A Study In Adults With Moderate To Severe Dermatomyositis

Pfizer

Status and phase: Completed; Phase 2

Conditions: Dermatomyositis

Treatments: Drug: PF-06823859 low; Drug: PF-06823859 high; Drug: Placebo Arm

Study type: Interventional

Funder types: Industry

Identifiers

NCT03181893

2020-004228-41 (EudraCT Number)

C0251002

Details and patient eligibility

About

A Study looking at Investigational drug and Placebo administered to adult Patients with moderate to severe Dermatomyositis

Enrollment: 75 patients

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria for Patients with Skin Predominant Activity:

• Must have CDASI Activity score of greater than or equal to 14, and have failed at least 1 standard of care systemic treatment, (eg, corticosteroids).

• Confirmation of DM by the investigator and two of the following:

  1. Gottron's papules;
  2. Gottron's sign;
  3. Heliotrope eruption;
  4. Nailfold changes, (dilated capillary loops, capillary dropout, cuticular hypertrophy and/or rugged cuticles;
  5. Photodistributed violaceous erythema, (skin that is exposed to sunlight and appears purplish/reddish, and patchy in appearance;
  6. Positive DM serology -

• Post DM diagnosis; standard of care workup for DM must have been completed prior to entry into this research study.

• Willing to provide 8 biopsies during the course of the research study

Inclusion Criteria for Patients with Muscle Predominant Activity:

• MMT-8 ≤136/150 and PhGA, VAS ≥3 cm (0-10 cm) by visual analog scale (VAS)

• Sum of PhGA, VAS, PtGA, and extramuscular global assessment VAS scores is ≥10 cm (0-10 cm) VAS for each.

  • Participant has failed at least two or more adequate courses of an immunosuppressive agent or immunomodulatory agent, including IVIG, at a dose known to be effective for rheumatologic diseases.

Exclusion Criteria for Patients with Skin Predominant Activity:

• Investigator site staff or members of their family.
• Acute and Chronic present medical conditions
• Intake of greater than 15 mg of prednisone or equivalent per day
• Pregnant or breastfeeding females. Fertile men and women who will not comply with the use of 2 effective birth control methods as per the research protocol
• Have required management of acute or chronic infections
• Have pre existing demyelinating disorder such as multiple sclerosis, or other severe neurological deficits.
• Clinically significant lab abnormalities
• Any health condition that may be worsened by immunosuppression

Exclusion Criteria for Patients with Muscle Predominant Activity:

Similar to patients with skin predominant activity; Intake of >20 mg oral prednisone/day, or equivalent

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Crossover Assignment

Masking: Triple Blind

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75 participants in 3 patient groups, including a placebo group

Placebo ARM

Placebo Comparator group

Treatment:

Drug: Placebo Arm

PF-06823859 ARM high

Experimental group

Treatment:

Drug: PF-06823859 high

PF-06823859 ARM low

Experimental group

Treatment:

Drug: PF-06823859 low