8 completed, 2 terminated, 1 active not recruiting, 1 not yet recruiting. 13 Phase 1, 6 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 13 | Completed 8, Terminated 2, Active not recruiting 1, Not yet recruiting 1, Unknown 1 |
| Phase 2 | 6 | Completed 4, Not yet recruiting 1, Terminated 1 |
Follow Ichthyosis: new trials by email Literature Review of Ichthyosis trials Research Report on Ichthyosis
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Epidermal Growth Factor Receptor Inhibition for Keratinopathies Erlotinib - Epidermolytic Ichthyosis, Palmoplantar Keratoderma | Phase 1 / Phase 2 | Not yet recruiting | Northwestern University | 44 | 2027-07-01 | 2026-08-03 |
| A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725 BCX17725, Placebo - Netherton Syndrome | Phase 1 | Active not recruiting | BioCryst Pharmaceuticals | 76 | 2024-09-26 | 2026-11-06 |
| Evaluation of Topical ATR12-351 in Adults With Netherton Syndrome ATR12-351 - Netherton Syndrome | Phase 1 | Completed | Azitra Inc. | 6 | 2024-06-16 | 2026-07-29 |
| Neoadjuvant ADI-PEG 20 + Ifosfamide + Radiotherapy in Soft Tissue Sarcoma ADI PEG20, Ifosfamide, Radiotherapy - Soft Tissue Sarcoma, Sts | Phase 1 | Terminated | Washington University School of Medicine | 6 | 2024-03-14 | 2026-09-03 |
| A Study to Explore Safety, Pharmacokinetics, and Early Clinical Signal of Efficacy of DS-2325a in Patients With Netherton Syndrome DS-2325a, Placebo - Netherton Syndrome | Phase 1 / Phase 2 | Terminated | Daiichi Sankyo | 9 | 2023-09-28 | 2026-05-14 |
| ADX-629 Therapy for Sjogren-Larsson Syndrome ADX-629 - Sjogren-Larsson Syndrome | Phase 1 / Phase 2 | Completed | University of Nebraska | 8 | 2023-01-02 | 2026-05-05 |
| A Study to Assess the Safety and Pharmacokinetics of Multiple Ascending Subcutaneous Doses of DS-2325a in Healthy Subjects DS-2325a, Placebo - Netherton Syndrome | Phase 1 | Completed | Daiichi Sankyo | 24 | 2022-11-08 | 2023-05-18 |
| A Study to Assess the Safety and Pharmacokinetics of Single Ascending Subcutaneous and Intravenous Doses of DS-2325a in Healthy Subjects DS-2325a, Placebo - Netherton Syndrome | Phase 1 | Completed | Daiichi Sankyo | 64 | 2022-06-20 | 2023-03-06 |
| A Study to Evaluate Topically Applied SXR1096 Cream in Patients With Netherton Syndrome SXR1096 cream, Placebo cream - Netherton Syndrome | Phase 1 / Phase 2 | Completed | Sixera Pharma | 9 | 2021-12-07 | 2025-12-16 |
| Gene Therapy for Netherton Syndrome One 20cm2/10cm2 autologous skin sheet graft - Netherton Syndrome | Phase 1 | Unknown | Great Ormond Street Hospital for Children NHS Foundation Trust | 5 | 2014-04-01 | 2016-10-19 |
| A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton Syndrome BPR277 ointment (controlled application), Placebo (Vehicle), BPR277 ointment - Healthy Volunteers, Atopic Dermatitis | Phase 1 | Completed | Novartis Pharmaceuticals | 12 | 2011-05-01 | 2020-12-08 |
| Effects of ROSE-010 on GI Transit in Constipation Predominant Irritable Bowel Syndrome (C-IBS) Patients ROSE-010, Placebo - Irritable Bowel Syndrome Constipation Predominant | Phase 1 / Phase 2 | Completed | Mayo Clinic | 52 | 2010-01-01 | 2013-05-31 |
| Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton Syndrome Pimecrolimus 1% Cream - Netherton Syndrome | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 3 | 2005-09-01 | 2019-08-14 |
Netherton Syndrome (9) · Cerebral Palsy (1) · Constipation (1) · Dermatitis, Atopic (1) · Eczema (1) · Hyperkeratosis, Epidermolytic (1) · Ichthyosis Bullosa of Siemens (1) · Ichthyosis, X-Linked (1) · Intellectual Disability (1) · Irritable Bowel Syndrome (1) · Keratoderma, Palmoplantar (1) · Muscle Spasticity (1) · Pachyonychia Congenita (1) · Retinal Diseases (1)
Congenital Abnormalities (13) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (13) · Genetic Diseases, Inborn (13) · Infant, Newborn, Diseases (13) · Keratosis (13) · Skin Abnormalities (13) · Skin and Connective Tissue Diseases (13) · Skin Diseases (12) · Skin Diseases, Genetic (12) · Abnormalities, Multiple (10) · Ichthyosiform Erythroderma, Congenital (10) · Metabolic Diseases (2) · Metabolism, Inborn Errors (2) · Nutritional and Metabolic Diseases (2)
ADI PEG20 (1) · ADX-629 (1) · Erlotinib Hydrochloride (1) · Glucagon-Like Peptide 1 (1) · Ifosfamide (1) · Mesna (1) · Radiotherapy (1) · WAS protein, human (1) · glucagon-like peptide (7-37), valyl(10)- (1) · pimecrolimus (1)
Daiichi Sankyo (3) · Azitra Inc. (1) · BioCryst Pharmaceuticals (1) · Children's Hospital of Philadelphia (1) · Great Ormond Street Hospital for Children NHS Foundation Trust (1) · Mayo Clinic (1) · Northwestern University (1) · Novartis Pharmaceuticals (1) · Sixera Pharma (1) · University of Nebraska (1) · Washington University School of Medicine (1)
United States (10) · France (3) · Germany (2) · Netherlands (2) · Australia (1) · Austria (1) · Sweden (1) · United Kingdom (1)
Albert C Yan, MD (1) · Amy Paller (1) · Jemima Mellerio, Dr (1) · Maarten de Chateau, MD PhD (1) · Mary Spellman, MD (1) · Mia Weiss, M.D. (1) · Michael Camilleri, MD (1) · William B Rizzo, MD (1)
PMID 20083693, PMID 22517769, PMID 24329107
2025 (1), UL1RR024150 (1)