A Phase 2 interventional study of VAY736 and Placebo in Pemphigus Vulgaris, sponsored by Novartis Pharmaceuticals. Terminated at 5 sites in 4 countries. Open to participants aged 20 Years to 70 Years. Per ClinicalTrials.gov, last updated 2021-10-08.
Sponsored by Novartis Pharmaceuticals (part of Novartis) · Phase 2, Interventional, and Treatment
Among 25 studies in Pemphigus with a site in United States — 4 open to participants now, 4 under way, none of them phase 3. Pemphigus · United States.
Among the 25 studies in Pemphigus with a site in United States, this one is the 16th largest by enrollment of the 23 interventional ones, is one of 2 that started in 2013, is one of 9 in phase 2, and was the 12th registered.
76 studies on the registry are indexed under Pemphigus; 18 are open to participants now.
This study's enrollment of 13 is below the median of 30 across 53 interventional studies indexed under Pemphigus.
Browse Pemphigus studies →4 of the 25 studies in Pemphigus with a site in United States are open now, in 1 country. Each count lists the studies.
Of the 9 Pemphigus studies recruiting now, 4 (44%) set no upper age limit and 2 (22%) stop at 75 or younger; 1 take children under 18; 1 take healthy volunteers.
This study: 20 Years to 70 Years · all sexes · no healthy volunteers.
The full table on the hub →25 studies; the count open now beside each. This study's row is marked.
First in 1996; this study’s year is marked. 2 started in 2013.
Median enrollment 29 across the 23 interventional studies, 10,075 across the 2 observational. Of the 21 that completed or were terminated, 15 (71%) have results posted.
14 of 25 have an industry lead sponsor. The 10 most frequent:
Novartis Pharmaceuticals is the lead sponsor of 2,718 studies on the registry; 221 are open to participants now.
Of its 585 completed or terminated interventional studies of FDA-regulated products, 438 (75%) have results posted.
Novartis Pharmaceuticals has 2 studies filed under Pemphigus — fewer than ten, so there is no comparison to draw.
1 of the studies in Pemphigus with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT06581562IRIS Research and Development, LLCOpen-label Single-Center Study to Evaluate the Safety and Efficacy of Combining Rituximab and AB-101 in B-cell Associated Autoimmune Diseases. | 2027-07 |
Among the 25 studies in Pemphigus with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
The study evaluated the efficacy, safety and pharmacokinetics of VAY736 in the treatment of patients with pemphigus vulagaris (PV).
This was a non-confirmatory, randomized, partial-blind, placebo-controlled trial evaluating the efficacy, safety and pharmacokinetics (PK) of VAY736 in the treatment of PV patients.
A total of 13 patients were enrolled and randomized into the study. Of these 13 patients,seven were randomized to the 3 mg/kg VAY736 group, two were randomized to the 10 mg/kg VAY736 group and four were randomized to the placebo group.In the placebo group, three out of the four patients consented to open-label VAY736 treatment and received 10 mg/kg VAY736 after Week 24 onwards. Thus, a total of 12 patients received VAY736, 7 patients received 3 mg/kg and 5 patients 10 mg/kg.
The Screening period consisted of a Screening Visit performed within 28 days prior to randomization to assess patient eligibility. Following Screening, patients underwent pre-dose procedures which included assessment of their PV by Pemphigus Disease Area Index (PDAI), Autoimmune Bullous Skin disease Intensity Score (ABSIS) and Investigator Global Assessment (IGA), and blood sampling for PK endpoints. Patients then received the study drug, which was administered over approximately a 2 hour period. The patients remained in the study center overnight post-infusion for observation and for measurement of safety parameters and PK samples approximately 24 h post-infusion (start of infusion: ±2 h). Patients were then discharged from the study site and returned as per the schedule. Patients were evaluated at Week 1, Week 2 and Week 3, then every 3 weeks through to Week 12, and every 4 weeks through to Week 24. At Week 24, the blind was broken to confirm treatment allocation. If a patient was on placebo, such patient completing the Week 24 visit and after unblinding had the option of receiving open label VAY736 10mg/kg.
Recruitment was paused in Mar-2015 and at the time 13 patients were enrolled. The study recruitment was then terminated in Dec-2015 for strategic reasons related to the development of the compound.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Washout period
A planned gap after a treatment, so that its effects can lessen before the study treatment or a study assessment. Ask the study team and the doctor who prescribed it before changing any medicine.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
single dose iv of Placebo
Drug: Placebo
single dose iv of VAY736 at a dose of 3mg/kg
Drug: VAY736
single dose iv of VAY736 at a dose of 10mg/kg initiated following a safety review of patients receiving VAY736 3 mg/kg or placebo.
Drug: VAY736
Also known as: Ianalumab
Pemphigus Disease Area Index (PDAI) at Week 12
PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Damage, such as post inflammatory hyperpigmentation or erythema from resolving lesion, scored separately from the main score as absent (0) or present (1) for each body area or scalp resulting in a score of 0 to 12 or 0 to 1, respectively. Thus, PDAI ranged from 0 to 263, with 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity) and 13 points representing disease damage.
Time frame: Week 12
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.
The ABSIS Score is a quality- and quantity-based score for cutaneous and oral mucosal lesions combining the extent of the affected body surface area (BSA), the quality of the skin lesions and oral involvement. The ABSIS score ranged from 0 to 206 with 150 points for skin involvement, 11 points for oral involvement and 45 points for subjective discomfort during eating and drinking. A reduction from baseline (or, a negative change from baseline) in ABSIS indicates improvement in patients.
Time frame: Baseline, Week 12
Change From Baseline in Investigator Global Assessment (IGA) at Week 12
The IGA score ranges from 0 to 4 and the decrease or reduction from baseline in IGA score indicates improvement in patients. IGA score scale: 0=Clear, 1=Near Clear, 2=Mild, 3=Moderate, 4=Severe active disease
Time frame: Baseline, Week 12
VAY736 Serum Concentration - AUCinf
The area under the serum concentration-time curve from time zero to infinity \[mass × time / volume\]. The concentration of VAY736 was measured in the serum.
Time frame: predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks
VAY736 Serum Concentration - AUClast
The area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration \[mass × time / volume\]. The concentration of VAY736 was measured in the serum.
Time frame: predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks
VAY736 Serum Concentration - Cmax
The observed maximum serum concentration following drug administration \[mass / volume\]. The concentration of VAY736 was measured in the serum.
Time frame: predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks
VAY736 Serum Concentration - Tmax
Tmax is the time to reach the maximum concentration after drug administration \[time\]. The concentration of VAY736 was measured in the serum.
Time frame: predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks
VAY736 Serum Concentration - T1/2
T1/2 is the terminal elimination half-life \[time\]. The concentration of VAY736 was measured in the serum.
Time frame: predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks
A total of 13 participants were enrolled and randomized into the study from Austria (1 center); Bulgaria (1 center); Taiwan (1 center); USA (2 centers).
| Milestone | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| Started | 7 | 2 | 4 |
| Safety analysis set | 7 | 2 | 4 |
| Pharmacokinetics (pk) analysis set | 7 | 2 | 0 |
| Completed | 7 | 1 | 3 |
| Not completed | 0 | 1 | 1 |
| Withdrew: Lost to follow-up | 0 | 1 | 0 |
| Withdrew: Adverse event | 0 | 0 | 1 |
| Milestone | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| Started | 0 | 0 | 3 |
| Completed | 0 | 0 | 3 |
| Not completed | 0 | 0 | 0 |
PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Damage, such as post inflammatory hyperpigmentation or erythema from resolving lesion, scored separately from the main score as absent (0) or present (1) for each body area or scalp resulting in a score of 0 to 12 or 0 to 1, respectively. Thus, PDAI ranged from 0 to 263, with 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity) and 13 points representing disease damage.
| Score on the scale | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| Pemphigus Disease Area Index (PDAI) at Week 12 | 5.90 ± 1.836 | 10.15 ± 8.273 | 22.07 ± 25.628 |
The ABSIS Score is a quality- and quantity-based score for cutaneous and oral mucosal lesions combining the extent of the affected body surface area (BSA), the quality of the skin lesions and oral involvement. The ABSIS score ranged from 0 to 206 with 150 points for skin involvement, 11 points for oral involvement and 45 points for subjective discomfort during eating and drinking. A reduction from baseline (or, a negative change from baseline) in ABSIS indicates improvement in patients.
| Score on the scale | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| Baseline | 13.26 ± 7.621 | 16.38 ± 12.905 | 33.75 ± 21.620 |
| Week 12 | 2.19 ± 3.465 | 5.55 ± 7.707 | 16.17 ± 25.838 |
The IGA score ranges from 0 to 4 and the decrease or reduction from baseline in IGA score indicates improvement in patients. IGA score scale: 0=Clear, 1=Near Clear, 2=Mild, 3=Moderate, 4=Severe active disease
| Score on the scale | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| Change From Baseline in Investigator Global Assessment (IGA) at Week 12 | -1.4 ± 0.79 | -1.0 ± 0.00 | -0.7 ± 0.58 |
The area under the serum concentration-time curve from time zero to infinity \[mass × time / volume\]. The concentration of VAY736 was measured in the serum.
| day*ug/mL | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| VAY736 Serum Concentration - AUCinf | 440 ± 114 | 1480 ± 231 | — |
The area under the serum concentration-time curve from time zero to the time of the last quantifiable concentration \[mass × time / volume\]. The concentration of VAY736 was measured in the serum.
| day*ug/mL | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| VAY736 Serum Concentration - AUClast | 440 ± 114 | 1480 ± 231 | — |
The observed maximum serum concentration following drug administration \[mass / volume\]. The concentration of VAY736 was measured in the serum.
| ug/mL | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| VAY736 Serum Concentration - Cmax | 77.1 ± 13.0 | 230 ± 2.83 | — |
Tmax is the time to reach the maximum concentration after drug administration \[time\]. The concentration of VAY736 was measured in the serum.
| Hours | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| VAY736 Serum Concentration - Tmax | 2.05 (2.00 to 2.22) | 2.11 (2.00 to 2.22) | — |
T1/2 is the terminal elimination half-life \[time\]. The concentration of VAY736 was measured in the serum.
| Days | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo |
|---|---|---|---|
| VAY736 Serum Concentration - T1/2 | 11.2 ± 2.01 | 15.4 ± 1.93 | — |
Collected over Adverse events were collected from first dose of study treatment until end of study for up to 5 years.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| VAY736 3 mg/kg | 0/7 (0%) | 1/7 (14.3%) | 6/7 (85.7%) |
| VAY736 10 mg/kg | 0/2 (0%) | 0/2 (0%) | 2/2 (100%) |
| Placebo | 0/4 (0%) | 1/4 (25%) | 3/4 (75%) |
| Open Label VAY736 10 mg/kg | 0/3 (0%) | 1/3 (33.3%) | 3/3 (100%) |
| Event | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo | Open Label VAY736 10 mg/kg |
|---|---|---|---|---|
| CataractEye disorders | 0/7 | 0/2 | 0/4 | 1/3 |
| PemphigusSkin and subcutaneous tissue disorders | 0/7 | 0/2 | 1/4 | 0/3 |
| Duodenal ulcerGastrointestinal disorders | 1/7 | 0/2 | 0/4 | 0/3 |
| Event | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo | Open Label VAY736 10 mg/kg |
|---|---|---|---|---|
| ChillsGeneral disorders | 0/7 | 1/2 | 0/4 | 0/3 |
| PainGeneral disorders | 0/7 | 1/2 | 0/4 | 0/3 |
| PyrexiaGeneral disorders | 0/7 | 1/2 | 0/4 | 0/3 |
| Tooth abscessInfections and infestations | 0/7 | 1/2 | 0/4 | 0/3 |
| Upper respiratory tract infectionInfections and infestations | 1/7 | 1/2 | 0/4 | 0/3 |
| Infusion related reactionInjury, poisoning and procedural complications | 0/7 | 1/2 | 0/4 | 0/3 |
| DizzinessNervous system disorders | 1/7 | 1/2 | 0/4 | 0/3 |
| HeadacheNervous system disorders | 2/7 | 1/2 | 0/4 | 1/3 |
| SciaticaNervous system disorders | 0/7 | 1/2 | 0/4 | 0/3 |
| Oropharyngeal painRespiratory, thoracic and mediastinal disorders | 2/7 | 1/2 | 0/4 | 1/3 |
| Age, Continuous(Years) | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Mean | 51.6 ± 10.45 | 35.0 ± 8.49 | 56.0 ± 8.83 | 50.4 ± 11.44 |
| Sex: Female, Male(Participants) | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Female | 3 | 0 | 2 | 5 |
| Male | 4 | 2 | 2 | 8 |
| Race/Ethnicity, Customized(Participants) | VAY736 3 mg/kg | VAY736 10 mg/kg | Placebo | Total |
|---|---|---|---|---|
| Asian | 2 | 1 | 1 | 4 |
| Caucasian | 4 | 1 | 3 | 8 |
| Other | 1 | 0 | 0 | 1 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect 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
Ran from Dec 2013 to primary completion in Sep 2019, 69 months.
Results were first posted in Oct 2020, 1 year after primary completion.
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This study is terminated, as verified in Oct 2021. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ianalumab