Clinical trials: Genetic Association Studies
Functional Gastrointestinal Disorders
Effect of Controlled Diet Combined With a Fermented Milk Product on Gas-related Symptoms
To select among different dietary and clinical conditions the most appropriate to assess the impact of a fermented milk product on gas-related symptoms
- Trial name
- FLATBACK (NCT02936713)
- Testing
- Fermented milk product with probiotics (FMPP)
- Where
- Spain
- Volunteers
- 126 people, healthy volunteers welcome
- Sponsor
- Danone Global Research & Innovation Center
Diabetes Mellitus
Observational Study to Assess Glycosylated Hemoglobin Changes After 6 Months of Treatment With Pioglitazone.
The purpose of this study is to asses changes in glycosylated hemoglobin, fasting blood lipids and genetic polymorphism's in peroxisomal proliferator activated receptors--gamma receptor after 6 months of pioglitazone, once daily…
- Trial name
- NCT00449553
- Testing
- Pioglitazone and sulphonylurea, Pioglitazone and metformin
- Where
- 4 sites in Denmark, Iceland and 2 more
- Volunteers
- 326 people
- Sponsor
- Takeda
Pain, Chronic Pain
Observational Study of the Impact of Genetic Testing on Healthcare Decisions and Care in Interventional Pain Management
The purpose of this study is to evaluate the impact of genetic testing on healthcare decisions and patient outcomes in interventional pain management clinical care.
- Conditions
- Pain, Chronic Pain, Back Pain, Nociceptive Pain
- Trial name
- MOSAIC (NCT02485795)
- Testing
- Observational
- Where
- 31 sites in United States
- Volunteers
- 50,000 people
- Sponsor
- Proove Bioscience, Inc.
Stargardt Disease, Stargardt Macular Degeneration
Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)
The purpose of this open-label, multicenter study is to determine the long-term safety, pharmacokinetics and effects of ALK-001 (C20-D3-retinyl acetate) on the progression of Stargardt disease.
- Conditions
- Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related)
- Trial name
- NCT04239625
- Testing
- ALK-001
- Where
- 8 sites in United States
- Volunteers
- 200 people
- Sponsor
- Alkeus Pharmaceuticals, Inc.
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets, PAS-004 Capsule
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Stargardt Disease, Age-related Macular Degeneration
Phase 1 Safety Study of ALK-001 in Healthy Volunteers
This protocol is a phase 1 clinical study to assess the safety and pharmacokinetics of ALK-001 in healthy volunteers. Please contact trials@alkeus.com for any questions.
- Conditions
- Stargardt Disease, Age-related Macular Degeneration, Other Retinal Dystrophies
- Trial name
- NCT02230228
- Testing
- ALK-001 (No generic name)
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Alkeus Pharmaceuticals, Inc.
Growth Hormone (GH) Deficiency, Short Stature Homeobox Containing Gene (SHOX) Deficiency
Observational Study of Somatropin Treatment in Children
GeNeSIS is an open-label, multinational, multicenter, observational study to evaluate the safety and effectiveness of Humatrope treatment.
- Conditions
- Growth Hormone (GH) Deficiency, Short Stature Homeobox Containing Gene (SHOX) Deficiency, SHOX Deficiency-related Disorder, Non-GH-deficient Growth Disorders
- Trial name
- GeNeSIS (NCT01088412)
- Testing
- Somatropin (recombinant deoxyribonucleic acid [rDNA] origin)
- Volunteers
- 22,845 people
- Sponsor
- Eli Lilly and Company
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy
Efficacy and Safety of AG10 in Subjects with Transthyretin Amyloid Polyneurophathy
See updated study design under NCT04882735. Phase 3 efficacy and safety of AG10 compared with placebo in subjects with symptomatic Transthyretin Amyloid Polyneuropathy (ATTR-PN)
- Trial name
- ATTRibute-PN (NCT04418024)
- Testing
- AG10
- Where
- United States
- Sponsor
- Eidos Therapeutics, a BridgeBio company
PIK3CA-Related Overgrowth Spectrum
A Study of the Epidemiology and Hospital Management of Patients With PROS in France
The purpose of this study was to estimate the incidence and prevalence of a group of genetic disorders known as PIK3CA-Related Overgrowth Spectrum (PROS) in France.
- Trial name
- EpiPROS (NCT07222423)
- Where
- United States
- Volunteers
- 3,605 people
- Sponsor
- Novartis Pharmaceuticals
Focal Segmental Glomerulosclerosis
A Study to Find Out if BI 764198 Helps Adults and Adolescents With a Kidney Condition Called Focal Segmental Glomerulosclerosis (FSGS)
PODOMOUNT-pFSGS This study is open to adults and adolescents with a kidney condition called focal segmental glomerulosclerosis (FSGS).
- Trial name
- NCT07220083
- Testing
- BI 764198
- Where
- 316 sites in United States, Argentina and 38 more
- Volunteers
- 286 people
- Sponsor
- Boehringer Ingelheim
Atopic Dermatitis, Eczema
A Study to Assess the Impact of Lebrikizumab on Health-Related Well-Being and Control of Skin Manifestations in Participants With Moderate-to-Severe Atopic Dermatitis
The main purpose of this study is to evaluate the impact of lebrikizumab treatment on the overall well-being of adult participants with moderate-to-severe AD in real-world clinical practice settings across Europe, as measured…
- Trial name
- ADTrust (NCT06815380)
- Testing
- No Intervention
- Where
- Denmark
- Volunteers
- 1,000 people
- Sponsor
- Almirall, S.A.
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT01960348
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- 52 sites in United States, Argentina and 19 more
- Volunteers
- 225 people
- Sponsor
- Alnylam Pharmaceuticals
Immune System and Related Disorders
Creation of a Prospective Cohort of Healthy and Sick Subjects and of a Collection of Associated Biological Resources, for the Study of the Immune System and of Its Genetic and Environmental Determinants.
CoSImmGEn is a protocol set up to respond to the current lack of healthy and sick population cohorts. Biological resources from these cohorts allow researchers to study the immune system and its genetic and environmental…
- Trial name
- CoSImmGEn (NCT03925272)
- Testing
- Collection of samples (blood, stool, etc.), Genetic determinants analysis, Sample obtained after surgery performed in the context of care
- Where
- 4 sites in France
- Volunteers
- 2,200 people, healthy volunteers welcome
- Sponsor
- Institut Pasteur
All Diagnosed Health Conditions, ADD/ADHD
At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions
We are the missing link in clinical trials, connecting patients and researchers seamlessly and conveniently using a mobile health platform to advance medical research.
- Conditions
- All Diagnosed Health Conditions, ADD/ADHD, Alopecia Areata, Ankylosing Spondylitis
- Trial name
- NCT01931644
- Where
- United States
- Volunteers
- 17,667 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Stargardt Disease, Cone Rod Dystrophy
Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and…
- Conditions
- Stargardt Disease, Cone Rod Dystrophy, Juvenile Macular Degeneration, Stargardt Disease 1
- Trial name
- NCT06467344
- Testing
- ACDN-01
- Where
- 11 sites in United States
- Volunteers
- 15 people
- Sponsor
- Ascidian Therapeutics, Inc
Palmar-Plantar Erythrodysesthesia, Breast Cancer
Study of 0.1% Uracil Topical Cream (UTC) for the Prevention of Hand-Foot Syndrome
The purpose of this research study is to see if combining uracil cream (UTC) with capecitabine (Xeloda) can prevent Hand-Foot Syndrome.
- Trial name
- NCT01100463
- Testing
- Cream, 0.1% Uracil Cream
- Where
- 6 sites in United States
- Volunteers
- 18 people
- Sponsor
- Nanometics (d.b.a. PHD Biosciences)
Solid Tumor
Tuvusertib (M1774) Human Mass Balance and Absolute Bioavailability Study (DDRIVER Solid Tumors 303)
This is a single sequence 2-period open label study in participants with advanced solid tumors. The purpose of Period 1 of this study is to assess the mass balance to determine drug-related entities present in circulation and…
- Trial name
- NCT06308263
- Testing
- Tuvusertib [14C]Tuvusertib microtracer, Tuvusertib, Tuvusertib + [14C]Tuvusertib microdose bolus injection
- Where
- Hungary
- Volunteers
- 12 people
- Sponsor
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany
Variegate Porphyria
Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Variegate Porphyria (VP)
The primary objective is to evaluate the impact of afamelanotide on the severity of skin disease in patients with Variegate Porphyria (VP).
- Trial name
- NCT05854784
- Testing
- Afamelanotide 16 MG
- Where
- Netherlands
- Volunteers
- 6 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Exocrine Pancreatic Insufficiency, Cystic Fibrosis
SOLUTION: Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in Patients With Cystic Fibrosis
Liprotamase powder is a non-porcine, soluble and stable mixture of three digestive enzymes including lipase, protease, and amylase.
- Trial name
- NCT02279498
- Testing
- Liprotamase, porcine (pig) PERT
- Where
- 54 sites in United States, Canada and 5 more
- Volunteers
- 128 people
- Sponsor
- Anthera Pharmaceuticals