A Phase 2 interventional study of LOU064 in Chronic Spontaneous Urticaria, sponsored by Novartis Pharmaceuticals. Completed at 69 sites in 15 countries. Open to participants aged 18 Years to 99 Years. Per ClinicalTrials.gov, last updated 2024-06-20.
Sponsored by Novartis Pharmaceuticals · Phase 2, Interventional, and Treatment
The main objective to assess the long-term safety and tolerability of LOU064 in patients with chronic spontaneous urticaria (CSU) who have participated in study CLOU064A2201 (NCT03926611)
This was an open-label, single-arm, multicenter, long-term safety and tolerability extension study for CSU patients rolling over from study CLOU064A2201 (NCT03926611).
Subjects rolling over from CLOU064A2201 with a weekly Urticaria Activity Score (UAS7)\<16 after the follow-up period at Week 16 were further followed up without receiving LOU064 for up to 12 weeks (observational period). If there was a relapse (UAS7≥16 at least once), the 12-week observational period was terminated, and subjects entered the treatment period. Subjects who never relapsed within 12 weeks completed the study after the observational period without treatment.
Subjects who rolled over from CLOU064A2201 with a UAS7≥16 at Week 12 or Week 16, as well as those subjects who relapsed during the 12-week observational period, were treated with 100 mg LOU064 twice a day (b.i.d.) open-label for 52 weeks. No background medication with a second-generation H1-antihistamine was permitted up to Week 4 of the treatment period. Subjects who completed the treatment period or who discontinued treatment early were followed-up for a minimum duration of 4 weeks. Subjects who had a UAS7≤6 at Week 52 of the treatment period had their follow-up period extended until relapse (UAS7≥16) for up to a total of 16 weeks.
237 studies on the registry are indexed under Urticaria; 26 are open to participants now.
This study's enrollment of 229 is above the median of 61 across 174 interventional studies indexed under Urticaria.
Browse Urticaria studies →Novartis Pharmaceuticals is the lead sponsor of 2,673 studies on the registry; 228 are open to participants now.
Of its 576 completed or terminated interventional studies of FDA-regulated products, 431 (75%) have results posted.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Participants transitioning from the CLOU064A2201 trial must have completed either the Week 12 visit (end of treatment period) or the Week 16 visit (end of follow-up period). They will be assigned to either the treatment period or the observational period based on their UAS7 score (average score from the 7 days prior to the respective visit) as follows:
Key Exclusion Criteria:
Participants with UAS7\<16 at Week 16 of CLOU064A2201 were followed up to 12 weeks without receiving treatment (observational period). If participants relapsed (UAS7≥16 at least once), they were transitioned to the treatment period. Otherwise, they were discontinued from the study. Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered 100 mg of LOU064 b.i.d. open-label for up to 52 weeks.
Drug: LOU064
Participants with a UAS7≥16 at Week 12 or Week 16 in the CLOU064A2201, as well as participants who experienced a relapse during the 12-week observational period, were administered LOU064 50mg capsules b.i.d. (i.e. two capsules of LOU064 50mg in the morning and two capsules of LOU064 50mg in the evening) from Day 1 up to Week 52 of the Treatment period.
Number of Participants With Treatment-emergent Adverse Events (AEs)
An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant. Serious AEs (SAEs) is defined as any AE that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or any other medically significant condition. Treatment-emergent AEs were defined as AEs that either begin on the same day or after the first dose of study medication during the treatment period in the extension study or worsen on the same day or after the first dose of study medication in the extension study and within the minimum of either 28 days post last dose or the end of the study visit. The number of participants with treatment-emergent AEs was summarized.
Time frame: From first dose of treatment up to 28 days after last dose, assessed up to 56 weeks
Change From Baseline in Weekly Urticaria Activity Score (UAS7) at Week 4 of the Treatment Period
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 at Week 4 of the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
Time frame: Baseline, Week 4 of treatment period
Percentage of Participants With Well-controlled Disease (UAS7≤6) at Week 4 of the Treatment Period
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7≤ 6 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.
Time frame: Week 4 of the treatment period
Percentage of Participants With Complete Response (UAS7=0) at Week 4 of the Treatment Period
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7= 0 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.
Time frame: Week 4 of the treatment period
Percentage of Participants With Well-controlled Disease (UAS7≤ 6) Overtime
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The percentage of subjects with UAS7≤ 6 during the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
Time frame: From baseline until Week 52 of the treatment period
Change From Baseline in UAS7 Overtime
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 during the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
Time frame: From baseline until Week 52 of the treatment period
229 subjects were enrolled in the observational period or the treatment period across 72 sites in 15 countries. One subject was a screening failure and, as a result, was not enrolled in either the observational or treatment period.
| Milestone | All Participants |
|---|---|
| Started | 229 |
| Treatment-free cohort | 68 |
| Treatment cohort | 194 |
| Completed | 188 |
| Not completed | 41 |
| Withdrew: Pregnancy | 1 |
| Withdrew: Adverse event | 11 |
| Withdrew: Lack of efficacy | 11 |
| Withdrew: Lost to follow-up | 1 |
| Withdrew: Physician decision | 2 |
| Withdrew: Subject decision | 13 |
| Withdrew: Covid-19 situation | 2 |
An AE refers to any undesirable medical occurrence, such as an unintended sign (including abnormal laboratory findings), symptom, or disease, experienced by a participant. Serious AEs (SAEs) is defined as any AE that results in death, is life-threatening, requires hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, results in a congenital anomaly/birth defect, or any other medically significant condition. Treatment-emergent AEs were defined as AEs that either begin on the same day or after the first dose of study medication during the treatment period in the extension study or worsen on the same day or after the first dose of study medication in the extension study and within the minimum of either 28 days post last dose or the end of the study visit. The number of participants with treatment-emergent AEs was summarized.
| Participants | Treated Cohort |
|---|---|
| AEs | 139 |
| Deaths | 0 |
| Non-fatal Serious AEs (SAEs) | 6 |
| SAE(s) | 6 |
| Discontinued treatment due to any AE(s) | 11 |
| Discontinued treatment due to any SAE(s) | 2 |
| Treatment interruption due to AE(s) | 13 |
| Treatment interruption due to SAE(s) | 1 |
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 at Week 4 of the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
| Score on a Scale | Treated Cohort |
|---|---|
| Change From Baseline in Weekly Urticaria Activity Score (UAS7) at Week 4 of the Treatment Period | -17.58 ± 13.400 |
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7≤ 6 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.
| Percentage of participants | Treated Cohort |
|---|---|
| Percentage of Participants With Well-controlled Disease (UAS7≤6) at Week 4 of the Treatment Period | 51.0 (44.9 to 57.1) |
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. Missing values were imputed by non-responder imputation method regardless of the reason for missingness. The percentage of subjects with UAS7= 0 at Week 4 of the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction.
| Percentage of participants | Treated Cohort |
|---|---|
| Percentage of Participants With Complete Response (UAS7=0) at Week 4 of the Treatment Period | 27.3 (22.2 to 33.1) |
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The percentage of subjects with UAS7≤ 6 during the treatment period was calculated. The 90% confidence interval was derived based on the score method with continuity correction. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
| Percentage of participants | Treated Cohort |
|---|---|
| Baseline | 1.0 (0.2 to 3.5) |
| Week 1 | 37.4 (31.4 to 43.8) |
| Week 4 | 52.7 (46.4 to 58.8) |
| Week 12 | 56.6 (50.1 to 63.0) |
| Week 20 | 62.7 (56.1 to 68.9) |
| Week 28 | 68.5 (61.9 to 74.5) |
| Week 40 | 66.5 (59.6 to 72.7) |
| Week 52 | 68.0 (61.1 to 74.3) |
The Urticaria Activity Score (UAS) is a composite, diary-recorded score with numeric severity intensity ratings (0=none to 3=intense/severe) for the number of wheals (hives) and the intensity of the pruritus (itch) over the past 12 hours (twice daily). The daily UAS is calculated as the average of the morning and evening scores. The UAS7 is the weekly sum of the daily UAS, which is the composite score of the intensity of pruritus and the number of wheals. UAS7 scores ranged from 0 to 42. A higher UAS7 indicated greater urticaria disease activity. A minimum of 4 out of 7 daily scores were needed to calculate the UAS7 values. Otherwise, the weekly score was missing for that week. The change from baseline in UAS7 during the treatment period was calculated. A negative change score from baseline indicates improvement. The UAS7 at baseline was considered as the UAS7 derived over the last 7 days before day 1 of the treatment period.
| Score on a Scale | Treated Cohort |
|---|---|
| Week 1 | -14.76 ± 11.502 |
| Week 4 | -17.58 ± 13.400 |
| Week 12 | -19.37 ± 12.502 |
| Week 20 | -20.61 ± 11.634 |
| Week 28 | -21.54 ± 11.525 |
| Week 40 | -21.25 ± 11.400 |
| Week 52 | -21.82 ± 10.699 |
Collected over In the Treated cohort, treatment-emergent AEs were collected from first dose of treatment up to 28 days post last dose, up to 56 weeks; and deaths were collected from first dose of treatment until end of study, assessed up to 68 weeks. In the Treatment-free cohort, AEs and deaths were collected from start of observation period until relapse (UAS7≥16) or end of observation period, up to 12 weeks.. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment-free Cohort (Observational Period) | 0/68 (0%) | 1/68 (1.5%) | 0/68 (0%) |
| Treated Cohort (Treatment+Follow-up Period) | 0/194 (0%) | 6/194 (3.1%) | 56/194 (28.9%) |
| Event | Treatment-free Cohort (Observational Period) | Treated Cohort (Treatment+Follow-up Period) |
|---|---|---|
| Superficial spreading melanoma stage unspecifiedNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/68 | 0/194 |
| COVID-19 pneumoniaInfections and infestations | 0/68 | 2/194 |
| MelaenaGastrointestinal disorders | 0/68 | 1/194 |
| Chest painGeneral disorders | 0/68 | 1/194 |
| AppendicitisInfections and infestations | 0/68 | 1/194 |
| Tibia fractureInjury, poisoning and procedural complications | 0/68 | 1/194 |
| Ovarian cystReproductive system and breast disorders | 0/68 | 1/194 |
| Event | Treatment-free Cohort (Observational Period) | Treated Cohort (Treatment+Follow-up Period) |
|---|---|---|
| Chronic spontaneous urticariaSkin and subcutaneous tissue disorders | — | 22/194 |
| COVID-19Infections and infestations | — | 16/194 |
| HeadacheNervous system disorders | — | 13/194 |
| EczemaSkin and subcutaneous tissue disorders | — | 10/194 |
| Age, Continuous(Years) | All Participants |
|---|---|
| Median | 45 (18 to 77) |
| Sex: Female, Male(Participants) | All Participants |
|---|---|
| Female | 165 |
| Male | 64 |
| Race/Ethnicity, Customized(Participants) | All Participants |
|---|---|
| White | 181 |
| Black | 2 |
| Asian | 44 |
| Multiple | 1 |
| American Indian or Alaska Native | 1 |
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Plan to share: Yes — Novartis is committed to sharing access to patient-level data and supporting clinical documents from eligible studies with qualified external researchers. Requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to protect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com
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