A Phase 3 Study to Evaluate the Efficacy and Safety of Ritlecitinib in Children 6 to <12 Years of Age With Severe Alopecia Areata

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Pfizer Inc.

Participant type

Pediatric, Patients

Age range

0-17 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

12 Jan 2026 → ongoing

Decision date (initial)

2025-10-15

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Pharmacokinetic, Safety

To evaluate the efficacy of ritlecitinib compared to placebo in pediatric participants with AA on regrowth of lost scalp hair.

Secondary objectives 9

1. To evaluate the effect of ritlecitinib on patient centered outcomes (Patient’s Global Impression of Change [PGI‑C]).
2. To further evaluate the efficacy of ritlecitinib compared to placebo in pediatric participants with AA on regrowth of lost scalp hair at all visits (except for that included as the primary endpoint).
3. To evaluate the effect of ritlecitinib on patient centered outcomes, measured by PGI‑C response at all visits (except for that included as a key secondary endpoint).
4. To evaluate the effect of ritlecitinib on Alopecia Areata Patient Priority Outcomes (AAPPO) scales and psychological/social impairment proxy reported (Patient-Reported Outcomes Measurement Information System [PROMIS], Behavior Rating Inventory of Executive Function®, Second Edition [BRIEF®2]), and participant reported (modified Children’s Dermatology Life Quality Index [CDLQI]) at all visits.
5. To evaluate the effect of ritlecitinib versus placebo on achieving eyebrow assessment (EBA) response.
6. To evaluate the effect of ritlecitinib versus placebo on achieving eyelash assessment (ELA) response.
7. To characterize the pharmacokinetics (PK) of ritlecitinib in children with AA 6 to <12 years of age.
8. To evaluate safety and tolerability of ritlecitinib over time.
9. To evaluate acceptability and palatability of the age-appropriate formulation.

Conditions and MedDRA coding

Alopecia areata

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002451-PIP01-18

Plan to share IPD

Yes

IPD plan description

Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Participants who are 6 to <12 years old at the time of the screening visit.
2. A diagnosis of AA (including AT and AU) with at least 50% scalp hair loss due to AA (ie, a SALT score of ≥50) at both the screening and baseline visits, without evidence of terminal hair regrowth within the previous 12 months.
3. History of clinical response failure to AA treatment (such as topical, off-label pharmacologic, or hairpiece prosthetics)

Exclusion criteria 1

1. Other (non-AA) types of alopecia; any present or history of malignancies or lymphoproliferative disorders; evidence of untreated or inadequately treated active or latent Mycobacterium tuberculosis (TB) infection; history (one or more episodes) of severe or serious cytomegalovirus (CMV) infection, herpes zoster (shingles) or disseminated herpes simplex; history of any infection requiring hospitalization, parenteral antimicrobial therapy, or as otherwise judged clinically significant within prior 3 months; infection with hepatitis B virus (HBV) or hepatitis C virus (HCV).

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Response based on achieving an absolute SALT score ≤10 at Week 24.

Secondary endpoints 10

1. PGI‑C response defined as a score of “moderately improved” or “greatly improved” at Week 24.
2. • Change from baseline (CFB) in SALT score at all visits. • Response based on achieving absolute SALT score ≤10 at all visits (except for that included as the primary endpoint). • Response based on achieving absolute SALT score ≤20 at all visits (except for that included as the primary endpoint).
3. PGI-C response at all visits (except for that included as a key secondary endpoint).
4. •Response based on improvement from baseline for each AAPPO Item (11 endpoints). •CFB in AAPPO activity limitation and emotional symptoms scores at all visits. • CFB in PROMIS Parent Proxy Depressive Symptoms T-score at all visits.
5. •CFB in PROMIS Parent Proxy Anxiety Symptoms T-score at all visits. •CFB in BRIEF®2 T-scores for the 3 index scores (Behavior Regulation Index [BRI], Emotional Regulation Index [ERI], Cognitive Regulation Index [CRI]) at all visits. •CFB in modified CDLQI total score at all visits.
6. • Response based on achieving at least 2 grade improvement or a score of 3 in EBA score at all visits in participants with an abnormal EBA at baseline.
7. Response based on achieving at least 2 grade improvement or a score of 3 in ELA score at all visits in participants with an abnormal ELA at baseline.
8. Plasma concentration of ritlecitinib at 1 hr (±15 min) and 3 hr (±30 min) post-dose at Week 4 or Week 8.
9. •Incidence of treatment-emergent adverse events (AEs) (including audiological and neurological treatment-emergent AEs). •Incidence of serious AEs (SAEs) and AEs leading to permanent discontinuation from the study.
10. Acceptability and palatability assessment.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Pfizer Inc.

Sponsor organisation

Pfizer Inc.

Address

66 Hudson Boulevard East

City

New York

Postcode

10001-2189

Country

United States

Scientific contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Public contact point

Organisation

Pfizer Inc.

Contact name

Clinical Medical Lead

Third parties 20

Locations

5 EU/EEA countries · 33 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Corrective measures 1 · Art. 77 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 56 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

8 submissions — initial application plus substantial / non-substantial modifications