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Efficacy and Safety of Adalimumab for the Treatment of Ocular Inflammation (PANDA-U)

tiistai 27. lokakuuta 2026 päivittänyt: Samsung Medical Center

Efficacy and Safety of Adalimumab in Pediatric Non-Infectious Uveitis: A Multicenter Ambispective Single-Arm Clinical Trial

This study aims to evaluate the clinical efficacy and safety of adalimumab in pediatric non-infectious uveitis and to generate domestic real-world evidence (RWE), thereby establishing a clinical foundation for label expansion and reimbursement coverage.

Tutkimuksen yleiskatsaus

Yksityiskohtainen kuvaus

Background: Although anti-TNFα agents play a crucial role globally in managing refractory non-infectious uveitis, adalimumab was removed from the approved indication for pediatric uveitis in South Korea several years ago-unlike adult uveitis-making domestic prescription exceptionally difficult. Meanwhile, indications for pediatric idiopathic arthritis and pediatric Crohn's disease remain approved domestically, highlighting an urgent need to establish objective evidence to resolve this regulatory discrepancy. Furthermore, as a substantial number of pediatric patients in Korea are already receiving off-label, non-reimbursed adalimumab treatment, their real-world data serve both as evidence of efficacy and safety and as direct proof of this institutional imbalance.

Opintotyyppi

Interventio

Ilmoittautuminen (Arvioitu)

40

Vaihe

  • Ei sovellettavissa

Yhteystiedot ja paikat

Tässä osiossa on tutkimuksen suorittajien yhteystiedot ja tiedot siitä, missä tämä tutkimus suoritetaan.

Opiskeluyhteys

Opiskelupaikat

      • Seoul, Etelä -Korea, 06351
        • Samsung Medical Center

Osallistumiskriteerit

Tutkijat etsivät ihmisiä, jotka sopivat tiettyyn kuvaukseen, jota kutsutaan kelpoisuuskriteereiksi. Joitakin esimerkkejä näistä kriteereistä ovat henkilön yleinen terveydentila tai aiemmat hoidot.

Kelpoisuusvaatimukset

Opintokelpoiset iät

2 vuotta - 18 vuotta (Lapsi, Aikuinen)

Hyväksyy terveitä vapaaehtoisia

Ei

Sukupuolet, jotka voivat opiskella

Kaikki

Kuvaus

Inclusion Criteria:

  • For the Prospective New-User Cohort, verification of Inclusion Criteria 1 through 6 is required.

  • For the Retrospective (Switch) Cohort, verification of Inclusion Criteria 1 through 7 is required.

    1. Pediatric patients aged 2 years and < 18 years.

    2. Diagnosed with non-infectious anterior uveitis, intermediate uveitis, posterior uveitis, or panuveitis.

    3. Active intraocular inflammation demonstrated by anterior chamber (AC) cell grade ≥ 1+ (defined as 6 cells per 1 mm\times 1 mm field) according to the SUN (Standardization of Uveitis Nomenclature) criteria, or active posterior segment lesions persistently observed on at least two consecutive assessments despite 12 weeks of treatment with methotrexate and corticosteroids (systemic or topical) prior to screening (SUN grade maintained on 2 occasions, adapted from the SYCAMORE trial definition of active disease).

    4. Inadequate response to corticosteroid therapy or other immunosuppressive agents (e.g., methotrexate [MTX]), or inability to continue treatment due to unacceptable toxicity/intolerance.

    5. Written informed consent obtained from the subject's legal representative (parent or legal guardian) and written pediatric assent obtained according to applicable age-specific regulatory and institutional guidelines.

      [Prospective De Novo Cohort]

    6. Patients initiating adalimumab for the first time (adalimumab-naïve).

[Retrospective / Prospective Cohort] 6. Patients currently on maintenance therapy with adalimumab (including extended dosing intervals, such as every other week [Q2W] or every 3-4 weeks) who require at least 1 additional year of treatment.

7. Patients who agree to switch to the investigational product and adhere to a standard every-other-week (Q2W / once every 2 weeks) administration schedule at the time of study enrollment.

Exclusion Criteria:

  • For the Prospective New-User Cohort, verification of Exclusion Criteria 1 through 22 is required.

  • For the Retrospective (Switch) Cohort, verification of Exclusion Criteria 1 through 24 is required.

    1. Patients with uveitis associated with juvenile idiopathic arthritis (JIA) or pediatric Crohn's disease indicated for adalimumab

    2. Individuals with infectious uveitis or non-immunological intraocular inflammation resulting from trauma, surgery, etc., within 30 days prior to screening

    3. Individuals with active tuberculosis, untreated latent tuberculosis, sepsis, opportunistic infections, or other severe active infections, or those at high risk for such infections

    4. Individuals with a history of hypersensitivity to adalimumab or any of its excipients

    5. Individuals instilling topical glucocorticoids more than 6 times per eye per day, or currently receiving oral prednisone (or its equivalent) at a dose exceeding 0.2 mg/kg/day (adapted from the SYCAMORE criteria)

    6. Individuals with active malignancy, lymphoproliferative disorders, demyelinating disease (e.g., multiple sclerosis, Guillain-Barré syndrome), or a history of other progressive or degenerative neurological disorders

    7. Patients who have received TNF inhibitors other than the investigational product

    8. Patients with New York Heart Association (NYHA) Class III/IV cardiac disease

    9. Individuals with uncontrolled viral infections, including active hepatitis B or hepatitis C

    10. Individuals who have received a live vaccine (including live-attenuated vaccines) within 30 days prior to screening, or who are scheduled to receive one during the study period

    11. Individuals who are pregnant or breastfeeding, or women of childbearing potential (WOCBP) who do not agree to use highly effective contraception*

      *A highly effective method of contraception is defined as one that results in a low failure rate of less than 1% per year when used consistently and correctly, and includes: combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal); progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable); intrauterine device (IUD); intrauterine hormone-releasing system (IUS); bilateral tubal occlusion; vasectomized partner; and sexual abstinence.

    12. Individuals who have received other biologic agents within 5 half-lives prior to screening

    13. Individuals with a family member who has active tuberculosis, or who have a history of close contact with an individual with active tuberculosis

    14. Individuals with a major infection requiring antibiotic therapy within 30 days prior to screening

    15. Individuals with diabetes mellitus deemed uncontrolled by the investigator, or persistently uncontrolled severe hypertension (> 95th percentile for height and age)

    16. Individuals with a history of malignancy, or a history of solid organ, bone marrow, or corneal transplantation

    17. Individuals with a history of ocular surgery (e.g., cataract surgery) within 3 months prior to screening

    18. Individuals who have received intraocular or periocular corticosteroid injections within 30 days prior to screening

    19. Individuals with a history of ocular herpes simplex or herpes zoster infection

    20. Individuals with laboratory values evaluated during screening that meet any of the following criteria:

      A. Hemoglobin < 8.0 g/dL B. WBC < 4.0 × 10³/µL C. ANC < 1.0 × 10³/µL D. Platelet count < 100 × 10³/µL E. AST or ALT > 2 × ULN

    21. Individuals weighing less than 15 kg

    22. Individuals otherwise deemed unsuitable for study participation in the judgment of the investigator

      [Retrospective / Prospective Cohort-Specific Criteria]

    23. Inability to confirm disease activity (e.g., SUN grading) at the initial start of adalimumab treatment and subsequent treatment response via medical record review.

    24. Patients whose adalimumab dosage has been tapered to near-discontinuation (i.e., dosing intervals substantially exceeding once every 4 weeks) or for whom the treating physician deems the standard maintenance regimen (once every 2 weeks) clinically inappropriate.

Opintosuunnitelma

Tässä osiossa on tietoja tutkimussuunnitelmasta, mukaan lukien kuinka tutkimus on suunniteltu ja mitä tutkimuksella mitataan.

Miten tutkimus on suunniteltu?

Suunnittelun yksityiskohdat

  • Ensisijainen käyttötarkoitus: Hoito
  • Jako: Ei satunnaistettu
  • Inventiomalli: Yksittäinen ryhmätehtävä
  • Naamiointi: Ei mitään (avoin tarra)

Aseet ja interventiot

Osallistujaryhmä / Arm
Interventio / Hoito
Kokeellinen: Prospective Treatment-Naïve Cohort and

Patients initiating adalimumab for the first time receive the investigational product (Yuflyma Pen Inj., subcutaneous adalimumab biosimilar) prospectively for 12 months (approximately 52 weeks) following enrollment.

Yuflyma Pen Inj. (recombinant adalimumab biosimilar, Celltrion) is administered via subcutaneous (SC) injection with weight-tiered dosing (<30 kg: 20 mg; ≥30 kg: 40 mg).

  • Prospective Treatment-Naïve Cohort: Patients initiating adalimumab receive weight-tiered doses every other week (eow) for 52 weeks.
  • Retrospective (Switch) Cohort: Patients previously maintained on adalimumab prior to study entry switch to Yuflyma at enrollment and receive weight-tiered maintenance doses once every two weeks (q2w) for at least 12 months.
Kokeellinen: Retrospective (Switch) Cohort

Patients previously maintained on adalimumab prior to study entry and requiring continued treatment for at least one additional year switch to the investigational product (Yuflyma Pen Inj., subcutaneous adalimumab biosimilar) at enrollment.

Yuflyma Pen Inj. (recombinant adalimumab biosimilar, Celltrion) is administered via subcutaneous (SC) injection with weight-tiered dosing (<30 kg: 20 mg; ≥30 kg: 40 mg).

  • Prospective Treatment-Naïve Cohort: Patients initiating adalimumab receive weight-tiered doses every other week (eow) for 52 weeks.
  • Retrospective (Switch) Cohort: Patients previously maintained on adalimumab prior to study entry switch to Yuflyma at enrollment and receive weight-tiered maintenance doses once every two weeks (q2w) for at least 12 months.

Mitä tutkimuksessa mitataan?

Ensisijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Treatment Failure Rate in the Prospective Treatment-Naïve Cohort
Aikaikkuna: Up to 12 months after adalimumab initiation

Percentage of participants meeting the criteria for treatment failure. Treatment failure is defined as any of the following: active intraocular inflammation (worsening anterior chamber cell grade, vitreous haze, or new chorioretinal lesions), lack of clinical efficacy requiring addition of new systemic immunosuppressive agents, inability to taper concomitant systemic corticosteroids to target maintenance levels, or discontinuation of the investigational product due to lack of efficacy or unacceptable toxicity.

Up to 12 months after adalimumab initiation
Change from Baseline in Medically Important Infections (MIIS) in the Prospective Treatment-Naïve Cohort
Aikaikkuna: Baseline and 12 months after adalimumab initiation

Total count and incidence rate of Medically Important Infections (defined as opportunistic infections, infections requiring intravenous antimicrobial therapy, or infections resulting in hospitalization or death) observed per participant. The change is calculated as the incidence during the 12-month post-initiation period compared to the pre-baseline reference status.

Baseline and 12 months after adalimumab initiation

Toissijaiset tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Treatment Failure Rate in the Retrospective (Switch) Cohort
Aikaikkuna: Up to 12 months post-switch to the investigational product

Percentage of participants who experience treatment failure following switch to the investigational product. Treatment failure is defined as active intraocular inflammation, necessity to escalate therapy or introduce unpermitted rescue therapies, or drug discontinuation due to lack of efficacy or adverse events.

Up to 12 months post-switch to the investigational product
Relapse-Free Survival Rate in the Retrospective (Switch) Cohort
Aikaikkuna: At 12, 24, and 36 months post-adalimumab initiation

Percentage of participants maintaining clinical inactive disease without uveitis recurrence after achieving initial clinical response, estimated using Kaplan-Meier survival methodology at specified annual landmark intervals.

At 12, 24, and 36 months post-adalimumab initiation
Cumulative Incidence of Relapse and Management Strategies in the Retrospective (Switch) Cohort
Aikaikkuna: Up to 36 months post-adalimumab initiation

Cumulative incidence of ocular relapse and the distribution of clinical management strategies implemented upon relapse, categorized as dose escalation, administration interval shortening (e.g., from every other week to weekly), or permanent treatment discontinuation.

Up to 36 months post-adalimumab initiation
Drug Survival Rate of Adalimumab in the Retrospective (Switch) Cohort
Aikaikkuna: Up to 36 months post-adalimumab initiation

Percentage of participants remaining on adalimumab therapy over time without permanent treatment cessation, evaluated by Kaplan-Meier survival curves.

Up to 36 months post-adalimumab initiation
Time to Adalimumab Discontinuation in the Retrospective (Switch) Cohort
Aikaikkuna: Up to 36 months post-adalimumab initiation

Median time (in months) from adalimumab initiation to permanent discontinuation of therapy due to any reason, including loss of response, remission, adverse events, or patient withdrawal.

Up to 36 months post-adalimumab initiation
Steroid-Sparing Success Rate in the Retrospective (Switch) Cohort (Pooled Analysis)
Aikaikkuna: Up to 36 months post-adalimumab initiation

Percentage of participants achieving successful systemic steroid-sparing, defined as achieving disease inactivity with oral prednisone (or equivalent) tapered to $\le 0.1\text{ mg/kg/day}$ (or complete cessation) and topical glucocorticoids tapered to $\le 2\text{ drops/day/eye}$.

Up to 36 months post-adalimumab initiation
Change from Baseline in Systemic and Topical Corticosteroid Requirements in the Retrospective (Switch) Cohort
Aikaikkuna: Baseline (initial adalimumab start) and up to 36 months post-initiation

Quantitative reduction in daily systemic corticosteroid dose (mg/kg/day of prednisone equivalent) and change in daily frequency of topical ophthalmic corticosteroid instillations per affected eye.

Baseline (initial adalimumab start) and up to 36 months post-initiation
Cumulative Incidence of Cataract and Elevated Intraocular Pressure in the Retrospective (Switch) Cohort (Pooled Analysis)
Aikaikkuna: Up to 36 months post-adalimumab initiation

Percentage of participants who develop new-onset or worsening cataract (requiring surgical intervention or clinically significant progression) or elevated intraocular pressure ($\text{IOP} > 21\text{ mmHg}$ or an increase $\ge 10\text{ mmHg}$ from baseline requiring hypotensive medication).

Up to 36 months post-adalimumab initiation

Muut tulostoimenpiteet

Tulosmittaus
Toimenpiteen kuvaus
Aikaikkuna
Integrated Assessment of Adverse Events Across the Cumulative Exposure Period
Aikaikkuna: Up to 48 months (covering up to 36 months retrospective exposure plus 12 months prospective study period)

Number, incidence rates, and severity of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse drug reactions (ADRs) assessed per Common Terminology Criteria for Adverse Events (CTCAE) criteria across the combined follow-up duration.

Up to 48 months (covering up to 36 months retrospective exposure plus 12 months prospective study period)

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Opintoihin ilmoittautumispäivät

Ensimmäinen lähetetty

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Ensimmäinen toimitettu, joka täytti QC-kriteerit

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Ensimmäinen Lähetetty (Todellinen)

Tiistai 3. marraskuuta 2026

Tutkimustietojen päivitykset

Viimeisin päivitys julkaistu (Todellinen)

Tiistai 3. marraskuuta 2026

Viimeisin lähetetty päivitys, joka täytti QC-kriteerit

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Viimeksi vahvistettu

Torstai 1. lokakuuta 2026

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IPD-suunnitelman kuvaus

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