Une étude de phase 3 pour évaluer l'innocuité et l'efficacité de l'efgartigimod PH20 sous-cutané chez des patients adultes atteints de thrombocytopénie immunitaire primaire (ADVANCE SC+)
Un essai de phase 3, multicentrique, ouvert et à long terme pour évaluer l'innocuité et l'efficacité de l'efgartigimod (ARGX-113) PH20 sous-cutané chez des patients adultes atteints de thrombocytopénie immunitaire primaire
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.
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Type d'étude
Inscription (Réel)
Phase
- Phase 3
Contacts et emplacements
Lieux d'étude
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Johannesburg, Afrique du Sud
- Investigator site 270003
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Observatory, Afrique du Sud
- Investigator site 270004
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Pretoria, Afrique du Sud
- Investigator site 270001
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Randburg, Afrique du Sud
- Investigator site 270002
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Buenos Aires, Argentine
- Investigator Site 0540001
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Buenos Aires, Argentine
- Investigator site 540004
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Garran, Australie
- Investigator Site 0610012
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West Perth, Australie
- Investigator Site 0610003
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Westmead, Australie
- Investigator site 610005
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Plovdiv, Bulgarie
- Investigator Site 3590017
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Reñaca, Chili
- Investigator site 0560003
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Santiago, Chili
- Investigator site 560002
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Temuco, Chili
- Investigator site 0560004
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Beijing, Chine
- Investigator site 860013
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Bengbu, Chine
- Investigator Site 0860008
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Huizhou, Chine
- Investigator site 860055
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Shenzhen, Chine
- Investigator Site 0860015
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Tianjin, Chine
- Investigator Site 0860001
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Wuhan, Chine
- Investigator Site 0860010
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Wuxi, Chine
- Investigator Site 0860002
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Zhengzhou, Chine
- Investigator Site 0860011
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Zhenjiang, Chine
- Investigator site 860058
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Seoul, Corée du Sud
- Investigator site 820004
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Seoul, Corée du Sud
- Investigator site KO0820007
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Athens, Grèce
- Investigator site 300008
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Thessaloniki, Grèce
- Investigator Site 0300009
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Tbilisi, Géorgie
- Investigator site 9950009
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Tbilisi, Géorgie
- Investigator site 9950011
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Tbilisi, Géorgie
- Investigator Site 9950007
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Dublin, Irlande
- Investigator site 3530003
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Ferrara, Italie
- Investigator site 390043
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Milan, Italie
- Investigator Site 0390032
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Naples, Italie
- Investigator Site 0390044
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Naples, Italie
- Investigator site 390041
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Hirakata, Japon, 5731191
- Investigator site JP0810015
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Iruma, Japon
- Investigator Site 0810017
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Kanagawa, Japon
- Investigator Site 0810053
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Kitakyushu, Japon
- Investigator Site 0810051
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Shibukawa, Japon, 377-0280
- Investigator site 0810016
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Shimotsuke, Japon
- Investigator site 810023
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Tama, Japon
- Investigator Site 0810038
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Irbid, Jordan
- Investigator site 9620001
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Lisbon, Le Portugal
- Investigator Site 3510007
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Porto, Le Portugal
- Investigator site 3510004
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Vila Nova de Gaia, Le Portugal
- Investigator site 3510001
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Aguascalientes, Mexique
- Investigator site 520002
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Sarpsborg, Norvège, 1714
- Investigator site 0470003
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Christchurch, Nouvelle-Zélande
- Investigator site 640005
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Katowice, Pologne, 40-519
- Investigator site PL0480013
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Nowy Sącz, Pologne
- Investigator Site 0480026
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Skorzewo, Pologne
- Investigator Site 0480037
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Bucharest, Roumanie
- Investigator Site 0400005
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Bucharest, Roumanie
- Investigator site 400012
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Craiova, Roumanie
- Investigator Site 0400007
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Coventry, Royaume-Uni
- Investigator site 440005
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London, Royaume-Uni
- Investigator site UK044041
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Penzance, Royaume-Uni, TR18 2PF
- Investigator site UK0440014
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Kirov, Russie
- Investigator site 0070040
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Moscow, Russie
- Investigator Site 0070026
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Nizhny Novgorod, Russie
- Investigator Site 0070038
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Novosibirsk, Russie
- Investigator Site 0070037
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Bangkok, Thaïlande
- Investigator Site 0660002
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Bangkok, Thaïlande
- Investigator Site 0660003
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Bangkok, Thaïlande
- Investigator site 660005
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Bangkok Noi, Thaïlande
- Investigator Site 0660001
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Chiang Mai, Thaïlande, 50200
- Investigator site 0660004
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Khon Kaen, Thaïlande
- Investigator site TH0660009
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Sousse, Tunisie
- Investigator site 2610001
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Tunis, Tunisie
- Investigator site 2160002
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Adapazarı, Turquie (Türkiye)
- Investigator Site 0900007
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Ankara, Turquie (Türkiye)
- Investigator Site 0900003
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Ankara, Turquie (Türkiye)
- Investigator Site 0900015
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Ankara, Turquie (Türkiye)
- Investigator Site 0900008
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Izmir, Turquie (Türkiye)
- Investigator site 900004
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Kocaeli, Turquie (Türkiye)
- Investigator Site 0900014
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Mersin, Turquie (Türkiye)
- Investigator site 900010
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Tekirdağ, Turquie (Türkiye), 59100
- Investigator site 0900017
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Trabzon, Turquie (Türkiye)
- Investigator site 900019
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Arkansas
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Bentonville, Arkansas, États-Unis, 72758
- Investigator Site 0010116
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District of Columbia
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Washington D.C., District of Columbia, États-Unis, 20007
- Investigator site 0010045
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Indiana
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Fort Wayne, Indiana, États-Unis, 46804
- Investigator Site 0010062
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Iowa
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Iowa City, Iowa, États-Unis, 52242
- Investigator site US0010042
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Oklahoma
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Oklahoma City, Oklahoma, États-Unis, 73142
- Investigator Site 0010095
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
La description
Inclusion Criteria:
- 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.
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.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Objectif principal: Traitement
- Répartition: N / A
- Modèle interventionnel: Affectation à un seul groupe
- Masquage: Aucun (étiquette ouverte)
Armes et Interventions
Groupe de participants / Bras |
Intervention / Traitement |
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Expérimental: efgartigimod PH20 SC
Patients receiving efgartigimod PH20 SC treatment |
Subcutaneous injection with efgartigimod PH20 SC
Autres noms:
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Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Incidence of AEs, AESIs, SAEs
Délai: Up to 216 weeks
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AE : adverse event ; SAE : Serious adverse event ; AESI : adverse event of special interest. |
Up to 216 weeks
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Vital sign measurement: blood pressure in the overall population
Délai: Up to 216 weeks
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Up to 216 weeks
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ECG: PR, QT and QRS interval in the overall population
Délai: Up to 216 weeks
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Up to 216 weeks
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Laboratory safety evaluations: CRP analysis in the overall population
Délai: Up to 216 weeks
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Up to 216 weeks
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Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
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Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L
Délai: Up to 52 weeks
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Up to 52 weeks
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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
Délai: Up to 52 weeks
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Up to 52 weeks
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Mean change from baseline in platelet count at each visit
Délai: Up to 52 weeks
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Up to 52 weeks
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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
Délai: Up to 52 weeks
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Up to 52 weeks
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The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline
Délai: Up to 52 weeks
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Up to 52 weeks
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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
Délai: Up to 52 weeks
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Up to 52 weeks
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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
Délai: 5 weeks (from week 19 to week 24)
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5 weeks (from week 19 to week 24)
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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
Délai: 7 weeks (from week 17 to week 24)
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7 weeks (from week 17 to week 24)
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Proportion of participants for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline
Délai: Up to 52 weeks
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Up to 52 weeks
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Rate of receipt of rescue therapy (rescue per participant per month)
Délai: Up to 52 weeks
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Up to 52 weeks
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Incidence of the WHO-classified bleeding events
Délai: Up to 52 weeks
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WHO : World Health Organization |
Up to 52 weeks
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Serum efgartigimod concentration observed predose (Ctrough)
Délai: Up to 52 weeks
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Up to 52 weeks
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Change from baseline in FACIT-fatigue at planned visits
Délai: Up to 52 weeks
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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." |
Up to 52 weeks
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Change from baseline in FACT-Th6 at planned visits
Délai: Up to 52 weeks
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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. |
Up to 52 weeks
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Change from baseline in SF-36 at planned visits
Délai: Up to 52 weeks
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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. |
Up to 52 weeks
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Change from baseline in ITP-PAQ at planned visits
Délai: Up to 52 weeks
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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. |
Up to 52 weeks
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Pharmacodynamics markers: total IgG
Délai: Up to 52 weeks
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Up to 52 weeks
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Percentage of participants who performed self-administration at home over time
Délai: Up to 52 weeks
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Up to 52 weeks
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Percentage of caregivers who administered the injection to the patient at home over time
Délai: Up to 52 weeks
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Up to 52 weeks
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Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC
Délai: Up to 52 weeks
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Up to 52 weeks
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Percentage of self- or caregiver-supported administrations at home
Délai: Up to 52 weeks
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Up to 52 weeks
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Incidence of antibodies to efgartigimod
Délai: Up to 216 weeks
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Up to 216 weeks
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Titers of antibodies to efgartigimod
Délai: Up to 216 weeks
|
Up to 216 weeks
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Presence of NAb against efgartigimod
Délai: Up to 216 weeks
|
NAb : Neutralizing antibodies |
Up to 216 weeks
|
Collaborateurs et enquêteurs
Parrainer
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
- Cytopénie
- Processus pathologiques
- Maladies auto-immunes
- Maladies du système immunitaire
- Hémorragie
- Manifestations cutanées
- Maladies hématologiques
- Troubles de la coagulation sanguine
- Troubles hémorragiques
- Troubles des plaquettes sanguines
- Microangiopathies thrombotiques
- Purpura thrombocytopénique
- Purpura
- Thrombocytopénie
- Conditions pathologiques, signes et symptômes
- Signes et symptômes
- Maladies hémiques et lymphatiques
- Purpura, thrombocytopénique, idiopathique
Autres numéros d'identification d'étude
- ARGX-113-2005
- 2024-513147-90-00 (Ctis)
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
Informations sur les médicaments et les dispositifs, documents d'étude
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