myOpportunITy2: A Study to Assess the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Sponsor

UCB Biopharma SRL (Industry)

Overall Status

Terminated

CT.gov ID

NCT04224688

Collaborator

(none)

Enrollment

Locations

Arms

Actual Duration (Months)

0.6

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment and assess safety and tolerability of rozanolixizumab in adult study participants with primary immune thrombocytopenia (ITP).

Condition or Disease	Intervention/Treatment	Phase
Primary Immune Thrombocytopenia	Drug: Rozanolixizumab Other: Placebo	Phase 3

Study Design

Study Type:

Interventional

Actual Enrollment :

30 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Masking:

Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)

Masking Description:

Investigators are blinded to the treatment code, they will see the platelet values.

Primary Purpose:

Treatment

Official Title:

A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Actual Study Start Date :

Jun 3, 2020

Actual Primary Completion Date :

Apr 25, 2022

Actual Study Completion Date :

May 5, 2022

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Rozanolixizumab Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.	Drug: Rozanolixizumab Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points. Other Names: UCB7665
Placebo Comparator: Placebo Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.	Other: Placebo Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Arm

Intervention/Treatment

Experimental: Rozanolixizumab

Study participants randomized to this arm will receive fixed-unit doses of rozanolixizumab across body weight tiers at pre-specified time points during the Treatment Period. Doses will be adjusted based on platelet count values or medical needs.

Drug: Rozanolixizumab

Study participants receive rozanolixizumab by subcutaneous infusion at pre-specified time points.

Other Names:

UCB7665

Placebo Comparator: Placebo

Study participants randomized to this arm receive placebo at pre-specified time points during the Treatment Period.

Other: Placebo

Study participants receive placebo by subcutaneous infusion at pre-specified time points.

Outcome Measures

Primary Outcome Measures

Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks [During the last 12 weeks (Week 13 to Week 25)]
Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at least 8 out of 12 weeks during the last 12 weeks (Week 13 to 25)

Secondary Outcome Measures

Cumulative number of weeks with Clinically Meaningful Platelet Response of ≥50×10^9/L over the 24-week Treatment Period [From Baseline during Treatment Period (up to Week 25)]
Total number of weeks with platelet counts ≥50×10^9/L over the 24-week Treatment Period of the study (Week 1 to Week 25).
Time to first Clinically Meaningful Platelet Response of ≥50x10^9/L: time from starting treatment to achievement of first response of ≥50×10^9/L [Time from starting treatment to achievement of first response of ≥50×10^9/L (up to Week 25)]
Time to first Clinically Meaningful Platelet Response of ≥50×10^9/L: time from starting treatment to achievement of first response of ≥50×10^9/L
Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8 [Baseline to Day 8]
Clinically meaningful Response defined as: platelet count ≥50×10^9/L.
Response defined as platelet count ≥30×10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit [From Baseline during Treatment Period (up to Week 25)]
Response, defined as platelet count ≥30×10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit.
Time to first rescue therapy [From Baseline to first rescue therapy (up to Week 25)]
Time to first rescue therapy use will be analyzed using a Cox Proportional Hazards model with fixed terms for treatment, splenectomy,degree of thrombocytopenia (platelet count < or ≥ 15×10^9/L), and geographical region.
Change from Baseline to Week 25 in ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score [From Baseline during Treatment Period (up to Week 25)]
The ITP-PAQ is a 44-item disease-specific Health-Related Quality of life (HRQoL) questionnaire developed for use in adults with chronic ITP. It includes 11 scales: Symptoms, Fatigue, Physical Health - Bother, Physical Health - Activity, Emotional Health - Psychological, Emotional Health - Fear, Overall QoL, Social Activity, Women's Reproductive Health - Fertility, Women's Reproductive Health - Menstrual Symptoms, and Work. Each item is rated on a Likert-type scale containing 4 to 7 responses. All item scores are transformed to a 0 to 100 continuum and are weighted equally to derive individual scale scores. Higher scores indicate better health status.
Occurrence of treatment-emergent adverse events (TEAEs) [From Baseline to end of Safety Follow-Up Period (up to Week 32)]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP.
Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP) (ie, study discontinuation) [From Baseline to end of Safety Follow-Up Period (up to Week 32)]
An adverse event (AE) is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of investigational medicinal product (IMP), whether or not considered related to the IMP. An AE can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease (new or exacerbated) temporally associated with the use of IMP.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Study participant must be ≥18 years of age at the time of the Screening Visit
Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit
Study participant has a documented intolerance or insufficient response to two or more appropriate standard of care ITP treatments prior to Screening
Study participants must have prior history of a response to a previous ITP therapy.
If taking allowed drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)
Study participant has a documented history of low platelet count (<30×10^9/L) prior to Screening
Study participant has a platelet count measurement at Screening and at Baseline (Day

with an average of the two <30×10^{9/L and no single count may be >35×10}9/L (using local laboratories)

Study participant has a current or history of a peripheral blood smear consistent with ITP
Study participants may be male or female:

A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period
A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test and not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:

Not a woman of childbearing potential (WOCBP) OR A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment

Exclusion Criteria:

Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires current anticoagulant treatment
Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant drop in hemoglobin)
Study participant has a known hypersensitivity to any components of the study medication or any other anti-neonatal Fc receptor (FcRn) medications
Study participant has evidence of a secondary cause of immune thrombocytopenia (clear association with other medical conditions eg, of untreated H. pylori infection, leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease or is drug induced), participant has a multiple immune cytopenia (eg, Evan's syndrome) etc.
Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the first dose of investigational medicinal product (IMP)
Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant
Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit
Study participant has a history of coagulopathy disorders other than ITP
Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit
Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tp0006 50242	Beverly Hills	California	United States	90211
2	Tp0006 50412	Saint Petersburg	Florida	United States	33709
3	Tp0006 50417	Peoria	Illinois	United States	61615
4	Tp0006 50243	Boston	Massachusetts	United States	02114
5	Tp0006 50500	Kansas City	Missouri	United States	64114
6	Tp0006 50384	Nyack	New York	United States	10960
7	Tp0006 40185	Gent		Belgium
8	Tp0006 40189	Plovdiv		Bulgaria
9	Tp0006 40008	Sofia		Bulgaria
10	Tp0006 20201	Bengbu		China
11	Tp0006 20189	Changchun		China
12	Tp0006 20188	Changsha		China
13	Tp0006 20186	Changzhou		China
14	Tp0006 20203	Changzhou		China
15	Tp0006 20179	Fuzhou		China
16	Tp0006 20187	Hangzhou		China
17	Tp0006 20177	Jinan		China
18	Tp0006 20185	Jinan		China
19	Tp0006 20180	Wuhan		China
20	Tp0006 20194	Wuxi		China
21	Tp0006 40465	Bayonne		France
22	Tp0006 40374	Dijon		France
23	Tp0006 40364	Lille		France
24	Tp0006 40365	Marseille		France
25	Tp0006 40409	Saint Brieuc		France
26	Tp0006 40554	Aschaffenburg		Germany
27	Tp0006 40369	Berlin		Germany
28	Tp0006 40367	Frankfurt am Main		Germany
29	Tp0006 40366	Rostock		Germany
30	Tp0006 40219	Słupsk		Poland
31	Tp0006 40223	Warszawa		Poland
32	Tp0006 20052	Moscow		Russian Federation
33	Tp0006 20054	Moscow		Russian Federation
34	Tp0006 20053	Saint Petersburg		Russian Federation
35	Tp0006 40571	Belgrade		Serbia
36	Tp0006 40572	Belgrade		Serbia
37	Tp0006 40158	Madrid		Spain
38	Tp0006 40231	Madrid		Spain
39	Tp0006 40268	Madrid		Spain
40	Tp0006 40232	Palma De Mallorca		Spain
41	Tp0006 40381	Zaragoza		Spain
42	Tp0006 20096	Changhua		Taiwan
43	Tp0006 20097	Kaohsiung		Taiwan
44	Tp0006 20094	Tainan		Taiwan
45	Tp0006 20095	Taipei City		Taiwan
46	Tp0006 20061	Cherkasy		Ukraine
47	Tp0006 20064	Kyiv		Ukraine
48	Tp0006 40239	Leeds		United Kingdom
49	Tp0006 40055	Norwich		United Kingdom