Efgartigimod IV for adolescents (12–17) with chronic immune thrombocytopenia
A Multicenter, Randomized, Double-blinded, Parallel-Arm, Placebo-Controlled, Pharmacokinetic and Pharmacodynamic Study Followed by an Open-Label Arm to Evaluate Efgartigimod IV in Pediatric Participants From 12 Years to Less Than 18 Years of Age With Chronic ITP
This trial will try intravenous efgartigimod to see if it safely raises platelet counts in people aged 12 to 17 with chronic ITP.
Quick facts
| Phase | Phase2; Phase3 |
|---|---|
| Study type | Interventional |
| Enrollment | 24 (estimated) |
| Ages | 12 Years to 17 Years |
| Sex | All |
| Sponsor | argenx Industry-sponsored |
| Drugs / interventions | rituximab, prednisone |
| Locations | 8 sites (Genoa and 7 other locations) |
| Trial ID | NCT07194850 on ClinicalTrials.gov |
What this trial studies
This phase 2/3 double-blind randomized study enrolls participants aged 12 to less than 18 with primary chronic ITP lasting more than 12 months and low platelet counts. In the double-blind treatment period participants are randomized 2:1 to receive efgartigimod IV or placebo for up to 24 weeks, after which all participants receive open-label efgartigimod for at least one year and may continue into a second year, followed by an ~8-week off-drug follow-up for a total study duration up to 138 weeks. Eligible participants must have prior ITP therapy (corticosteroids, IVIg, anti-D, TPO‑RAs, or rituximab) with documented prior response to at least one therapy and documented insufficient response to others. The primary goal is to confirm the appropriate pediatric dose and characterize safety and effects on platelet counts compared with placebo while collecting longer-term open-label safety data.
Who should consider this trial
Good fit: Ideal candidates are 12 to less than 18 years old with primary chronic ITP for over 12 months, a mean platelet count under 30 x10^9/L, and prior inadequate responses to standard ITP therapies.
Not a fit: Patients with secondary ITP, those with higher platelet counts, people outside the 12–17 age range, or those excluded by other protocol criteria are unlikely to receive benefit from this protocol.
Why it matters
Potential benefit: If successful, this could provide a targeted IV treatment that raises platelet counts, reduces bleeding risk, and may decrease reliance on steroids in adolescents with chronic ITP.
How similar studies have performed: Efgartigimod has demonstrated improved platelet counts in adult ITP studies, so this pediatric program builds on positive adult data.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Is aged 12 to less than 18 years when completing the informed consent process * Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete * Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, IVIg, anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab. * Has documented prior response, defined as 1 platelet count of ≥50 × 10\^9/L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin * Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg, anti-D immunoglobulin, TPO-RAs, rituximab, or splenectomy * Has documented mean platelet count of less than 30 x10\^9/L Exclusion Criteria: * Secondary ITP according to the following definition by the International Working Group (IWG): all forms of immune-mediated thrombocytopenia except primary ITP * Nonimmune thrombocytopenia * ITP-associated critical or severe bleeding * History of hereditary thrombocytopenia
Where this trial is running
Genoa and 7 other locations
- Gaslini Children's Hospital — Genoa, Italy (Recruiting)
- Uniwersytecki Szpital Dzieciecy w Lublinie — Lublin, Poland (Recruiting)
- Institutul Clinic Fundeni — Bucharest, Romania (Recruiting)
- Hospital Universitari Vall d'Hebron — Barcelona, Spain (Recruiting)
- Hospital Sant Joan de Deu Barcelona — Esplugues de Llobregat, Spain (Recruiting)
- Hospital Infantil Universitario Nino Jesus (HIUNJS) — Madrid, Spain (Recruiting)
- Hospital Materno-Infantil Universitario Gregorio Maranon — Madrid, Spain (Recruiting)
- Cardiff and Vale NHS Trust - University Hospital of Wales (UHW) — Cardiff, United Kingdom (Recruiting)
Study contacts
- Study coordinator: Sabine Coppieters, MD
- Email: clinicaltrials@argenx.com
- Phone: 857-350-4834
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.