- ICH GCP
- US-Register für klinische Studien
- Klinische Studie NCT07612319
Romiplostim for Oral TPO-RA Resistant ITP
A Prospective Cohort Study of Romiplostim in Immune Thrombocytopenia Patients Resistant to Oral Thrombopoietin Receptor Agonists
This study is a prospective, multicenter, open-label, single-arm cohort study to evaluate the effectiveness and safety of romiplostim in patients with immune thrombocytopenia (ITP) who have not responded to oral thrombopoietin receptor agonists (TPO-RAs) such as eltrombopag, hetrombopag, or avatrombopag.
ITP is a blood disorder in which the immune system attacks and destroys the body's own platelets, leading to low platelet counts and an increased risk of bleeding. While oral TPO-RAs are effective for many patients, approximately 20-30% of patients do not respond adequately or lose response over time. For these patients, alternative treatments are urgently needed.
Participants in this study will discontinue their current oral TPO-RA and receive romiplostim as a weekly subcutaneous injection. The starting dose is 3 µg/kg, and the dose may be adjusted weekly based on platelet counts (maximum 10 µg/kg). The total treatment and follow-up period is 24 weeks.
The primary outcome measure is the durable platelet response rate at week 24, defined as maintaining platelet counts at ≥50×10⁹/L for at least two scheduled visits during weeks 22-24 without rescue therapy. Secondary outcomes include the sustained response rate at week 12, complete response rate at week 24, time to first response, improvement in bleeding events, and quality of life assessment.
Safety outcomes include monitoring for thromboembolic events, bone marrow fibrosis, hepatotoxicity, injection site reactions, and other adverse events according to CTCAE v5.0.
Approximately 60 participants will be enrolled across multiple centers in China. This study aims to provide evidence for romiplostim as a treatment option for ITP patients who have failed oral TPO-RAs.
Studienübersicht
Status
Intervention / Behandlung
Studientyp
Einschreibung (Geschätzt)
Phase
- Unzutreffend
Kontakte und Standorte
Studienkontakt
- Name: The First Affiliated Hospital of Soochow University
- Telefonnummer: 86-0512-67781521
- E-Mail: tianhong0718@163.com
Studienorte
-
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Jiangsu
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Suzhou, Jiangsu, China, 215000
- Rekrutierung
- The First Affiliated Hospital of Soochow University
-
-
Teilnahmekriterien
Zulassungskriterien
Studienberechtigtes Alter
- Erwachsene
- Älterer Erwachsener
Akzeptiert gesunde Freiwillige
Beschreibung
Inclusion Criteria:
Age ≥18 years Diagnosed with primary immune thrombocytopenia (ITP) according to current guidelines
Failed prior oral TPO-RA therapy (eltrombopag, hetrombopag, or avatrombopag), defined as:
Platelet count persistently <30×10⁹/L after ≥4 weeks of treatment at the maximum recommended or tolerated dose, OR Prior achieved response (platelet ≥50×10⁹/L) then lost response with platelet count <30×10⁹/L on two consecutive visits (≥1 week apart), OR Intolerance to oral TPO-RA leading to discontinuation (with documented reason) Baseline platelet count <30×10⁹/L (at least two measurements, ≥5 days apart) ECOG performance status 0-2 Willing and able to receive weekly subcutaneous injections and comply with study procedures For women of childbearing potential: negative pregnancy test at screening and agreement to use effective contraception during the study and for 3 months after the last dose Able to provide written informed consent
Exclusion Criteria:
Secondary ITP (e.g., associated with autoimmune diseases, lymphoproliferative disorders, drug-induced, or infections such as HIV/HCV/HBV) Known hypersensitivity to romiplostim or any of its excipients Prior treatment with romiplostim Received rituximab or other B-cell depleting therapy within 6 months prior to screening Received immunosuppressants (e.g., cyclosporine, mycophenolate mofetil, azathioprine, danazol) within 4 weeks prior to screening Splenectomy within 8 weeks prior to screening Use of corticosteroids (prednisone >10 mg/day or equivalent) or IVIG within 2 weeks prior to screening History of bone marrow fibrosis (MF grade ≥2) or known reticulin fibrosis History of arterial or venous thromboembolic event (e.g., myocardial infarction, stroke, deep vein thrombosis, pulmonary embolism) within 6 months prior to screening Known thrombophilic conditions (e.g., antiphospholipid syndrome, protein C/S deficiency, ATIII deficiency) Severe hepatic impairment (ALT or AST >3×ULN, or total bilirubin >1.5×ULN) Severe renal impairment (creatinine clearance <30 mL/min) New York Heart Association (NYHA) functional class III or IV heart failure Malignancy within 5 years prior to screening (except non-melanoma skin cancer, cervical carcinoma in situ, or ductal carcinoma in situ of the breast) Pregnancy or breastfeeding Participation in another interventional clinical trial within 30 days prior to screening Any other condition that, in the investigator's judgment, would make the participant unsuitable for the study
Studienplan
Wie ist die Studie aufgebaut?
Designdetails
- Hauptzweck: Behandlung
- Zuteilung: N / A
- Interventionsmodell: Einzelgruppenzuweisung
- Maskierung: Keine (Offenes Etikett)
Waffen und Interventionen
Teilnehmergruppe / Arm |
Intervention / Behandlung |
|---|---|
|
Experimental: Romiplostim Treatment Arm
|
Romiplostim is administered as a subcutaneous injection once weekly for 24 weeks. The starting dose is 3 µg/kg. Platelet count is assessed weekly, and the dose is adjusted based on platelet count and bleeding symptoms as follows: If platelet count <50×10⁹/L: increase dose by 1 µg/kg (maximum dose: 10 µg/kg per week) If platelet count 50-200×10⁹/L: maintain the lowest dose that reduces bleeding risk If platelet count 200-400×10⁹/L: decrease dose by 1 µg/kg If platelet count >400×10⁹/L: withhold dosing. After platelet count decreases to <200×10⁹/L, resume at a dose 1 µg/kg lower than the dose before withholding. |
Was misst die Studie?
Primäre Ergebnismessungen
Ergebnis Maßnahme |
Maßnahmenbeschreibung |
Zeitfenster |
|---|---|---|
|
Durable Platelet Response Rate at Week 24
Zeitfenster: 24 weeks
|
Defined as the proportion of participants achieving platelet count ≥50×10⁹/L for at least two scheduled visits during weeks 22-24, without receiving rescue therapy (IVIG, high-dose corticosteroids, or platelet transfusion) during weeks 20-24.
|
24 weeks
|
Mitarbeiter und Ermittler
Mitarbeiter
Studienaufzeichnungsdaten
Haupttermine studieren
Studienbeginn (Tatsächlich)
Primärer Abschluss (Geschätzt)
Studienabschluss (Geschätzt)
Studienanmeldedaten
Zuerst eingereicht
Zuerst eingereicht, das die QC-Kriterien erfüllt hat
Zuerst gepostet (Tatsächlich)
Studienaufzeichnungsaktualisierungen
Letztes Update gepostet (Tatsächlich)
Letztes eingereichtes Update, das die QC-Kriterien erfüllt
Zuletzt verifiziert
Mehr Informationen
Begriffe im Zusammenhang mit dieser Studie
Schlüsselwörter
Zusätzliche relevante MeSH-Bedingungen
- Zytopenie
- Pathologische Prozesse
- Autoimmunerkrankungen
- Erkrankungen des Immunsystems
- Blutung
- Hautmanifestationen
- Hämatologische Erkrankungen
- Blutgerinnungsstörungen
- Hämorrhagische Störungen
- Erkrankungen der Blutplättchen
- Thrombotische Mikroangiopathien
- Purpura
- Thrombozytopenie
- Pathologische Zustände, Anzeichen und Symptome
- Anzeichen und Symptome
- Hämische und lymphatische Krankheiten
- Purpura, thrombozytopenisch, idiopathisch
- Purpura, Thrombozytopenie
- Romiplostim
Andere Studien-ID-Nummern
- 20251200
Plan für individuelle Teilnehmerdaten (IPD)
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Arzneimittel- und Geräteinformationen, Studienunterlagen
Studiert ein von der US-amerikanischen FDA reguliertes Arzneimittelprodukt
Studiert ein von der US-amerikanischen FDA reguliertes Geräteprodukt
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