Idiopathic Thrombocytopenic Purpura, Idiopathic Thrombocytopenic Purpura (ITP), Immune Thrombocytopenia (ITP), Immune Thrombocytopenic Purpura, Immune Thrombocytopenic Purpura ( ITP ), ITP, ITP - Immune Thrombocytopenia
Conditions
Brief summary
The main purpose of this study is to confirm the correct dose of efgartigimod IV for treating patients aged 12 to younger than 18 years with chronic immune thrombocytopenia (ITP). The study consists of a double-blinded treatment period (DBTP) in which the participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV. At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first year open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second year (OLTP2). After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancejunior
Interventions
BIOLOGICALEfgartigimod IV
Intravenous infusion of efgartigimod
OTHERPlacebo IV
Intravenous infusion of placebo
Sponsors
argenx
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
12 Years to 17 Years
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Efgartigimod serum concentrations in the DBTP | Up to 24 weeks |
| Total IgG levels in the DBTP | Up to 24 weeks |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Efgartigimod serum concentrations over time during the DBTP | Up to 24 weeks | — |
| Percent change from baseline in total IgG levels in serum over time during the DBTP | Up to 24 weeks | — |
| Incidence of AEs, SAEs and AEs leading to IMP discontinuation | Up to 136 weeks | SAE: Serious adverse event; AE: adverse event |
| Sustained platelet count response between study weeks 19 and 24 of the DBTP and in OLTP1 for participants receiving placebo in the DBTP | Up to 48 weeks | Sustained platelet count defined as achieving platelet counts of ≥50 × 10\^9/L for at least 4 of the 6 study visits |
| Extent of disease control during the DBTP and during the first 24 weeks of OLTP1 for those participants receiving placebo in the DBTP | Up to 48 weeks | Extend of disease defined as the number of cumulative weeks with a platelet count of ≥50 × 10\^9/L |
| Changes from baseline for platelet counts over time | Up to 76 weeks | — |
| Incidence of bleeding, assessed by the Modified Buchanan and Adix Bleeding Score for pediatric ITP | Up to 76 weeks | The Modified Buchanan and Adix Bleeding Score for pediatric ITP is a semiquantitative assessment tool that measures bleeding signs and symptoms, comprising a score based on a scale of 0 to 5, each representing a different level of severity (0 = no risk; 5 = highest severity). |
| Incidence of ADA and Nab against efgartigimod in serum | Up to 76 weeks | ADA: anti-drug antibodies; Nab: neutralizing antibodies |
| Change from baseline in EQ-5D-5L | Up to 76 weeks | The European Quality of Life 5 Dimensions 5 Level (EQ-5D-5L) is a questionnaire comprised of 5 dimensions. Participants are asked to select the statement in each dimension which best describes their health on the day they complete the questionnaire. Responses in each dimension are coded as a 1-digit number ranging from 1 (no problems) to 5 (extreme problems). |
| Change from baseline in KIT Child Self-Report and KIT Parent Impact Report | Up to 76 weeks | The Kids' ITP Tools (KIT) comprises two disease- specific tools: a self-report form for children aged 12 and older, and a parent impact form. Respondents provide insights into their disease experience using a 1-week recall period. The instrument generates a total score, calculated by summing the items and converting them to a scale of 0 to 100, where higher scores reflect a better disease-specific quality of life (QoL). |
| Change from baseline in peds FACIT-F | Up to 76 weeks | In all participants, the pediatric Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-F) will use to assess health-related quality of life (HRQoL) before and after treatment. The FACIT-F questionnaire has a score range of 0 to 52, where 0 represents the worst possible fatigue and 52 indicates no fatigue. |
Countries
France, Germany, Italy, Lithuania, Poland, Romania, Serbia, Spain, United Kingdom
Contacts
CONTACTSabine Coppieters, MD
Outcome results
None listed