Study Evaluating Efficacy of Topical Squaric Acid Dibutyl Ester in Children and Adolescents With Alopecia Areata

NCT05414266 | Unknown Early Phase 1 FDA Drug

• 1 other identifier: HSC-MS-21-1035
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to evaluate the efficacy and tolerability of squaric acid dibutylester (SADBE) in children and adolescents with alopecia areata (AA) and to evaluate effect of squaric acid dibutyl ester on patient-centered outcomes and payer relevant measures to assess treatment benefit from the patient perspective and to demonstrate value

Conditions

Alopecia Areata

Keywords

Alopecia

Outcome Measures

Primary Outcomes (12)

• Hair loss as assessed by number of subjects that score <30 on the the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  end of treatment (12 months of treatment)

• Hair loss as assessed by the number of subjects that score <50 on the the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  month 4

• Overall hair loss as assessed by the SALT score

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• AA hair loss as assessed by the SALT score

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Change in overall hair loss as assessed by the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1,Month2,Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, month 11, Month 12,Month 14,early termination visit (occurs only if subject discontinues form study)

• Change in AA hair loss as assessed by the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1,Month2,Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, month 11, Month 12,Month 14,early termination visit (occurs only if subject discontinues form study)

• Overall hair loss as assessed by the number of subjects with at least 50% improvement in the SALT score as assessed by the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• AA hair loss as assessed by the number of subjects with at least 50% improvement in the SALT score as assessed by the Severity of alopecia tool (SALT)

  The SALT measures the percentage of hair loss on the scalp utilizing a visual aid showing the division of the scalp hair into four quadrants (back, top of scalp, and both sides), with each of the four quadrants given an accurate determination of the % of scalp surface area covered, representing 24%, 40%, 18%, and 18% of the total scalp surface area, respectively. Total SALT score is reported and calculated as: Total SALT Score=\[0.40x%scalp hair loss top quadrant\]+\[0.24x%scalp hair loss back quadrant\]+\[0.18x%scalp hair loss left quadrant\]+\[0.18x% scalp hair loss right quadrant\]

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of subjects that achieved a 1-point increase in eyebrow assessment as assessed by the eyebrow assessment (EBA) score on the right side

  This survey has a rating scale that ranges from 0 (none eyebrow) to 3 (normal eyebrow).This assessment will be performed for participants with eyebrows designated as one of the treatment areas, grading each eyebrow separately.

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of subjects that achieved a 2-point increase in eyebrow assessment as assessed by the eyebrow assessment (EBA) score on the left side

  This survey has a rating scale that ranges from 0 (none eyebrow) to 3 (normal eyebrow).This assessment will be performed for participants with eyebrows designated as one of the treatment areas, grading each eyebrow separately.

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of participants with hair loss as described as a 1-point decrease in the Clinician Global Impression - Alopecia Areata (CGI-AA)

  The Clinician Global Impression - Alopecia Areata (CGI-AA) is a single clinician-reported item developed to assess clinical impression of severity of scalp hair loss. The rater is asked to rate the participant's current hair loss on a 5 point static discrete scale ranging from 0 "None (no hair loss)", 1 "Minimal Hair Loss, 2 "Moderate Hair Loss", 3 "Severe Hair Loss" 4 "Very Severe or complete hair loss" with higher scores indicating more severe hair loss.

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of participants with hair loss as described as a 2-point decrease in the Clinician Global Impression - Alopecia Areata (CGI-AA)

  The Clinician Global Impression - Alopecia Areata (CGI-AA) is a single clinician-reported item developed to assess clinical impression of severity of scalp hair loss. The rater is asked to rate the participant's current hair loss on a 5 point static discrete scale ranging from 0 "None (no hair loss)", 1 "Minimal Hair Loss, 2 "Moderate Hair Loss", 3 "Severe Hair Loss" 4 "Very Severe or complete hair loss" with higher scores indicating more severe hair loss.

  Baseline, Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

Secondary Outcomes (6)

• AA as assessed by the Patient's Global Impression of Change (PGI-C) scale

  Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of subjects with moderately improved AA as assessed by the Patient's Global Impression of Change (PGI-C) scale

  Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Number of subjects with greatly improved AA as assessed by the Patient's Global Impression of Change (PGI-C) scale

  Month 1, Month 2, Month 3, Month 4, Month 5, Month 6, Month 7, Month 8,Month 9, Month 10, Month 11, Month 12(end of treatment),Month 14(end of study),early termination visit (occurs only if subject discontinues form study)

• Quality of life as assessed by the Dermatology Life Quality Index( DLQI)

  Baseline,Mont4, Month 6, Month 9,Month 12

• Quality of life as assessed by the Children's Dermatology Life Quality Index (CDLQI)

  Baseline,Mont4, Month 6, Month 9,Month 12

Study Arms (2)

4 months Placebo, then 8 months SADBE 5%

EXPERIMENTAL

Drug: Placebo; Drug: SADBE 5%

12 months SADBE 5%

EXPERIMENTAL

Drug: SADBE 5%

Interventions

Placebo DRUG

Placebo is acetone. At baseline, the investigator will record the area that will be treated in mm2 . Use of a 1 mm x 1 mm gridded transparency will be used to record the treatment area at baseline. At subsequent visits, the study intervention will be applied to the entire treatment area defined at baseline, regardless of any new hair growth. The assigned study intervention is applied monthly by the PI (or designee) to the treatment areas affected by AA on the scalp and/or eyebrows identified at Baseline. The topical solution is applied with a cotton tip applicator by the PI or their qualified designee. Solution is applied by gently pressing a cotton tip applicator that has been soaked in the study solution against the skin. Treatment is repeated monthly.

4 months Placebo, then 8 months SADBE 5%

SADBE 5% DRUG

SADBE 5% is a solution of 5% Squaric Acid Dibutyl Ester (SADBE) in acetone. At baseline, the investigator will record the area that will be treated in mm2 . Use of a 1 mm x 1 mm gridded transparency will be used to record the treatment area at baseline. At subsequent visits, the study intervention will be applied to the entire treatment area defined at baseline, regardless of any new hair growth. The assigned study intervention is applied monthly by the PI (or designee) to the treatment areas affected by AA on the scalp and/or eyebrows identified at Baseline. The topical solution is applied with a cotton tip applicator by the PI or their qualified designee. Solution is applied by gently pressing a cotton tip applicator that has been soaked in the study solution against the skin. Treatment is repeated monthly.

12 months SADBE 5%; 4 months Placebo, then 8 months SADBE 5%

Eligibility Criteria

• Age: 12 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Evidence of a personally signed and dated informed consent document indicating that the participant or a legally acceptable representative/parent(s)/legal guardian has been informed of all pertinent aspects of the study.
• Willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.
• not pregnant or breast feeding
• Participants who are women of child bearing potential(WOCBP) must agree to use one highly effective method of contraception(with a failure rate of \<1% per year during the intervention period and for at least 28 days after the last dose of study intervention.
• Negative highly sensitive pregnancy (urine) at the Day 1 visit before the first dose of study intervention for WOCBP
• Have a clinical diagnosis of AA with no other etiology of hair loss (e.g., telogen effluvium,androgenetic alopecia, etc.)
• greater than 10% hair loss of the scalp, including without evidence of terminal hair regrowth within 6 months at both screening and baseline visits
• Current episode of hair loss less than 5 years.
• must be on a stable regimen of permitted concomitant medication
• Must agree to avoid prolonged exposure to the sun and not to use tanning booths, sun lamps or other ultraviolet light sources in the treatment area during the study.

You may not qualify if:

• Other scalp disease that may impact AA assessment (e.g., scalp psoriasis, dermatitis, etc).
• Active systemic diseases that may cause hair loss (e.g., lupus erythematosus, thyroiditis, systemic sclerosis, lichen planus, etc).
• Any psychiatric condition including recent or active suicidal ideation or behavior
• Ongoing or recent history of any other uncontrolled and/or clinically significant medical or psychiatric disease or condition which, in the PI's medical opinion, should exclude participation in the study.
• Any present malignancies or history of malignancies with the exception of adequately treated or excised non metastatic basal cell or squamous cell cancer of the skin or cervical carcinoma in situ.
• History (single episode) of disseminated herpes zoster or disseminated herpes simplex, or a recurrent (more than one episode of) localized, dermatomal herpes zoster
• History of systemic infection requiring hospitalization, parenteral antimicrobial therapy, or as otherwise judged clinically significant by the investigator within 6 months prior to Day 1
• Known primary or secondary immunodeficiency disorder or a first-degree relative with a hereditary immunodeficiency
• Significant trauma or major surgery within 1 month of the first dose of study intervention.
• Considered in imminent need for surgery. Participants with elective surgery scheduled can only be enrolled in Study SADBE-high dose (HD) with the approval of the investigator.
• Participant has a known hypersensitivity or previous allergic reaction to any of the active or inactive ingredients in the study intervention
• Anticipated treatment with prohibited concomitant medication(s) during the course of the study
• Received Herbal medications with either unknown properties or pharmaceutical properties that impact AA within 1 week of first dose of study intervention
• Received topical steroids (e.g., steroid cream, steroid ointment) on areas under assessment (i.e., scalp, eyebrows). within 2 week of first dose of study intervention
• Received Ultraviolet B (UVB) phototherapy, Psoralen Ultraviolet A (PUVA) therapy, other phototherapy, contact immunotherapy \[e.g.diphenylcyclopropenone (DPCP), and 1-chloro-2,4-dinitrobenzene (DNCB)\], topical irritants(e.g., anthralin), and liquid nitrogen cryotherapy within 4 weeks of first dose of study intervention

Sponsors & Collaborators

• The University of Texas Health Science Center, Houston: lead

Study Sites (1)

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

RECRUITING

MeSH Terms

Conditions

Alopecia Areata; Alopecia

Condition Hierarchy (Ancestors)

Hypotrichosis; Hair Diseases; Skin Diseases; Skin and Connective Tissue Diseases; Pathological Conditions, Anatomical; Pathological Conditions, Signs and Symptoms

Study Officials

• Adelaide Hebert, MD

  The University of Texas Health Science Center, Houston

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Adelaide A Hebert, MD

CONTACT

713-500-8276; Adelaide.A.Hebert@uth.tmc.edu

Eugenio G Galindo, MD

CONTACT

713-500-8278; Eugenio.G.Galindo@uth.tmc.edu

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: June 7, 2022
• First Posted: June 10, 2022
• Study Start: August 15, 2022
• Primary Completion: June 1, 2023
• Study Completion: May 22, 2024
• Last Updated: October 13, 2022

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)