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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04812925




Registration number
NCT04812925
Ethics application status
Date submitted
15/03/2021
Date registered
24/03/2021

Titles & IDs
Public title
A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients with Primary Immune Thrombocytopenia
Scientific 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
Secondary ID [1] 0 0
ARGX-113-2005
Universal Trial Number (UTN)
Trial acronym
ADVANCE SC+
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Primary Immune Thrombocytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Other - efgartigimod PH20 SC

Experimental: efgartigimod PH20 SC - Patients receiving efgartigimod PH20 SC treatment


Treatment: Other: efgartigimod PH20 SC
Subcutaneous injection with efgartigimod PH20 SC
Intervention code [1] 0 0
Treatment: Other
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)
Timepoint [1] 0 0
216 weeks
Primary outcome [2] 0 0
Vital sign measurement: blood pressure in the overall population
Timepoint [2] 0 0
216 weeks
Primary outcome [3] 0 0
ECG: PR, QT and QRS interval in the overall population
Timepoint [3] 0 0
216 weeks
Primary outcome [4] 0 0
Laboratory safety evaluations: CRP analysis in the overall population
Timepoint [4] 0 0
216 weeks
Secondary outcome [1] 0 0
Extent of disease control defined as the percentage of weeks in the trial with platelet counts of =50×10E9/L
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
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
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
Mean change from baseline in platelet count at each visit
Timepoint [3] 0 0
52 weeks
Secondary outcome [4] 0 0
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
Timepoint [4] 0 0
52 weeks
Secondary outcome [5] 0 0
The percentage of weeks in the trial with platelet counts of =30×10E9/L and =20×10E9/L above baseline
Timepoint [5] 0 0
52 weeks
Secondary outcome [6] 0 0
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
Timepoint [6] 0 0
52 weeks
Secondary outcome [7] 0 0
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
Timepoint [7] 0 0
5 weeks (week 19-24)
Secondary outcome [8] 0 0
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
Timepoint [8] 0 0
7 weeks (week 17-24)
Secondary outcome [9] 0 0
Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
Timepoint [9] 0 0
52 weeks
Secondary outcome [10] 0 0
Rate of receipt of rescue therapy (rescue per patient per month)
Timepoint [10] 0 0
52 weeks
Secondary outcome [11] 0 0
Incidence of the World Health Organization (WHO)-classified bleeding events
Timepoint [11] 0 0
52 weeks
Secondary outcome [12] 0 0
Severity of the World Health Organization (WHO)-classified bleeding events
Timepoint [12] 0 0
52 weeks
Secondary outcome [13] 0 0
Serum efgartigimod concentration observed predose (Ctrough)
Timepoint [13] 0 0
52 weeks
Secondary outcome [14] 0 0
Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits
Timepoint [14] 0 0
52 weeks
Secondary outcome [15] 0 0
Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits
Timepoint [15] 0 0
52 weeks
Secondary outcome [16] 0 0
Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits
Timepoint [16] 0 0
52 weeks
Secondary outcome [17] 0 0
Pharmacodynamics markers: total IgG
Timepoint [17] 0 0
52 weeks
Secondary outcome [18] 0 0
Number of patients who performed self-administration at home over time
Timepoint [18] 0 0
52 weeks
Secondary outcome [19] 0 0
Percentage of patients who performed self-administration at home over time
Timepoint [19] 0 0
52 weeks
Secondary outcome [20] 0 0
Number of caregivers who administered the injection to the patient at home over time
Timepoint [20] 0 0
52 weeks
Secondary outcome [21] 0 0
Percentage of caregivers who administered the injection to the patient at home over time
Timepoint [21] 0 0
52 weeks
Secondary outcome [22] 0 0
Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
Timepoint [22] 0 0
52 weeks
Secondary outcome [23] 0 0
Number of self- or caregiver-supported administrations at home
Timepoint [23] 0 0
52 weeks
Secondary outcome [24] 0 0
Percentage of self- or caregiver-supported administrations at home
Timepoint [24] 0 0
52 weeks
Secondary outcome [25] 0 0
Incidence and prevalence of antibodies to efgartigimod
Timepoint [25] 0 0
216 weeks
Secondary outcome [26] 0 0
Titers of antibodies to efgartigimod
Timepoint [26] 0 0
216 weeks
Secondary outcome [27] 0 0
Presence of neutralizing antibodies (NAb) against efgartigimod
Timepoint [27] 0 0
216 weeks

Eligibility
Key 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.
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
17/11/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/10/2026
Actual
Sample size
Target
Accrual to date
Final
173
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Investigator Site 0610012 - Garran
Recruitment hospital [2] 0 0
Investigator Site 0610003 - West Perth
Recruitment hospital [3] 0 0
Investigator site 610005 - Westmead
Recruitment postcode(s) [1] 0 0
- Garran
Recruitment postcode(s) [2] 0 0
- West Perth
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Arkansas
Country [2] 0 0
United States of America
State/province [2] 0 0
District of Columbia
Country [3] 0 0
United States of America
State/province [3] 0 0
Indiana
Country [4] 0 0
United States of America
State/province [4] 0 0
Iowa
Country [5] 0 0
United States of America
State/province [5] 0 0
Oklahoma
Country [6] 0 0
Argentina
State/province [6] 0 0
Buenos Aires
Country [7] 0 0
Bulgaria
State/province [7] 0 0
Plovdiv
Country [8] 0 0
Chile
State/province [8] 0 0
Reñaca
Country [9] 0 0
Chile
State/province [9] 0 0
Santiago
Country [10] 0 0
Chile
State/province [10] 0 0
Temuco
Country [11] 0 0
China
State/province [11] 0 0
Beijing
Country [12] 0 0
China
State/province [12] 0 0
Bengbu
Country [13] 0 0
China
State/province [13] 0 0
Huizhou
Country [14] 0 0
China
State/province [14] 0 0
Shenzhen
Country [15] 0 0
China
State/province [15] 0 0
Tianjin
Country [16] 0 0
China
State/province [16] 0 0
Wuhan
Country [17] 0 0
China
State/province [17] 0 0
Wuxi
Country [18] 0 0
China
State/province [18] 0 0
Zhengzhou
Country [19] 0 0
China
State/province [19] 0 0
Zhenjiang
Country [20] 0 0
Georgia
State/province [20] 0 0
Tbilisi
Country [21] 0 0
Greece
State/province [21] 0 0
Athens
Country [22] 0 0
Greece
State/province [22] 0 0
Thessaloníki
Country [23] 0 0
Ireland
State/province [23] 0 0
Dublin
Country [24] 0 0
Italy
State/province [24] 0 0
Ferrara
Country [25] 0 0
Italy
State/province [25] 0 0
Milan
Country [26] 0 0
Italy
State/province [26] 0 0
Napoli
Country [27] 0 0
Japan
State/province [27] 0 0
Hirakata
Country [28] 0 0
Japan
State/province [28] 0 0
Iruma
Country [29] 0 0
Japan
State/province [29] 0 0
Kanagawa
Country [30] 0 0
Japan
State/province [30] 0 0
Kitakyushu
Country [31] 0 0
Japan
State/province [31] 0 0
Shibukawa
Country [32] 0 0
Japan
State/province [32] 0 0
Shimotsuke
Country [33] 0 0
Japan
State/province [33] 0 0
Tama
Country [34] 0 0
Jordan
State/province [34] 0 0
Irbid
Country [35] 0 0
Korea, Republic of
State/province [35] 0 0
Seoul
Country [36] 0 0
Mexico
State/province [36] 0 0
Aguascalientes
Country [37] 0 0
New Zealand
State/province [37] 0 0
Christchurch
Country [38] 0 0
Norway
State/province [38] 0 0
Sarpsborg
Country [39] 0 0
Poland
State/province [39] 0 0
Katowice
Country [40] 0 0
Poland
State/province [40] 0 0
Nowy Sacz
Country [41] 0 0
Poland
State/province [41] 0 0
Skorzewo
Country [42] 0 0
Portugal
State/province [42] 0 0
Lisboa
Country [43] 0 0
Portugal
State/province [43] 0 0
Porto
Country [44] 0 0
Portugal
State/province [44] 0 0
Vila Nova De Gaia
Country [45] 0 0
Romania
State/province [45] 0 0
Bucharest
Country [46] 0 0
Romania
State/province [46] 0 0
Bucuresti
Country [47] 0 0
Romania
State/province [47] 0 0
Craiova
Country [48] 0 0
Russian Federation
State/province [48] 0 0
Kirov
Country [49] 0 0
Russian Federation
State/province [49] 0 0
Moscow
Country [50] 0 0
Russian Federation
State/province [50] 0 0
Nizhny Novgorod
Country [51] 0 0
Russian Federation
State/province [51] 0 0
Novosibirsk
Country [52] 0 0
South Africa
State/province [52] 0 0
Johannesburg
Country [53] 0 0
South Africa
State/province [53] 0 0
Observatory
Country [54] 0 0
South Africa
State/province [54] 0 0
Pretoria
Country [55] 0 0
South Africa
State/province [55] 0 0
Randburg
Country [56] 0 0
Thailand
State/province [56] 0 0
Bangkok Noi
Country [57] 0 0
Thailand
State/province [57] 0 0
Bangkok
Country [58] 0 0
Thailand
State/province [58] 0 0
Chiang Mai
Country [59] 0 0
Thailand
State/province [59] 0 0
Khon Kaen
Country [60] 0 0
Tunisia
State/province [60] 0 0
Sousse
Country [61] 0 0
Tunisia
State/province [61] 0 0
Tunis
Country [62] 0 0
Turkey
State/province [62] 0 0
Adapazari
Country [63] 0 0
Turkey
State/province [63] 0 0
Ankara
Country [64] 0 0
Turkey
State/province [64] 0 0
Izmir
Country [65] 0 0
Turkey
State/province [65] 0 0
Kocaeli
Country [66] 0 0
Turkey
State/province [66] 0 0
Mersin
Country [67] 0 0
Turkey
State/province [67] 0 0
Tekirdag
Country [68] 0 0
Turkey
State/province [68] 0 0
Trabzon
Country [69] 0 0
United Kingdom
State/province [69] 0 0
Coventry
Country [70] 0 0
United Kingdom
State/province [70] 0 0
London
Country [71] 0 0
United Kingdom
State/province [71] 0 0
Penzance

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
argenx
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT04812925