Clinical trials: Congenital Deformity
Retinitis Pigmentosa
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading…
- Trial name
- NCT03349242
- Where
- 8 sites in United States, Canada and 1 more
- Volunteers
- 140 people
- Sponsor
- MeiraGTx UK II Ltd
Atopic Dermatitis
A Clinical Study to Evaluate the Safety and Efficacy of a Baby Cleanser and a Moisturizer
A Clinical Study to Evaluate the Safety and Efficacy of a Baby Cleanser and a Moisturizer Under the Supervision of Dermatologist and Pediatrician
- Trial name
- NCT03719742
- Testing
- Sponsor Test Products
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Burt's Bees Inc.
Cystic Fibrosis
A Study Evaluating the Long Term Safety and Efficacy of VX-659 Combination Therapy
This study will evaluate the long-term safety and tolerability of VX-659 in triple combination (TC) with tezacaftor (TEZ) and ivacaftor (IVA) in subjects with cystic fibrosis (CF) who are homozygous or heterozygous for the…
- Trial name
- NCT03447262
- Testing
- VX-659/TEZ/IVA, IVA
- Where
- 100 sites in United States, Australia and 9 more
- Volunteers
- 484 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Cerebral Cavernous Malformation
The Symptomatic Cerebral Cavernous Malformation Trial of REC-994
This is a two-part, multi-center, randomized, double-blind, placebo-controlled study to investigate the safety, efficacy and pharmacokinetics of REC-994 (200 mg and 400 mg) compared to placebo in participants with symptomatic…
- Trial name
- SYCAMORE (NCT05085561)
- Testing
- REC-994
- Where
- 15 sites in United States
- Volunteers
- 62 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Blindness, Leber Congenital Amaurosis 10
Natural History Study of CEP290-Related Retinal Degeneration
A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future clinical treatment trials involving patients with CEP290-related…
- Conditions
- Blindness, Leber Congenital Amaurosis 10, Vision Disorders, Eye Diseases
- Trial name
- NCT03396042
- Where
- 7 sites in United States, France and 2 more
- Volunteers
- 26 people
- Sponsor
- Editas Medicine, Inc.
Gout
RDEA3170 and Allopurinol Combination Study in Gout Subjects
This is a Phase 1b, randomized, open-label, drug-drug interaction study in adult male subjects with gout. It is designed to assess the pharmacokinetics (PK) and pharmacodynamics (PD) of RDEA3170 or allopurinol alone and in…
- Trial name
- NCT02279641
- Testing
- RDEA3170 10 mg, allopurinol 300 mg
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Ardea Biosciences, Inc.
Hypertension
A Study of the Effects of 6R-BH4 on Blood Pressure in Subjects With Poorly Controlled Systemic Hypertension
The purpose of this study is to determine whether 6R-BH4 (sapropterin dihydrochloride) is safe and effective in the treatment of poorly controlled hypertension in the presence or absence of type 2 diabetes.
- Trial name
- NCT00325962
- Testing
- 6R-BH4 (sapropterin dihydrochloride)
- Where
- 24 sites in United States
- Volunteers
- 84 people
- Sponsor
- BioMarin Pharmaceutical
Huntington Chorea
Efficacy and Safety on SOM3355 in Huntington's Disease Chorea
Phase IIb, randomized, double-blind, placebo-controlled study in parallel groups assessing the efficacy and safety of two doses of SOM3355 in patients suffering from Huntington's Disease with choreic movements.
- Trial name
- NCT05475483
- Testing
- SOM3355 200 mg capsules, SOM3355 300 mg capsules
- Where
- 23 sites in France, Germany and 5 more
- Volunteers
- 139 people
- Sponsor
- SOM Innovation Biotech SA
Hemophilia A
To Evaluate the Clinical Study of GS1191-0445 Injection in the Treatment of Hemophilia A
This study is a single-arm, open-label, multicenter study evaluating the efficacy and safety of GS1191-0445 injection as a single dose in Chinese subjects with hemophilia A.
- Trial name
- NCT06833983
- Testing
- GS1191-0445 injection
- Where
- 13 sites in China
- Volunteers
- 50 people
- Sponsor
- Gritgen Therapeutics Co., Ltd.
Mucopolysaccharidosis IH
Gene Therapy With Modified Autologous Hematopoietic Stem Cells for the Treatment of Patients With Mucopolysaccharidosis Type I, Hurler Variant
This is a phase I/II study evaluating safety and efficacy of autologous hematopoietic stem and progenitor cells genetically modified with IDUA lentiviral vector encoding for the human α-L-iduronidase gene for the treatment of…
- Trial name
- TigetT10_MPSIH (NCT03488394)
- Testing
- Frozen autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LVV, encoding for the α-L-iduronidase cDNA, in their final formulation medium.
- Where
- Italy
- Volunteers
- 8 people
- Sponsor
- Orchard Therapeutics
Tyrosinemia, AKU
Evaluation of TYR Sphere in France
10 participants aged 3 years and over with tyrosinaemia or alkaptonuria will be prescribed Tyr sphere following an assessment of their individual needs by their dietitian.
- Trial name
- TYR sphere Fra (NCT04761588)
- Testing
- Tyr sphere
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Vitaflo International, Ltd
Focal Segmental Glomerulosclerosis, Minimal Change Disease
Study of Sparsentan Treatment in Pediatrics With Proteinuric Glomerular Diseases
To evaluate the safety, efficacy and tolerability of sparsentan oral suspension and tablets, and assess changes in proteinuria after once-daily dosing over 108 weeks.
- Conditions
- Focal Segmental Glomerulosclerosis, Minimal Change Disease, Immunoglobulin A Nephropathy, IgA Vasculitis
- Trial name
- EPPIK (NCT05003986)
- Testing
- Sparsentan
- Where
- 47 sites in United States, Germany and 6 more
- Volunteers
- 67 people
- Sponsor
- Travere Therapeutics, Inc.
Pulmonary Valve Insufficiency, Complex Congenital Heart Defect
ALTERRA Post-Approval Study
This study will monitor device performance and outcomes in subjects undergoing implantation of the Edwards SAPIEN 3 Transcatheter Pulmonary Valve System with Alterra Adaptive Prestent in the post-approval setting
- Conditions
- Pulmonary Valve Insufficiency, Complex Congenital Heart Defect, Tetralogy of Fallot, Pulmonary Regurgitation
- Trial name
- NCT05378386
- Testing
- Edwards Alterra Adaptive Prestent with SAPIEN 3 Transcatheter Pulmonary Valve System
- Where
- 24 sites in United States, Israel
- Volunteers
- 150 people
- Sponsor
- Edwards Lifesciences
Epidermolysis Bullosa
Open-Label Extension Study to Evaluate the Safety of SD-101 Cream in Participants With Epidermolysis Bullosa
The purpose of this study was to assess the continued safety of topical use of SD-101 cream (6%) in participants with Epidermolysis Bullosa (EB). Funding Source: FDA Office of Orphan Products Development
- Trial name
- NCT02090283
- Testing
- SD-101 dermal cream (6%)
- Where
- 7 sites in United States
- Volunteers
- 42 people
- Sponsor
- Scioderm, Inc.
Atopic Dermatitis
Efficacy Emollient on Xerosis in Children With Atopic Dermatitis
Atopic dermatitis is a frequent, chronic inflammatory disease influenced by local, immunological, genetic and environmental factors.
- Trial name
- NCT01467362
- Testing
- V0034CR01B, Vehicle cream, desonide 0.1% cream
- Where
- 25 sites in Estonia, France and 3 more
- Volunteers
- 251 people
- Sponsor
- Pierre Fabre Medicament
Atopic Dermatitis
Efficacy and Safety of MEDI3506 in Adult Subjects With Atopic Dermatitis
This is a research study to determine the efficacy and safety of investigational drug MEDI3506 for the treatment of adult subjects with Atopic Dermatitis.
- Trial name
- NCT04212169
- Testing
- MEDI3506
- Where
- 38 sites in United States, Australia and 4 more
- Volunteers
- 148 people
- Sponsor
- AstraZeneca
Prader-Willi Syndrome
Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome
This Phase 3 study is designed to test the effectiveness of intranasal carbetocin (LV-101) in participants with Prader-Willi syndrome (PWS). Carbetocin is an oxytocin analog (a man-made chemical that is like oxytocin).
- Trial name
- CARE-PWS (NCT03649477)
- Testing
- 3.2 mg intranasal carbetocin, 9.6 mg intranasal carbetocin
- Where
- 24 sites in United States, Australia and 1 more
- Volunteers
- 130 people
- Sponsor
- Levo Therapeutics, Inc.
Lysosomal Storage Disease, Lung Diseases
Biomarker for Maroteaux-Lamy Disease (BioMaroteaux)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Maroteaux-Lamy disease from blood
- Conditions
- Lysosomal Storage Disease, Lung Diseases, Obstructive Sleep Apnoea, Macroglossia
- Trial name
- BioMaroteaux (NCT01458613)
- Where
- 5 sites in Egypt, Germany and 2 more
- Sponsor
- CENTOGENE GmbH Rostock
Eczema, Dermatitis
Efficacy and Safety Study of Desloratadine (MK-4117) in Japanese Participants With Eczema/Dermatitis and Dermal Pruritus (MK-4117-202)
This is an efficacy and safety study of up to 12 weeks of desloratadine in Japanese participants with eczema/dermatitis and dermal pruritus.
- Conditions
- Eczema, Dermatitis, Dermal Pruritus
- Trial name
- NCT01916980
- Testing
- Desloratadine 5 mg
- Volunteers
- 94 people
- Sponsor
- Organon and Co
Healthy, Long QT Syndrome
Study Evaluating the Effects of Multiple Oral Doses of ERB-041 on Cardiac Repolarization in Healthy Subjects
To assess the effect of ERB-041 after multiple oral doses on cardiac repolarization as assessed by the QTc interval.
- Trial name
- NCT00316459
- Testing
- ERB-041
- Where
- United States
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
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