Thrombocytopenia
Conditions
Keywords
ITP
Brief summary
The primary objective of the study is to check if an subcutaneous (sc) infusion of UCB7665 is safe and tolerated in subjects with primary immune thrombocytopenia.
Interventions
DRUGUCB7665
* Intervention Type: Biological/Vaccine * Pharmaceutical Form: Powder for solution for infusion * Concentration: 100 mg/ml - Route of Administration: Subcutaneous infusion
Sponsors
Parexel
UCB Biopharma SRL
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subject has a diagnosis of primary immune thrombocytopenia (ITP) for a minimum of 3 months prior to Screening Visit * Subject has a platelet count \<30x10\^9/L at Screening and \<35x10\^9/L at Baseline (Visit 2) * Subject has a current or history of a peripheral blood smear consistent with ITP * Subject has responded to previous ITP therapy (according to the judgment of the investigator)
Exclusion criteria
* Subject has an immunoglobulin G (IgG) level \<=6g/L at Screening Visit * Subject has a partial thromboplastin time (PTT) \>=1.5x upper limit of normal (ULN) or International Normalized Ratio (INR) \>=1.5 at Screening Visit * Subject has renal and/or liver impairment defined as: * Serum creatinine level of \>=1.4 mg/dL for females and \>=1.5 mg/dL for males at Screening Visit * Subject has planned an elective surgical procedure in the coming 6 months * Subject has evidence of a secondary cause of primary immune thrombocytopenia purpura * Subject has a history of clinically relevant ongoing chronic infections * Subject has a family history of primary immunodeficiency * Subject has a clinically relevant active infection or has had a serious infection within 6 weeks prior to the first dose of IMP * Subject has a history of known inflammatory bowel disease, diverticular disease, and gastric or esophageal ulceration * Subject has experienced gastrointestinal bleed in the last 6 months prior to Screening Visit and/or has current gastritis or esophagitis * Subject has a medical history of thrombosis * Subject has a history of coagulopathy disorders other than ITP * Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP * Subject has had prior treatment with rituximab in the 6 months prior to the Baseline Visit * Subject has not completed the washout period for the immunosuppressants, biologics and other therapies
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | From Visit 2 (Week 1) until End of Study Visit or Early Termination (up to 12 weeks after the first investigational medicinal product (IMP) administration) | TEAEs were defined as Adverse Events starting after the time of first Investigational Medicinal Product (IMP) administration up to and including 8 weeks after the final dose. |
Countries
Australia, Bulgaria, Czechia, Georgia, Germany, Italy, Moldova, Poland, Romania, Spain, United Kingdom
Participant flow
Recruitment details
The study started to enroll patients in March 2016 and concluded in February 2019.
Pre-assignment details
The study included a Screening Period (1 to 28 days), a Dosing Period of 1 to 4 weeks, and an Observation Period of 8 weeks. Participant Flow refers to the Safety Set.
Participants by arm
| Arm | Count |
|---|---|
| UCB7665 4 mg/kg Participants in this arm received 5 sc doses of UCB7665 (rozanolixizumab) 4 mg/kg at 1-week intervals. | 15 |
| UCB7665 7 mg/kg Participants in this arm received 3 sc doses of UCB7665 (rozanolixizumab) 7 mg/kg at 1-week intervals. | 15 |
| UCB7665 10 mg/kg Participants in this arm received 2 sc doses of UCB7665 (rozanolixizumab) 10 mg/kg at 1-week intervals. | 12 |
| UCB7665 15 mg/kg Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 15 mg/kg. | 12 |
| UCB7665 20 mg/kg Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 20 mg/kg. | 12 |
| Total Title | 66 |
| Total | 132 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 | FG003 | FG004 |
|---|---|---|---|---|---|---|
| Overall Study | Lack of Efficacy | 1 | 0 | 0 | 0 | 0 |
Baseline characteristics
| Characteristic | UCB7665 4 mg/kg | UCB7665 7 mg/kg | UCB7665 10 mg/kg | UCB7665 15 mg/kg | UCB7665 20 mg/kg | Total Title |
|---|---|---|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 8 Participants | 1 Participants | 2 Participants | 1 Participants | 5 Participants | 17 Participants |
| Age, Categorical Between 18 and 65 years | 7 Participants | 14 Participants | 10 Participants | 11 Participants | 7 Participants | 49 Participants |
| Age, Continuous | 59.1 years STANDARD_DEVIATION 18.4 | 46.0 years STANDARD_DEVIATION 15.9 | 46.3 years STANDARD_DEVIATION 16.8 | 45.8 years STANDARD_DEVIATION 14.6 | 56.1 years STANDARD_DEVIATION 18.2 | 50.8 years STANDARD_DEVIATION 17.4 |
| Race/Ethnicity, Customized Asian | 2 Participants | 0 Participants | 0 Participants | 0 Participants | 0 Participants | 2 Participants |
| Race/Ethnicity, Customized White | 13 Participants | 15 Participants | 12 Participants | 12 Participants | 12 Participants | 64 Participants |
| Sex: Female, Male Female | 8 Participants | 11 Participants | 7 Participants | 7 Participants | 9 Participants | 42 Participants |
| Sex: Female, Male Male | 7 Participants | 4 Participants | 5 Participants | 5 Participants | 3 Participants | 24 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk |
|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 15 | 0 / 15 | 0 / 12 | 0 / 12 | 0 / 12 |
| other Total, other adverse events | 11 / 15 | 9 / 15 | 7 / 12 | 10 / 12 | 12 / 12 |
| serious Total, serious adverse events | 1 / 15 | 0 / 15 | 1 / 12 | 2 / 12 | 0 / 12 |
Outcome results
Primary
Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study
TEAEs were defined as Adverse Events starting after the time of first Investigational Medicinal Product (IMP) administration up to and including 8 weeks after the final dose.
Time frame: From Visit 2 (Week 1) until End of Study Visit or Early Termination (up to 12 weeks after the first investigational medicinal product (IMP) administration)
Population: The Safety Set (SS) consisted of all study participants who had received at least 1 infusion (full or partial infusion) of rozanolixizumab and was used for the analysis of safety data.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| UCB7665 4 mg/kg (SS) | Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | 80.0 percentage of participants |
| UCB7665 7 mg/kg (SS) | Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | 60.0 percentage of participants |
| UCB7665 10 mg/kg (SS) | Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | 58.3 percentage of participants |
| UCB7665 15 mg/kg (SS) | Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | 91.7 percentage of participants |
| UCB7665 20 mg/kg (SS) | Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study | 100 percentage of participants |