Clinical trial news in pediatrics: October 2026

October's pediatric clinical trial news came largely out of fall congress season — EADV, MDS, United European Gastroenterology Week, and ASN Kidney Week all generated late-breaking pediatric or adolescent data — spanning vitiligo, psoriasis, Tourette syndrome, ulcerative colitis, eosinophilic esophagitis, a rare bone disease, and sepsis-associated kidney injury. Most of the month's readouts extend follow-up on drugs that are already approved (or nearly so) for older patients or adults, and several rely on open-label extensions, post hoc analyses, or cross-study comparisons rather than placebo-controlled pediatric data.

This recap rounds up the month's study and trial results and pipeline data readouts in pediatrics — as distinct from FDA regulatory decisions, which are covered separately — as reported in Contemporary Pediatrics' drug-pipeline-news coverage, so clinicians can scan what's emerging without combing through individual stories.

Clinical trial news in pediatrics: October 2026

Upadacitinib sustains repigmentation in non-segmental vitiligo through 76 weeks

On October 1, 2026, Contemporary Pediatrics reported late-breaking phase 3 extension data presented at the EADV Congress showing that facial and body repigmentation in non-segmental vitiligo continued increasing through week 76 in patients taking upadacitinib (RINVOQ), a once-daily oral JAK inhibitor approved in Europe in July 2026 for patients 12 and older who are candidates for systemic therapy, with a US indication still under review. In the Viti-Up-1 and Viti-Up-2 extensions, observed-case facial F-VASI 75 response rose from 33.1% to 55.1% and from 27.6% to 47.4%, respectively, between weeks 48 and 76; at week 48, upadacitinib separated from placebo on T-VASI 50 (19% vs 6%, and 21% vs 6%) but not on the stricter T-VASI 75 threshold. The article cautions that observed-case analyses can overstate response relative to intention-to-treat estimates, that more than half of patients had not reached T-VASI 50 by week 76, and that results were not broken out separately for adolescents.

Icotrokinra maintains high-impact site psoriasis clearance through 2 years in ICONIC-TOTAL

On October 2, 2026, the outlet covered 2-year data from the phase 3 ICONIC-TOTAL trial, presented at EADV 2026, showing that oral icotrokinra (Icotyde, Johnson & Johnson) sustained clearance of psoriasis at high-impact sites — scalp, genital, and hand/foot involvement — through week 112 in patients 12 and older, the population covered by its approved moderate-to-severe plaque psoriasis indication. Among 311 trial participants, overall IGA 0/1 response rose from 57% at week 16 to 70% at week 112, with site-specific clearance reaching 60% (scalp), 89% (genital), and 63% (hand/foot) by week 112. The article notes that only 6 pediatric patients were enrolled, limiting conclusions specific to adolescents, that 18% of patients did not complete treatment with no description of how they were handled in the analysis, and that the company reported no new safety signals without releasing adverse event rates.

Ecopipam data at MDS 2026 show early and sustained tic reduction in pediatric Tourette syndrome

Also on October 2, 2026, Contemporary Pediatrics reported new post hoc and interim analyses of investigational ecopipam, a dopamine D1 receptor antagonist under FDA priority review for Tourette syndrome, presented at the MDS Congress in Seoul. A pooled post hoc analysis of 292 patients found 69.8% reached at least a 25% improvement in YGTSS Total Tic Score within 8 weeks, an 18-month open-label extension (n=118) showed a mean 45.6% YGTSS-TTS reduction, and a phase 3 randomized-withdrawal analysis found ecopipam reduced relapse risk versus placebo (HR 0.47; 95% CI, 0.26-0.84; P = .008). The article emphasizes that most findings are post hoc or interim, that the extension is open-label without a placebo arm, that somnolence and headache clustered in the first 8 weeks, and that the FDA decision on ecopipam remains pending.

Mirikizumab meets 1-year remission end point in pediatric UC

On October 19, 2026, the outlet reported phase 3 SHINE-2 data on mirikizumab (Omvoh, Eli Lilly), presented at United European Gastroenterology Week, in children and adolescents aged 6 to 17 with moderately to severely active ulcerative colitis (the trial enrolled ages 4-17); the study met its primary endpoint, and Lilly plans to submit the data to support a pediatric label expansion. Fifty percent of induction responders were in clinical remission at week 52, and 81% had achieved clinical response by week 12, with the pediatric remission estimate's lower bound (42.8%) exceeding the upper bound of an adult placebo benchmark drawn from the LUCENT-2 trial rather than a pediatric placebo arm. The article notes this was a single-arm, open-label study with no randomized pediatric control, that the 63-patient enrollment is too small to detect uncommon adverse events, and that mirikizumab's pediatric safety and effectiveness have not yet been established.

Tezepelumab sustains EoE remission through 52 weeks

On October 20, 2026, Contemporary Pediatrics covered full 52-week results from the phase 3 CROSSING trial of tezepelumab (Tezspire, AstraZeneca/Amgen) in adolescents and adults (ages 12-80) with eosinophilic esophagitis uncontrolled on background therapy, presented as a late-breaking abstract at United European Gastroenterology Week in Barcelona; tezepelumab is currently FDA-approved only for severe asthma and chronic rhinosinusitis with nasal polyps in patients 12 and up, and eosinophilic esophagitis is not an approved indication. Histologic remission (≤6 eosinophils per high-power field) at week 52 reached 67.8% (210-mg dose) and 63.3% (420-mg dose) versus 5.4% with placebo, while total endoscopic remission reached 34.4% and 29.5% versus 4.1%. The article notes symptom improvements lagged behind histologic gains — the 210-mg dose's dysphagia-score benefit only narrowly reached significance — that patients could remain on stable background therapy during the trial, and that safety event rates were not disclosed.

Zilurgisertib reduces HO volume in children aged 6 to 11 with FOP

On October 26, 2026, the outlet reported topline results from Cohort 2 of the phase 2 PROGRESS study of zilurgisertib (Atebrioz, Mirum Pharmaceuticals), an oral ALK2 inhibitor already FDA-approved for fibrodysplasia ossificans progressiva in patients 12 and older, now tested in 25 children aged 6 to 11; Mirum plans a supplemental NDA for the younger age group in the first half of 2027. Over 24 weeks, total heterotopic ossification volume changed by −8.09 cm³ with zilurgisertib versus +9.14 cm³ with placebo (nominal P = .003), and no new heterotopic lesions developed in the treated group versus one in the placebo group. The article flags the small sample (12 treated, 13 placebo), wide between-patient variability, and that the P value is nominal and the data are topline and company-announced; safety showed no new signals relative to the drug's already-approved older-age cohort.

SCD-PED linked to higher survival in pediatric sepsis-associated AKI

Also on October 26, 2026, Contemporary Pediatrics covered an exploratory registry analysis, presented as a late-breaking poster at ASN Kidney Week 2026, finding that children with sepsis-associated acute kidney injury who received the selective cytopheretic device for pediatrics (SCD-PED, marketed as QUELIMMUNE and FDA-cleared in February 2024 under a Humanitarian Device Exemption) alongside continuous renal replacement therapy had higher 90-day survival than a matched historical cohort — 65.1% versus 47.4% (OR 2.07; 95% CI, 1.04-4.11; P = .043) in the primary analysis of 43 treated patients versus 196 historical controls. Among 24 survivors not dependent on renal replacement therapy at baseline, 83% were RRT-independent by day 90. The article cautions that the analysis used external historical controls rather than a randomized comparison, that a landmark sensitivity analysis did not reach statistical significance (P = .074), that the device's labeling states effectiveness for this use has not been demonstrated, and that the commenting expert is a compensated advisor to the manufacturer.