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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT02718716




Registration number
NCT02718716
Ethics application status
Date submitted
11/03/2016
Date registered
24/03/2016
Date last updated
29/01/2024

Titles & IDs
Public title
Study to Evaluate Safety, Tolerability and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
Scientific title
A Multicenter, Open-label, Multiple-dose Study to Evaluate the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
Secondary ID [1] 0 0
TP0001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Thrombocytopenia 0 0
Condition category
Condition code
Blood 0 0 0 0
Haematological diseases
Blood 0 0 0 0
Other blood disorders
Inflammatory and Immune System 0 0 0 0
Autoimmune diseases

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - UCB7665

Experimental: UCB7665 4 mg/kg - Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 (rozanolixizumab) 4 milligram per kilograms (mg/kg) at 1-week intervals.

Experimental: UCB7665 7 mg/kg - Participants in this arm received 3 sc doses of UCB7665 (rozanolixizumab) 7 mg/kg at 1-week intervals.

Experimental: UCB7665 10 mg/kg - Participants in this arm received 2 sc doses of UCB7665 (rozanolixizumab) 10 mg/kg at 1-week intervals.

Experimental: UCB7665 15 mg/kg - Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 15 mg/kg.

Experimental: UCB7665 20 mg/kg - Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 20 mg/kg.


Treatment: Drugs: UCB7665
* Intervention Type: Biological/Vaccine
* Pharmaceutical Form: Powder for solution for infusion
* Concentration: 100 mg/ml - Route of Administration:

Subcutaneous infusion
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study
Timepoint [1] 0 0
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)

Eligibility
Key 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)
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key 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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
2/03/2016
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
4/02/2019
Sample size
Target
Accrual to date
Final
66
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Tp0001 1101 - Adelaide
Recruitment postcode(s) [1] 0 0
- Adelaide
Recruitment outside Australia
Country [1] 0 0
Bulgaria
State/province [1] 0 0
Pleven
Country [2] 0 0
Bulgaria
State/province [2] 0 0
Sofia
Country [3] 0 0
Czechia
State/province [3] 0 0
Olomouc
Country [4] 0 0
Czechia
State/province [4] 0 0
Praha 10
Country [5] 0 0
Georgia
State/province [5] 0 0
Tbilisi
Country [6] 0 0
Germany
State/province [6] 0 0
Berlin
Country [7] 0 0
Germany
State/province [7] 0 0
Dusseldorf
Country [8] 0 0
Germany
State/province [8] 0 0
Muenchen
Country [9] 0 0
Italy
State/province [9] 0 0
Firenze
Country [10] 0 0
Italy
State/province [10] 0 0
Torino
Country [11] 0 0
Italy
State/province [11] 0 0
Udine
Country [12] 0 0
Italy
State/province [12] 0 0
Vicenza
Country [13] 0 0
Moldova, Republic of
State/province [13] 0 0
Chisinau
Country [14] 0 0
Poland
State/province [14] 0 0
Bialystok
Country [15] 0 0
Poland
State/province [15] 0 0
Gdansk
Country [16] 0 0
Poland
State/province [16] 0 0
Lodz
Country [17] 0 0
Poland
State/province [17] 0 0
Poznan
Country [18] 0 0
Poland
State/province [18] 0 0
Warsaw
Country [19] 0 0
Romania
State/province [19] 0 0
Brasov
Country [20] 0 0
Romania
State/province [20] 0 0
Bucharest
Country [21] 0 0
Romania
State/province [21] 0 0
Craiova
Country [22] 0 0
Spain
State/province [22] 0 0
Madrid
Country [23] 0 0
Spain
State/province [23] 0 0
Valencia
Country [24] 0 0
United Kingdom
State/province [24] 0 0
London
Country [25] 0 0
United Kingdom
State/province [25] 0 0
Truro

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
UCB Biopharma SRL
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Parexel
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/study/NCT02718716
Trial related presentations / publications
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.
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.
Public notes

Contacts
Principal investigator
Name 0 0
UCB Cares
Address 0 0
001 844 599 2273 (UCB)
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT02718716