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

NCT04224688 | Terminated Phase 3 Results DMC FDA Drug

• 2 other identifiers: TP00062019-003451-11
• Study Type: interventional
• Target: 30
• Locations: 11 countries
• Sites: 29

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).

Conditions

Primary Immune Thrombocytopenia

Keywords

ITP; UCB7665; Rozanolixizumab; Primary Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10^9/L, for at Least 8 Out of 12 Weeks During the Last 12 Weeks

  Percentage of Participants With Durable Clinically Meaningful Platelet Response of ≥50×10\^9/L, for at least 8 out of 12 weeks during the last 12 weeks were reported.

  During the last 12 weeks (Week 13 to Week 25)

Secondary Outcomes (8)

• Cumulative Number of Weeks With Clinically Meaningful Platelet Response of ≥50×10^9/L Over the 24-week Treatment Period

  Week 1 up to Week 25

• Time to First Clinically Meaningful Platelet Response (CMPR) of ≥50×10^9/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)

• Percentage of Participants With Clinically Meaningful Platelet Response of ≥50×10^9/L by Day 8

  Baseline to Day 8

• Percentage of Participants With Response Defined as Platelet Count ≥30*10^9/L and at Least Doubling of Baseline, at Least 2 Separate Occasions at Two Adjacent Nominal Visits at Least 7 Days Apart, and Absence of Bleeding

  From Baseline during Treatment Period (up to Week 25)

• Time to First Rescue Therapy

  From Baseline to first rescue therapy (up to Week 25)

Study Arms (2)

Rozanolixizumab

EXPERIMENTAL

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

Placebo

PLACEBO COMPARATOR

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

Other: Placebo

Interventions

Rozanolixizumab DRUG

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

Also known as: UCB7665

Rozanolixizumab

Placebo OTHER

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

Placebo

Eligibility Criteria

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

You may qualify if:

• 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 1) 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

You may not qualify if:

• 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

Sponsors & Collaborators

• UCB Biopharma SRL: lead

Study Sites (29)

Tp0006 50412

St. Petersburg, Florida, 33709, United States

Location

Tp0006 50243

Boston, Massachusetts, 02114, United States

Location

Tp0006 40189

Plovdiv, Bulgaria

Location

Tp0006 40008

Sofia, Bulgaria

Location

Tp0006 20201

Bengbu, China

Location

Tp0006 20189

Changchun, China

Location

Tp0006 20188

Changsha, China

Location

Tp0006 20186

Changzhou, China

Location

Tp0006 20179

Fuzhou, China

Location

Tp0006 20187

Hangzhou, China

Location

Tp0006 20177

Jinan, China

Location

Tp0006 20185

Jinan, China

Location

Tp0006 20194

Wuxi, China

Location

Tp0006 40364

Lille, France

Location

Tp0006 40369

Berlin, Germany

Location

Tp0006 40366

Rostock, Germany

Location

Tp0006 40219

Słupsk, Poland

Location

Tp0006 40223

Warsaw, Poland

Location

Tp0006 20052

Moscow, Russia

Location

Tp0006 20054

Moscow, Russia

Location

Tp0006 20053

Saint Petersburg, Russia

Location

Tp0006 40231

Madrid, Spain

Location

Tp0006 40268

Madrid, Spain

Location

Tp0006 40381

Zaragoza, Spain

Location

Tp0006 20094

Tainan, Taiwan

Location

Tp0006 20095

Taipei, Taiwan

Location

Tp0006 20061

Cherkasy, Ukraine

Location

Tp0006 20064

Kyiv, Ukraine

Location

Tp0006 40239

Leeds, United Kingdom

Location

Related Links

• FDA Safety Alerts and Recalls

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: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: Investigators are blinded to the treatment code, they will see the platelet values.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 8, 2020
• First Posted: January 13, 2020
• Study Start: June 3, 2020
• Primary Completion: April 25, 2022
• Study Completion: May 5, 2022
• Last Updated: August 8, 2023
• Results First Posted: August 8, 2023

Record last verified: 2023-08

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, CSR
• Time Frame: Data from this study 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

PL (2); FR (1); ES (3); CN (9); GB (1); TW (2); UA (2); RU (3); US (2); DE (2); BU (2)