Clinical trials: Hereditary Diseases
Cutaneous Neurofibroma, Neurofibromatosis 1
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF.
- Trial name
- NCT05005845
- Testing
- NFX-179 gel, Vehicle gel
- Where
- 23 sites in United States
- Volunteers
- 199 people
- Sponsor
- NFlection Therapeutics, Inc.
Endothelial Dysfunction, Hyperhomocysteinemia
Effect of L-Arginine and Pycnogenol on Light to Moderate Hypertension and Endothelial Function
The effect of a combination product (Verum ) with L-arginine, Pycnogenol, vitamin K2, R-(+)-alpha-lipoic acid and vitamins B6, B12 and folic acid is investigated in a double blind placebo-controlled cross-over study.
- Conditions
- Endothelial Dysfunction, Hyperhomocysteinemia, Hypertension Grade I, Subgroup "Borderline" (WHO)
- Trial name
- NCT02392767
- Testing
- Verum
- Where
- Germany
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Dr. Loges & Co. GmbH
Skin Laxity
Safety and Effectiveness Evaluation of the Device in Achieving Submental Lift
A prospective evaluation of the ThermiRF device in treating skin laxity under the chin and neck. Seventy male and female subjects between the age of 35 and 65 (inclusive) will be enrolled in this trial.
- Trial name
- NCT02832674
- Testing
- Percutaneous Radiofrequency single treatment
- Where
- United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- ThermiGen, LLC
Sickle Cell Anemia
Study of Efficacy, Safety and Tolerability of ACZ885 (Canakinumab) in Pediatric and Young Adult Patients With Sickle Cell Anemia
The study assesses the efficacy, safety and tolerability of ACZ885 (canakinumab) in pediatric and young adult patients with sickle cell anemia (SCA).
- Trial name
- NCT02961218
- Testing
- ACZ885
- Where
- 15 sites in United States, Canada and 5 more
- Volunteers
- 49 people
- Sponsor
- Novartis Pharmaceuticals
Hereditary Angioedema
A Study of Icatibant (TAK-667) in Japanese Children and Teenagers With Acute Attacks of Hereditary Angioedema
The main aim of the study is to check for side effects from icatibant in children and teenagers with hereditary angioedema (HAE).
- Trial name
- NCT04654351
- Testing
- TAK-667
- Where
- 3 sites in Japan
- Volunteers
- 2 people
- Sponsor
- Takeda
Congenital Adrenal Hyperplasia
Comparison of Chronocort Versus Standard Hydrocortisone Replacement Therapy in Participants Aged 16 Years and Over With Congenital Adrenal Hyperplasia
This study is a randomized, double-blind, active-controlled, phase III study of Chronocort® compared with immediate-release hydrocortisone replacement therapy in participants aged 16 years and over with Congenital Adrenal…
- Trial name
- CONnECT (NCT05063994)
- Testing
- Chronocort, Cortef
- Where
- 21 sites in United States, France and 1 more
- Volunteers
- 55 people
- Sponsor
- Neurocrine UK Limited
Alpha1-antitrypsin Deficiency, Healthy Volunteers
GLASSIA Infusion Rate Study
The purpose of this study was to generate sufficient safety and tolerability information in support of an increase in the infusion rate of intravenous GLASSIA in the prescribing information from 0.04 to 0.2 mL/kg/min.
- Trial name
- NCT01651351
- Testing
- Alpha1-proteinase inhibitor
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baxalta now part of Shire
Gaucher Disease
Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00705939
- Testing
- Taliglucerase alfa
- Where
- 11 sites in United States, Australia and 6 more
- Volunteers
- 45 people
- Sponsor
- Pfizer
Adenomatous Polyposis Coli
Trial In Pediatric Patients With Familial Adenomatous Polyposis (FAP)
To test whether celecoxib can be used to prevent colon polyp formation in children with familial adenomatous polyposis (FAP).
- Trial name
- CHIP (NCT00585312)
- Testing
- Celecoxib
- Where
- 42 sites in United States, Belgium and 12 more
- Volunteers
- 106 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Hemophilia B
BeneFIX Drug Use Results Survey [All-Case Surveillance]
The survey is intended to investigate the following matters, etc. under the actual use status after marketing in all patients who are administered this drug for a certain period of time after the launch. 1.
- Trial name
- NCT01154231
- Testing
- Nonacog Alfa (Genetical Recombination)
- Where
- Japan
- Volunteers
- 314 people
- Sponsor
- Pfizer
Erythropoietic Protoporphyria
Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP.
- Trial name
- NCT05308472
- Testing
- DISC-1459
- Where
- 9 sites in United States
- Volunteers
- 75 people
- Sponsor
- Disc Medicine, Inc
Schizophrenia, Paranoid, Schizophrenia, Disorganized
Fixed Dose Efficacy and Safety Study of Asenapine for the Treatment of Schizophrenia in Adolescents (P05896)
This study is designed to evaluate whether asenapine, which is approved by the United States Food and Drug Administration (US FDA) for acute treatment of schizophrenia in adults, is also effective in adolescents with…
- Conditions
- Schizophrenia, Paranoid, Schizophrenia, Disorganized, Schizophrenia, Undifferentiated
- Trial name
- NCT01190254
- Testing
- asenapine 2.5 mg, asenapine 5.0 mg
- Volunteers
- 306 people
- Sponsor
- Organon and Co
Atopic Dermatitis
Long-term Extension Trial in Subjects With Atopic Dermatitis Who Participated in Previous Tralokinumab Trials - ECZTEND
The purpose of this extension trial is to evaluate the long-term safety of tralokinumab.
- Trial name
- NCT03587805
- Testing
- Tralokinumab
- Where
- 309 sites in United States, Belgium and 9 more
- Volunteers
- 1,672 people
- Sponsor
- LEO Pharma
Tourette's Disorder
Efficacy & Safety Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current study is to determine efficacy and safety of once-weekly aripiprazole in reducing Total Tic Severity (TTS) score in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01418339
- Testing
- Aripiprazole
- Where
- 39 sites in United States, Canada and 4 more
- Volunteers
- 135 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Homozygous Familial Hypercholesterolemia
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH)
Participants with documented homozygous familial hypercholesterolemia (HoFH) who have provided informed consent will receive 2 open-label doses of ARO-ANG3 and be evaluated for safety and efficacy parameters through 36 weeks.
- Trial name
- Gateway (NCT05217667)
- Testing
- ARO-ANG 3 Injection
- Where
- 7 sites in United States, Australia and 2 more
- Volunteers
- 18 people
- Sponsor
- Arrowhead Pharmaceuticals
Cystic Fibrosis
A Study Evaluating the Efficacy and Safety of VX-445/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects, Homozygous for F508del
This study will evaluate the efficacy, safety, and pharmacodynamics of elexacaftor (ELX, VX-445) in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous for…
- Trial name
- NCT04105972
- Testing
- ELX/TEZ/IVA, TEZ/IVA, IVA
- Where
- 35 sites in Australia, Belgium and 2 more
- Volunteers
- 176 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Prader-Willi Syndrome
Study of the Efficacy and Safety of Somatropin in Japanese Participants With PWS
This is a multicenter, open label, multi cohort study to evaluate the efficacy and safety of somatropin in a cohort of Japanese participants with PWS.
- Trial name
- NCT04697381
- Testing
- somatropin - GH naïve pediatric cohort, somatropin - GH treated cohort, somatropin - adult cohort
- Where
- 5 sites in Japan
- Volunteers
- 33 people
- Sponsor
- Pfizer
Severe Hemophilia A
Study to Investigate the Long-term Efficacy and Safety of Human-cl rhFVIII in Previously Treated Patients (PTPs)
The purpose of the study is to study the long-term efficacy, safety and tolerability of Human-cl rhFVIII in previously treated patients with severe hemophilia A.
- Trial name
- NCT01341912
- Testing
- Human-cl rhFVIII
- Where
- 2 sites in United States, Bulgaria
- Volunteers
- 3 people
- Sponsor
- Octapharma
Gout
Food Effect Study of Febuxostat XR in Healthy Participants
The purpose of this study is to assess the effect of food on the bioavailability of febuxostat after a single oral dose of 80 mg febuxostat XR and to evaluate the pharmacokinetics (PK) of febuxostat after single oral doses of 40…
- Trial name
- NCT02374164
- Testing
- Febuxostat XR
- Where
- United States
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- Takeda
Fragile X Syndrome
Study of SPG601 in Adult Men With Fragile X Syndrome
This Phase 2 study described herein will evaluate the safety, efficacy, tolerability, pharmacokinetics and pharmacodynamics of SPG601 in adult men with Fragile X syndrome.
- Trial name
- NCT06413537
- Testing
- SPG601
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Spinogenix