BridgeMD
A study to assess the effect and safety of efgartigimod IV in participants from 12 years to less than 18 years of age with chronic Immune Thrombocytopenia (ITP)
EU CTIS ID: 2025-521055-23-00
What this study is testing
To confirm an appropriate dose of efgartigimod IV in participants with chronic ITP in the DBTP
- Phase II and Phase III (Integrated)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1A. Is aged 12 to <18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF. Participants who are at the age of majority, according to local regulations, may sign the ICF
- 10) Has documented baseline mean platelet count of <30 × 109/L before randomization on study day 1
- 11) Has 1 documented qualifying platelet count (ie, a platelet count used in the formula for calculating the arithmetic mean) on study day 1 before randomization
- 12) Has at least 2 documented qualifying platelet counts between study day −14 and study day 1 before randomization
- 13) Has at least 3 documented qualifying platelet counts in the 3 months before randomization on study day 1
- 14) Has no documented platelet count of >35 × 109/L within 30 days before randomization on study day 1
You likely can't join if
- 1) Besides the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of chronic ITP or puts the participant at undue risk
- 10) Different IMP received in another clinical study <12 weeks or <5 half-lives (whichever is longer) before screening
- 11) Anti-CD20 or anti-CD19 antibody received <6 months before screening
- 12) IVIg, SCIg, or PLEX received <3 weeks before screening
- 13) Live or live-attenuated vaccine received <4 weeks before treatment
- 14) Prior ITP therapy not discontinued per the defined washout period (refer to Table 10)
See the full eligibility criteria
Who can join
- 1A. Is aged 12 to <18 years when completing the informed consent process, defined as providing informed assent according to local regulations and having a parent or guardian sign the ICF. Participants who are at the age of majority, according to local regulations, may sign the ICF
- 10) Has documented baseline mean platelet count of <30 × 109/L before randomization on study day 1
- 11) Has 1 documented qualifying platelet count (ie, a platelet count used in the formula for calculating the arithmetic mean) on study day 1 before randomization
- 12) Has at least 2 documented qualifying platelet counts between study day −14 and study day 1 before randomization
- 13) Has at least 3 documented qualifying platelet counts in the 3 months before randomization on study day 1
- 14) Has no documented platelet count of >35 × 109/L within 30 days before randomization on study day 1
- 2A. Is capable of completing the informed consent process as described in Section 10.1.3, and can comply with protocol requirements
- 3) If an FAOCBP (defined in Section 10.4.1.1), agrees to use contraceptive measures consistent with local regulations and study requirements defined in Section 10.4.2.1
- 4) If an FAOCBP, has a negative serum pregnancy test at screening and a negative urine pregnancy test at baseline before receiving IMP (Section 10.4.2.1)
- 5) Has a documented diagnosis of primary ITP, as defined by the IWG1,3: a platelet count of <100 × 109 /L in the absence of other causes or disorders that may be associated with thrombocytopenia
- 6A. Has a documented duration of primary ITP of more than 12 months on the date the informed consent process is complete
- 7) Has documented prior ITP treatment with at least 1 of the following: corticosteroids, IVIg, anti-D immunoglobulin (for participants who are nonsplenectomized and Rho[D]-positive), TPO-RAs, or rituximab
- 8) Has documented prior response, defined as 1 platelet count of ≥50 × 109 /L to at least 1 of the following ITP treatments: prednisone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin (for participants who are nonsplenectomized and Rho[D]-positive)
- 9) Has documented insufficient response to a prior ITP treatment with corticosteroids, IVIg,anti-D immunoglobulin (for participants who are nonsplenectomized and Rho[D]-positive), TPO-RAs, rituximab, or splenectomy, as defined by any of the following criteria: a. No platelet count of ≥50 × 109/L after treatment b. Platelet count of ≥50 × 109/L after treatment followed by platelet count <50 × 109/L c. Less than 2-fold increase in platelet count from the pretreatment count d. No platelet count of ≥30 × 109/L after splenectomy e. Platelet count of ≥30 × 109/L after splenectomy followed by platelet count <30 × 109/L f. Reduction in platelet count to <30 × 109/L if tapering corticosteroids g. Ongoing need for continuous prednisone (or corticosteroid equivalent) 5 mg/day to maintain a platelet count of ≥30 × 109 /L and/or to avoid bleeding h. Repeated corticosteroid administration for at least 2 months to maintain a platelet count of ≥30 × 109/L and/or to avoid bleeding
What rules you out
- 1) Besides the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of chronic ITP or puts the participant at undue risk
- 10) Different IMP received in another clinical study <12 weeks or <5 half-lives (whichever is longer) before screening
- 11) Anti-CD20 or anti-CD19 antibody received <6 months before screening
- 12) IVIg, SCIg, or PLEX received <3 weeks before screening
- 13) Live or live-attenuated vaccine received <4 weeks before treatment
- 14) Prior ITP therapy not discontinued per the defined washout period (refer to Table 10)
- 15) Secondary ITP according to the following definition by the IWG1: all forms of immune-mediated thrombocytopenia except primary ITP
- 16) Documented prior arterial or venous thrombosis within 12 months before screening
- 17) Concurrent ITP therapy, except as specified in this protocol (refer to Section 6.9.3)
- 18) Nonimmune thrombocytopenia
- 19) History of hereditary thrombocytopenia
- 2) Serious or severe active infection that is not sufficiently resolved in the investigator’s opinion
- 20) ITP-associated critical or severe bleeding (refer to Table 12)
- 21) Positive serum test result at screening for active infection with any of the following: a. HBV indicative of an acute or chronic infection unless associated with a negative HBV DNA test result b. HCV based on HCV antibody assay unless a negative RNA test result is available (Section 10.2.1.2) c. HIV based on confirmed positive serology results (Section 10.2.1.3)
- 22) At the screening visit, clinically significant laboratory abnormalities as follows: a. Hemoglobin concentration ≤9 g/dL b. Severe renal impairment with eGFR <30 mL/min/1.73 m2. eGFR will be calculated using the bedside Schwartz formula. c. Aspartate aminotransferase and alanine aminotransferase >3.0× the ULN d. Total serum bilirubin concentration >1.5× the ULN e. Total IgG level below the lower limit of normal
- 23) History of malignancy unless considered cured by adequate treatment a. With no evidence of recurrence for ≥3 years before first IMP administration b. One of the following cancers: • Basal cell or squamous cell skin cancer • Carcinoma in situ of the cervix • Carcinoma in situ of the breast • Incidental histological findings of prostate cancer (TNM stage T1a or T1b)
- 24) Modification of concurrent ITP therapy during the screening period
- 25A. Occurrence of rescue ITP therapy during the screening period (from the date the informed consent process is complete to randomization)
- 3A. Recent major surgery (within 3 months of screening) or intention to have major surgery during the study
- 4) Current participation in another interventional clinical study
- 5) Known hypersensitivity to IMP or 1 of its excipients
- This criterion was removed in version 2.0
- 7) Pregnant or lactating state or intention to become pregnant during the study
- 8) Previous participation in an efgartigimod clinical study and at least 1 dose of IMP received
- 9) Monoclonal antibody that is not an anti-CD20 or Fc-fusion protein received <4 weeks before screening
The study team makes the final eligibility decision.
Where it's taking place
- Serbia
- Turkey
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Serbia; Turkey; United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.