E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Primary Immune Thrombocytopenia |
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E.1.1.1 | Medical condition in easily understood language |
Primary immune thrombocytopenia is an autoimmune disease which is characterized by an isolated low platelet count (thrombocytopenia) and the absence of other causes of thrombocytopenia. |
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E.1.1.2 | Therapeutic area | Diseases [C] - Blood and lymphatic diseases [C15] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10043554 |
E.1.2 | Term | Thrombocytopenia |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Assess the long-term safety and tolerability of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only)
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E.2.2 | Secondary objectives of the trial |
-Assess the long-term clinical efficacy of repeated treatment with rozanolixizumab (Maintenance Treatment Arm only)
-Assess effect of rozanolixizumab on health-related quality of life (HRQoL)
-Assess the pharmacodynamic (PD) effect of rozanolixizumab (Maintenance Treatment Arm only)
-Assess the effect of rozanolixizumab on patient-reported outcomes (PROs)
-Assess the reduction in use of steroids in study participants receiving rozanolixizumab
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
Study participants who have consented can participate in the pharmacogenomics sub-study. |
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E.3 | Principal inclusion criteria |
-Study participant completed TP0003 [NCT04200456] or TP0006 [NCT04224688] until Visit 28 (Week 25) and, in the opinion of the investigator and sponsor, 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
-If taking allowed concomitant medications, study participant must have been on stable doses
- Study participants may be male or female:
a) 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
b) A female participant is eligible to participate if she is not pregnant as confirmed by a negative urine pregnancy test or not planning to get pregnant during the 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
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E.4 | Principal 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 had any relevant resolved IMP-related SAE or severe IMP-related TEAE experienced during TP0003 or TP0006 that was not discussed and approved as acceptable for enrollment into open-label extension (OLE) study by Medical Monitor or designee
-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) |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Occurrence of treatment-emergent adverse events (TEAEs)
2. Occurrence of TEAEs leading to withdrawal of rozanolixizumab
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. and 2. From Baseline to end of Safety Follow-Up Period (up to Week 61)
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E.5.2 | Secondary end point(s) |
1. Stable Clinically Meaningful Response without rescue therapy at ≥70% of the visits starting after the second dose of rozanolixizumab until Week 54
2. Change from Baseline in European Quality of Life-5 Dimension 5 Levels Assessment (EQ5D-5L)
3. Change from Baseline in Short-Form 36-Item (SF-36) Survey
4. Change from Baseline in ITP-Patient Assessment Questionnaire (ITP-PAQ)
5. Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
6. Minimum value in total serum immunoglobulin G (IgG) concentration over time
7. Maximum decrease from Baseline in total serum IgG concentration over time
8. Change from Baseline in serum IgG subclasses concentration over time
9. Change from Baseline in serum Ig concentrations (IgA, IgE, IgM) over time
10. Change from Baseline in Physical Fatigue Instrument
11. Change from Baseline in Patient Global Impression of Severity (PGI-S)
12. Change from Baseline in Patient Global Impression of Change (PGI-C)
13. Area under the curve (AUC) of the oral steroid dose over time |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. After second dose of rozanolixizumab until Week 54 (up to Week 54)
2.-7.; 12.; 13. From Baseline during the Treatment Period (up to Week 55)
8.-11. From Baseline during the Treatment Period (up to Week 53) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability, Immunogenicity |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 90 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Austria |
Belgium |
Bulgaria |
Canada |
China |
Czech Republic |
France |
Georgia |
Germany |
Hong Kong |
Hungary |
Italy |
Japan |
Moldova, Republic of |
Netherlands |
Poland |
Romania |
Russian Federation |
Spain |
Taiwan |
Ukraine |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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Last visit of the last subject (LVLS) |
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 15 |