Clinical Trials Tracker

Active recruiting trials, recent completions, and published results — 2025–2026.

1 Recruiting 1 Active 2 Completed 3 In Development
Important: Trial information is current as of March 2026 and subject to change. Verify eligibility and details directly with trial sponsors before enrolling. This page does not constitute an endorsement or recommendation to participate.

Active Trials (Limited / Not Yet Enrolling)

Ongoing trials with potential future enrolment opportunities:

Active NCT04047732 FIRST Foundation

KB105: Topical Gene Therapy for TGM1-Related Lamellar Ichthyosis

Phase
Phase I/II
Condition
Lamellar Ichthyosis (TGM1 mutations)
Intervention
KB105 topical lipid replacement
Expected completion
2025
Mechanism of Action

Topical gene therapy using a replication-incompetent, non-integrating HSV-1 viral vector expressing human transglutaminase-1 (TGM1). Applied directly to skin, KB105 delivers a functional copy of the TGM1 gene to keratinocytes, restoring the enzyme missing in TGM1-deficient ARCI. Non-integrating design avoids insertional mutagenesis risk.

Eligibility Criteria
  • Lamellar ichthyosis (autosomal recessive congenital ichthyosis)
  • TGM1 mutations confirmed by genetic testing
  • Adults — moderate-to-severe disease severity
  • Capacity to apply topical therapy and complete assessments
Study Design & Expected Outcomes
Study size:Up to 6 adult subjects with lamellar ichthyosis (Phase I/II)
Duration:Approximately 3.5 months treatment period
Primary outcomes:Safety, tolerability, and efficacy via IGA and VIIS scoring
Design:Intra-patient comparisons; placebo-treated target areas; imaging documentation
Register / learn more:

ClinicalTrials.gov NCT04047732 · FIRST Foundation: firstskinfoundation.org (Research Studies section)

Active — Observational NCT06669949 UCSF

SPLIS Natural History Study — Sphingosine Phosphate Lyase Insufficiency Syndrome

Study type
Observational natural history
Condition
SPLIS (SGPL1 mutations) incl. ichthyosis
Intervention
None — observational only
Expected completion
2026
About this study

Rare multisystem disorder caused by SGPL1 gene mutations. SPLIS presents with ichthyosis, nephrosis, endocrine dysfunction, neuropathy, and immune abnormalities. This observational study collects natural history data to guide future therapeutic development.

Eligibility Criteria
  • Confirmed biallelic SGPL1 mutations (any variant classification)
  • At least one SPLIS feature: ichthyosis, nephrosis, endocrine defect, neuropathy, male gonadal dysgenesis, or lymphopenia
  • OR biochemical evidence of S1P lyase deficiency in skin fibroblasts
  • OR sibling of confirmed SGPL1 mutation carrier
Register / learn more:

UCSF Clinical Trials Database: clinicaltrials.ucsf.edu/trial/NCT06669949

Completed Trials with Published Results

Trials that have finished and reported outcomes:

Completed — Phase 3 Failed NCT05295732 Leo Pharma / Timber Pharmaceuticals

TMB-001 ASCEND: Topical Isotretinoin 0.05% for Congenital Ichthyosis

Phase
Phase 3 (Pivotal)
Condition
ARCI / X-Linked Ichthyosis (moderate-severe)
Intervention
TMB-001 topical isotretinoin 0.05%
Age
≥6 years
Phase 3 Trial Failed (August 2024)

The ASCEND Phase 3 trial missed both its primary and secondary endpoints. 209 participants were enrolled (≥6 years, moderate-to-severe congenital ichthyosis). Leo Pharma had acquired Timber Pharmaceuticals for $36M ahead of the results. TMB-001 will not proceed to regulatory submission in its current form. The Phase 2b results (CONTROL study, JAAD 2023) remain scientifically relevant context but did not predict Phase 3 success.

Phase 2b Key Results (PMID 36794376)
VIIS-50 response: 100% TMB-001 vs 40% vehicle (P=0.04)
IGA ≥2 improvement: 100% vs 10%
Safety: No clinically significant lab changes; systemic exposure <1% of 80mg oral dose
Learn more:

ClinicalTrials.gov NCT05295732 · Phase 2b paper (PMID 36794376)

Completed NCT01222000 French Dermatology Research Group

Polyphénon E 10% (Green Tea Extract) for Lamellar Ichthyosis

Study type
Pilot clinical trial
Condition
Lamellar Ichthyosis (ARCI)
Intervention
Topical Polyphénon E 10% (green tea extract)
Completed
2012 — results published
Key Findings
Primary outcome:Improvement in scaling and cutaneous roughness after 4 weeks of daily topical Polyphénon E 10%
Safety:Well tolerated; minor adverse effects only
Mechanism:Polyphenols (green tea) act as antioxidants and may modulate inflammatory cascades in ichthyotic skin
Clinical implication:Topical green tea extract may offer adjunctive benefit; requires larger RCT confirmation

Actively Recruiting

Recruiting NCT06953466 Quoin Pharmaceuticals

QRX003 Lotion (4%) for Netherton Syndrome

Phase
Phase 2/3 → Phase 3 Pivotal
Condition
Netherton Syndrome (SPINK5)
Intervention
QRX003 lotion 4% — topical, twice daily
Status
Phase 3 recruitment 2026; NDA target 2027
Key Developments (March 2026)
FDA Fast Track:Granted March 2026 — facilitates accelerated review
Single Phase 3:FDA indicated a single pivotal study may be sufficient for approval (Type C meeting, 25 March 2026)
Trial design:FDA open to randomised withdrawal or delayed start design — no traditional upfront placebo control required
Designations:Orphan Drug (FDA + EMA), Pediatric Rare Disease, Fast Track
Significance:If approved, QRX003 would be the first treatment specifically approved for Netherton Syndrome
Learn more:

ClinicalTrials.gov NCT06953466 · Quoin Pharmaceuticals

Trials in Development — Expected 2026–2027

Upcoming — IND Submitted

ATR-01 — Azitra Inc. (Filaggrin / Ichthyosis Vulgaris)

First-in-class living therapeutic: engineered S. epidermidis expressing recombinant filaggrin, applied topically. IND submission planned 2026; first-in-human Phase 1/2 trial expected to follow. Targets confirmed FLG mutations in ichthyosis vulgaris.

Watch: ClinicalTrials.gov — search "Azitra" or "ATR-01".

Preclinical — Human Data Published

LNP Base-Editor — TGM1 ARCI (Lamellar Ichthyosis)

Topical LNP delivering mRNA base-editor corrects TGM1 mutations in human skin models, restoring transglutaminase-1 activity (Cell Stem Cell, Jan 2026 — PMID 41605220). Non-viral approach. IND filing projected 2–3 years. First in situ gene editing for ARCI demonstrated in human tissue.

Watch: ClinicalTrials.gov and FIRST Foundation for IND announcements.

Pre-clinical / Fundraising

Epithelica — ARCI Ex Vivo Gene Therapy

Spin-off from the successful EB ex vivo gene therapy programme (2022 NEJM). Adapting the correction platform for ABCA12 (Harlequin) and TGM1 (Lamellar) — the world's first dedicated ARCI gene therapy company. Pre-clinical and fundraising stage as of early 2026.

Watch: Epithelica company announcements for IND filing.

Planning Stage

Biologic Therapy Trials (Risankizumab / Anti-IL-23)

Preclinical and early clinical data promising in ichthyosis vulgaris. Formal RCTs in planning stages for inflammatory ichthyosis subtypes.

Watch: Major dermatology institutions and immunology-focused research group announcements.

How to Find & Enrol in Ichthyosis Trials

1
Check ClinicalTrials.gov

Visit clinicaltrials.gov and search "ichthyosis". Filter by: Recruiting, Active, Not Yet Recruiting. Review eligibility, contacts, and study details for each result.

2
Contact FIRST Foundation

The Foundation for Ichthyosis & Related Skin Types maintains a dedicated research registry and coordinates ichthyosis-specific trials. Visit firstskinfoundation.org → Research Studies section.

3
Consult Your Dermatologist

Discuss trial options and suitability for your type and severity. Ask about genetic testing (often required for enrolment). Request a referral to a trial site if your specialist is involved.

4
Understand Informed Consent

Carefully read the Informed Consent Form. Understand risks, benefits, and your rights as a participant. Ask all your questions before signing. You can withdraw at any time without penalty.

Essential Research Resources

Trial Registries

ClinicalTrials.gov

US registry of all clinical trials. Search "ichthyosis" for current list.

FIRST Foundation (USA)

FIRST Foundation

Ichthyosis-specific research coordination and patient support.

UK Support

ISG UK

UK patient organisation with research updates and trial signposting.

Frequently Asked Questions

Many trials require a confirmed genetic diagnosis (FLG mutations, TGM1 mutations, etc.). If you haven't had genetic testing, discuss with your dermatologist. NHS genetic testing turnaround is typically 3–8 weeks depending on the test.
Trial designs vary significantly. Some allow continued standard care; others require washout periods before the study drug. Always check the eligibility criteria and full protocol, and discuss with the trial investigators before applying.
You can withdraw at any time and return to your standard care. Trials are designed to benefit participants, but individual responses vary. Withdrawing does not affect your right to continued NHS care.
Most clinical trials cover study-related costs and in some cases provide compensation for participation. Always confirm the payment and expenses policy with trial investigators before enrolling.
Completed trials publish results in peer-reviewed journals (PubMed, ClinicalTrials.gov results section). Trial sponsors notify participants. FIRST Foundation distributes major findings to the ichthyosis community via their newsletter and website.

Keep up with ichthyosis research

Read the full research database — gene discoveries, treatment evidence, and the latest science.

→ Research Database