Clinical trials are a crucial step in drug development, serving as the bridge between lab research and the approval of new treatments for public use. They’re essential because they allow researchers to assess a treatment’s safety in humans, understand its effectiveness, find the right dosage, identify any side effects, and see how it stacks up against existing treatments.

But before a drug even reaches clinical trials, it undergoes extensive pre-clinical research in the lab, ensuring that only the most promising treatments move forward for testing in people.

Clinical Trial Phases: A Step-by-Step Overview

Trials progress through Phases I-IV to test a drug candidate, find the appropriate dosage, and look for side effects. The goal is to determine whether a medicine is both safe and effective. Each new phase of a clinical trial builds on information from previous stages; each designed to find out specific information. The findings from clinical trials are submitted to the U.S. Federal Drug Administration or it’s counterpart European Medicines Agency to review and determine if a new treatment will be approved to go to market.

Pre-Clinical Stage

This stage includes lab and animal studies to gauge a new compound’s safety and potential effectiveness. It usually takes 3-6 years and includes:

• In Vitro Studies: Test tube or cell culture experiments
• In Vivo Studies: Testing on animals
• Toxicology Studies: Checking for toxicity
• Pharmacokinetic Studies: Understanding how the compound moves through the body

Only about 1 in 1,000 compounds tested in the pre-clinical stage make it to human clinical trials.

Phase 1

• Purpose: Primarily assesses safety and dosage
• Participants: 20-100 healthy volunteers or individuals with the condition
• Duration: Several months

Success Rate: Around 70% of drugs move to the next phase

Phase 2

• Purpose: Continues testing safety and begins evaluating effectiveness
• Participants: Up to several hundred people with the condition
• Duration: Several months to 2 years

Success Rate: About 33% of drugs advance to the next phase

Phase 3

• Purpose: Confirms effectiveness, monitors side effects, and compares the new treatment with standard options
• Participants: 300-3,000 people with the condition
• Duration: 1 to 4 years

Success Rate: 25-30% of drugs move to the next phase

Phase 4

• Purpose: Monitors long-term safety and effectiveness after the treatment is FDA-approved
• Participants: Several thousand people who use the treatment
• Duration: Ongoing, often several years

From discovery to market approval, this entire process generally takes 10-15 years. However, only about 1 in 10 drugs that enter clinical trials ultimately receive FDA approval.

Why Consider a Clinical Trial?

Finding an effective treatment for vitiligo can be challenging, especially if past therapies haven’t given the results you hoped for. If you’re open to exploring new options, a clinical trial might be a great next step. In late-stage trials, you’ll have the chance to try innovative treatments—like JAK inhibitors (ruxolitinib, ritlecitinib) and prostaglandin analogues—that are already showing promising results. Participants receive close, high-quality care, with regular check-ups and personalized support from specialists.

By joining a clinical trial, you’re also helping advance vitiligo research, which could make a difference for others in the future. Many trials cover the cost of the treatment and care, making it a more affordable option. For those who haven’t had success with current therapies, clinical trials can bring new hope and fresh possibilities.

Latest Developments

Recent studies have shown real promise:

• JAK Inhibitors: Topical ruxolitinib has led to significant repigmentation in phase III trials, with around 30% of patients seeing a 75% or greater improvement in their facial Vitiligo Area Scoring Index after 24 weeks. (Update: it has been approved by the FDA and available under the Opzelura name.)
• Oral Treatments: A phase IIb trial of oral ritlecitinib showed accelerated improvement by week 28.
• Combination Therapies: Combining approaches like microneedling with topical treatments has demonstrated enhanced effectiveness over standalone treatments.

What to Expect

When participating in a clinical trial, you can generally expect:

• An initial screening to determine if you’re eligible
• Regular visits for treatment and evaluation
• Careful monitoring of your progress and any side effects
• Some studies may use a placebo, so results are measured accurately

Remember, your participation is always voluntary, and you’re free to withdraw from the study at any time.

How to Get Involved

If you’re interested in exploring clinical trials for vitiligo:

• Talk with your dermatologist about trial options
• Visit clinicaltrials.gov to look for studies in your area
• Contact vitiligo research centers or foundations for details on ongoing trials

Clinical trials offer exciting new possibilities but also come with potential risks. Discussing these options with your healthcare provider can help you decide if a clinical trial is the right fit for you.

 

Suggested reading

• Vitiligo Drug Pipeline Analysis and Market Insights
• Vitiligo Patient Journey Map
• Ayahuasca as a Potential Therapy for Vitiligo