Baricitinib for Pediatric Hair Loss

(BRAVE-AA-PEDS Trial)

Recruiting at 231 trial locations

Overseen By Howard Sofen

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Eli Lilly and Company

Disqualifiers: Diffuse alopecia, Uncontrolled hypertension, Major surgery, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

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

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 2 Jurisdictions This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

595 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests baricitinib, a medication, to determine its safety and effectiveness for treating severe hair loss in children with alopecia areata. The study includes three groups: one receiving a placebo (a pill with no active ingredient) and two receiving different doses of baricitinib. Children who have experienced significant hair loss for at least a year and have not found success with other treatments might be suitable candidates. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants the opportunity to contribute to a potentially groundbreaking treatment.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

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

Research has shown that baricitinib is generally safe and effective for treating severe alopecia areata, a type of hair loss. One study found that 71% of teenagers who took baricitinib for a year experienced significant hair regrowth, indicating the drug's safety. Another study found that after 48 weeks, more than 75% of patients had moderate hair regrowth. These findings suggest that baricitinib can be effective and safe, with many patients experiencing positive results without serious side effects. ^{1 2 3 4 5}

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

Baricitinib is unique because it targets the Janus kinase (JAK) pathway, which plays a critical role in the inflammation processes that contribute to hair loss. Unlike standard treatments for pediatric hair loss, such as topical corticosteroids or minoxidil, Baricitinib is an oral medication, offering a different approach to treatment. Researchers are excited about Baricitinib because it has the potential to provide more effective and lasting results, especially for those who haven't responded well to current options. Plus, the ability to offer both low and high doses allows for tailored treatment to better meet individual needs.

What evidence suggests that baricitinib might be an effective treatment for alopecia areata in children?

Research has shown that baricitinib can help treat severe hair loss, known as alopecia areata, in teenagers. One study found that 71% of teens taking a 4 mg dose of baricitinib experienced successful hair regrowth after one year. Another study reported that after 48 weeks, 63% of patients had significant hair regrowth on their scalp, with over 75% seeing moderate improvement. Additionally, 54.1% of patients on the 4 mg dose and 31% on a lower dose had significant hair regrowth. This trial will evaluate the effectiveness of different doses of baricitinib, including a low dose and a high dose, compared to a placebo. These findings suggest that baricitinib could be an effective treatment for hair regrowth in young people with severe hair loss. ^{1 2 3 4 5}

Who Is on the Research Team?

Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST)

Principal Investigator

Eli Lilly and Company

Are You a Good Fit for This Trial?

This trial is for children aged 6 to less than 18 with severe alopecia areata (AA), a hair loss condition. They must have tried at least one treatment without success, had AA for over a year, and experienced an ongoing episode of AA for at least six months. Those with uncontrolled high blood pressure, recent major surgery, hepatitis B or C infections, HIV infection, or other serious health issues cannot participate.

Inclusion Criteria

I have had severe alopecia areata for less than 8 years or have seen hair regrowth in the last 8 years.

I have tried at least one treatment for alopecia areata without success.

I have had severe hair loss for at least a year.

Exclusion Criteria

I am not bedridden and can take care of myself.

I do not have any serious health issues that could risk my participation.

My blood pressure is under control.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

5 weeks

Double-Blind Treatment

Participants receive either baricitinib or placebo for the treatment of severe or very severe alopecia areata

36 weeks

Long-term Extension

Participants may continue to receive baricitinib for an extended period to assess long-term efficacy and safety

Approximately 2 years

Post-treatment Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Baricitinib

Trial Overview

The study tests the effectiveness and safety of Baricitinib in treating severe alopecia areata in children. It includes a screening period followed by a double-blind treatment phase where participants randomly receive either Baricitinib or placebo without knowing which one they're getting.

How Is the Trial Designed?

Group I: Baricitinib Low Dose Experimental Treatment 1 Intervention

Participants will receive baricitinib low dose orally.

Group II: Baricitinib High Dose Experimental Treatment 1 Intervention

Participants will receive baricitinib high dose orally.

Group III: Placebo Placebo Group 1 Intervention

Participants will receive placebo

Baricitinib is already approved in United States, European Union for the following indications:

Approved in United States as Olumiant for:

• Rheumatoid arthritis
• Severe alopecia areata
• COVID-19

Approved in European Union as Olumiant for:

• Rheumatoid arthritis
• Severe alopecia areata
• COVID-19

Who Is Running the Clinical Trial?

Eli Lilly and Company

Lead Sponsor

Trials

2,708

Recruited

3,720,000+

Dr. Daniel Skovronsky

Eli Lilly and Company

Chief Medical Officer since 2018

MD from Harvard Medical School

David A. Ricks

Eli Lilly and Company

Chief Executive Officer since 2017

BSc from Purdue University, MBA from Indiana University

Published Research Related to This Trial

The FDA approved the combination of dabrafenib and trametinib for treating pediatric patients with low-grade glioma (LGG) with a BRAFV600E mutation, marking the first systemic therapy approved for this condition.

In a clinical trial with 110 patients, the combination therapy showed a significantly higher overall response rate of 47% compared to 11% for the standard treatment of carboplatin and vincristine, along with longer progression-free survival of 20.1 months versus 7.4 months.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

FDA Approval Summary: Dabrafenib in combination with trametinib for BRAF V600E mutation-positive low-grade glioma. Barbato, MI., Nashed, J., Bradford, D., et al.[2023]

In a study involving 32 pediatric patients with BRAF V600-mutant low-grade glioma, dabrafenib showed a 44% objective response rate and an impressive 85% one-year progression-free survival rate, indicating its effectiveness in treating this type of brain tumor.

The treatment was generally well-tolerated, with 91% of patients experiencing treatment-related adverse events, primarily fatigue, and only 28% experiencing severe (grade 3/4) side effects.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and Safety of Dabrafenib in Pediatric Patients with BRAF V600 Mutation-Positive Relapsed or Refractory Low-Grade Glioma: Results from a Phase I/IIa Study. Hargrave, DR., Bouffet, E., Tabori, U., et al.[2023]

Baricitinib (Olumiant®) has been shown to significantly improve scalp coverage in patients with alopecia areata, with a higher percentage achieving at least 80% coverage after 36 weeks of treatment compared to placebo in two phase-3 trials.

While generally effective, baricitinib can cause side effects such as acne and urinary tract infections, and it is important to follow the recommended dosage of 2 mg or 4 mg daily based on hair loss severity.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Olumiant&#174; (Baricitinib)-A Newly Approved Janus Kinase Inhibitor for the Treatment of Alopecia Areata. Gupta, AK., Wang, T., Vincent, K., et al.[2022]

Ceritinib, an off-target inhibitor of the IGF1 receptor and insulin receptor, showed promising efficacy in treating a pediatric patient with an unclassified brain tumor, leading to a positive MRI response after 58 days of treatment.

The drug effectively inhibited tumor cell proliferation and accumulated in the tumor at a concentration of 2.72 µM, suggesting its potential as a viable option for targeting IGF-driven brain tumors despite the challenge of crossing the blood-brain barrier.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Ceritinib-Induced Regression of an Insulin-Like Growth Factor-Driven Neuroepithelial Brain Tumor. Russo, A., Paret, C., Alt, F., et al.[2020]

This study developed a population pharmacokinetic (PPK) model to determine initial sirolimus dosages for pediatric patients with PIK3CD mutation-related immunodeficiency, providing tailored dosing recommendations based on body weight and the use of posaconazole.

For children not taking posaconazole, recommended sirolimus dosages ranged from 0.04 to 0.07 mg/kg/day depending on weight, while those on posaconazole should receive a lower dose of 0.02 mg/kg/day, highlighting the importance of individualized treatment plans.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Initial sirolimus dosage recommendations for pediatric patients with PIK3CD mutation-related immunodeficiency disease. Chen, X., Wang, J., Lan, J., et al.[2022]

Novel agents like bispecific antibodies (blinatumomab and inotuzumab ozogamicin) targeting CD19 and CD22 are showing promise in treating relapsed and refractory B-cell precursor acute lymphoblastic leukaemia (BCP-ALL), potentially offering less toxic options compared to traditional therapies.

Targeted therapies for specific genetic subtypes of leukaemia, such as KMT2A-rearranged leukaemias and BCR-ABL-positive ALL, are being developed, but the pace of new drug approvals for pediatric cancers remains slower than for adults, highlighting the need for prioritization and international collaboration in research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Targeted inhibitors and antibody immunotherapies: Novel therapies for paediatric leukaemia and lymphoma. Brivio, E., Baruchel, A., Beishuizen, A., et al.[2022]

In a study involving 855 adults with severe alopecia areata, treatment with baricitinib (2 mg and 4 mg) showed continued improvement in scalp hair growth up to week 52, indicating its long-term efficacy.

Patients with a baseline Severity of Alopecia Tool (SALT) score of 50-94 responded better to baricitinib compared to those with scores of 95-100, and the higher 4 mg dose resulted in faster and greater response rates than the 2 mg dose.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy of Baricitinib in Patients with Various Degrees of Alopecia Areata Severity: Post-Hoc Analysis from BRAVE AA1 and BRAVE AA2. Taylor, S., Korman, NJ., Tsai, TF., et al.[2023]

Baricitinib, a JAK1/JAK2 inhibitor, has been shown to have a favorable safety profile in treating severe alopecia areata, with data collected from 1303 patients over a median of 532 days, indicating no deaths and a low incidence of serious infections.

Common treatment-emergent adverse events included upper respiratory infections and acne, with some differences noted compared to its use in atopic dermatitis, suggesting that the safety profile may vary based on the specific condition being treated.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Integrated safety analysis of baricitinib in adults with severe alopecia areata from two randomized clinical trials. King, B., Mostaghimi, A., Shimomura, Y., et al.[2023]

In a post hoc analysis of two clinical trials involving baricitinib treatment for alopecia areata, significant scalp hair regrowth was observed in patients after 52 weeks, with 70% of responders showing improvement in eyebrow and eyelash hair regrowth.

Even patients who did not achieve complete scalp hair regrowth experienced meaningful improvements in eyebrow and eyelash hair, indicating that baricitinib can enhance emotional well-being and quality of life, particularly for those with significant scalp hair improvement.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Benefits of Baricitinib Therapy According to Scalp Hair Regrowth in Patients with Severe Alopecia Areata. Senna, MM., Kwon, O., Piraccini, BM., et al.[2023]

In a pooled analysis of two phase 3 trials involving 1200 adults with severe alopecia areata, increasing the dose of baricitinib from 2 mg to 4 mg led to significant improvements in hair regrowth, with 25.9% of previously non-responding patients achieving a SALT score of 20 or lower by week 76.

Clinician-reported outcomes for eyebrow and eyelash hair loss also improved, with response rates increasing from 19.3% to 37.9% for eyebrows and from 24.1% to 40.9% for eyelashes, demonstrating the efficacy of uptitration in enhancing treatment outcomes.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Clinical Outcomes for Uptitration of Baricitinib Therapy in Patients With Severe Alopecia Areata: A Pooled Analysis of the BRAVE-AA1 and BRAVE-AA2 Trials. Ko, JM., Mayo, TT., Bergfeld, WF., et al.[2023]

Citations

1.

naaf.org

naaf.org/news/lilly-shares-52-week-results-of-baricitinib-olumiant-for-treatment-of-severe-alopecia-areata-in-adolescents-demonstrating-near-complete-hair-regrowth/

Lilly shares 52-week results of baricitinib (Olumiant®) for ...

The data showed that 71% of adolescents with severe AA treated with baricitinib 4 mg, “achieved successful scalp hair regrowth at one year, with ...

2.

prnewswire.com

prnewswire.com/news-releases/lillys-baricitinib-delivered-near-complete-scalp-hair-regrowth-at-one-year-for-adolescents-with-severe-alopecia-areata-in-phase-3-brave-aa-peds-trial-302593426.html

Lilly's baricitinib delivered near-complete scalp hair ...

Baricitinib provides significant hair regrowth in adolescents with severe alopecia areata: 52-week efficacy and safety results from a Phase 3 ...

3.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40985491/

Effectiveness and safety of baricitinib in severe alopecia ...

Key findings: After 48 weeks, 63% of patients had major scalp hair regrowth (SALT ≤20), and over 75% had moderate regrowth (SALT ≤30).

4.

dermnppa.org

dermnppa.org/new-longterm-data-for-lillys-baricitinib-in-pediatric-aa-lebrikizumab-in-adult-ad/

New Longterm Data for Lilly's Baricitinib in Pediatric AA ...

At one year, 54.1% of patients receiving baricitinib 4mg and 31% receiving baricitinib 2mg achieved successful hair regrowth (defined as 80% or ...

5.

clinicaltrials.gov

clinicaltrials.gov/study/NCT05723198

NCT05723198 | A Study of Baricitinib (LY3009104) in ...

The main purpose of this study is to determine the efficacy and safety of baricitinib for the treatment of severe or very severe alopecia areata (hair loss) ...