Clinical trial NCT02718716: Study to Evaluate Safety, Tolerability and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
Official title: A Multicenter, Open-label, Multiple-dose Study to Evaluate the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Thrombocytopenia
Keywords: ITP
Lead sponsor: UCB Pharma
Sponsor class: INDUSTRY
Collaborators: name: Parexel
First posted: 2016-03-24
Enrollment: 66
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study
timeFrame: 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)
Secondary outcomes:
Other outcomes:
Locations:
city: Adelaide
country: Australia
facility: Tp0001 1101
city: Pleven
country: Bulgaria
facility: Tp0001 1302
city: Sofia
country: Bulgaria
facility: Tp0001 1301
city: Olomouc
country: Czechia
facility: Tp0001 203
city: Praha 10
country: Czechia
facility: Tp0001 201
city: Tbilisi
country: Georgia
facility: Tp0001 1201
city: Berlin
country: Germany
facility: Tp0001 401
city: Dusseldorf
country: Germany
facility: Tp0001 403
city: Muenchen
country: Germany
facility: Tp0001 404
city: Firenze
country: Italy
facility: Tp0001 502
city: Torino
country: Italy
facility: Tp0001 506
city: Udine
country: Italy
facility: Tp0001 503
city: Vicenza
country: Italy
facility: Tp0001 505
city: Chisinau
country: Moldova, Republic of
facility: Tp0001 601
city: Bialystok
country: Poland
facility: Tp0001 702
city: Gdańsk
country: Poland
facility: Tp0001 703
city: Lodz
country: Poland
facility: Tp0001 701
city: Poznan
country: Poland
facility: Tp0001 704
city: Warsaw
country: Poland
facility: Tp0001 705
city: Brasov
country: Romania
facility: Tp0001 802
city: Bucharest
country: Romania
facility: Tp0001 801
city: Craiova
country: Romania
facility: Tp0001 803
city: Madrid
country: Spain
facility: Tp0001 902
city: Madrid
country: Spain
facility: Tp0001 903
city: Valencia
country: Spain
facility: Tp0001 901
city: London
country: United Kingdom
facility: Tp0001 1001
city: London
country: United Kingdom
facility: Tp0001 1002
city: London
country: United Kingdom
facility: Tp0001 1003
city: Truro
country: United Kingdom
facility: Tp0001 1004
Study officials:
affiliation: 001 844 599 2273 (UCB)
name: UCB Cares
role: STUDY_DIRECTOR
References:
citation: Robak T, Kazmierczak M, Jarque I, Musteata V, Trelinski J, Cooper N, Kiessling P, Massow U, Woltering F, Snipes R, Ke J, Langdon G, Bussel JB, Jolles S. Phase 2 multiple-dose study of an FcRn inhibitor, rozanolixizumab, in patients with primary immune thrombocytopenia. Blood Adv. 2020 Sep 8;4(17):4136-4146. doi: 10.1182/bloodadvances.2020002003.
citation: Smith B, Kiessling A, Lledo-Garcia R, Dixon KL, Christodoulou L, Catley MC, Atherfold P, D'Hooghe LE, Finney H, Greenslade K, Hailu H, Kevorkian L, Lightwood D, Meier C, Munro R, Qureshi O, Sarkar K, Shaw SP, Tewari R, Turner A, Tyson K, West S, Shaw S, Brennan FR. Generation and characterization of a high affinity anti-human FcRn antibody, rozanolixizumab, and the effects of different molecular formats on the reduction of plasma IgG concentration. MAbs. 2018 Oct;10(7):1111-1130. doi: 10.1080/19420862.2018.1505464. Epub 2018 Sep 12.