CTP-543 for Alopecia Areata

No longer recruiting at 38 trial locations

Overseen By Colleen E. Hamilton, MS

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Concert Pharmaceuticals

Must not be taking: Immunosuppressives, Biologics

Disqualifiers: Psoriasis, Pregnancy, Psychiatric disease, others

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called CTP-543, an experimental drug, to determine its effectiveness in promoting hair regrowth in adults with moderate to severe alopecia areata, a condition that causes hair loss. Researchers aim to find out if hair regrowth persists even when the dose is reduced. The trial includes different groups to test various doses and a placebo (a pill with no active drug) for comparison. Individuals who have lost at least half of their scalp hair due to alopecia areata for 6 months to 10 years might be suitable candidates. As a Phase 2 trial, this research focuses on assessing the treatment's effectiveness in an initial, smaller group of participants.

Will I have to stop taking my current medications?

The trial requires that you stop taking any medications that may affect hair regrowth or immune response within 1 month of starting the study. Additionally, you must not take systemic immunosuppressive medications within 3 months or biologics within 6 months of starting the study.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

Research has shown that CTP-543 is generally well-tolerated by people with alopecia areata. One study found that both doses of CTP-543 were well accepted and had a promising safety record. Most side effects were mild, such as common colds, occurring in about 10.3% of cases. However, the 12 mg dose did report some serious side effects, including a decrease in white blood cells and skin infections. These serious effects were rare. Overall, earlier studies have demonstrated that CTP-543 has a manageable safety profile. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for alopecia areata?

Unlike the standard treatments for alopecia areata, which often involve corticosteroids or immunosuppressants, CTP-543 is unique because it is a targeted oral treatment. CTP-543 works by blocking specific enzymes, known as Janus kinases (JAKs), which play a role in the autoimmune response that leads to hair loss in alopecia areata. Researchers are excited about CTP-543 because it offers the potential for a more precise approach, potentially resulting in fewer side effects and improved hair regrowth compared to existing therapies. Additionally, being an oral medication, it provides a convenient alternative to topical or injectable treatments.

What evidence suggests that this trial's treatments could be effective for alopecia areata?

Research has shown that CTP-543 may help treat alopecia areata, a condition that causes hair loss. In this trial, participants will receive CTP-543 at doses of 8 mg or 12 mg twice a day, or a placebo, to evaluate its effectiveness. Earlier studies demonstrated that participants taking CTP-543 experienced significantly less hair loss and observed thicker, more even hair compared to those who took a placebo. These findings suggest that CTP-543 could be effective for individuals with moderate to severe alopecia areata, offering hope for those seeking to regrow their hair. ^{1 6 7 8 9}

Are You a Good Fit for This Trial?

Inclusion Criteria

At least 50% scalp hair loss, as defined by a SALT score ≥50, at Screening and Baseline.

Willing to comply with the study visits and requirements of the study protocol.

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.

Exclusion Criteria

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.

You cannot take any medication or treatment that may affect hair growth or your immune system 1 month before or during the study.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A, Period 1

Participants receive CTP-543 8 mg or 12 mg tablets, orally, BID for up to 24 weeks

24 weeks

Treatment Part A, Period 2

Participants receive dose reduction or placebo for up to 24 weeks

24 weeks

Treatment Part B

Participants who met LOM criteria receive re-treatment with CTP-543 for up to 24 weeks

24 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• CTP-543

How Is the Trial Designed?

Group I: Part B: CTP-543 8 mg BID to Placebo to 8 mg BID Experimental Treatment 1 Intervention

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.

Group II: Part B: CTP-543 8 mg BID to 4 mg BID to 8 mg BID Experimental Treatment 1 Intervention

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.

Group III: Part B: CTP-543 12 mg BID to Placebo to 12 mg BID Experimental Treatment 1 Intervention

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.

Group IV: Part B: CTP-543 12 mg BID to 8 mg BID to 12 mg BID Experimental Treatment 1 Intervention

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.

Group V: Part A: Period 2 - CTP-543 8 mg BID to 4 mg BID Experimental Treatment 1 Intervention

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.

Group VI: Part A: Period 2 - CTP-543 12 mg BID to 8 mg BID Experimental Treatment 1 Intervention

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.

Group VII: Part A: Period 1 - CTP-543 8 mg BID Experimental Treatment 1 Intervention

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

Group VIII: Part A: Period 1 - CTP-543 12 mg BID Experimental Treatment 1 Intervention

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

Group IX: Part A: Period 2 - CTP-543 8 mg BID to Placebo Placebo Group 1 Intervention

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.

Group X: Part A: Period 2 - CTP-543 12 mg BID to Placebo Placebo Group 1 Intervention

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.

Who Is Running the Clinical Trial?

Concert Pharmaceuticals

Lead Sponsor

Trials

35

Recruited

4,400+

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/35364216/

Phase 2 randomized, dose-ranging trial of CTP-543, a ... Conclusions: Patients treated with CTP-543 (8 or 12 mg, twice daily) had a significant reduction in the severity of AA. Keywords: AA; JAK; JAK ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT03137381

Study to Evaluate the Safety and Efficacy of CTP-543 in ... This study will evaluate the safety and efficacy of CTP-543 on hair loss in adults with chronic, moderate to severe alopecia areata.

3. clinicaltrials.gov clinicaltrials.gov/study/NCT04784533

A Study to Evaluate the Durability of Response of CTP-543 ... 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 ...

4. jaad.org jaad.org/article/S0190-9622(23)02175-8/fulltext

42746 Patient Reported Outcomes in the Phase 3 THRIVE- ... Patients on both CTP-543 doses were significantly different from placebo at Week 24 for satisfaction with both thickness (p<0.001) and evenness (p <0.001) of ...

5. sciencedirect.com sciencedirect.com/science/article/pii/S0190962222005370

Phase 2 randomized, dose-ranging trial of CTP-543, a ... The results of treatment with CTP-543 suggest that this is an effective treatment for patients with ≥50% scalp hair loss. Alopecia areata (AA), an autoimmune ...

6. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC10155665/

Safety of Janus Kinase inhibitors in Patients with Alopecia ... The most common adverse effects of systemic JAK inhibitors were mild and consisted of upper respiratory infections (mean for all studies: 10.3%) ...

7. cdn.clinicaltrials.gov cdn.clinicaltrials.gov/large-docs/50/NCT04797650/Prot_000.pdf

CTP-543 Safety and tolerability of CTP-543 will be assessed by evaluating adverse events, vital signs, concomitant medications, and clinical laboratory ...

8. jaad.org jaad.org/article/S0190-9622(23)01699-7/fulltext

42736 Safety Assessments in the Multinational Phase 3 ... Both doses of CTP-543 were generally well tolerated. The overall safety profile of CTP-543 in patients with moderate to severe alopecia areata is encouraging.

9. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2024/217900Orig1s000MultidisciplineR.pdf

217900Orig1s000 MULTI-DISCIPLINE REVIEW Summary ... Study title/ number: An oral study of the effects of CTP-543 on embryo-fetal development in ... Safety of CTP-543 in Adult Patients with.