Clinical trials: Inherited Genetic Conditions (Diagnosis)
Cancer, Colorectal Cancer
FOG-001 in Locally Advanced or Metastatic Solid Tumors
The goal of this clinical trial is to determine if FOG-001 is safe and effective in participants with locally advanced or metastatic solid tumors or in participants with familial adenomatous polyposis (FAP).
- Conditions
- Cancer, Colorectal Cancer, Solid Tumor, Locally Advanced Solid Tumor
- Trial name
- NCT05919264
- Testing
- FOG-001, mFOLFOX-6, Nivolumab
- Where
- 33 sites in United States, Australia
- Volunteers
- 619 people
- Sponsor
- Parabilis Medicines, Inc.
Homozygous Familial Hypercholesterolemia (HoFH)
Identifying and Genotyping Homozygous Familial Hypercholesterolemia (HoFH) Patients
This study is designed to help identify patients with HoFH due to mutations in the LDLR as confirmed by genotyping.
- Trial name
- NCT04148001
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- REGENXBIO Inc.
Congenital Adrenal Hyperplasia
Safety, Pharmacokinetics and Pharmacodynamics of NBI-77860 in Adolescent Females With Congenital Adrenal Hyperplasia
This is a Phase 1, multicenter, open-label, single-dose study to evaluate the safety and tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-77860 in subjects with congenital adrenal hyperplasia (CAH).
- Trial name
- NCT02349503
- Testing
- NBI-77860
- Where
- 2 sites in United States
- Sponsor
- Neurocrine Biosciences
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.
Fed
Bioequivalence Study of Etodolac Tablet USP 500 mg Under Fed Condition
This is a open Label, balanced, randomized, single dose, two-treatment, two-sequence, two-period, crossover pivotal study. The purpose of this study is to assess the bioequivalence between Test Product and the corresponding…
- Trial name
- NCT01735383
- Testing
- Etodolac Tablets USP 500mg, Etodolac Tablets USP 500 mg
- Where
- India
- Volunteers
- 36 people, healthy volunteers welcome
- Sponsor
- IPCA Laboratories Ltd.
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Atopic Dermatitis
A Study to Evaluate the Safety and Efficacy of Ruxolitinib Phosphate Cream Applied Topically to Adults With Atopic Dermatitis
The purpose of this study is to establish the efficacy of each strength of ruxolitinib cream once daily (QD) or twice daily (BID) in participants with atopic dermatitis as compared with vehicle cream BID.
- Trial name
- NCT03011892
- Testing
- Ruxolitinib 0.15% Cream QD, Ruxolitinib 0.5% Cream QD, Ruxolitinib 1.5% Cream QD
- Where
- 52 sites in United States, Canada
- Volunteers
- 307 people
- Sponsor
- Incyte Corporation
Atopic Dermatitis
A Dose Ranging Placebo-Controlled Double-Blind Study to Evaluate the Safety and Efficacy of Tezepelumab in Atopic Dermatitis
This phase 2b study is designed to evaluate the safety and efficacy of tezepelumab as a monotherapy and explore its efficacy as adjunct therapy in subjects with moderate-to-severe atopic dermatitis (AD).
- Trial name
- NCT03809663
- Testing
- Tezepelumab
- Where
- 86 sites in United States, Australia and 13 more
- Volunteers
- 251 people
- Sponsor
- Amgen
Retinitis Pigmentosa (RP), Usher Syndrome
24-Month Trial of NPI-001 for the Preservation of Photoreceptors in Retinitis Pigmentosa Associated With Usher Syndrome
The goal of this clinical trial is to learn if NPI-001 works to prevent progression of retinitis pigmentosa in adults diagnosed with Usher syndrome. It will also provide information about the safety of NPI-001.
- Trial name
- NCT07290530
- Testing
- N-acetylcysteine amide
- Volunteers
- 80 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Healthy, Fasting
The Food Effect on Pharmacokinetics of Fixed Dose Combination of Gemigliptin/Dapagliflozin/Metformin in Healthy Volunteers
This is a single dose, randomized, open-label, two-way crossover study to investigate the food-effect bioavailability of the fixed-dose combination of Gemigliptin/Dapagliflozin/Metformin 50/10/1000 mg in healthy volunteers under…
- Conditions
- Healthy, Fasting, Fed
- Trial name
- NCT07396844
- Testing
- Fixed-dose combination of gemigliptin/dapagliflozin/metformin 50/10/1000 mg
- Where
- Thailand
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- LG Chem
Pseudomonas Infection
Cipro Inhaler for Cystic Fibrosis Children Ages 6-12
Ciprofloxacin PulmoSphere Inhalation Powder appears to be an effective and adequate antibiotic treatment for cystic fibrosis patients with P. aeruginosa colonisation.
- Trial name
- NCT00910351
- Testing
- Ciprofloxacin (Cipro, BAYQ3939)
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- Bayer
Haemophilia A, Haemophilia B
Management of Health-Related QoL Impairment, Including Pain, Depression and Anxiety, in People With Haemophilia A and B
The puropse of this non-interventional register and survey study is to identify the patterns of prescribed pain, anti-depressive and anti-anxiety medication and management of pain, depression and anxiety for people with…
- Trial name
- MIND (NCT03276130)
- Where
- 5 sites in Denmark, Finland and 1 more
- Volunteers
- 2,300 people
- Sponsor
- Swedish Orphan Biovitrum
Wrinkle, Fine Lines
Study To Assess A Radiofrequency Microneedling Device For Dermatologic Conditions
Subjects are to be enrolled in this clinical study if they are 18 - 55 years old. Up to 120 subjects will be enrolled at multiple study centers.
- Conditions
- Wrinkle, Fine Lines, Crepey Skin, Acne Scars
- Trial name
- NCT05097157
- Testing
- Potenza
- Where
- 4 sites in United States
- Volunteers
- 79 people
- Sponsor
- Cynosure, Inc.
Atopic Dermatitis
A Study to Learn How Different Forms of the Study Medicine Called PF 08049820 Are Taken up Into the Bloodstream in Healthy Adults
The purpose of this study is to see how different forms of a medicine called PF-08049820 move through the body when taken by mouth.
- Trial name
- NCT07172321
- Testing
- PF-08049820 MR1, PF-08049820 MR2, PF-08049820 IR
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Familial Adenomatous Polyposis
A Trial of Low Dose Sulindac Combined With Eflornithine in Patients With Familial Adenomatous Polyposis (FAP)
The purpose of this phase III study is to evaluate the safety and efficacy of the combination of eflornithine and sulindac compared to single agent sulindac or eflornithine in reducing the number of polyps in patients with…
- Trial name
- NCT01245816
- Testing
- Eflornithine plus Sulindac
- Sponsor
- Cancer Prevention Pharmaceuticals, Inc.
Choroideremia
Natural Disease Progression in Participants With Choroideremia
The primary objective of this study is to quantify disease progression measured by best corrected visual acuity (BCVA) in the real-world setting among Choroideremia (CHM) participants.
- Trial name
- NCT04795206
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 1,178 people
- Sponsor
- Biogen
Dermatitis, Atopic
A Study of an Eczema Spot Treatment in Providing Eczema Symptoms Relief and Lasting Itch Relief to Affected Skin Area
The purpose of this study is to evaluate the efficacy (assessed by clinical evaluation of eczema/atopic lesion severity [atopic dermatitis severity index {ADSI} scoring] and perceived benefits in itch [visual analog score {VAS}…
- Trial name
- NCT05226143
- Testing
- Gel Cream
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)
Mucopolysaccharidosis II
Long-term Follow-Up for RGX-121
RGX-121-5101 is an observational long-term follow-up study to the RGX-121-101 or RGX-121-1102 studies where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene…
- Trial name
- NCT04597385
- Testing
- Long-term Follow-Up
- Where
- 4 sites in United States, Brazil
- Volunteers
- 27 people
- Sponsor
- REGENXBIO Inc.
Obesity Associated With Defects in Leptin-melanocortin Pathway
Open-Label Extension Study of Setmelanotide
This is an open-label extension study designed to evaluate the long-term safety and tolerability of continued setmelanotide treatment in male and female patients ≥2 years of age who have completed or transitioned from a previous…
- Trial name
- NCT06596135
- Testing
- Setmelanotide
- Where
- 13 sites in United States
- Volunteers
- 28 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Fabry Disease
Study of the Safety and Efficacy of PRX-102 Compared to Agalsidase Beta on Renal Function
This was a randomized, double-blind, active control study of the enzyme replacement therapy (ERT) drug PRX-102 (pegunigalsidase alfa) in Fabry disease patients with impaired renal function.
- Trial name
- BALANCE (NCT02795676)
- Testing
- PRX-102 (pegunigalsidase alfa), agalsidase beta
- Where
- 29 sites in United States, Czechia and 10 more
- Volunteers
- 78 people
- Sponsor
- Protalix