Search clinical trials for: pachyonychia congenita

Total 9119 results

Kamari Pharma Ltd

Recruiting

Assessment of KM-001 - Safety, Tolerability, and Efficacy in Patients With PPPK1 or PC

Pachyonychia Congenita | Punctate Palmoplantar Keratoderma Type 1

United Kingdom
Palvella Therapeutics, Inc.

Recruiting

A Study Evaluating the Safety and Pharmacokinetics of QTORIN Rapamycin 3.9% Anhydrous Gel in the Treatment of Adults With Pachyonychia Congenita

Pachyonychia Congenita

United States
Kamari Pharma Ltd

Recruiting

Study Designed to Evaluate Safety and Efficacy of 1% Topical Formulation of KM-001 on Type 1 Punctate Palmoplantar Keratoderma or Pachyonychia Congenita Diseases

KPPP1 | Pachyonychia, Congenital

Israel
Palvella Therapeutics, Inc.

Recruiting

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 (VAPAUS)

Pachyonychia Congenita

United States, United Kingdom
Pachyonychia Congenita Project

Recruiting

International Pachyonychia Congenita Research Registry (IPCRR)

Pachyonychia Congenita

United States
National Institute of Arthritis and Musculoskeletal...

Completed

National Registry for Ichthyosis and Related Disorders

Ichthyosis | Ichthyosis, X-Linked | Ichthyosis, Lamellar | Darier Disease | Hailey-Hailey Disease | Hyperkeratosis, Epidermolytic | Keratoderma

United States
Palvella Therapeutics, Inc.

Active, not recruiting

VALO-2: Study Evaluating the Safety and Efficacy of PTX022 in the Treatment of Adults With Pachyonychia Congenita (VALO-2)

Pachyonychia Congenita

United States
Palvella Therapeutics, Inc.

Unknown

Phase 2/3 Study Evaluating the Safety and Efficacy of PTX-022 in Treatment of Adults With Pachyonychia Congenita

Pachyonychia Congenita

United States
Johns Hopkins University

Completed

Effect of Broccoli Sprout Extract on Keratinocyte Differentiation in Normal Skin

Epidermolysis Bullosa Simplex | Pachyonychia Congenita

United States
TransDerm, Inc.
Pachyonychia Congenita Project

Completed

Topical Sirolimus for the Treatment of Pachyonychia Congenita (PC)

Pachyonychia Congenita

United States
Tel-Aviv Sourasky Medical Center

Unknown

Simvastatin Treatment of Pachyonychia Congenita

Pachyonychia Congenita
Uppsala University

Unknown

Botulinumtoxin A Treatment in Epidermolysis Bullosa Simplex and Pachyonychia Congenita

Epidermolysis Bullosa Simplex

Sweden
Pachyonychia Congenita Project

Completed

Study of TD101, a Small Interfering RNA (siRNA) Designed for Treatment of Pachyonychia Congenita

Pachyonychia Congenita

United States
Xinhua Hospital, Shanghai Jiao Tong University...

Recruiting

Artificial Intelligence-enabled ECG Detection of Congenital Heart Disease in Children: a Novel Diagnostic Tool (AI-ECG-CHD)

Artificial Intelligence | Diagnosis | Congenital Heart Disease in Children | Deep Learning | Electrocardiogram

China
Istituto Ortopedico Rizzoli
Istituto Clinico Humanitas; Istituto Giannina Gaslini; Ospedale Pediatrico Bambino... and other collaborators

Not yet recruiting

Advanced Surgical Simulation Processes in the Correction of Skeletal Defects and Deformities (SIMULA)

Musculoskeletal Abnormalities | Musculoskeletal Disorder | Musculoskeletal Deformity

Italy
Assiut University

Not yet recruiting

Coronary Arteries Anomalies by CT in Children With CHD

Cardiac Congenital Defects
University of Campania "Luigi Vanvitelli"
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; University of Milan and other collaborators

Recruiting

The PRO-FUTURE Project (PRO-FUTURE)

Chronic Kidney Diseases | Ureteropelvic Junction Obstruction

Italy
University Ghent
Research Foundation Flanders

Not yet recruiting

Effect and Cost-utility of of High Intensity vs. Low Intensity Speech Intervention in Children With Cleft Palate

Cleft Lip and Palate | Cleft Palate Children | Speech Therapy | Speech Disorders in Children
Chongqing Medical University

Recruiting

Effect of Bilirubin on Prognosis in Heart Failure With Preserved Ejection Fraction

Heart Failure With Preserved Ejection Fraction | Prognathism

China
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
IRCCS Eugenio Medea

Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] (MDCJ-NeuBeRe)

Myotonic Dystrophy 1

Italy
Assiut University

Not yet recruiting

Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia

B Thalassemia
Didier TCHETCHE
Abbott

Not yet recruiting

NavIIcusp: Bicuspid Aortic Valve Stenosis With Navitor Platform International Experience

Bicuspid Aortic Valve

France
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
University College, London
Washington University School of Medicine; University of Washington; Alzheimer... and other collaborators

Not yet recruiting

A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK (NIO-SILK)

Alzheimer Disease | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Amyloid Precursor Protein (Disorder)
Kocaeli University

Recruiting

Maternal Consequences of Membrane Stripping

Maternal Care for Other Abnormalities of Cervix

Turkey
Fondation Hôpital Saint-Joseph

Not yet recruiting

Procedural Pain Management by Multimodal Sedation Analgesia Combining Hypnosis in Children With Congenital Heart Disease (PEACE-HYPNO)

Congenital Heart Disease | Hypnosis | Procedural Pain | Heart Surgery
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain (PRESENCE)

Pain | Sickle Cell Disease
Stanford University
National Institutes of Health (NIH)

Not yet recruiting

Cardiac Simulator for Surgical Planning

Congenital Heart Disease in Children
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Lawson Health Research Institute
Greenstone Biosciences, Inc.

Not yet recruiting

iPSC Biobank of Biomarkers Diversity in Cardiovascular Disease (INFERENCE)

Cardiovascular Diseases | Heart Failure | Cardiomyopathies | Congenital Heart Disease | Cerebrovascular Accident | Arrythmia | Cardiometabolic Syndrome
Cairo University

Not yet recruiting

En-mass Retraction by Lingual Retractor Versus Clear Aligner Appliances

Maxillary Prognathism

Egypt
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

School Readiness Intervention for Preschool Children With Sickle Cell Disease

Sickle Cell Disease

United States
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Dan Bing

Not yet recruiting

Genetic Feature of Congenital Hearing Loss in Chinese Population

Congenital Hearing Loss | Congenital Deafness
Centre Hospitalier Universitaire de Besancon

Not yet recruiting

EPBONF : Evaluation of the Safety and Effectiveness of a Medical Device Aimed at Guiding Orbito-naso-frontal Band Surgery, for the Treatment of Craniostenoses (EPBONF)

Craniosynostoses
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Perinatal Thoraco-abdomino-pelvic Tumors Study (CONTRAST)

Perinatal Solid Tumors of the Thoraco-abdomino-pelvic Region

France
Editas Medicine, Inc.

Not yet recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
Octapharma

Not yet recruiting

Phase 3 Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema
University Hospital, Toulouse

Not yet recruiting

Efficacy of Injectable Gentamicin in Hereditary Ichthyosis (GENTIC)

Ichthyosis

France
Universidad Nacional Autonoma de Mexico

Recruiting

Effects of Different Remineralization Agents on Molar Incisor Hypomineralization Defects: a Randomized Clinical Study

Molar Incisor Hypomineralization

Mexico
Children's National Research Institute
The Hospital for Sick Children; Ann & Robert H Lurie Children's Hospital of... and other collaborators

Recruiting

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY (SUN-RAY)

Sickle Cell Disease

United States
Children's Hospital of Fudan University

Not yet recruiting

Phenotype and Multi-omics Analysis of Children With Congenital Diarrhea and Enteropathy in China

Diarrhea Infantile
Karamanoğlu Mehmetbey University

Not yet recruiting

Knowledge, Attitudes and Practices of Paediatric Cardiologists About Cardiac Rehabilitation in Children With Congenital Heart Disease

Physician's Role
IRCCS Eugenio Medea

Recruiting

Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy

Epilepsy | Angelman Syndrome | Dup15q Syndrome

Italy
Institut de Myologie, France
Association Française contre les Myopathies (AFM), Paris

Not yet recruiting

Natural History Study of Children With LAMA2-related Dystrophies (LAMA2)

Merosin Deficient Congenital Muscular Dystrophy

France
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Prevalence and Characterization of Pain in RASopathies (3717)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
Hasan Kalyoncu University

Recruiting

Effects of Orofacial Therapy and Therapeutic Yoga in Children With Down Syndrome

Down Syndrome

Turkey
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients (TRANSPLANTORN2)

Sickle Cell Disease

Search clinical trials for: pachyonychia congenita

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