Safety, Tolerability, and Efficacy of JBT-101 in Subjects With Dermatomyositis

Corbus Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Dermatomyositis

Treatments: Drug: JBT-101; Drug: Placebo

Study type: Interventional

Funder types: Other; Industry; NIH

Identifiers

NCT02466243

JBT101-DM-001

R21AR066286 (U.S. NIH Grant/Contract)

116313

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, tolerability and efficacy of JBT-101 in adult subjects with skin-predominant, dermatomyositis (DM) that is refractory to at least 3 months treatment with hydroxychloroquine.

Full description

Part A: An interventional, double-blind, randomized, placebo-control design will be used to test JBT-101 in about 22 eligible male or female subjects ≥ 18 and ≤ 70 years of age with moderate-to-severe active skin-predominant dermatomyositis.

Part B: A one-year open-label design to test JBT-101 in subjects who completed Part A without permanent discontinuation of study product because of safety or tolerability reasons.

Enrollment: 22 patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria (Part A):

• CDASI activity score ≥ 14;
• No difficulty with lifting or walking, and no more than 1.5 x the upper limit of normal of creatine phosphokinase or aldolase;
• Failed at least 3 months treatment with hydroxychloroquine;
• Stable treatment for dermatomyositis for at least 28 days before Visit 1 (Day 1).

Inclusion Criteria (Part B):

• Completion of dosing in Part A without permanent discontinuation of study product because of safety or tolerability reasons

Exclusion Criteria (Part A and B):

• Significant diseases or conditions other than DM that may influence response to the study product or safety;

• Any one of the following values for laboratory tests at Screening:

  1. A positive pregnancy test (or at Visit 1);
  2. Hemoglobin < 10 g/dL;
  3. Neutrophils < 1.0 x 10^9/L;
  4. Platelets < 75 x 10^9/L;
  5. Creatinine clearance < 50 ml/min according to modified Cockcroft-Gault equation;
  6. Aspartate aminotransferase, alanine aminotransferase, or alkaline phosphatase > 2.5 x upper normal limit;
  7. Total bilirubin ≥ 1.5 x upper limit of normal.

• Any other condition that, in the opinion of the Principal Investigator, is clinically significant and may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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22 participants in 2 patient groups, including a placebo group

JBT-101

Experimental group

Description:

Part A: JBT-101 20 mg capsule once a day on Days 1-28, then 20 mg capsule twice a day on Days 29-84. Part B: JBT-101 20 mg twice daily on Days 1 - 365 of the OLE.

Treatment:

Drug: JBT-101

Placebo

Placebo Comparator group

Description:

Part A: Placebo capsule once a day on Days 1-28, then placebo capsule twice a day on Days 29-84. Part B: Placebo twice daily on Days 1 - 365 of the OLE.

Treatment:

Drug: Placebo