: total IgG
timeFrame: 52 weeks
measure: Number of patients who performed self-administration at home over time
timeFrame: 52 weeks
measure: Percentage of patients who performed self-administration at home over time
timeFrame: 52 weeks
measure: Number of caregivers who administered the injection to the patient at home over time
timeFrame: 52 weeks
measure: Percentage of caregivers who administered the injection to the patient at home over time
timeFrame: 52 weeks
measure: Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
timeFrame: 52 weeks
measure: Number of self- or caregiver-supported administrations at home
timeFrame: 52 weeks
measure: Percentage of self- or caregiver-supported administrations at home
timeFrame: 52 weeks
measure: Incidence and prevalence of antibodies to efgartigimod
timeFrame: 216 weeks
measure: Titers of antibodies to efgartigimod
timeFrame: 216 weeks
measure: Presence of neutralizing antibodies (NAb) against efgartigimod
timeFrame: 216 weeks
Other outcomes:
Locations:
city: Bentonville
country: United States
facility: Investigator Site 0010116
state: Arkansas
city: Washington
country: United States
facility: Investigator site 0010045
state: District of Columbia
city: Fort Wayne
country: United States
facility: Investigator Site 0010062
state: Indiana
city: Iowa City
country: United States
facility: Investigator site US0010042
state: Iowa
city: Oklahoma City
country: United States
facility: Investigator Site 0010095
state: Oklahoma
city: Buenos Aires
country: Argentina
facility: Investigator Site 0540001
city: Buenos Aires
country: Argentina
facility: Investigator site 540004
city: Garran
country: Australia
facility: Investigator Site 0610012
city: West Perth
country: Australia
facility: Investigator Site 0610003
city: Westmead
country: Australia
facility: Investigator site 610005
city: Plovdiv
country: Bulgaria
facility: Investigator Site 3590017
city: Reñaca
country: Chile
facility: Investigator site 0560003
city: Santiago
country: Chile
facility: Investigator site 560002
city: Temuco
country: Chile
facility: Investigator site 0560004
city: Beijing
country: China
facility: Investigator site 860013
city: Bengbu
country: China
facility: Investigator Site 0860008
city: Huizhou
country: China
facility: Investigator site 860055
city: Shenzhen
country: China
facility: Investigator Site 0860015
city: Tianjin
country: China
facility: Investigator Site 0860001
city: Wuhan
country: China
facility: Investigator Site 0860010
city: Wuxi
country: China
facility: Investigator Site 0860002
city: Zhengzhou
country: China
facility: Investigator Site 0860011
city: Zhenjiang
country: China
facility: Investigator site 860058
city: Tbilisi
country: Georgia
facility: Investigator Site 9950007
city: Tbilisi
country: Georgia
facility: Investigator site 9950009
city: Tbilisi
country: Georgia
facility: Investigator site 9950011
city: Athens
country: Greece
facility: Investigator site 300008
city: Thessaloníki
country: Greece
facility: Investigator Site 0300009
city: Dublin
country: Ireland
facility: Investigator site 3530003
city: Ferrara
country: Italy
facility: Investigator site 390043
city: Milan
country: Italy
facility: Investigator Site 0390032
city: Napoli
country: Italy
facility: Investigator Site 0390044
city: Napoli
country: Italy
facility: Investigator site 390041
city: Hirakata
country: Japan
facility: Investigator site JP0810015
city: Iruma
country: Japan
facility: Investigator Site 0810017
city: Kanagawa
country: Japan
facility: Investigator Site 0810053
city: Kitakyushu
country: Japan
facility: Investigator Site 0810051
city: Shibukawa
country: Japan
facility: Investigator site 0810016
city: Shimotsuke
country: Japan
facility: Investigator site 810023
city: Tama
country: Japan
facility: Investigator Site 0810038
city: Irbid
country: Jordan
facility: Investigator site 9620001
city: Seoul
country: Korea, Republic of
facility: Investigator site 820004
city: Seoul
country: Korea, Republic of
facility: Investigator site KO0820007
city: Aguascalientes
country: Mexico
facility: Investigator site 520002
city: Christchurch
country: New Zealand
facility: Investigator site 640005
city: Sarpsborg
country: Norway
facility: Investigator site 0470003
city: Katowice
country: Poland
facility: Investigator site PL0480013
city: Nowy Sącz
country: Poland
facility: Investigator Site 0480026
city: Skorzewo
country: Poland
facility: Investigator Site 0480037
city: Lisboa
country: Portugal
facility: Investigator Site 3510007
city: Porto
country: Portugal
facility: Investigator site 3510004
city: Vila Nova De Gaia
country: Portugal
facility: Investigator site 3510001
city: Bucharest
country: Romania
facility: Investigator Site 0400005
city: Bucuresti
country: Romania
facility: Investigator site 400012
city: Craiova
country: Romania
facility: Investigator Site 0400007
city: Kirov
country: Russian Federation
facility: Investigator site 0070040
city: Moscow
country: Russian Federation
facility: Investigator Site 0070026
city: Nizhny Novgorod
country: Russian Federation
facility: Investigator Site 0070038
city: Novosibirsk
country: Russian Federation
facility: Investigator Site 0070037
city: Johannesburg
country: South Africa
facility: Investigator site 270003
city: Observatory
country: South Africa
facility: Investigator site 270004
city: Pretoria
country: South Africa
facility: Investigator site 270001
city: Randburg
country: South Africa
facility: Investigator site 270002
city: Bangkok Noi
country: Thailand
facility: Investigator Site 0660001
city: Bangkok
country: Thailand
facility: Investigator Site 0660002
city: Bangkok
country: Thailand
facility: Investigator Site 0660003
city: Bangkok
country: Thailand
facility: Investigator site 660005
city: Chiang Mai
country: Thailand
facility: Investigator site 0660004
city: Khon Kaen
country: Thailand
facility: Investigator site TH0660009
city: Sousse
country: Tunisia
facility: Investigator site 2610001
city: Tunis
country: Tunisia
facility: Investigator site 2160002
city: Adapazarı
country: Turkey
facility: Investigator Site 0900007
city: Ankara
country: Turkey
facility: Investigator Site 0900003
city: Ankara
country: Turkey
facility: Investigator Site 0900008
city: Ankara
country: Turkey
facility: Investigator Site 0900015
city: İzmir
country: Turkey
facility: Investigator site 900004
city: Kocaeli
country: Turkey
facility: Investigator Site 0900014
city: Mersin
country: Turkey
facility: Investigator site 900010
city: Tekirdağ
country: Turkey
facility: Investigator site 0900017
city: Trabzon
country: Turkey
facility: Investigator site 900019
city: Coventry
country: United Kingdom
facility: Investigator site 440005
city: London
country: United Kingdom
facility: Investigator site UK044041
city: Penzance
country: United Kingdom
facility: Investigator site UK0440014
Study officials:
References: