8 completed, 2 recruiting, 1 not yet recruiting, 1 unknown. 6 Phase 1, 4 Phase 3, 2 Phase 2, 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 5, Not yet recruiting 1 |
| Phase 3 | 4 | Completed 3, Recruiting 1 |
| Phase 2 | 2 | Completed 1, Not yet recruiting 1 |
| Not applicable | 1 | Unknown 1 |
Follow Pachyonychia Congenita: new trials by email Literature Review of Pachyonychia Congenita trials Research Report on Pachyonychia Congenita
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 |
| Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC KM-001 cream 1% 12 weeks treatment., Physical Examination, Vital Signs - Punctate Palmoplantar Keratoderma Type 1, Pachyonychia Congenita | Phase 1 | Completed | Orvida Pharma (formerly Kamari Pharma) | 18 | 2023-03-06 | 2025-01-15 |
| A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita PTX-022 - Pachyonychia Congenita | Phase 3 | Recruiting | Palvella Therapeutics, Inc. | 45 | 2022-11-15 | 2022-12-09 |
| Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punctate Palmoplantar Keratoderma or Pachyonychia Congenita Diseases Serum chemistry, Hematology, Urinalysis - Punctate Palmoplantar Keratoderma Type 1, Pachyonychia Congenita | Phase 1 | Completed | Orvida Pharma (formerly Kamari Pharma) | 14 | 2022-07-17 | 2025-01-07 |
| A Multicenter, Phase 3 Randomized, Double-Blind, Vehicle-Controlled Study Evaluating the Safety and Efficacy of QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Pachyonychia Congenita QTORIN 3.9% rapamycin anhydrous gel, Vehicle - Pachyonychia Congenita | Phase 3 | Completed | Palvella Therapeutics, Inc. | 87 | 2021-11-29 | 2024-08-29 |
| VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita PTX-022 - Pachyonychia Congenita | Phase 3 | Completed | Palvella Therapeutics, Inc. | 36 | 2020-09-01 | 2024-08-29 |
| Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita PTX-022, Placebo - Pachyonychia Congenita | Phase 2 / Phase 3 | Completed | Palvella Therapeutics, Inc. | 73 | 2019-04-01 | 2024-08-29 |
| Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin Jojoba oil with broccoli sprout extract, Jojoba oil (placebo) - Epidermolysis Bullosa Simplex, Pachyonychia Congenita | Phase 1 | Completed | Johns Hopkins University | 5 | 2015-09-01 | 2017-12-06 |
| Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC) 1% sirolimus cream (TD201 1%) - Pachyonychia Congenita | Phase 1 | Completed | TransDerm, Inc. | 15 | 2014-01-01 | 2016-10-07 |
| Simvastatin Treatment of Pachyonychia Congenita Simvastatine - Pachyonychia Congenita | Not applicable | Unknown | Tel-Aviv Sourasky Medical Center | 10 | 2011-07-01 | 2011-06-27 |
| Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita TD101, Normal saline (placebo) - Pachyonychia Congenita | Phase 1 | Completed | Pachyonychia Congenita Project | 1 | 2008-01-01 | 2008-11-19 |
| International Pachyonychia Congenita Research Registry Pachyonychia Congenita | Recruiting | Pachyonychia Congenita Project | 2000 | 2004-04-01 | 2018-11-21 |
Keratosis palmoplantaris papulosa (2) · Cysts (1) · Epidermolysis Bullosa Simplex (1) · Hyperkeratosis, Epidermolytic (1) · Keratoderma, Palmoplantar (1) · Leukoplakia (1) · Pain (1)
Abnormalities, Multiple (12) · Congenital Abnormalities (12) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (12) · Ectodermal Dysplasia (12) · Genetic Diseases, Inborn (12) · Nail Diseases (12) · Skin Abnormalities (12) · Skin Diseases (12) · Skin Diseases, Genetic (12) · Skin and Connective Tissue Diseases (12) · Epidermolysis Bullosa (1) · Ichthyosiform Erythroderma, Congenital (1) · Ichthyosis (1) · Infant, Newborn, Diseases (1) · Keratosis (1)
Hematologic Tests (2) · Pain Measurement (2) · Physical Examination (2) · Therapeutics (2) · Urinalysis (2) · Vital Signs (2) · Erlotinib Hydrochloride (1) · KPNA1 protein, human (1) · Saline Solution (1) · Simvastatin (1) · jojoba wax (1)
Palvella Therapeutics, Inc. (4) · Orvida Pharma (formerly Kamari Pharma) (2) · Pachyonychia Congenita Project (2) · Johns Hopkins University (1) · Northwestern University (1) · Tel-Aviv Sourasky Medical Center (1) · TransDerm, Inc. (1)
United States (9) · United Kingdom (2) · Israel (1)
David Hansen, MD (2) · Joyce Teng, MD (2) · Amy Paller (1) · Amy Paller, MD (1) · Bernard Cohen, MD (1) · C. David Hansen, MD (1) · Roger L Kaspar, PhD (1) · Sancy A Leachman, MD, PhD (1)