Trial · NCT01930175

Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris

NCT01930175, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2020-10-19

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris. Trials, record NCT01930175. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01930175

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Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris
NCT number: NCT01930175
Status: Terminated
Phases: Phase 2
Enrollment: 13 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Pemphigus Vulgaris
Interventions: VAY736, Placebo
First posted: 2013-08-28
Last updated: 2021-10-08
Results first posted: 2020-10-19

Source ClinicalTrials.gov

Registered outcomes8

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Pemphigus Disease Area Index (PDAI) at Week 12	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.
Secondary	Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	Baseline, 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.
Secondary	Change From Baseline in Investigator Global Assessment (IGA) at Week 12	Baseline, 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
Secondary	VAY736 Serum Concentration - AUCinf	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	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.
Secondary	VAY736 Serum Concentration - AUClast	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	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.
Secondary	VAY736 Serum Concentration - Cmax	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	The observed maximum serum concentration following drug administration \[mass / volume\]. The concentration of VAY736 was measured in the serum.
Secondary	VAY736 Serum Concentration - Tmax	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	Tmax is the time to reach the maximum concentration after drug administration \[time\]. The concentration of VAY736 was measured in the serum.
Secondary	VAY736 Serum Concentration - T1/2	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	T1/2 is the terminal elimination half-life \[time\]. The concentration of VAY736 was measured in the serum.

Source ClinicalTrials.gov

Outcomes8

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Pemphigus Disease Area Index (PDAI) at Week 12	Week 12	All evaluable patients.	Score on the scale	MEAN
SECONDARY	Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	Baseline, Week 12	All evaluable patients.	Score on the scale	MEAN
SECONDARY	Change From Baseline in Investigator Global Assessment (IGA) at Week 12	Baseline, Week 12	All evaluable patients.	Score on the scale	MEAN
SECONDARY	VAY736 Serum Concentration - AUCinf	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	PK analysis set: Patients with at least one PK measurement and no major protocol deviations affecting PK	day*ug/mL	MEAN
SECONDARY	VAY736 Serum Concentration - AUClast	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	PK analysis set: Patients with at least one PK measurement and no major protocol deviations affecting PK	day*ug/mL	MEAN
SECONDARY	VAY736 Serum Concentration - Cmax	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	PK analysis set: Patients with at least one PK measurement and no major protocol deviations affecting PK	ug/mL	MEAN
SECONDARY	VAY736 Serum Concentration - Tmax	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	PK analysis set: Patients with at least one PK measurement and no major protocol deviations affecting PK	Hours	MEDIAN
SECONDARY	VAY736 Serum Concentration - T1/2	predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks	PK analysis set: Patients with at least one PK measurement and no major protocol deviations affecting PK	Days	MEAN

Source ClinicalTrials.gov

Results by group22

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Pemphigus Disease Area Index (PDAI) at Week 12	VAY736 3 mg/kg		5.90	Standard Deviation 1.836	Score on the scale
Pemphigus Disease Area Index (PDAI) at Week 12	VAY736 10 mg/kg		10.15	Standard Deviation 8.273	Score on the scale
Pemphigus Disease Area Index (PDAI) at Week 12	Placebo		22.07	Standard Deviation 25.628	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	VAY736 3 mg/kg	Baseline	13.26	Standard Deviation 7.621	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	VAY736 10 mg/kg	Baseline	16.38	Standard Deviation 12.905	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	Placebo	Baseline	33.75	Standard Deviation 21.620	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	VAY736 3 mg/kg	Week 12	2.19	Standard Deviation 3.465	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	VAY736 10 mg/kg	Week 12	5.55	Standard Deviation 7.707	Score on the scale
Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.	Placebo	Week 12	16.17	Standard Deviation 25.838	Score on the scale
Change From Baseline in Investigator Global Assessment (IGA) at Week 12	VAY736 3 mg/kg		-1.4	Standard Deviation 0.79	Score on the scale

1 to 10 of 22Source ClinicalTrials.gov

See all 22 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Core Study	STARTED	VAY736 3 mg/kg	7
Core Study	STARTED	VAY736 10 mg/kg	2
Core Study	STARTED	Placebo	4
Core Study	Safety Analysis Set	VAY736 3 mg/kg	7
Core Study	Safety Analysis Set	VAY736 10 mg/kg	2
Core Study	Safety Analysis Set	Placebo	4
Core Study	Pharmacokinetics (PK) Analysis Set	VAY736 3 mg/kg	7
Core Study	Pharmacokinetics (PK) Analysis Set	VAY736 10 mg/kg	2
Core Study	Pharmacokinetics (PK) Analysis Set	Placebo	0
Core Study	COMPLETED	VAY736 3 mg/kg	7

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events216

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Arrhythmia	Cardiac disorders	VAY736 3 mg/kg	0	7
other	Arrhythmia	Cardiac disorders	VAY736 10 mg/kg	0	2
other	Arrhythmia	Cardiac disorders	Placebo	1	4
other	Arrhythmia	Cardiac disorders	Open Label VAY736 10 mg/kg	0	3
other	Conjunctivitis allergic	Eye disorders	VAY736 3 mg/kg	0	7
other	Conjunctivitis allergic	Eye disorders	VAY736 10 mg/kg	0	2
other	Conjunctivitis allergic	Eye disorders	Placebo	0	4
other	Conjunctivitis allergic	Eye disorders	Open Label VAY736 10 mg/kg	1	3
other	Diarrhoea	Gastrointestinal disorders	VAY736 3 mg/kg	1	7
other	Diarrhoea	Gastrointestinal disorders	VAY736 10 mg/kg	0	2

1 to 10 of 216Source ClinicalTrials.gov

See all 216 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	VAY736 3 mg/kg	0	7
Total, all-cause mortality	VAY736 10 mg/kg	0	2
Total, all-cause mortality	Placebo	0	4
Total, all-cause mortality	Open Label VAY736 10 mg/kg	0	3
Total, other adverse events	VAY736 3 mg/kg	6	7
Total, other adverse events	VAY736 10 mg/kg	2	2
Total, other adverse events	Placebo	3	4
Total, other adverse events	Open Label VAY736 10 mg/kg	3	3
Total, serious adverse events	VAY736 3 mg/kg	1	7
Total, serious adverse events	VAY736 10 mg/kg	0	2

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

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