E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Chronic Spontaneous Urticaria |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
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 | 10072757 |
E.1.2 | Term | Chronic spontaneous urticaria |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To characterize the dose-response relationship of LOU064 administered once or twice daily in subjects with CSU with respect to change from baseline in UAS7 at Week 4 |
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E.2.2 | Secondary objectives of the trial |
To evaluate:
- the efficacy of LOU064 compared to placebo with respect to change from baseline in UAS7 at Week 12
- the efficacy of LOU064 compared to placebo with respect to change from baseline in UAS7 over time
- the efficacy of LOU064 compared to placebo with respect to achievement of complete clinical response (UAS7= 0) over time
- the efficacy of LOU064 compared to placebo with respect to achievement of disease control (UAS7≤ 6) over time
- the effect of LOU064 on angioedema (AAS7) with respect to the number of weeks with an AAS7= 0 response from baseline through Week 12
- the effect of LOU064 on disease-related quality of life with respect to achievement of a DLQI score of 0 or 1 at Week 4 and Week 12
- the effect of LOU064 on CSU related quality of life with respect to change from baseline in DLQI at Week 4 and Week 12
- the pharmacokinetics of LOU064 resulting from oral dosing at Week 4 and Week 12
- safety and tolerability of LOU064 in subjects with CSU |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Signed informed consent must be obtained prior to participation in the study
2. Male and female subjects aged ≥18 years of age
3. CSU diagnosis for ≥ 6 months prior to screening
4. Diagnosis of CSU inadequately controlled by second generation H1-antihistamines at the time of randomization as defined in the following:
- The presence of itch and hives for ≥6 consecutive weeks prior to screening in spite of use of non-sedating H1-antihistamines according to local treatment guidelines during this time period
- UAS7 score (range 0-42) ≥16 and HSS7 score (range 0-21) ≥ 8 during 7 days prior to randomization (Day 1)
5. Willing and able to complete an Urticaria Participant Daily eDiary (UPDD) for the duration of the study
Other protocol-defined inclusion criteria may apply |
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E.4 | Principal exclusion criteria |
1. Hypersensitivity to any of the study treatments
2. Clearly defined, predominant or sole trigger of their chronic urticaria (chronic inducible urticaria)
3. Other diseases with symptoms of urticaria or angioedema
4. Other skin disease associated with chronic itching that might influence in the investigators opinion the study evaluations and results
5. Known or suspected history of an ongoing, chronic or recurrent infectious disease
including but not limited to opportunistic infections (eg tuberculosis, atypical
mycobacterioses, listeriosis or aspergillosis), HIV, Hepatitis B/C.
6. Pregnant or nursing (lactating) women
7. Women of child-bearing potential not using highly effective methods of contraception
Other protocol-defined exclusion criteria may apply |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in weekly Urticaria Activity Score (UAS7) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Change from baseline in UAS7 at Week 12
- Change from baseline in UAS7 over time
- Complete absence of hives and itch, assessed as UAS7= 0 response over time
- Disease control (UAS7≤ 6) over time
- Cumulative number of weeks with an AAS7= 0 response between baseline and Week 12
- DLQI score of 0 or 1 at Week 4 and 12
- Change from baseline in DLQI score at Week 4 and 12
- Concentrations of LOU064 in blood and calculation of respective PK parameters at
Week 4 and Week 12
- Safety endpoints will include but not be limited to:
- Occurrence of treatment emergent adverse events during the study
- Occurrence of treatment emergent serious adverse events during the study |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 |
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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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 7 |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 47 |
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 |
Argentina |
Belgium |
Canada |
Czech Republic |
Denmark |
France |
Germany |
Hungary |
Japan |
Netherlands |
Poland |
Slovakia |
Spain |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | No |
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
|
Study completion is defined as when the last subject finishes their Study Completion visit, and any repeat assessments associated with this visit have been documented and followed-up appropriately by the investigator, or in the event of an early study termination decision, the date of that decision. |
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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 | 1 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 6 |
E.8.9.2 | In all countries concerned by the trial days | 0 |