Clinical trials: Genetic Conditions
Prader-Willi Syndrome
Phase 2 Trial to Evaluate Safety and Efficacy of Setmelanotide (RM-493) in Obese Participants With Prader-Willi Syndrome
The purpose of this study was to evaluate the effects of a once daily subcutaneous injectable formulation of setmelanotide in obese participants with Prader-Willi syndrome on tolerability, weight loss, and hyperphagia-related…
- Trial name
- NCT02311673
- Testing
- Setmelanotide
- Where
- 5 sites in United States
- Volunteers
- 40 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Myotubular Myopathy
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Myotubular Myopathy (MTM)
This is a prospective, non-interventional, longitudinal study of the natural history and function of approximately 60 patients with MTM from the United States, Canada and Europe.
- Trial name
- MTM (NCT02057705)
- Where
- 13 sites in United States, Belgium and 5 more
- Volunteers
- 48 people
- Sponsor
- Valerion Therapeutics, LLC
To Determine the Bioequivalence Study Under Fed
Bioavailability Study of Metronidazole Capsules Under Fed Conditions
The purpose of this study is to compare the single-dose Bioavailability of Kali and G.D. Searle, Inc.
- Trial name
- NCT01380522
- Testing
- Metronidazole, Flagyl
- Where
- Canada
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Primary Gout, Hyperuricemia
Phase II/III Study of AR882 Capsules Compared to Febuxostat Tablets in Patients with Primary Gout and Hyperuricemia
The goal of this clinical trial is to evaluate the efficacy and safety of AR882 Capsules in patients with primary gout and hyperuricemia.
- Trial name
- NCT06603142
- Testing
- Febuxostat 20MG Tablets, AR882 25MG, AR882 12.5MG
- Where
- China
- Volunteers
- 636 people
- Sponsor
- Guangzhou Ruianbo Pharmaceutical Technology Co., Ltd
Atopic Dermatitis
Study of TS-022 in Adult Patients With Atopic Dermatitis With Pruritus (POC)
The purpose and (primary) objectives of this study are to evaluate the efficacy, safety, and tolerability of TS-022 in adults with atopic dermatitis who have moderate to very severe pruritus (itching), following a 28-day regimen…
- Trial name
- NCT00914186
- Testing
- TS022, Vehicle
- Where
- 15 sites in United States
- Volunteers
- 122 people
- Sponsor
- Taisho Pharmaceutical R&D Inc.
Farber Disease, Farber's Disease
Farber Disease Natural History Study
The primary objective of this study is to establish the natural history of Farber disease (acid ceramidase deficiency) through the collection and analysis of retrospective and prospective data on patients diagnosed with Farber…
- Conditions
- Farber Disease, Farber's Disease, Farber Lipogranulomatosis, Acid Ceramidase Deficiency
- Trial name
- NCT03233841
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 45 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Dermatitis, Atopic, Asthma
A Study to Evaluate Use of Induced Skin Blisters in Adult Participants With Atopic Dermatitis, Allergic Asthma and Atopic Healthy Participants
The purpose of this study is to characterize the cell (the basic building block of all living things) populations and inflammatory (pain and swelling) mediator responses in suction skin blister fluid after allergic skin reaction…
- Conditions
- Dermatitis, Atopic, Asthma, Healthy
- Trial name
- NCT02343783
- Testing
- Allergic Skin Reaction (ASR) Testing and Skin Blister Induction
- Where
- 7 sites in Belgium, Germany
- Volunteers
- 46 people, healthy volunteers welcome
- Sponsor
- Janssen Research & Development, LLC
Lafora Disease
Natural History and Functional Status Study of Patients With Lafora Disease
A natural history and functional status study to characterize the clinical disease course in Lafora disease patients using standardized, quantitative evaluations and to identify useful biomarkers and clinical outcome measures for…
- Trial name
- NCT03876522
- Where
- 4 sites in United States, Italy and 1 more
- Volunteers
- 33 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Atopic Dermatitis
A Study of Lebrikizumab in Adults and Adolescents With Face and Neck Atopic Dermatitis
The main aim of the study is to evaluate the efficacy and safety of lebrikizumab treatment for up to 32 weeks in participants with moderate-to-severe face and neck atopic dermatitis (FNAD) who also have moderate-to-severe AD…
- Trial name
- ADface (NCT07873801)
- Testing
- Lebrikizumab
- Volunteers
- 230 people
- Sponsor
- Almirall, S.A.
Pain, Chronic Pain
Observational Study Linking Genetic Variants With Clinical Outcomes in Pain Management
The purpose of this study is to (a) evaluate the treatment approaches and changes in treatment regimens utilized by clinicians when genetic testing is performed in the clinic; and (b) create a patient data registry to identify…
- Trial name
- TROJAN (NCT02480075)
- Testing
- Observational
- Where
- United States
- Volunteers
- 2,000 people
- Sponsor
- Proove Bioscience, Inc.
Gout
A Study of Dotinurad Versus Allopurinol in Participants With Gout
The primary objective of this study is to evaluate the efficacy of dotinurad in lowering serum uric acid (sUA) at Week 24 compared with allopurinol in adult participants with hyperuricemia associated with gout.
- Trial name
- NCT07089875
- Testing
- Allopurinol, Dotinurad
- Where
- 119 sites in United States, Poland and 1 more
- Volunteers
- 500 people
- Sponsor
- Crystalys Therapeutics
Dermatitis, Atopic
A Study to Learn About the Study Medicine Abrocitinib in People With Severe Atopic Dermatitis Who Have Switched From Other Therapies
The purpose of this observational study is to learn about the effects of the study medicine (called abrocitinib) for the treatment of severe atopic dermatitis in people that were moved from previous novel therapies, known as…
- Trial name
- NCT07887867
- Testing
- abrocitinib
- Volunteers
- 120 people
- Sponsor
- Pfizer
Hemophilia A, Hemophilia B
Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively
A study to learn about the long-term safety and efficacy of giroctocogene fitelparvovec or fidanacogene elaparvovec in patients with hemophilia A or hemophilia B respectively, who have received treatment through prior…
- Trial name
- NCT05568719
- Testing
- Testing of hepatic AAV Vector integration
- Where
- 39 sites in United States, Australia and 11 more
- Volunteers
- 173 people
- Sponsor
- Pfizer
Orphan Cholestatic Liver Diseases, Progressive Familial Intrahepatic Cholestasis
An Open Label, Single-dose, Single Period ADME Study of A4250 in Healthy Subjects
The primary objectives of the study are to assess the mass balance recovery after a single dose of carbon-14 [14C]-A4250 as a capsule and to provide plasma, urine and faecal samples for metabolite profiling and structural…
- Conditions
- Orphan Cholestatic Liver Diseases, Progressive Familial Intrahepatic Cholestasis, Alagille Syndrome, Primary Biliary Cirrhosis
- Trial name
- NCT03082937
- Testing
- 3 mg [14C]-A4250 capsule
- Where
- United Kingdom
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Albireo
Splenomegaly, Hepatomegaly
Biomarker for Gaucher Disease: BioGaucher (BioGaucher)
Development of a new mass spectrometry-based biomarker for the early and sensitive diagnosis of Gaucher Disease from blood (plasma)
- Trial name
- BioGaucher (NCT01331642)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Androgenetic Alopecia
Association of Polymorphisms in the Androgen Receptor Gene and Finasteride Response in Women With Androgenetic Alopecia
Previous studies of finasteride treatment in women with hair loss have failed to show positive results, yet, some women have responded anecdotally.
- Trial name
- NCT01052870
- Testing
- Finasteride
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- HairDx, LLC
Hyperuricemia, Gout
Phase IIa Study of ALLN-346 (Engineered Urate Oxidase) in Subjects With Hyperuricemia, Gout and Chronic Kidney Disease
The purpose of this Phase IIa study is to evaluate the safety, tolerability and pharmacodynamics of ALLN-346 in subjects with hyperuricemia and gout, and with mild to moderate chronic kidney disease.
- Conditions
- Hyperuricemia, Gout, Chronic Kidney Diseases
- Trial name
- NCT04987294
- Testing
- ALLN-346
- Where
- 17 sites in United States
- Volunteers
- 19 people
- Sponsor
- Allena Pharmaceuticals
Ototoxicity
SPI-1005 for Prevention and Treatment of Tobramycin Induced Ototoxicity
The primary objective of this study is to determine the safety and efficacy of SPI-1005 treatment in CF patients with active pulmonary exacerbation that are receiving an IV course of tobramycin, determined by comparing hearing…
- Trial name
- NCT02819856
- Testing
- SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sound Pharmaceuticals, Incorporated
Relapsed and Refractory Acute Myeloid Leukemia (RR-AML)
HPB-092 for the Treatment of Relapsed and Refractory Acute Myeloid Leukemia
HPB-092 effectively inhibits Fms-like tyrosine kinase 3 (FLT3) mutants with comparable or superior potency to approved FLT3 inhibitors and demonstrates improved selectivity, potentially reducing toxicity.
- Trial name
- NCT07137637
- Testing
- HPB-092 tablet
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Hangzhou Polymed Biopharmaceuticals, Inc.
Breast Cancer
Genetic and Non-Genetic Breast Cancer Risk Prediction Evaluation in Indonesian Samples
Breast cancer is the most common cancer and cause of cancer- related deaths among women, accounting for 1.67 million (25.2%) new cases and 521,907 (14.7%) deaths worldwide.
- Trial name
- NCT05570266
- Testing
- Breast Cancer Risk Prediction Software
- Where
- Indonesia
- Volunteers
- 322 people, healthy volunteers welcome
- Sponsor
- Nalagenetics Pte Ltd