Clinical trials: Pachyonychia Congenita
Epidermolysis Bullosa Simplex
Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita
This study evaluates the clinical effect of foot injection of the bacteria protein Botulinum toxin A on plantar pain in patients with EBS (epidermolysis bullosa simplex) or PC (pachyonychia congenita).
- Trial name
- NCT00936533
- Testing
- Dysport® (Botulinumtoxin A (Btx A))
- Where
- Sweden
- Volunteers
- 40 people
- Sponsor
- Uppsala University
Darier Disease, Hailey-Hailey Disease
National Registry for Ichthyosis and Related Disorders
The ichthyoses are a family of genetic skin diseases characterized by dry, thickened, scaling skin. Dermatologists estimate that there are over twenty varieties of ichthyosis, with a wide range of severity and associated…
- Conditions
- Darier Disease, Hailey-Hailey Disease, Hyperkeratosis, Epidermolytic, Ichthyosis
- Trial name
- NCT00074685
- Where
- United States
- Volunteers
- 610 people
- Sponsor
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Pachyonychia Congenita
Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita
This study evaluates the safety and efficacy of PTX-022, topical rapamycin, in the treatment of adults with moderate to severe Pachyonychia Congenita.
- Trial name
- NCT03920228
- Testing
- PTX-022
- Where
- 9 sites in United States
- Volunteers
- 73 people
- Sponsor
- Palvella Therapeutics, Inc.
Pachyonychia Congenita
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
This study evaluates the safety and efficacy of QTORIN 3.9% rapamycin anhydrous gel in the treatment of adults with Pachyonychia Congenita. This study includes a screening period, baseline period and 6-month treatment period.
- Trial name
- VAPAUS (NCT05180708)
- Testing
- QTORIN 3.9% rapamycin anhydrous gel, Vehicle
- Where
- 8 sites in United States, United Kingdom
- Volunteers
- 87 people
- Sponsor
- Palvella Therapeutics, Inc.
Pachyonychia Congenita
Safety, Tolerability, and Efficacy of Oral KM-023 in Adult Patients With Pachyonychia Congenita (pEDD-PC)
The goal of this clinical trial is to learn if KM-023 is safe and well tolerated in adults with Pachyonychia Congenita (PC). The study will also explore whether KM-023 may help reduce pain, improve mobility, and lessen the impact…
- Trial name
- NCT07893483
- Testing
- KM-023
- Where
- 2 sites in Israel
- Volunteers
- 10 people
- Sponsor
- Orvida Pharma (formerly Kamari Pharma)
Epidermolysis Bullosa Simplex, Pachyonychia Congenita
Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin
Adult participants will apply a broccoli sprout extract-jojoba oil compound to one arm every night under occlusion for 1 week. Jojoba oil alone will be applied to the other arm.
- Trial name
- NCT02592954
- Testing
- Jojoba oil with broccoli sprout extract
- Where
- United States
- Volunteers
- 5 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Pachyonychia Congenita
Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)
A study to evaluate safety and efficacy of topical sirolimus to treat plantar keratoderma in adults with PC. Subjects may receive either placebo or treatment with at least 1 foot receiving topical sirolimus at some time.
- Trial name
- NCT02152007
- Testing
- 1% sirolimus cream (TD201 1%)
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- TransDerm, Inc.
Punctate Palmoplantar Keratoderma Type 1, Pachyonychia Congenita
Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC
This Phase 1b, open-label, single-center, prospective trial will be assessing the safety, tolerability, and efficacy of topical KM-001 1% in patients with PPPK1 or PC diseases. In this study 2 cohorts will be recruited: 1.
- Trial name
- NCT05956314
- Testing
- KM-001 cream 1% 12 weeks treatment., Physical Examination, Vital Signs
- Where
- United Kingdom
- Volunteers
- 18 people
- Sponsor
- Orvida Pharma (formerly Kamari Pharma)
Pachyonychia Congenita
VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita
VALO-2 is a multicenter, open-label extension (OLE) study enrolling patients with genotyped keratin mutations KRT6A, KRT6B and KRT16 who were previously treated with investigational PTX-022 during the VALO study.
- Trial name
- VALO-2 (NCT04520750)
- Testing
- PTX-022
- Where
- 4 sites in United States
- Volunteers
- 36 people
- Sponsor
- Palvella Therapeutics, Inc.
Pachyonychia Congenita
A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita
PALV-08 is a multicenter, open-label treatment (OLT) study enrolling adults with Pachyonychia Congenita (PC) with genotyped keratin mutations KRT6A, KRT6B, KRT6C or KRT16 who were previously enrolled in the PALV-05 (VAPAUS)…
- Trial name
- NCT05643872
- Testing
- PTX-022
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Palvella Therapeutics, Inc.
Epidermolytic Ichthyosis, Palmoplantar Keratoderma
Epidermal Growth Factor Receptor Inhibition for Keratinopathies
Epidermal growth factor receptor (EGFR) signaling plays a key role in regulating epidermal cell proliferation, survival, and differentiation.
- Conditions
- Epidermolytic Ichthyosis, Palmoplantar Keratoderma, Pachyonychia Congenita
- Trial name
- NCT06545695
- Testing
- Erlotinib
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- Northwestern University
Pachyonychia Congenita
Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita
Pachyonychia congenita (PC) is a rare, autosomal dominant keratin disorder affecting the nails, skin, oral mucosae, larynx, hair and teeth.
- Trial name
- NCT00716014
- Testing
- TD101
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Pachyonychia Congenita Project
Punctate Palmoplantar Keratoderma Type 1, Pachyonychia Congenita
Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punctate Palmoplantar Keratoderma or Pachyonychia Congenita Diseases
In this phase 1 open label study for patients with type I punctate palmoplantar keratoderma or pachyonychia congenital, 2 arms will be recruited to be treated twice daily, with 1% topical KM-001.
- Trial name
- NCT05435638
- Testing
- Serum chemistry, Hematology, Urinalysis
- Where
- 3 sites in Israel
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Orvida Pharma (formerly Kamari Pharma)
Pachyonychia Congenita
Simvastatin Treatment of Pachyonychia Congenita
Simvastatin and related statins, such as compactin, are able to inhibit the constitutive expression of inducible/repressible keratin genes such as K6a and K17.
- Trial name
- NCT01382511
- Testing
- Simvastatine
- Volunteers
- 10 people
- Sponsor
- Tel-Aviv Sourasky Medical Center
Pachyonychia Congenita
International Pachyonychia Congenita Research Registry
International Pachyonychia Congenita Research Registry (IPCRR) is a patient registry for those suffering from Pachyonychia Congenita (PC).
- Trial name
- IPCRR (NCT02321423)
- Where
- United States
- Volunteers
- 2,000 people, healthy volunteers welcome
- Sponsor
- Pachyonychia Congenita Project