A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).

A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.

Sponsors

Lead Sponsor: argenx

Source argenx
Brief Summary

This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.

Overall Status Recruiting
Start Date June 2, 2020
Completion Date October 2022
Primary Completion Date June 2022
Phase Phase 3
Study Type Interventional
Primary Outcome
Measure Time Frame
Frequency and severity of Adverse Events Up to 56 weeks
Frequency and severity of vital signs Up to 56 weeks
Frequency and severity of laboratory assessments Up to 56 weeks
Secondary Outcome
Measure Time Frame
Extent of disease control defined as the number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥ 50×10^9/L Over the 52 weeks of treatment
Percentage of patients 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. Over the 52 weeks of treatment
Mean change from baseline in platelet count at each visit. Up to 56 weeks, at each visit
For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L UP to 56 weeks, at each visit
The number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline. Over the 52 weeks of treatment
In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline. Over the 52 weeks of treatment
In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial. Up to 5 weeks, between visit 19 and 24 of the trial
In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial. Up to 7 weeks, between visit 17 and 24 of the trial
Rate of receipt of rescue therapy (rescue per patient per month). Up to 56 weeks, at each visit
Reduction in concurrent ITP therapy. Up to 56 weeks, at each visit
Incidence and severity of the WHO-classified bleeding events. Up to 56 weeks, at each visit
Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits. Up to 52 weeks
Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits. Up to 52 weeks
Change from baseline in Quality of Life (SF-36) at planned visits. Up to 52 weeks
Incidence of anti-drug antibodies (ADA) to efgartigimod. Up to 56 weeks
Pharmacokinetic parameters of efgartigimod: maximum observed serum concentration (Cmax). Up to 56 weeks
Serum levels of pharmacodynamics markers: total IgG, IgG isotypes (IgG1, IgG2, IgG3, IgG4). Up to 56 weeks
Enrollment 156
Condition
Intervention

Intervention Type: Biological

Intervention Name: efgartigimod

Description: Intravenous infusion of efgartigimod

Arm Group Label: efgartigimod

Other Name: ARGX-113

Eligibility

Criteria:

Inclusion Criteria:

1. Ability to understand the requirements 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).

2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period.

3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are postmenopausal (defined by continuous amenorrhea) for at least 1 year with a follicle-stimulating hormone (FSH) of >40 IU/L or are surgically sterilized (i.e. women who had a hysterectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle stimulating hormone can be used to confirm postmenopausal status in amenorrheic patients not on hormonal replacement therapy.

4. Women of childbearing potential should use a highly effective method of contraception (i.e. pregnancy rate of less than 1% per year) during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month:

- combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: oral, intravaginal, transdermal.

- progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable, implantable

- intrauterine device (IUD)

- intrauterine hormone-releasing system

- bilateral tubal occlusion

- vasectomized partner (provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post procedure)

- continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable.

5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, being a condom for male patients and a highly effective form of contraception for the female partner of childbearing (same as for female patients described in inclusion criterion 4). Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had vasectomy with documented aspermia post procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of informed consent form, throughout the duration of the trial, and for 90 days after the last administration of IMP.

Exclusion Criteria:

1. Introduction or continuation of non-permitted medications during the ARGX- 113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).

2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.

3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.

4. Use of any other investigational drug or participation in any other investigational trial.

Gender: All

Minimum Age: 18 Years

Maximum Age: N/A

Healthy Volunteers: No

Overall Contact

Last Name: Antonio Guglietta, MD

Phone: +1 857-350-4834

Email: [email protected]

Location
Facility: Status: Contact:
Investigator Site 1 | Ocala, Florida, 34474, United States Recruiting Antonio Guglietta 857-350-4834 [email protected]
Investigator Site 2 | Turnhout, Belgium Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 3 | Yvoir, Belgium Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 5 | Budapest, Hungary Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 4 | Győr, Hungary Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 6 | Nyíregyháza, Hungary Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 9 | Ravenna, Italy Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 10 | Rimini, Italy Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 8 | Siena, Italy Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 13 | Iruma, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 11 | Maebashi, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 12 | Niigata, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 16 | Sapporo, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 15 | Shibukawa, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 14 | Shimotsuke, Japan Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 18 | Den Haag, Netherlands Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 17 | Rotterdam, Netherlands Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 20 | Kaluga, Russian Federation Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 28 | Moscow, Russian Federation Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 27 | Saint Petersburg, Russian Federation Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 7 | Tula, Russian Federation Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 19 | Ufa, Russian Federation Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 25 | Barcelona, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 30 | Barcelona, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 21 | Madrid, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 23 | Madrid, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 22 | Palma De Mallorca, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 29 | Pozuelo De Alarcón, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 24 | Sevilla, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 31 | Valencia, Spain Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Investigator Site 26 | Mykolaiv, Ukraine Recruiting Antonio Guglietta, MD 857-350-4834 +1 [email protected]
Location Countries

Belgium

Hungary

Italy

Japan

Netherlands

Russian Federation

Spain

Ukraine

United States

Verification Date

September 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Number Of Arms 1
Arm Group

Label: efgartigimod

Type: Experimental

Description: patients receiving efgartigimod

Acronym ADVANCE+
Patient Data Undecided
Study Design Info

Allocation: N/A

Intervention Model: Single Group Assignment

Primary Purpose: Treatment

Masking: None (Open Label)

Source: ClinicalTrials.gov