A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults With Dermatomyositis (VALOR)
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| ClinicalTrials.gov Identifier: NCT05437263 |
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Recruitment Status :
Recruiting
First Posted : June 29, 2022
Last Update Posted : June 1, 2023
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Sponsor:
Priovant Therapeutics, Inc.
Information provided by (Responsible Party):
Priovant Therapeutics, Inc.
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Brief Summary:
This is a phase 3, multicenter, randomized, placebo-controlled, double-blind study of treatment with brepocitinib (TYK2/JAK1 inhibitor) in adults with dermatomyositis (DM). The primary objective of this study is to assess the efficacy of two dose levels of brepocitinib in comparison to placebo, as measured by differences in the Total Improvement Score (TIS). After 52 weeks of double-blind treatment, participants have the option to continue therapy in a 52 week open-label extension phase where all participants will receive brepocitinib.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Dermatomyositis | Drug: Brepocitinib Drug: Placebo | Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 225 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Investigate the Efficacy and Safety of Oral Brepocitinib in Adults With Dermatomyositis |
| Actual Study Start Date : | October 31, 2022 |
| Estimated Primary Completion Date : | December 2024 |
| Estimated Study Completion Date : | December 2024 |
Resource links provided by the National Library of Medicine
| Arm | Intervention/treatment |
|---|---|
| Experimental: Brepocitinib Dose Level 1 PO QD |
Drug: Brepocitinib
Oral Brepocitinib |
| Experimental: Brepocitinib Dose Level 2 PO QD |
Drug: Brepocitinib
Oral Brepocitinib |
| Placebo Comparator: Placebo PO QD |
Drug: Placebo
Oral Placebo |
Primary Outcome Measures :
- Total Improvement Score (TIS) at Week 52 [ Time Frame: 52 weeks ]TIS is a composite endpoint based on improvement in the 6 Disease Activity Core Set Measure (CSM) scores and ranges from 0 to 100 (2016 American College of Rheumatology [ACR] Myositis Response Criteria/European League Against Rheumatism [EULAR]) where a higher score indicates more improvement
Secondary Outcome Measures :
- TIS responder rate after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]Proportion of responders, defined as achieving TIS ≥ 40 points (moderate improvement) at Week 52
- Time to initiation of rescue medication through 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]Time to initiation of rescue medication through Week 52
- Manual Muscle Testing (MMT-8) after 52 weeks of brepocitinib administration QD, in comparison to placebo [ Time Frame: 52 weeks ]Change from baseline in MMT-8 score at Week 52. The MMT-8 evaluation includes 7 muscles tested bilaterally plus neck flexors, each scored from 0 to 10 for a potential score ranging from 0 to 150 where a higher score indicates more improvement.
Information from the National Library of Medicine
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| Ages Eligible for Study: | 18 Years to 75 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- A diagnosis of dermatomyositis according to 2017 EULAR/ACR Classification Criteria for Idiopathic Inflammatory Myopathies
- Adult subjects (18-75 years old)
- Active muscle and skin disease at screening and baseline
- Prior therapy OR current therapy with corticosteroids, hydroxychloroquine, and/or one non-steroid immunosuppressant
- Weight > 40 kg to < 130 kg, and with a body mass index (BMI) < 40 kg/m2.
Exclusion Criteria:
- Dermatomyositis with end-stage organ involvement
- Dermatomyositis with irreversible muscle involvement
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History of:
- Any lymphoproliferative disorder
- Active malignancy;
- History of cancer within 5 years prior to screening (exceptions for basal cell carcinoma, squamous cell carcinoma, ductal carcinoma in situ of the breast, carcinoma in situ of the uterine cervix, or thyroid carcinoma.)
- Cancer-associated dermatomyositis
- Overlap myositis/connective tissue disease (except for overlap with Sjögren's syndrome)
- Participants at a risk of thrombosis and cardiovascular disease
- Participants with a high risk for herpes zoster reactivation
- Participants with active or recent infections
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05437263
Contacts
| Contact: Clinical Trial Administrator | (212) 634-9743 | ValorStudyManager@PriovantTx.com |
Locations
Show 63 study locations
Sponsors and Collaborators
Priovant Therapeutics, Inc.
Investigators
| Study Director: | Noriko Iikuni, MD | VP, Clinical Development |
Additional Information:
| Responsible Party: | Priovant Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT05437263 |
| Other Study ID Numbers: |
PVT-2201-301 2022-500367-12-00 ( Registry Identifier: EU CT Number ) |
| First Posted: | June 29, 2022 Key Record Dates |
| Last Update Posted: | June 1, 2023 |
| Last Verified: | May 2023 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Priovant Therapeutics will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) from eligible studies upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Data requests will be reviewed and approved on the basis of scientific merit. |
| Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Clinical Study Report (CSR) |
| Time Frame: | The data will be made available within 24 months after study completion and will be accessible for a time frame appropriate for the approved proposal. |
| Access Criteria: | Access will be provided following review and approval of a research proposal and execution of a Data Sharing Agreement (DSA). Further details on Priovant Therapeutics' data sharing criteria and process for requesting access can be obtained by emailing info@priovanttx.com. |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Priovant Therapeutics, Inc.:
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brepocitinib dermatomyositis TYK2/JAK1 inhibitor PF-06700841 PVT-2201 |
Additional relevant MeSH terms:
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Dermatomyositis Polymyositis Myositis Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Connective Tissue Diseases Skin Diseases |