A Phase 1, Open-label, Multiple Ascending Dose Basket Study to Evaluate the Safety and Activity of DR-0201 in Patients With Select Autoimmune Rheumatic Diseases
This is a multi-center, open-label, multi-ascending dose study to evaluate the safety, tolerability, PK, PD, immunogenicity, and preliminary clinical response of DR-0201 following IV administration in subjects with SLE, CLE, pSS, PM/DM, and/or dcSSc.
• American College of Rheumatology (ACR)/European League Against Rheumatism (EULAR) diagnosis of 1 of the following active autoimmune diseases: SLE or CLE (both must have CLASI \> 8 at both screening and baseline visits); pSS; PM/DM (probable or definite); and/or dcSSc (of \< 3 years duration).
• Subjects with SLE, CLE, or dcSSc must be willing to undergo skin biopsies at baseline and 2 skin biopsies during the treatment period; subjects with pSS must be willing to undergo salivary gland biopsies at baseline and 2 salivary gland biopsies during the treatment period; subjects with PM must be willing to undergo muscle biopsies at baseline and 2 muscle biopsies during the treatment period; and subjects with DM must be willing to undergo muscle biopsies at baseline and 2 muscle biopsies during the treatment period or they may choose to undergo skin biopsies instead.
• Between 18 and 75 years of age, inclusive, of either gender and of any race.
• Stable, but active disease, such that in the opinion of the Investigator, it is unlikely that a change in the subject's therapeutic regimen would be required during the subsequent 3 to 4 months.
• Photographs of skin lesions, when applicable, must be submitted for review to confirm that the lesions are suitable for punch biopsy and caused by SLE, CLE, or DM disease activity. Such confirmation by the Sponsor must be received prior to enrollment.
• Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF).
• Use of a highly effective contraceptive measure (\< 1% failure rate; see Section 13.1) for all non-sterilized males and all females of childbearing potential during study through 120 days after last DR-0201 dose. Females of childbearing potential need to have a negative serum pregnancy test within 7 days prior to the first dose of DR-0201. Females who are not of childbearing potential (i.e., who are considered to be post-menopausal \[≥ 12 months of non-therapy amenorrhea\] or surgically sterile \[absence of ovaries and/or uterus\]) are not required to have a pregnancy test or use contraception.