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

NCT04596995 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: TP00042019-000883-40
• Study Type: interventional
• Target: 43
• Locations: 14 countries
• Sites: 27

Brief Summary

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

Conditions

Primary Immune Thrombocytopenia

Keywords

ITP; UCB7665; Rozanolixizumab; Primary immune thrombocytopenia

Outcome Measures

Primary Outcomes (2)

• Percentage of Participants With Treatment-emergent Adverse Events (TEAEs)

  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. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.

  From Baseline to end of Safety Follow-Up Period (up to Week 60)

• Percentage of Participants With TEAEs Leading to Permanent Withdrawal of Rozanolixizumab (ie, Study Discontinuation)

  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. TEAEs are defined as AEs starting after the time of first IMP administration up to and including 8 weeks (56 days) after the final dose.

  From Baseline to end of Safety Follow-Up Period (up to Week 60)

Secondary Outcomes (2)

• Percentage of Participants With 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 52-week Treatment Period (starting at Week 4)

• Change From Baseline in Immune Thrombocytopenia-Patient Assessment Questionnaire (ITP-PAQ) to Week 53 or 55 Symptoms Domain Score

  Week 53 or 55, compared to Baseline

Study Arms (1)

Rozanolixizumab Treatment Arm

EXPERIMENTAL

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

Interventions

Rozanolixizumab DRUG

Study participants receive rozanolixizumab by subcutaneous infusion during the Treatment Period.

Also known as: UCB7665

Rozanolixizumab Treatment Arm

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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)

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (27)

Tp0004 50243

Boston, Massachusetts, 02114, United States

Location

Tp0004 20179

Fuzhou, China

Location

Tp0004 20185

Jinan, China

Location

Tp0004 20194

Wuxi, China

Location

Tp0004 20050

Tbilisi, Georgia

Location

Tp0004 40369

Berlin, Germany

Location

Tp0004 40202

Győr, Hungary

Location

Tp0004 40178

Nyíregyháza, Hungary

Location

Tp0004 40208

Florence, Italy

Location

Tp0004 20039

Iruma-gun, Japan

Location

Tp0004 20051

Chisinau, Moldova

Location

Tp0004 40218

Gdansk, Poland

Location

Tp0004 40222

Skorzewo, Poland

Location

Tp0004 40219

Słupsk, Poland

Location

Tp0004 40223

Warsaw, Poland

Location

Tp0004 20052

Moscow, Russia

Location

Tp0004 20053

Saint Petersburg, Russia

Location

Tp0004 40268

Madrid, Spain

Location

Tp0004 20095

Taipei, Taiwan

Location

Tp0004 20099

Taipei, Taiwan

Location

Tp0004 20061

Cherkasy, Ukraine

Location

Tp0004 20060

Dnipropetrovsk, Ukraine

Location

Tp0004 20062

Ivano-Frankivsk, Ukraine

Location

Tp0004 20063

Kyiv, Ukraine

Location

Tp0004 20064

Kyiv, Ukraine

Location

Tp0004 20100

Zaporizhzhia, Ukraine

Location

Tp0004 40234

Plymouth, United Kingdom

Location

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

rozanolixizumab

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: UCB
• Organization: Cares

UCBCares@ucb.com

001 844 599 2273

Study Officials

• UCB Cares

  001 844 599 2273

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: GT60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 15, 2020
• First Posted: October 22, 2020
• Study Start: January 6, 2021
• Primary Completion: December 21, 2022
• Study Completion: December 21, 2022
• Last Updated: February 5, 2024
• Results First Posted: February 5, 2024

Record last verified: 2024-01

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data from this trial may be requested by qualified researchers six months after product approval in the US and/or Europe or global development is discontinued, and 18 months after trial completion.
• Access Criteria: Qualified researchers may request access to anonymized IPD and redacted study documents which may include: raw datasets, analysis-ready datasets, study protocol, blank case report form, annotated case report form, statistical analysis plan, dataset specifications, and clinical study report. Prior to use of the data, proposals need to be approved by an independent review panel at www.Vivli.org and a signed data sharing agreement will need to be executed. All documents are available in English only, for a pre-specified time, typically 12 months, on a password protected portal.

Locations

DE (1); RU (2); ES (1); IT (1); CN (3); MO (1); HU (2); PL (4); US (1); GE (1); TW (2); GB (1); UA (6); JP (1)