Clinical trials: Congenital Disorder
Leigh Syndrome
A Study of the Natural Course of SURF1 Deficiency
The purpose of the study is to prospectively and systematically collect standardized clinical information, to describe important features of the disease course of SURF1 deficiency.
- Trial name
- NCT05277363
- Testing
- None (Observational study)
- Where
- United States
- Sponsor
- Taysha Gene Therapies, Inc.
Achondroplasia
A Study in Children With Achondroplasia
The goal of this observational study is to collect the anthropometric parameters, clinical characteristics, related medical complications, health-related quality of life and treatments of children with ACH, and complete a natural…
- Trial name
- NCT07301463
- Testing
- No Interventions, complete a natural history observation of ACH for at least 6 months and up to 2 years
- Where
- 8 sites in China
- Volunteers
- 260 people
- Sponsor
- Abbisko Therapeutics Co, Ltd
Dermatitis, Atopic, Dermatitis, Contact
Efficacy and Tolerability of Prednisolone Acetate 0.5% Cream Versus Betamethasone Valerate 0.1% Cream in Cortisosensitive Dermatosis
Topical corticosteroids are largely used in dermatology. The major problem related to their use is that the same mechanisms underlying their therapeutic effects (antiinflammatory and antiproliferative) may lead to adverse events.
- Conditions
- Dermatitis, Atopic, Dermatitis, Contact, Dermatitis, Seborrheic, Psoriasis
- Trial name
- NCT01011621
- Testing
- 0.5% prednisolone acetate cream, 0.1% betamethasone valerate cream
- Volunteers
- 170 people
- Sponsor
- Mantecorp Industria Quimica e Farmaceutica Ltd.
Pain
An Exploratory Study to Assess the Effects of JNJ-39439335 on the Relief of Pain Using a Thermal-Grill Experimental Model
This exploratory study will use a thermal-grill experimental model to assess the effects of JNJ-39439335 (50 mg, single oral dose) on the relief of pain.
- Trial name
- NCT01006304
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Pharmaceutical Research & Development, L.L.C.
Becker Muscular Dystrophy
Safety and Biomarker Response to (+)-Epicatechin in Becker Muscular Dystrophy
This is a Phase 1, open-label, dose escalation study aimed at evaluating the safety, early efficacy and potential biomarkers of (+)-epicatechin in patients with Becker or Becker-like Muscular Dystrophy (BMD).
- Trial name
- NCT04386304
- Testing
- (+)-Epicatechin
- Where
- 3 sites in United States
- Volunteers
- 22 people
- Sponsor
- Epirium Bio Inc.
Bone Mineral Density, Hemophilia A
Osteoporosis and MRI Study in Hemophilia
The purpose of this study is to evaluate bone mineral density in adult subjects with hemophilia versus a comparator population without hemophilia (non-hemophilia age- and gender-matched database) by using the following diagnostic…
- Conditions
- Bone Mineral Density, Hemophilia A, Hemophilia B
- Trial name
- NCT01460147
- Testing
- DXA scan + MRI
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Baxalta now part of Shire
Atopic Dermatitis
Safety and Efficacy of ADSTEM Inj. in Patients With Moderately Subacute and Chronic Atopic Dermatitis
This study aims to evaluate safety, tolerance, and efficacy in subjects with over moderately subacute and chronic atopic dermatitis after an intravenous injection of autologous mesenchymal stem cells.
- Trial name
- NCT02888704
- Testing
- ADSTEM Inj. (Adult human mesenchymal stem cells)
- Where
- South Korea
- Volunteers
- 13 people
- Sponsor
- EHL Bio Co., Ltd.
Advanced Solid Tumor
A Clinical Study to Test if an Investigational Treatment Called BNT326 is Safe and Potentially Beneficial When Used Alone or in Combination With Other Investigational Treatments Such as BNT327, for People With Advanced Malignant Tumors
This study will evaluate the safety, efficacy, optimal dose, and pharmacokinetics (PK) of BNT326 as monotherapy (Part 1) and as combination treatment with immunotherapeutic agents (Part 2) in participants with histologically or…
- Trial name
- NCT07070232
- Testing
- BNT326, Pumitamig, Itraconazole
- Where
- 77 sites in United States, Australia and 7 more
- Volunteers
- 1,438 people
- Sponsor
- BioNTech SE
Cryopyrin-Associated Periodic Syndromes (CAPS)
HL2351 CAPS Phase II Study
This is an open label, single arm trial to evaluate the efficacy, safety, and pharmacokinetics of HL2351 in patients with cryopyrin associated periodic syndromes (CAPS).
- Trial name
- NCT02853084
- Testing
- HL2351
- Where
- South Korea
- Volunteers
- 8 people
- Sponsor
- Handok Inc.
Chronic Kidney Diseases, Alport Syndrome
An Extended Access Program for Bardoxolone Methyl in Patients With CKD (EAGLE)
This extended access study will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with…
- Conditions
- Chronic Kidney Diseases, Alport Syndrome, Autosomal Dominant Polycystic Kidney
- Trial name
- EAGLE (NCT03749447)
- Testing
- Bardoxolone methyl
- Where
- 87 sites in United States, Australia and 4 more
- Volunteers
- 270 people
- Sponsor
- Biogen
Hypercholesterolemia, Mixed Hyperlipemia
MK0859 Dose-Ranging Study (0859-003)
To assess the cholesterol changing effects of MK0859 in patients with primary hypercholesterolemia or mixed dyslipidemia.
- Trial name
- NCT00325455
- Testing
- MK0859
- Volunteers
- 500 people
- Sponsor
- Merck Sharp & Dohme LLC
Lowe Syndrome
An Open-label, Phase 2 Pilot Study on the Efficacy and Safety of Piclidenoson in Patients With Lowe Syndrome
The primary objective of this trial is to: 1. Evaluate the efficacy of piclidenoson to increase renal uptake of 99mTc-labeled DMSA, in comparison to baseline, after 6 months (26 weeks) of treatment as a measure the reabsorption…
- Trial name
- NCT07410455
- Testing
- Piclidenoson
- Where
- Italy
- Volunteers
- 5 people
- Sponsor
- Can-Fite BioPharma
Hemophilia B
Study Evaluating BENEFIX in Previously Treated Patients With Hemophilia B
To assess efficacy and safety of BeneFix® for prophylaxis in "Short-term" therapy and on demand therapy for all bleeding episodes of subjects with hemophilia B.
- Trial name
- NCT00581126
- Testing
- Recombinant Factor IX Coagulation
- Volunteers
- 14 people
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Pseudomonas Aeruginosa Infection
Study to Evaluate Arikayce™ in CF Patients With Chronic Pseudomonas Aeruginosa Infections
A major factor in the respiratory health of Cystic Fibrosis (CF) participants is the prevalence of chronic Pseudomonas aeruginosa (Pa) infections.
- Trial name
- NCT01315678
- Testing
- Liposomal amikacin for inhalation (Arikayce™) using the PARI Investigational eFlow® Nebulizer., Tobramycin inhalation solution using a PARI LC® Plus nebulizer.
- Where
- 62 sites in Austria, Belgium and 16 more
- Volunteers
- 302 people
- Sponsor
- Insmed Incorporated
Atopic Dermatitis
Targeting Staphylococci in Atopic Dermatitis
The aim of the study is to deliver a better understanding of the molecular mechanisms used by S. epidermidis strains in their adhesion and colonization on the stratum corneum across a broad spectrum of atopic dermatitis in mild…
- Trial name
- NCT05502185
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- L'Oreal
Migraine, Patent Foramen Ovale
GORE® CARDIOFORM Septal Occluder Migraine Clinical Study
Multi-center, prospective, randomized, placebo- and sham-controlled study to evaluate the GORE® CARDIOFORM Septal Occluder for migraine headache relief
- Conditions
- Migraine, Patent Foramen Ovale, PFO - Patent Foramen Ovale
- Trial name
- RELIEF (NCT04100135)
- Testing
- Actual device PFO closure, Thienopyridine (clopidogrel or prasugrel)
- Where
- 15 sites in United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- W.L.Gore & Associates
Cystic Fibrosis
Efficacy and Safety of Inhaled Nitric Oxide (NO) in Cystic Fibrosis (CF) Patients
Prospective, randomized, placebo controlled, phase II clinical study of subjects crossing over from an approved inhaled antibiotic to inhaled nitric oxide as compared to a placebo control arm.
- Trial name
- NCT02498535
- Testing
- Nitric Oxide 160 ppm
- Where
- 7 sites in United States, Canada
- Volunteers
- 49 people
- Sponsor
- Novoteris, LLC
Hyperphagia, Prader-Willi Syndrome
The Hunger Elimination or Reduction Objective (HERO ) Open -Label Extension (OLE) Trial
The goal of this clinical trial is to learn if ARD-101 works to treat hyperphagia-related behavior in patients with Prader-Willi syndrome (PWS) when used in a long term setting. It will also teach us about the safety of ARD-101.
- Conditions
- Hyperphagia, Prader-Willi Syndrome, Hyperphagia in Prader-Willi Syndrome
- Trial name
- NCT07197034
- Testing
- ARD-101
- Where
- 30 sites in United States, Australia and 3 more
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Cerebral Arteriovenous Malformation
Treatment of Cerebral Arteriovenous Malformations With SQUID Liquid Embolic Agent (CHOICE)
The objective of this study is to further establish that SQUID, an alternative liquid embolic agent with specific properties, is a safe and effective alternative in bAVM endovascular treatment strategy available to date.
- Trial name
- CHOICE (NCT03413852)
- Testing
- SQUID non-adhesive liquid embolic agent
- Where
- 19 sites in Belgium, Denmark and 3 more
- Volunteers
- 111 people
- Sponsor
- Embo-Flüssigkeiten A.G.
Niemann-Pick Disease, Type C1
Study of the Pharmacokinetics of Trappsol and Effects on Potential Biomarkers of Niemann-Pick C1 (NPC1)
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann Pick disease Type C (NPC-1) is safe at 2 different dose levels and what effects it has on people who…
- Trial name
- NCT02939547
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Cyclo Therapeutics, Inc.
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