To determine the dose-response relationship of VAY736 for key efficacy and safety parameters.
ID
Source
Brief title
Condition
- Autoimmune disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
To demonstrate a dose response of VAY736 defined as change in ESSDAI from
baseline at 24 weeks
Secondary outcome
To assess a dose response of VAY736 in the change from baseline of ESSPRI at 24
weeks
To assess a dose response of VAY736 in the change from baseline of the
Functional Assessment of Chronic Illness Therapy-Fatigue Scale (FACIT-F) at 24
weeks
To assess changes from baseline in PhGA of the patient*s overall disease
activity at week 24
To assess a dose response of VAY736 in the change from baseline of SF-36 at 24
weeks
To evaluate the effects of VAY736 on salivary gland function at 24 weeks
To evaluate CD19+ B-cell counts before and after VAY736 treatment, and time to
recovery
To assess safety and tolerability of VAY736 through incidence of AEs, SAEs, and
monthly safety laboratory tests
To assess immunogenicity (IG) of VAY736 by measuring serum anti-VAY736
antibodies
To assess PK of VAY736 after multiple s.c. doses at multiple time points
Background summary
Primary Sjögren*s Syndrome (pSS) is a chronic autoimmune disease of unknown
etiology, characterized by lymphoid infiltration and progressive destruction of
exocrine glands. Current standard-of-care (SoC) treatment for pSS patients is
limited to symptomatic care for the mucosal signs and symptoms (dryness).
Steroids and conventional disease modifying antirheumatic drugs (DMARDs),
although used in selected patients, have not been proven efficacious, and no
pharmacologic intervention is effective against the severe, disabling fatigue.
Hence, there are no approved treatments available for moderate to severe (i.e.,
systemic) pSS.
The 12-week efficacy results in pSS clinical outcomes observed in the proof of
concept study VAY736X2201 with single infusions of two different i.v. doses of
VAY736 (3 mg/kg and 10 mg/kg) in 27 patients suggest early and clinically
meaningful improvements in signs and symptoms compared to placebo. All patients
will be treated with VAY736 in one of the treatment arms.
Study objective
To determine the dose-response relationship of VAY736 for key efficacy and
safety parameters.
Study design
This is a randomized, double-blind, placebo-controlled, multicenter,
parallel-group trial in patients with active pSS. The study treatment period
will last for 24 weeks and for a subset of patients up to 52 weeks. A post
treatment safety follow up period for all patients will last for a minimum of
20 weeks.
Intervention
VAY736 or placebo
Study burden and risks
When participating in part 1, 2 and 3: Minimum of 18 visits, duration vary from
1-4 hours per visit, total study time minimal 72 weeks.
- Physical examination also Sjogren specifically: every visit
- Biopsy of the saliva: optional if participating in substudy
- Saliva production: 6 x
- Tear fluid production: 6x
- ECG: 5x
- Subcutaneous injections: starting from randomization every 4 weeks two
injections (150 ml each injection)
- At first injection intravenous Methylprednisolone administration.
- Blood collection: every visit
- Filling out questionnaires: every visit
Optional:
- 1 x extra blood collection for farmacogenetisch research
Patients who do not have part 3 have fewer visits.
There is forbidden co-medication.
Haaksbergweg 16
Amsterdam 1101 BX
NL
Haaksbergweg 16
Amsterdam 1101 BX
NL
Listed location countries
Age
Inclusion criteria
- Fulfilled revised European US consensus criteria for pSS
- Seropositive at screening for anti-Ro/SSA antibodies
- See protocol for other protocol-defined inclusion criteria
Exclusion criteria
- Secondary Sjogren*s syndrome
- Use of other investigational drugs
- Active viral, bacterial or other infections
- Positive hepatitis B, hepatitis C, HIV or tuberculosis test results at
screening
- See protocol for other protocol-defined exclusion criteria
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2016-003292-22-NL |
| ClinicalTrials.gov | NCT02962895 |
| CCMO | NL60083.078.17 |