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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT01920477
Registration number
NCT01920477
Ethics application status
Date submitted
3/07/2013
Date registered
12/08/2013
Date last updated
6/06/2019
Titles & IDs
Public title
Efficacy and Safety of Ofatumumab in Treatment of Pemphigus Vulgaris
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Scientific title
OPV116910: A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Ofatumumab Injection for Subcutaneous Use in Subjects With Pemphigus Vulgaris
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Secondary ID [1]
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116910
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Pemphigus Vulgaris
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Condition category
Condition code
Skin
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Dermatological conditions
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Skin
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Other skin conditions
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Inflammatory and Immune System
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Autoimmune diseases
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Other interventions - Ofatumumab
Other interventions - Placebo
Experimental: Ofatumumab - Subject received subcutaneous administration of ofatumumab 20 mg once every 4 weeks through Week 56, with an additional 20 mg dose (that is 40mg total) at both Week 0 and Week 4.
Placebo Comparator: Placebo - Subject received subcutaneous administration of matching placebo of ofatumumab once every 4 weeks through Week 56, with an additional dose at both Week 0 and Week 4.
Other interventions: Ofatumumab
Ofatumumab (human monoclonal antibody) was provided in prefilled glass syringes initial syringes contained 0.6ml (60mg) of concentration 100 mg/m: drug product subsequently modified to 0.4 mL (20mg) concentration (50mg/ML) drug product
Other interventions: Placebo
Placebo to match the active doses will consist of prefilled glass syringes filled with normal saline.
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Intervention code [1]
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Other interventions
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Number of Subjects Who Experienced Sustained Remission on Minimal Steroid Therapy
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Assessment method [1]
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Sustained remission = time from randomization to the time of the subject's initial reduction of prednisone/prednisolone dose to <=10 mg/day and maintenance of a dose <=10 mg/day with no new or nonhealing lesions for >=8 weeks and maintenance of the status until Week 60.
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Timepoint [1]
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Baseline up to approximately 60 weeks
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Primary outcome [2]
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Duration of Remission on Minimal Steroid Therapy
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Assessment method [2]
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Sum of all periods of absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day up to Week 60 was assessed.
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Timepoint [2]
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Baseline up to approximately 60 weeks
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Secondary outcome [1]
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Percentage of Subjects Achieving Remission on Minimal Steroid Therapy at Week 60
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Assessment method [1]
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Percentage of subjects who achieved absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day for > or = 8 weeks at Week 60 was assessed. Time to remission was not estimable.
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Timepoint [1]
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Week 60
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Secondary outcome [2]
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Time to Remission While on Minimal Steroid Therapy by Week 60.
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Assessment method [2]
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Time from randomization to the time of the subject's initial reduction of prednisone/prednisolone dose to <=10 mg/day and maintained dose at <=10 mg/day with no new or nonhealing lesions for >=8 weeks by Week 60 was assessed
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Timepoint [2]
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Baseline up to approximately 60 weeks
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Secondary outcome [3]
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Percentage of Subjects Achieving Remission While Off Steroid Therapy by Week 60
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Assessment method [3]
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Percentage of subjects with initial reduction of all steroids for >=8 weeks with an absence of new or nonhealing (established) lesions by Week 60 were to be assessed. All subjects remained on prednisone/prednisolone so this endpoint could not be analyzed.Time to remission off steroid therapy also could not be analyzed
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Timepoint [3]
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Baseline up to approximately 60 weeks
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Secondary outcome [4]
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Number of Days a Subject Maintained Minimal Steroid Therapy by Week 60.
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Assessment method [4]
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Number of days a subject maintained minimal steroid therapy (an oral prednisone/prednisolone dose of =10 mg/day in the absence of new or nonhealing lesions) by Week 60.
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Timepoint [4]
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Baseline up to approximately 60 weeks
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Secondary outcome [5]
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Time to Initial Flare/Relapse by Week 60
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Assessment method [5]
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Time from randomization to the time of appearance of >=3 new lesions within 1 month that did not heal spontaneously within 1 week, or to the time when there was an extension of lesions that were present at the randomization by Week 60 was assessed
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Timepoint [5]
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Baseline up to approximately 60 weeks
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Secondary outcome [6]
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Percentage of Participants With no Flare/Relapse by Week 60
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Assessment method [6]
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Percentage of participants achieving absence of new or nonhealing lesions while on an oral prednisone/prednisolone dose of <=10 mg/day and did not subsequently have a appearance of >=3 new lesions within 1 month that did not heal spontaneously within 1 week, or to the time when there was an extension of lesions that were present at the randomization by Week 60 was assessed
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Timepoint [6]
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Baseline up to approximately 60 weeks
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Secondary outcome [7]
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Plasma Trough Concentrations of Ofatumumab
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Assessment method [7]
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Only plasma (trough) concentrations of ofatumumab were presented
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Timepoint [7]
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4 hours post baseline, Days 1-4,7,14, Weeks 4,8,12,16,20,24,36,48,52,56, up to approximately 60 weeks
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Secondary outcome [8]
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Number of Participants With Values Below Lower Limit of Normal for Immunoglobulin A
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Assessment method [8]
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Assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
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Timepoint [8]
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Baseline up to approximately 60 weeks
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Secondary outcome [9]
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Number of Participants With Values Below Lower Limit of Normal for Immunoglobulin G
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Assessment method [9]
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Assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
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Timepoint [9]
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Baseline up to approximately 60 weeks
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Secondary outcome [10]
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Number of Participants With Values Below Lower Limit of Normal for Immunoglobulin M
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Assessment method [10]
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Assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
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Timepoint [10]
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Baseline up to approximately 60 weeks
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Secondary outcome [11]
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Largest Mean Decrease From Baseline for Immunoglobulin A, G and M
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Assessment method [11]
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Immunoglobulins A, G and M were assessed by the incidence, titer, and type of human anti-human antibody (HAHA) immune response
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Timepoint [11]
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Baseline up to approximately 60 weeks
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Secondary outcome [12]
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Change From Baseline for CD19+ B Cell Count
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Assessment method [12]
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CD19+ B cell count will be performed using Flow Cytometry
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Timepoint [12]
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Baseline up to approximately 60 weeks
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Eligibility
Key inclusion criteria
Inclusion criteria
- Adults (18 through 70 years of age) with clinically-documented diagnosis of PV for >2
months and <10 years.
- History of biopsy consistent with PV (Hematoxylin and Eosin staining and direct
immunofluorescence). If no history, a biopsy may be performed during the Screening
Period.
- At least 1 previous episode of a failed steroid taper (ie, disease flare/relapse) at a
prednisone/prednisolone dose >10 mg/day. The following criteria must have been met as
evidence of disease severity at the time of the failed steroid taper: a) A Pemphigus
Severity of Clinical Disease score of moderate (2) or severe (3) (may be
historical/retrospective assessment). b) Required a treatment change at the time of
the failed steroid taper of at least one of the following: i) A steroid increase to
>=20 mg/day OR ii) The addition of immunosuppressive/immunomodulatory agent/treatment
OR iii) A dose increase of immunosuppressive/immunomodulatory agent/treatment
- Screening anti-Dsg antibodies consistent with a diagnosis of PV (ie, elevated antiDsg3
antibodies).
- Has initiated and received a stable dose of prednisone/prednisolone from a minimum of
20 mg/day (example: 0.25 mg/kg/day for an 80 kg person) up to a maximum of 120 mg/day
or 1.5 mg/kg/day (whichever is higher) for >=2 weeks prior to randomization.
- Has exhibited PV disease control, defined as no new lesions for >=2 weeks.
- A female subject is eligible to enter the study if she: Is of non-child bearing
potential, who is either surgically sterile (bilateral tubal ligation, bilateral
oophorectomy, or post-hysterectomy) or is postmenopausal without menses for >2 years.
Women who are <2 years postmenopausal are required to have menopausal status confirmed
by follicle-stimulating hormone (FSH) and estradiol levels at the screening
evaluation. If FSH and estradiol levels do not provide confirmation of menopause,
subject will be considered to be of childbearing potential.
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Minimum age
18
Years
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Maximum age
70
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
- Diagnosis of pemphigus foliaceus, paraneoplastic pemphigus, or other autoimmune
blistering disease (other than pemphigus vulgaris).
- Past or current history of hypersensitivity to components of the investigational
product or medically significant adverse effects (including allergic reactions) from
cetirizine (or antihistamine equivalent) or paracetamol/acetaminophen.
- Prior treatment with rituximab without achieving disease control within 6 months of
initiating rituximab dosing.
- Prior treatment with immunosuppressant or immunomodulation agents within the protocol
specified periods
- Evidence or history of clinically significant infections
For Japan: Evidence or history of clinically significant infection or medical condition
including: Pneumocystis pneumonia or interstitial pneumonia
- Past or current malignancy, except for cervical carcinoma Stage 1B or less,
noninvasive basal cell and squamous cell skin carcinoma and cancer diagnoses with a
duration of complete response (remission) >5 years
- Significant concurrent, uncontrolled medical condition that could affect the subject's
safety, impair the subject's reliable participation in the study, impair the
evaluation of endpoints, or necessitate the use of medication not allowed by the
protocol. This includes subjects who require any systemic steroid treatment for a
concurrent medical condition (other than pemphigus vulgaris).
- Use of an investigational drug or other experimental therapy within 4 weeks, 5
pharmacokinetic half-lives, or the duration of biological effect (whichever is longer)
prior to Screening.
- Electrocardiogram (ECG) showing a clinically significant abnormality or showing a QTc
interval =450 msec (=480 msec for subjects with a bundle branch block)
- Woman who is breastfeeding.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Terminated
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
13/08/2013
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
11/01/2018
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Sample size
Target
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Accrual to date
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Final
35
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Novartis Investigational Site - East Melbourne
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Recruitment hospital [2]
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Novartis Investigational Site - Melbourne
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Recruitment postcode(s) [1]
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3002 - East Melbourne
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Recruitment postcode(s) [2]
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3050 - Melbourne
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Georgia
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Country [3]
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United States of America
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State/province [3]
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Michigan
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Country [4]
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United States of America
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State/province [4]
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New York
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Country [5]
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United States of America
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State/province [5]
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North Carolina
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Country [6]
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United States of America
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State/province [6]
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Pennsylvania
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Country [7]
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United States of America
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State/province [7]
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Utah
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Country [8]
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Greece
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State/province [8]
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Thessalonica
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Country [9]
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Israel
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State/province [9]
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Ramat-Gan
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Country [10]
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Israel
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State/province [10]
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Tel Aviv
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Country [11]
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Italy
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State/province [11]
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Lombardia
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Country [12]
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Japan
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State/province [12]
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Tokyo
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Country [13]
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Poland
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State/province [13]
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Warszawa
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Country [14]
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Romania
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State/province [14]
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Cluj-Napoca
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
Novartis Pharmaceuticals
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening
autoimmune disorder that is characterized by mucocutaneous blisters. Ofatumumab is a novel
monoclonal antibody (mAb) that specifically binds to the human CD20 antigen, which is
expressed only in B lymphocytes.
The purpose of this study was to evaluate the efficacy, tolerability, and safety of
ofatumumab injection for subcutaneous use (ofatumumab SC) 20 milligrams (mg) administered
once in every 4 weeks, (with an additional 20 mg loading dose [i.e. 40 mg total] at both Week
0 and Week 4) in subjects with PV. It was anticipated that with sustained B-cell depletion in
the presence of ofatumumab SC, and the resultant reduction of pathogenic anti Dsg
(desmoglein) autoantibodies in PV, that clinical remission of the disease would result.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT01920477
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Novartis Pharmaceuticals
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Address
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Novartis Pharmaceuticals
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT01920477
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