IRB Study Number 23-598
Status Recruiting
Institute Taussig Cancer Institute
Description
Primary objective(s)
● To demonstrate that the addition of ianalumab (either dose) to standard first-line corticosteroids prolongs Time to Treatment Failure (TTF) compared to corticosteroids alone in participants with primary ITP who responded to corticosteroids (+/-IVIG) prior to randomization.
Secondary objective(s)
● To assess quality of response, time to and duration of complete response in each treatment group
● To assess the safety profile of ianalumab
● To assess the incidence and severity of bleeding in each treatment arm
● To assess the need of rescue treatment in each treatment group
● To evaluate treatment effects on ITP related symptoms, functioning, and health-related quality of life (HRQoL)
● To assess B-cell and immunoglobulin levels in each treatment group
● To assess ianalumab pharmacokinetics (PK)
● To assess the immunogenicity of ianalumab
Inclusion Criteria
1a. Signed informed consent prior to participation in the study.
2a. Male or female participants aged 18 years and older on the day of signing informed consent
3a. Primary ITP diagnosed within 3 months before initiating first-line ITP therapy (corticosteroids, IVIG).
Platelet count below 30 G/L before starting any first-line ITP therapy (corticosteroids, IVIG).
Response (platelet count ≥50 G/L) to corticosteroids (+/- IVIG) at any time prior to randomization. Note: Platelet counts measured within 7 days of platelet transfusion will not be considered as response.
Exclusion Criteria
Evans syndrome or any other cytopenia
Current life-threatening bleeding
3a. Previous ITP treatment, including splenectomy, except for corticosteroids and/or IVIG for up to 28 days before randomization
4a. Prior use of B-cell depleting therapy (e.g., rituximab).
6a. Absolute neutrophil count below 1.0 G/L at randomization.
7a. Participants with concurrent coagulation disorders and/or receiving anti-platelet or anticoagulant medication with an exemption of low dose of acetylsalicylic acid (see Prohibited medications in Section 6.8.2)
8a. Active viral, bacterial or other infections (including tuberculosis -TB) requiring systemic treatment or SARS-CoV-2 infection during the screening period, or history of recurrent clinically significant infection (eg. bacterial infections with encapsulated organisms). Participants with positive hepatitis C virus antibody (HCV Ab) antibody, hepatitis B surface antigen (HBsAg) or hepatitis B core antibody (HBcAb)
Known history of primary or secondary immunodeficiency, or a positive HIV (ELISA and Western blot) test result
Live or live-attenuated vaccination within 4 weeks before randomization
Nursing or pregnancy (positive serum or urine B-human chorionic gonadotrophin (β-hCG) pregnancy test) at screening or pre-dose on Day 1.
Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective methods of contraception during dosing and for 6 months after last dose of ianalumab. Highly effective contraception methods include:
• Total abstinence (when this is in line with the preferred and usual lifestyle of the subject). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception
• Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) total hysterectomy or tubal ligation at least six weeks before taking investigational drug. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment
• For female participants on the study, the vasectomized male partner should be the sole partner for that participant
• Use of oral, (estrogen and progesterone), injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception
In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking investigational drug.
Women are considered post-menopausal if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g., age-appropriate history of vasomotor symptoms). Women are considered not of child-bearing potential if they are post-menopausal or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks prior to enrollment on study. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow-up hormone level assessment is she considered not of child-bearing potential. If local regulations are more stringent from the contraception methods listed above to prevent pregnancy, local regulations apply and will be described in the informed consent form (ICF).
Previous or concurrent malignancy except for curatively treated non-melanoma skin cancer, in situ cancer (e.g., cervix, breast, bladder, prostate), and cancer in complete remission for at least 3 years and without evidence of recurrence
Any serious and/or unstable pre-existing medical, psychiatric disorder or other conditions that could interfere with participants' safety and efficacy, obtaining informed consent or compliance with the study procedures as per investigator discretion
Known immediate or delayed hypersensitivity reaction or idiosyncrasy to ianalumab or drugs chemically related to ianalumab or excipients that contraindicate their participation
17a. Concurrent participation in an investigational study within 30 days prior to randomization or within 5-half-lives of the investigational product, whichever is longest. Note: parallel enrollment in a disease registry is permitted.
