A Long-Term Study of the Medicine Called Abrocitinib in Children Aged 2 Years and Older with Moderate to Severe Eczema

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] - Skin and Connective Tissue Diseases [C17]

Trial duration

19 Dec 2025 → ongoing

Decision date (initial)

2025-08-05

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Pfizer, Inc

External identifiers

EU CT number

2023-509124-18-00

ClinicalTrials.gov

NCT06807281

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate long-term safety of abrocitinib in children ≥2 years of age with moderate-to-severe disease

Secondary objectives 3

1. To evaluate long-term safety of abrocitinib in children ≥2 years of age with moderate-to-severe disease
2. To evaluate long term efficacy of abrocitinib in children ≥2 years of age with moderate-to-severe disease
3. To assess effect of abrocitinib on vaccine immunity

Conditions and MedDRA coding

Moderate-to-Severe Atopic Dermatitis

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

EMA paediatric investigation plan (PIP)

EMEA-002312-PIP01-17

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 4

1. Participants who have completed the treatment phase of the qualifying parent study (age 2 to <12 years old).
2. Children aged 6 to <12 years at the time of informed consent/assent.
3. Participants who meet all of the following AD criteria:  A documented diagnosis of chronic AD for at least 6 months prior to screening and confirmed at screening and baseline visits according to the Hanifin and Rajka criteria[19]; and  A diagnosis of moderate-to-severe AD at the baseline visit (must fulfill all of the following criteria: BSA ≥10%, vIGA ≥3, EASI ≥16, and WI-NRS ≥4); and  Documented history (within 6 months of the screening visit) of inadequate response to treatment with topical medical therapy for AD (eg, TCS and TCI), for at least 4 weeks and are candidates for systemic therapy.
4. Body weight ≥15 kg

Exclusion criteria 13

1. Any medical or psychiatric condition including any active suicidal ideation in the past year or suicidal behavior in the past 5 years or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
2. Required use of any prohibited concomitant treatments outlined in Section 6.9.3 and Appendix 9.
3. Required vaccination with live attenuated vaccines during study treatment and for 6 weeks after discontinuing study treatment.
4. Ongoing adverse event in the parent studies which in the opinion of the investigator, or sponsor, is an ongoing safety concern OR the participant is currently triggering safety monitoring criteria.  Available results from assessments performed as part of the Week 12 visit of the parent study should be evaluated to determine eligibility and appropriateness of the participant to be enrolled. Clinically significant abnormalities on physical examination at Week 12 visit or any unresolved, clinically significant abnormality from results of ECGs (if applicable), safety laboratory assessments, or vital signs measurements recorded in the parent study that may increase the risk associated with study participation or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the participant inappropriate for entry into this study, are exclusionary.
5. Discontinued from treatment early in the parent studies OR triggered a discontinuation criterion at any point during the parent studies OR meets exclusion criteria from the parent studies which in the opinion of the investigator, or sponsor, is an ongoing safety concern.
6. Any medical or psychiatric condition including any active suicidal ideation in the past year or suicidal behavior in the past 5 years or laboratory abnormality that may increase the risk of study participation or, in the investigator’s judgment, make the participant inappropriate for the study.
7. Have any of the following medical conditions:  Infections: - Skin infections that require treatment with systemic antimicrobials within 2 weeks prior to Day 1 (baseline) or have superficial skin infections within 1 week of Day 1. - History of systemic infection requiring hospitalization or parenteral antimicrobial therapy or as otherwise judged clinically significant by the investigator within 1 month prior to Day 1. - Have a history (single episode) of disseminated herpes zoster or disseminated herpes simplex, or a recurrent localized, dermatomal herpes zoster. - Known or suspected infection with HIV, hepatitis B, and/or hepatitis C (Section 8.3.6). - Evidence of active TB or inadequately treated latent TB.  Skin Conditions: - Including but not limited to psoriasis, seborrheic dermatitis or lupus on Day 1 that would interfere with evaluation of AD or response to treatment.  Other Conditions: - Documented history of skeletal dysplasia. - Documented history of retinal detachment. - History of or conditions associated with thrombocytopenia, coagulopathy or platelet dysfunction. - Prior history of leukemia, lymphoma, sarcoma or any other malignancy. - Immunodeficiency disorder or a first-degree relative with a hereditary immunodeficiency. - Any other medical conditions that in the investigator’s judgment make the participant inappropriate for the study.
8. Prior treatment with a systemic JAK inhibitor for AD.
9. Live attenuated vaccination within 6 weeks prior to Day 1 or require vaccination with live attenuated vaccines during treatment or within 6 weeks after the last dose of study intervention.
10. Concomitant use of strong inhibitors and inducers of CYP2C19 enzymes and strong inducers of CYP2C9 enzymes is not allowed in the study.
11. Previous administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, of Day 1.
12. Hepatic and/or renal and/or hematological abnormalities defined as:  AST >2 x ULN  Hemoglobin <10 g/dL  ALT >2 x ULN  ANC <1000/mm3  Total bilirubin ≥1.5 x ULN  ALC <500/mm3  eGFR 60 mL/min/1.73 m2  Platelets <150,000 /mm3
13. Investigator site staff directly involved in the conduct of the study and their family members, site staff otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly involved in the conduct of the study and their family members.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Overall treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and AEs that lead to discontinuation of study intervention.

Secondary endpoints 17

1. Clinically significant laboratory abnormalities
2. Response based on achieving vIGA score of clear (0) or almost clear (1) (on a 5-point scale) and ≥2-point reduction from baseline1 at all scheduled time points.
3. Response based on achieving a ≥4-point improvement from baseline1 in the Worst Itch-Numerical Rating Scale (WI-NRS) at all scheduled time points.
4. Response based on achieving a ≥4-point improvement from baseline1 in the Worst Scratch/Itch-Numerical Rating Scale (WSI-NRS) at all scheduled time points.
5. Response based on achieving ≥50%, ≥75%, ≥90%, 100% improvement from baseline1 in the Eczema Area and Severity Index (EASI) total score (EASI-50, EASI-75, EASI-90, EASI-100) at all scheduled time points.
6. Percent change from baseline (CFB)1 in EASI total score at all scheduled time points.
7. CFB1 in the percentage Body Surface Area (BSA) affected at all scheduled time points.
8. Loss of response due to protocol-defined flare2
9. CFB1 in Children’s Dermatology Life Quality Index (CDLQI) at all scheduled time points.
10. CFB1 in Infants’ Dermatitis Quality of Life (IDQOL) at all scheduled time points.
11. CFB1 in Patient-Oriented Eczema Measure (POEM) at all scheduled time points.
12. CFB1 in Dermatitis Family Impact (DFI) at all scheduled time points
13. CFB1 in Patient Reported Global Impression of Severity (PGIS) at all scheduled time points
14. CFB1 in Observer Reported Global Impression of Severity (OGIS) at all scheduled time points
15. CFB1 in the EuroQol- 5 Dimension Youth (EQ-5D-Y) at all scheduled time points
16. Number of topical corticosteroids- and / or topical calcineurin inhibitor-free days.
17. Proportion of participants achieving satisfactory response in Tetanus, Diphtheria and Acellular Pertussis Vaccine (Tdap)/Diphtheria, Tetanus & Pertussis vaccine (DTaP) and/or pneumococcal antibody titers as appropriate in participants who receive Tdap/DTaP and/or pneumococcal vaccinations

Investigational products

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

Test 1

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 9

Locations

4 EU/EEA countries · 17 investigational sites

By country

Investigational sites

Country notifications

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

Results and documents

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

Documents 64 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