Primary Immune ThrombocytopeniaPhase 3ADVANCE SC+NCT04812925
A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
A Phase 3 study testing efgartigimod PH20 SC in people with Primary Immune Thrombocytopenia, run by argenx at 83 sites in 25 countries.
Trial summary
The main aim of the study is to evaluate the safety and efficacy of efgartigimod PH20 SC (subcutaneous) in adult patients with primary immune thrombocytopenia (ITP). Participants who completed the 24-week trial period of the ARGX- 113-2004 trial are given the opportunity to enter into this open-label extension trial… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Primary Immune Thrombocytopenia
- What is being tested?
- efgartigimod PH20 SC
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 173 participants
- Where
- 83 sitesin 25 countries
- Study dates
- 17 Nov 2021to Oct 2027
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
- Agree to use contraceptives consistent with local regulations.
- In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period, the participant has completed a 52-week treatment period.
Who cannot join
- 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.
Trial locations and contacts
83 sites in 25 countries.
83 sites in 25 countries
China9 sites
Investigator site 860013
Beijing
Investigator Site 0860008
Bengbu
Investigator site 860055
Huizhou
Investigator Site 0860015
Shenzhen
Investigator Site 0860001
Tianjin
Investigator Site 0860010
Wuhan
Investigator Site 0860002
Wuxi
Investigator Site 0860011
Zhengzhou
Investigator site 860058
Zhenjiang
Turkey (Türkiye)9 sites
Investigator Site 0900007
Adapazarı
Investigator Site 0900003
Ankara
Investigator Site 0900008
Ankara
Investigator Site 0900015
Ankara
Investigator site 900004
Izmir
Investigator Site 0900014
Kocaeli
Investigator site 900010
Mersin
Investigator site 0900017
Tekirdağ, 59100
Investigator site 900019
Trabzon
Japan7 sites
Investigator site JP0810015
Hirakata, 5731191
Investigator Site 0810017
Iruma
Investigator Site 0810053
Kanagawa
Investigator Site 0810051
Kitakyushu
Investigator site 0810016
Shibukawa, 377-0280
Investigator site 810023
Shimotsuke
Investigator Site 0810038
Tama
Thailand6 sites
Investigator Site 0660002
Bangkok
Investigator Site 0660003
Bangkok
Investigator site 660005
Bangkok
Investigator Site 0660001
Bangkok Noi
Investigator site 0660004
Chiang Mai, 50200
Investigator site TH0660009
Khon Kaen
United States5 sites
Investigator Site 0010116
Bentonville, Arkansas, 72758
Investigator Site 0010062
Fort Wayne, Indiana, 46804
Investigator site US0010042
Iowa City, Iowa, 52242
Investigator Site 0010095
Oklahoma City, Oklahoma, 73142
Investigator site 0010045
Washington D.C., District of Columbia, 20007
Italy4 sites
Investigator site 390043
Ferrara
Investigator Site 0390032
Milan
Investigator Site 0390044
Naples
Investigator site 390041
Naples
Russia4 sites
Investigator site 0070040
Kirov
Investigator Site 0070026
Moscow
Investigator Site 0070038
Nizhny Novgorod
Investigator Site 0070037
Novosibirsk
South Africa4 sites
Investigator site 270003
Johannesburg
Investigator site 270004
Observatory
Investigator site 270001
Pretoria
Investigator site 270002
Randburg
Australia3 sites
Investigator Site 0610012
Garran
Investigator Site 0610003
West Perth
Investigator site 610005
Westmead
Chile3 sites
Investigator site 0560003
Reñaca
Investigator site 560002
Santiago
Investigator site 0560004
Temuco
Georgia3 sites
Investigator Site 9950007
Tbilisi
Investigator site 9950009
Tbilisi
Investigator site 9950011
Tbilisi
Poland3 sites
Investigator site PL0480013
Katowice, 40-519
Investigator Site 0480026
Nowy Sącz
Investigator Site 0480037
Skorzewo
Portugal3 sites
Investigator Site 3510007
Lisbon
Investigator site 3510004
Porto
Investigator site 3510001
Vila Nova de Gaia
Romania3 sites
Investigator Site 0400005
Bucharest
Investigator site 400012
Bucharest
Investigator Site 0400007
Craiova
United Kingdom3 sites
Investigator site 440005
Coventry
Investigator site UK044041
London
Investigator site UK0440014
Penzance, TR18 2PF
Argentina2 sites
Investigator Site 0540001
Buenos Aires
Investigator site 540004
Buenos Aires
Greece2 sites
Investigator site 300008
Athens
Investigator Site 0300009
Thessaloniki
South Korea2 sites
Investigator site 820004
Seoul
Investigator site KO0820007
Seoul
Tunisia2 sites
Investigator site 2610001
Sousse
Investigator site 2160002
Tunis
About this study
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
The main aim of the study is to evaluate the safety and efficacy of efgartigimod PH20 SC (subcutaneous) in adult patients with primary immune thrombocytopenia (ITP). Participants who completed the 24-week trial period of the ARGX- 113-2004 trial are given the opportunity to enter into this open-label extension trial and receive efgartigimod PH20 SC.
Participants will be offered the opportunity to continue in this trial if they have completed the first 52-week treatment period unless efgartigimod becomes commercially available for patients with primary ITP or becomes available through another program for patients with primary ITP, whichever comes first. A maximum of three additional 52-week treatment periods will be offered to the participant to continue this trial.
The total trial duration per participant is up to 4 years (52-week treatment period, extendable with three additional 52-week treatment periods) + 8 weeks follow-up period.
- Conditions
- Primary Immune Thrombocytopenia
- Being tested
- efgartigimod PH20 SC
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
efgartigimod PH20 SCBiological
Also called ARGX-113 PH20 SC
Subcutaneous injection with efgartigimod PH20 SC
| Study group | Role | What they receive |
|---|---|---|
| efgartigimod PH20 SC | Experimental | Patients receiving efgartigimod PH20 SC treatment Receives: Biological: efgartigimod PH20 SC |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs, AESIs, SAEs
AE : adverse event ; SAE : Serious adverse event ; AESI : adverse event of special interest.
Measured: Up to 216 weeks
Vital sign measurement: blood pressure in the overall population
Measured: Up to 216 weeks
ECG: PR, QT and QRS interval in the overall population
Measured: Up to 216 weeks
Laboratory safety evaluations: CRP analysis in the overall population
Measured: Up to 216 weeks
Secondary outcomes (24)
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L
Measured: Up to 52 weeks
Proportion of participants 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
Measured: Up to 52 weeks
Mean change from baseline in platelet count at each visit
Measured: Up to 52 weeks
For participants 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
Measured: Up to 52 weeks
The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline
Measured: Up to 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
Measured: Up to 52 weeks
In participants 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
Measured: 5 weeks (from week 19 to week 24)
In participants 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
Measured: 7 weeks (from week 17 to week 24)
Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
Measured: Up to 52 weeks
Rate of receipt of rescue therapy (rescue per participant per month)
Measured: Up to 52 weeks
Incidence of the WHO-classified bleeding events
WHO : World Health Organization
Measured: Up to 52 weeks
Serum efgartigimod concentration observed predose (Ctrough)
Measured: Up to 52 weeks
Change from baseline in FACIT-fatigue at planned visits
The Functional Assessment of Chronic Illness Therapy Fatigue (FACIT- fatigue) scale is a short, 13-item, easy-to-administer tool that measures an individual's level of fatigue during their usual daily activities over the past week. The level of fatigue is measured by recording item responses on a 4-point Likert scale ranging from 0 "not at all" to 4 "very much."
Measured: Up to 52 weeks
Change from baseline in FACT-Th6 at planned visits
The Functional Assessment of Cancer Therapy questionnaire-Th6 (FACT-Th6) uses the Likert scale, with participants rating their degree of concern in the past 7 days. The 6 selected items pertain to ability to do usual activities, worry about problems with bleeding or bruising, worry about the possibility of serious bleeding, avoidance of physical or social activity because of concern with bleeding or bruising and frustration due to the inability to carry out usual activities.
Measured: Up to 52 weeks
Change from baseline in SF-36 at planned visits
The SF-36 is a 36-item scale constructed to survey health-related QoL on 8 domains: limitations in physical activities due to health problems; limitations in social activities due to physical or emotional problems; limitations in usual role activities due to physical health problems; bodily pain; general mental health (psychological distress and well being); limitations in usual role activities due to emotional problems; vitality (energy and fatigue); and general health perceptions.
Measured: Up to 52 weeks
Change from baseline in ITP-PAQ at planned visits
The ITP-Patient Assessment Questionnaire (ITP-PAQ) is a 44-item questionnaire to assess the health-related QoL in adults with ITP. It includes scales for physical health (symptoms, fatigue/sleep, bother, and activity), emotional health (psychological and fear), overall QoL, social activity, women's reproductive health, and work.
Measured: Up to 52 weeks
Pharmacodynamics markers: total IgG
Measured: Up to 52 weeks
Percentage of participants who performed self-administration at home over time
Measured: Up to 52 weeks
Percentage of caregivers who administered the injection to the patient at home over time
Measured: Up to 52 weeks
Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
Measured: Up to 52 weeks
Percentage of self- or caregiver-supported administrations at home
Measured: Up to 52 weeks
Incidence of antibodies to efgartigimod
Measured: Up to 216 weeks
Titers of antibodies to efgartigimod
Measured: Up to 216 weeks
Presence of NAb against efgartigimod
NAb : Neutralizing antibodies
Measured: Up to 216 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2021
- Study start17 Nov 2021
- Primary completionOct 2027 (estimated)
- Study completionOct 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Immune ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by argenxThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 173 participants (actual)
Sponsor and investigators
- Lead sponsor
- argenx
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04812925
- Sponsor's protocol ID
- ARGX-113-2005
- Other IDs
- 2024-513147-90-00
- Acronym
- ADVANCE SC+
- Status last verified
- Sep 2026
- First submitted
- 15 Mar 2021
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided