Clinical trial NCT03953209: Spironolactone for the Treatment of Melasma
Official title: Spironolactone for the Treatment of Melasma: a Prospective, Open-label Proof-of-concept and Dose-ranging Study
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE1
Conditions: Melasma
Keywords: spironolactone
Lead sponsor: Medical University of South Carolina
Sponsor class: OTHER
First posted: 2019-05-16
Enrollment: 0
Sex: FEMALE
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to prospectively gather data on the efficacy and tolerability of spironolactone to treat refractory melasma and to compare treatment response of randomized 50 mg, 100mg, and 200mg per day dosing.

Detailed description: Melasma is a common, acquired condition of the skin characterized by symmetric reticulated hyperpigmented patches, most often of the central face and mandible.

An array of treatment modalities encompassing topical, oral, procedural, and combination therapies have been used to treat the disorder, with varying levels of immediate and long-term efficacy. Current treatment options are often unsuccessful in inducing complete disease remission. The tolerability of certain treatments is a valid concern, with adverse reactions such as irritation, peeling, burning, and post-inflammatory hyperpigmentation a common occurrence. Antiandrogens like spironolactone have been successfully used as adjunctive treatment for melasma. However, evidence for its off-label use for this purpose remains anecdotal. Clinical data regarding its efficacy and dosing are absent from the literature and are likely barriers to the drug's use in melasma. The high relapse rates and unfavorable side effect profiles of traditional treatment regimens underlie the need for an improved multimodal approach.

This prospective, randomized trial seeks to obtain information about the efficacy and tolerability of spironolactone to treat refractory melasma to guide future studies and clinical decision making. Participants will be randomized to one of the three dosage groups and take the study drug for 12 weeks. Outcomes will be assessed at baseline, week 6, week 12, and week 16. Subjects may opt to participate in a treatment extension period of up to 9 additional months, in which they will return to clinic monthly for outcome assessment.

Eligibility criteria: Inclusion Criteria:

1. Subject must have clinically diagnosed melasma, with an inadequate response to other treatment modalities, including but not limited to depigmenting agents, topical retinoids, topical steroids, peeling agents, laser and light therapies and combined topical creams.
2. Subject must be a female.
3. Subject must be 18-70 years of age.
4. Women of child-bearing potential must be on effective contraception. Acceptable methods of contraception include oral contraceptive pills (OCPs), hormonal or copper IUDs, contraceptive implants, contraceptive injections, birth control patches, vaginal rings, condom, sponge, diaphragm with spermicide, or prior surgical sterilization.
5. Subject must provide written informed consent prior to any study-related procedures being performed.
6. Subject must be willing to comply with all clinical study procedures.

Exclusion Criteria:

1. Subject is a pregnant or nursing female.
2. Subject has previously received spironolactone or another antiandrogenic treatment for melasma.
3. Subject is hyperkalemic, defined by a potassium level of greater than 5.1 mEq/liter.
4. Subjects with Addison's disease.
5. Subjects taking eplerenone or other potassium-sparing diuretics, lithium, cholestyramine, ACE inhibitors/angiotensin II antagonists/aldosterone blockers, or NSAIDS.
6. Subjects receiving potassium supplementation.
7. Subjects with history of renal disease or an eFGR \< 30.
8. Subjects with acute or chronic liver failure.
9. Subject has an acute psychiatric condition that impairs ability to give consent or follow study protocols.

Primary outcomes:
measure: Proportion of patients achieving clinical response
timeFrame: baseline and 12 weeks
Secondary outcomes:
measure: Proportion of patients achieving clinical response
timeFrame: week 6, week 16, and optional treatment extension period visits lasting up to 1 yr
measure: Change in Dermatology Life Quality Index (DLQI)
timeFrame: baseline, week 6, week 12, week 16, and week 6, week 16, and optional treatment extension period visits lasting up to 1 yr
measure: Tolerability of spironolactone as measured by incidence of treatment-related adverse events
timeFrame: baseline, week 6, week 12, week 16, and week 6, week 16, and optional treatment extension period visits lasting up to 1 yr
measure: Comparing proportion of patients achieving clinical response between the three treatment arms: 50 mg, 100 mg, and 200 mg of spironolactone
timeFrame: week 6, week 12, week 16, and week 6, week 16, and optional treatment extension period visits lasting up to 1 yr
Other outcomes:
Locations:
city: Charleston
country: United States
facility: Medical University of SC
state: South Carolina
Study officials:
affiliation: Medical University of South Carolina
name: Dirk Elston, MD
role: PRINCIPAL_INVESTIGATOR
References: