ThrombocytopeniaPhase 2NCT02718716
Study to Evaluate Safety, Tolerability and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
A Phase 2 study testing UCB7665 in people with Thrombocytopenia, run by UCB Biopharma SRL at 29 sites in 11 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Thrombocytopenia
- What is being tested?
- UCB7665
- Type of treatment
- Vaccines, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 66 participants
- Where
- 29 sitesin 11 countries
- Study dates
- 2 Mar 2016to 4 Feb 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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)
Who cannot join
- 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
Show all 16Show fewer
- 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
Trial locations and contacts
29 sites in 11 countries.
29 sites in 11 countries
Poland5 sites
Tp0001 702
Bialystok
Tp0001 703
Gdansk
Tp0001 701
Lodz
Tp0001 704
Poznan
Tp0001 705
Warsaw
Italy4 sites
Tp0001 502
Florence
Tp0001 506
Torino
Tp0001 503
Udine
Tp0001 505
Vicenza
United Kingdom4 sites
Tp0001 1001
London
Tp0001 1002
London
Tp0001 1003
London
Tp0001 1004
Truro
About this study
Official title: A Multicenter, Open-label, Multiple-dose Study to Evaluate the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
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.
- Conditions
- Thrombocytopenia
- Being tested
- UCB7665
- Keywords
- ITP
- Medical subject headings
- Thrombocytopenia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UCB7665Drug
Also called rozanolixizumab
- Intervention Type: Biological/Vaccine
- Pharmaceutical Form: Powder for solution for infusion
- Concentration: 100 mg/ml - Route of Administration:
Subcutaneous infusion
| Study group | Role | What they receive |
|---|---|---|
| UCB7665 4 mg/kg | Experimental | Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 (rozanolixizumab) 4 milligram per kilograms (mg/kg) at 1-week intervals. Receives: Drug: UCB7665 |
| UCB7665 7 mg/kg | Experimental | Participants in this arm received 3 sc doses of UCB7665 (rozanolixizumab) 7 mg/kg at 1-week intervals. Receives: Drug: UCB7665 |
| UCB7665 10 mg/kg | Experimental | Participants in this arm received 2 sc doses of UCB7665 (rozanolixizumab) 10 mg/kg at 1-week intervals. Receives: Drug: UCB7665 |
| UCB7665 15 mg/kg | Experimental | Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 15 mg/kg. Receives: Drug: UCB7665 |
| UCB7665 20 mg/kg | Experimental | Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 20 mg/kg. Receives: Drug: UCB7665 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2016 (estimated)
- Study start2 Mar 2016
- Primary completion4 Feb 2019
- Study completion4 Feb 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for ThrombocytopeniaStudies of the same condition, open and under way.
- More trials run by UCB Biopharma SRLThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 66 participants (actual)
Sponsor and investigators
- Lead sponsor
- UCB Biopharma SRL
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Parexel
- Responsible party
- Sponsor
- Investigators
- UCB Cares, Study director, 001 844 599 2273 (UCB)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02718716
- Sponsor's protocol ID
- TP0001
- Status last verified
- May 2023
- First submitted
- 11 Mar 2016
- Last updated
- 29 Jan 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
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.
PubMed 32886753Smith 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.
PubMed 30130439
Study at a glance
From the registry record, last updated by the sponsor on 29 Jan 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Open-label, Multiple-dose Study to Evaluate the Safety, Tolerability, and Efficacy of UCB7665 in Subjects With Primary Immune Thrombocytopenia
- ClinicalTrials.gov ID
- NCT02718716
- Acronym / sponsor ID
- TP0001
- Conditions
- Thrombocytopenia
- Keywords
- ITP
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 66 participants (actual)
- Lead sponsor
- UCB Biopharma SRLIndustry (pharma, biotech, devices)
- Collaborators
- Parexel
- Study start
- 2 Mar 2016 (actual)
- Primary completion
- 4 Feb 2019 (actual)
- Study completion
- 4 Feb 2019 (actual)
- First posted
- 24 Mar 2016 (estimated)
- Results
- Posted 29 Jan 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| UCB7665 4 mg/kg | Experimental | Drug: UCB7665Participants in this arm received 5 subcutaneous (sc) doses of UCB7665 (rozanolixizumab) 4 milligram per kilograms (mg/kg) at 1-week intervals. |
| UCB7665 7 mg/kg | Experimental | Drug: UCB7665Participants in this arm received 3 sc doses of UCB7665 (rozanolixizumab) 7 mg/kg at 1-week intervals. |
| UCB7665 10 mg/kg | Experimental | Drug: UCB7665Participants in this arm received 2 sc doses of UCB7665 (rozanolixizumab) 10 mg/kg at 1-week intervals. |
| UCB7665 15 mg/kg | Experimental | Drug: UCB7665Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 15 mg/kg. |
| UCB7665 20 mg/kg | Experimental | Drug: UCB7665Participants in this arm received 1 sc dose of UCB7665 (rozanolixizumab) 20 mg/kg. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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)
Key exclusion criteria (16 listed)
- 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:
- Subject has planned an elective surgical procedure in the coming 6 months
- Subject has evidence of a secondary cause of primary immune thrombocytopenia purpura
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| UCB7665Also: rozanolixizumab* Intervention Type: Biological/Vaccine * Pharmaceutical Form: Powder for solution for infusion * Concentration: 100 mg/ml - Route of Administration: Subcutaneous infusion | Drug | UCB7665 10 mg/kg, UCB7665 15 mg/kg, UCB7665 20 mg/kg, UCB7665 4 mg/kg, UCB7665 7 mg/kg |
Primary outcome
Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study
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)
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.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jan 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 32886753Robak 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.
- Derived from this study: PubMed 30130439Smith 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.
- FDA Safety Alerts and RecallsLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
UCB Cares study director
001 844 599 2273 (UCB)
29 sites in 11 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing UCB7665 in people with Thrombocytopenia, run by UCB Biopharma SRL at 29 sites in 11 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 Jan 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 29 sites in Poland, Italy, United Kingdom, Germany, Romania and 6 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 29 Jan 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Experiencing at Least One Treatment Emergent Adverse Event (TEAE) During the Study (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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02718716, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jan 2024.