A Study to Evaluate the Durability of Response of CTP-543 in Adult Patients With Moderate to Severe Alopecia Areata

Concert Pharmaceuticals

Status and phase: Completed; Phase 2

Conditions: Alopecia Areata

Treatments: Drug: Placebo; Drug: CTP-543

Study type: Interventional

Funder types: Industry

Identifiers

NCT04784533

CP543.2004

Details and patient eligibility

About

This study is designed to evaluate the regrowth of hair with CTP-543 and subsequent durability of that regrowth following dose reduction in adult patients with moderate to severe alopecia areata.

Enrollment: 317 patients

Sex: All

Ages: 18 to 65 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Definitive diagnosis of alopecia areata with a current episode of scalp hair loss lasting at least 6 months and not exceeding 10 years at the time of Screening. Total disease duration greater than 10 years is permitted.
• At least 50% scalp hair loss, as defined by a severity of alopecia tool (SALT) score ≥50, at Screening and Baseline.
• Willing to comply with the study visits and requirements of the study protocol.

Exclusion criteria

• Treatment with other medications or agents within 1 month of Baseline or during the study that may affect hair regrowth or immune response.
• Active scalp inflammation, psoriasis, or seborrheic dermatitis requiring topical treatment to the scalp, significant trauma to the scalp, or other scalp condition that may interfere with the SALT assessment, or untreated actinic keratosis anywhere on the body at Screening and/or Baseline.
• Treatment with systemic immunosuppressive medications within 3 months of Screening or during the study, or biologics within 6 months of Screening or during the study.
• Females who are nursing, pregnant, or planning to become pregnant while in the study, and for 30 days after last dose of study drug.
• Clinically significant medical condition, psychiatric disease, or social condition, as determined by the Investigator, that may unfavorably alter the risk-benefit of study participation, adversely affect study compliance, or confound interpretation of study results.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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317 participants in 10 patient groups, including a placebo group

Part A: Period 1 - CTP-543 8 mg BID

Experimental group

Description:

Participants received CTP-543 8 milligrams (mg) tablets, orally, twice daily (BID) for up to 24 weeks.

Treatment:

Drug: CTP-543

Part A: Period 1 - CTP-543 12 mg BID

Experimental group

Description:

Participants received CTP-543 12 mg tablets, orally, BID for up to 24 weeks.

Treatment:

Drug: CTP-543

Part A: Period 2 - CTP-543 8 mg BID to 4 mg BID

Experimental group

Description:

Participants who received CTP-543 8 mg during Period 1 of Part A and achieved an absolute severity of alopecia tool (SALT) score of ≤20 at Week 24, received CTP-543 4 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543

Part A: Period 2 - CTP-543 12 mg BID to 8 mg BID

Experimental group

Description:

Participants who received CTP-543 12 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543

Part A: Period 2 - CTP-543 8 mg BID to Placebo

Placebo Comparator group

Description:

Participants who received CTP-543 8 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 matched placebo tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: Placebo

Part A: Period 2 - CTP-543 12 mg BID to Placebo

Placebo Comparator group

Description:

Participants who received CTP-543 12 mg during Period 1 of Part A and achieved an absolute SALT score of ≤20 at Week 24, received CTP-543 matched placebo tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: Placebo

Part B: CTP-543 8 mg BID to 4 mg BID to 8 mg BID

Experimental group

Description:

Participants who received CTP-543 8 mg during Period 1, followed by CTP-543 4 mg during Period 2 of Part A and met the criteria for loss of regrowth maintenance (LOM) (absolute SALT score of \> 20), received re-treatment with CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543

Part B: CTP-543 12 mg BID to 8 mg BID to 12 mg BID

Experimental group

Description:

Participants who received CTP-543 12 mg during Period 1, followed by CTP-543 8 mg during Period 2 of Part A and met the criteria for LOM (absolute SALT score of \> 20), received re-treatment with CTP-543 12 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543

Part B: CTP-543 8 mg BID to Placebo to 8 mg BID

Experimental group

Description:

Participants who received CTP-543 8 mg during Period 1, followed by CTP-543 matched placebo during Period 2 of Part A and met the criteria for LOM (absolute SALT score of \> 20), received re-treatment with CTP-543 8 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543

Part B: CTP-543 12 mg BID to Placebo to 12 mg BID

Experimental group

Description:

Participants who received CTP-543 12 mg during Period 1, followed by CTP-543 matched placebo during Period 2 of Part A and met the criteria for LOM (absolute SALT score of \> 20), received re-treatment with CTP-543 12 mg tablets, orally, BID, for up to 24 weeks.

Treatment:

Drug: CTP-543