myOpportunITy3: A Study to Investigate the Long-term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Sponsor

UCB Biopharma SRL (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT04596995

Collaborator

(none)

180

Enrollment

Locations

Arm

35.4

Anticipated Duration (Months)

6.4

Patients Per Site

0.2

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

The purpose of this study is to assess the long-term safety, tolerability and clinical efficacy of treatment with rozanolixizumab.

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

Study Design

Study Type:

Interventional

Anticipated Enrollment :

180 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

An Open-Label Extension Study to Investigate the Long-Term Safety, Tolerability, and Efficacy of Rozanolixizumab in Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)

Actual Study Start Date :

Dec 30, 2020

Anticipated Primary Completion Date :

Dec 11, 2023

Anticipated Study Completion Date :

Dec 11, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Rozanolixizumab Treatment Arm All study participants 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 during the Treatment Period. Other Names: UCB7665

Arm

Intervention/Treatment

Experimental: Rozanolixizumab Treatment Arm

All study participants 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 during the Treatment Period.

Other Names:

UCB7665

Outcome Measures

Primary Outcome Measures

Occurrence of treatment-emergent adverse events (TEAEs) [From Baseline to end of Safety Follow-Up Period (up to Week 60)]
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 permanent withdrawal of rozanolixizumab (ie, study discontinuation) [From Baseline to end of Safety Follow-Up Period (up to Week 60)]
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.

Secondary Outcome Measures

Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4 [Over the planned 52-week Treatment Period (starting at Week 4)]
Stable Clinically Meaningful Response, defined as Clinically Meaningful Response (ie, platelet count ≥50×10^9/L) without rescue therapy at ≥70% of the visits over the planned 52-week Treatment Period starting at Week 4
Change from Baseline to Week 53 including all intermediate timepoints for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms domain score [From Baseline during the Treatment Period (up to Week 53)]
The ITP-PAQ is a 44-item disease-specific quality of life (QoL) 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.
Area under the curve (AUC) of the oral steroid dose over time [From Baseline during the Treatment Period (up to Week 53)]
Area under the curve (AUC) of the oral steroid dose over time
Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time [From Baseline during the Treatment Period (up to Week 53)]
Change in dose and/or frequency of concomitant ITP medications (excluding corticosteroids) over time will be measured.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Study participant completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 27 (Week 25) and, in the opinion of the investigator, has been compliant with the TP0003 or TP0006 study assessments
The study participant is considered reliable and capable of adhering to the protocol, visit schedule, or medication intake according to the judgment of the investigator
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 urine 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 final dose of study treatment

Exclusion Criteria:

Study participant has any ongoing investigational medicinal product (IMP)-related serious adverse event (SAE) or ongoing severe IMP-related treatment-emergent adverse event (TEAE) experienced during TP0003 or TP0006
Study participant has, at last available assessment of TP0003 or TP0006, 3.0x upper limit of normal (ULN) of any of the following: alanine aminotransferase (ALT), aspartate aminotransferase (AST), or alkaline phosphatase (ALP)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Tp0004 50243	Boston	Massachusetts	United States	02114
2	Tp0004 20179	Fuzhou		China
3	Tp0004 20185	Jinan		China
4	Tp0004 20194	Wuxi		China
5	Tp0004 20050	Tbilisi		Georgia
6	Tp0004 40369	Berlin		Germany
7	Tp0004 40202	Győr		Hungary
8	Tp0004 40178	Nyíregyháza		Hungary
9	Tp0004 40208	Firenze		Italy
10	Tp0004 20039	Iruma-Gun		Japan
11	Tp0004 20159	Shibuya-Ku		Japan
12	Tp0004 20051	Chisinau		Moldova, Republic of
13	Tp0004 40218	Gdańsk		Poland
14	Tp0004 40222	Skorzewo		Poland
15	Tp0004 40219	Słupsk		Poland
16	Tp0004 40223	Warszawa		Poland
17	Tp0004 20052	Moscow		Russian Federation
18	Tp0004 20053	Saint Petersburg		Russian Federation
19	Tp0004 40268	Madrid		Spain
20	Tp0004 20095	Taipei city		Taiwan
21	Tp0004 20099	Taipei		Taiwan
22	Tp0004 20061	Cherkasy		Ukraine
23	Tp0004 20060	Dnipropetrovs'k		Ukraine
24	Tp0004 20062	Ivano-Frankivsk		Ukraine
25	Tp0004 20063	Kyiv		Ukraine
26	Tp0004 20064	Kyiv		Ukraine
27	Tp0004 20100	Zaporizhzhia		Ukraine
28	Tp0004 40234	Plymouth		United Kingdom