Pilot Study of the Safety and Efficacy of Apremilast in Subjects With Moderate to Severe Alopecia Areata

NCT02684123 | Completed Results DMC

• 1 other identifier: GCO 15-1817
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

Alopecia areata (AA) is a medical condition, in which the hair falls out in patches. The hair can fall out on the scalp or elsewhere on the face and body. Alopecia areata is an autoimmune skin disease, which means that the immune system is recognizing the hair follicles as foreign and attacking them, causing round patches of hair loss. It can progress to total scalp hair loss (alopecia totalis) or complete body hair loss (alopecia universalis). The scalp is the most commonly affected area, but the beard or any hair-bearing site can be affected alone or together with the scalp. Alopecia areata occurs in males and females of all ages, and is a highly unpredictable condition that tends to recur. Alopecia areata can cause significant distress to both patients and their families. In this study, the aim to assess the effects of a new treatment called apremilast in patients with alopecia areata. A total of 30 patients will be included in the study, which will run for a total of 52 weeks.

Conditions

Alopecia Areata

Keywords

Alopecia areata

Outcome Measures

Primary Outcomes (1)

• Number of Patients With SALT50

  Number of patients achieving 50% or greater improvement in their Severity of Alopecia Tool (SALT) score (SALT50) at Week 24 SALT50 is defined as the percent of patients achieving \>=50% reduction in SALT score compared to baseline. Scalp is divided into 4 areas namely, Vertex - 40% (0.4) of scalp surface area; right profile of scalp - 18% (0.18) of scalp surface area; left profile of scalp - 18% (0.18) of scalp surface area; Posterior aspect of scalp - 24% (0.24) of scalp surface area. Percentage of hair loss in any of these areas is percentage hair loss multiplied by percent surface area of the scalp in that area. SALT score is the sum of percentage of hair loss in all above mentioned areas.

  Baseline and Week 24

Secondary Outcomes (5)

• Mean Change in SALT Score

  Baseline and week 4, 8, 12, 16, 20, 24

• Phase 2: Number of Patients Achieving aaPGA Score of 3 or Above

  Week 24 and Week 48

• Phase 2: Change in AASIS

  Week 24 and Weeks 48

• Phase 2: AA-QoL

  Baseline, Week 24 and Weeks 48

• Phase 2: Semiquantitative Score

  Baseline, Week 24 and Weeks 48

Study Arms (2)

Apremilast

ACTIVE COMPARATOR

Apremilast 30mg twice daily

Drug: Apremilast

Placebo

PLACEBO COMPARATOR

Placebo pills twice daily

Drug: Placebo

Interventions

Apremilast DRUG

Subjects will get drug or placebo

Also known as: Otezla

Apremilast

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Males or females, 18 years or older at the time of signing the informed consent document.
• Understand and voluntarily sign an informed consent document prior to any study-related assessments/procedures are conducted.
• Able to adhere to the study visit schedule and other protocol requirements. Subject with a diagnosis of patchy scalp alopecia areata present for at least 6 months, and up to a maximum of 10 years.
• Patients with ≥50% and \<95% total scalp hair loss at Baseline as measured using the SALT score to qualify as moderate to severe AA; and 95%-100% scalp hair loss to qualify as AA totalis/universalis.
• Must meet the following laboratory criteria White blood cell count ≥ 3000/mm3 (≥ 3.0 x 109/L) and \< 14,000/mm3 (\< 14 x 109/L). Platelet count ≥ 100,000/μL (≥ 100 x 109/L). Serum creatinine ≤ 1.5 mg/dL (≤ 132.6 μmol/L). AST (SGOT) and ALT (SGPT) ≤ 2 x upper limit of normal (ULN). If the initial test shows ALT or AST \> 2 times the ULN, one repeat test is allowed during the Screening Phase.
• Total bilirubin ≤ 2 mg/dL (34 μmol/L). If the initial test shows total bilirubin \> 2 mg/dL (34 μmol/L), one repeat test is allowed during the Screening Phase.
• Hemoglobin ≥ 10 g/dL (≥ 6.2 mmol/L).
• Females of childbearing potential (FCBP) must have a negative pregnancy test at Screening and Baseline. While on investigational product and for at least 28 days after taking the last dose of investigational product (IP), FCBP who engage in activity in which conception is possible must use one of the approved contraceptive options described below:
• Option 1: Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy;
• Option 2: Male or female condom (latex condom or nonlatex condom NOT made out of natural \[animal\] membrane \[for example, polyurethane\]); plus spermicide PLUS one additional barrier method: (a) diaphragm with spermicide; (b) cervical cap with spermicide; or (c) contraceptive sponge with spermicide.
• Male subjects (including those who have had a vasectomy) who engage in activity in which conception is possible must use barrier contraception (male latex condom or nonlatex condom NOT made out of natural \[animal\] membrane \[for example, polyurethane\]) while on IP and for at least 28 days after the last dose of IP.
• No evidence of hair regrowth present at Baseline.

You may not qualify if:

• The presence of any of the following will exclude a subject from enrollment:
• Clinically significant (as determined by the investigator) cardiac, endocrine, pulmonary, neurologic, psychiatric, hepatic, renal, hematologic, or immunologic disease, or other major uncontrolled diseases that will affect the health of the subject during the study or interfere with the interpretation of study results.
• Hepatitis B surface antigen positive at Screening (Visit 1). Hepatitis C antibody positive at Screening (Visit 1). History of positive human immunodeficiency virus (HIV), or congenital or acquired immunodeficiency (eg, Common Variable Immunodeficiency \[CVID\]). Subjects deemed at risk by the study investigator, may also undergo testing for human immunodeficiency virus (HIV). Subjects deemed at risk include those with a history of injection drug use, homosexual subjects, and subjects with known sexual contact with an HIV positive partner.
• Active TB or a history of inadequately treated TB. Active substance abuse or a history of substance abuse within six months prior to Screening.
• Pregnant or breast feeding. History of allergy to any component of the IP.
• Major surgery within eight weeks prior to Screening (Visit 1) and/or planned surgery during the length of the study.
• Malignancy or history of malignancy, except for:
• treated (ie, cured) basal cell or squamous cell in situ skin carcinomas; treated (ie, cured) cervical intraepithelial neoplasia (CIN) or carcinoma in situ of the cervix with no evidence of recurrence within 5 years prior to Screening (Visit 1).
• Unstable asthma (eg, acute episodes of exacerbation \[nocturnal episodes, sudden episodes triggered by unidentifiable factors\] despite a stable regimen of anti-asthmatic medications); prior episode(s) of life-threatening asthma; or asthma that requires inhaled budesonide or equivalent at \>1200 μg/day or fluticasone propionate at \> 880 μg/day along with another anti-asthmatic drug such as a long-acting beta-agonist.
• A history of and/or concurrent condition of serious hypersensitivity (eg, anaphylaxis) to drugs, foods, or other allergens without access to emergency rescue medication such as epinephrine.
• Persistent or recurring bacterial infection requiring systemic antibiotics, or clinically significant viral or fungal infections, within two weeks of Screening (Visit 1). Any treatment for such infections must have been completed at least two weeks prior to the Screening Visit and no new/recurrent infections should have occurred prior to the Baseline Visit.
• Active skin infection requiring systemic antimicrobials at Baseline/Randomization (Visit 2).
• Skin lesion(s) due to conditions other than AA that would interfere with the study specified assessments.
• Prior treatment with apremilast, or participation in a clinical study involving apremilast.
• Use of phototherapy (ie, UVB, UVA) or systemic immunosuppressive drugs (including, but not limited to, cyclosporine, corticosteroids, mycophenolate mofetil, azathioprine, Methotrexate, or tacrolimus), or oral preparations of herbal immunomodulatory medications within four weeks prior to Baseline/Randomization (Visit 2).

Sponsors & Collaborators

• Icahn School of Medicine at Mount Sinai: lead

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

Location

Related Publications (4)

• Haider AS, Lowes MA, Suarez-Farinas M, Zaba LC, Cardinale I, Khatcherian A, Novitskaya I, Wittkowski KM, Krueger JG. Identification of cellular pathways of "type 1," Th17 T cells, and TNF- and inducible nitric oxide synthase-producing dendritic cells in autoimmune inflammation through pharmacogenomic study of cyclosporine A in psoriasis. J Immunol. 2008 Feb 1;180(3):1913-20. doi: 10.4049/jimmunol.180.3.1913.

  PMID: 18209089 BACKGROUND

• Alkhalifah A. Alopecia areata update. Dermatol Clin. 2013 Jan;31(1):93-108. doi: 10.1016/j.det.2012.08.010. Epub 2012 Oct 2.

  PMID: 23159179 BACKGROUND

• Croxford AL, Mair F, Becher B. IL-23: one cytokine in control of autoimmunity. Eur J Immunol. 2012 Sep;42(9):2263-73. doi: 10.1002/eji.201242598.

  PMID: 22949325 BACKGROUND

• Vaccaro M, Cannavo SP, Imbesi S, Cristani M, Barbuzza O, Tigano V, Gangemi S. Increased serum levels of interleukin-23 circulating in patients with non-segmental generalized vitiligo. Int J Dermatol. 2015 Jun;54(6):672-4. doi: 10.1111/ijd.12392. Epub 2014 Nov 27.

  PMID: 25427848 BACKGROUND

MeSH Terms

Conditions

Alopecia Areata

Interventions

apremilast

Condition Hierarchy (Ancestors)

Alopecia; Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases

Limitations and Caveats

This was an exploratory study. Future studies with a larger sample size are needed.

Results Point of Contact

• Title: Dr. Emma Guttman-Yassky
• Organization: Icahn School of Medicine at Mount Sinai

Emma.Guttman@mountsinai.org

(212) 241-9728

Study Officials

• Emma Guttman, MD, PhD

  ISMMS

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Associate Professor

Study Record Dates

• First Submitted: February 11, 2016
• First Posted: February 17, 2016
• Study Start: February 1, 2016
• Primary Completion: September 15, 2017
• Study Completion: September 15, 2017
• Last Updated: November 7, 2023
• Results First Posted: November 7, 2023

Record last verified: 2023-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)