Clinical trials: Genetic Conditions
Cystic Fibrosis, Chronic Obstructive Pulmonary Disease
A Phase 1 Study to Characterize the Effect of GS-5737 Enhancement of Mucociliary Clearance (MCC) in Healthy Subjects
This is a randomized, double-blind, cross-over, vehicle-controlled study to determine whether GS-5737 in 2.8% saline accelerates mucociliary clearance (MCC) in healthy subjects, compared to vehicle of 2.8% saline alone.
- Trial name
- NCT01793649
- Testing
- GS-5737
- Where
- United States
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Gilead Sciences
Usher Syndromes
Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome
This study will examine the safety and efficacy of NPI-001 Tablets as compared to placebo for 24 months in subjects with vision loss due to RP associated with Usher syndrome.
- Trial name
- SLO RP (NCT04355689)
- Testing
- NPI-001
- Where
- 4 sites in Australia
- Volunteers
- 49 people
- Sponsor
- Nacuity Pharmaceuticals, Inc.
Prostatic Neoplasm
A Phase 1a/1b Study to Evaluate the Safety of EZN-4176, in Adult Patients With Castration-Resistant Prostate Cancer
This study will evaluate an experimental drug called EZN-4176 to determine the anticancer effects when it is given to patients with an advanced form of prostate cancer called castration-resistant prostate cancer (CRPC).
- Trial name
- NCT01337518
- Testing
- EZN-4176
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 150 people
- Sponsor
- Enzon Pharmaceuticals, Inc.
Recurrent Basal Cell Carcinoma
A Study of Patidegib Topical Gel, 2%, for the Reduction of Disease Burden of Persistently Developing Basal Cell Carcinomas in Patients With Non-Gorlin High Frequency BCC
This is a multicenter, randomized, double-blind, stratified, vehicle-controlled study of the efficacy and safety of Patidegib Topical Gel, 2%, applied topically twice daily to the face of adult participants with non-Gorlin HF-BCC…
- Trial name
- NCT04155190
- Testing
- Patidegib Topical Gel, 2%, Patidegib Topical Gel, Vehicle
- Where
- 14 sites in United States
- Volunteers
- 50 people
- Sponsor
- Sol-Gel Technologies, Ltd.
To Determine Bioequivalence Under Fed Conditions
Bioequivalence Study of Doxycycline Monohydrate Tablets Under Fed Conditions
The purpose of this study is to compare the single-dose bioequivalence of Par and Oclassen doxycycline monohydrate 100 mg.
- Trial name
- NCT01380496
- Testing
- doxycycline monohydrate
- Where
- Canada
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Par Pharmaceutical, Inc.
Diarrhea, Atopic Dermatitis
Cesarean Section Study
Effect, tolerance and safety of a supplementation with a probiotic on the risk of gastrointestinal infections, on growth, and on gut microbiota in healthy newborn term infants born by Cesarean section.
- Trial name
- NCT01992497
- Testing
- Probiotic
- Where
- Germany
- Volunteers
- 721 people, healthy volunteers welcome
- Sponsor
- Société des Produits Nestlé (SPN)
Bowel Incontinence
A Study to Evaluate the Safety, Tolerability and Effectiveness of the Renew Insert in Treating Accidental Bowel Leakage
The purpose of this study is to determine whether the use of the Renew Insert is effective, tolerable and safe in the treatment of accidental bowel leakage due to bowel incontinence.
- Trial name
- REST (NCT01475474)
- Testing
- Renew Insert
- Where
- 3 sites in United States
- Volunteers
- 97 people
- Sponsor
- Renew Medical
Prader-Willi Syndrome, Obesity
A Study of Setmelanotide in Patients With Prader-Willi Syndrome
This is a Phase 2, open-label study designed to assess the safety and efficacy of setmelanotide in patients with obesity due to Prader-Willi Syndrome (PWS) who are 6 to 65 years of age.
- Conditions
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Trial name
- NCT06772597
- Testing
- Setmelanotide
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Xeroderma Pigmentosum
A Study to Evaluate the Safety and Efficacy of Afamelanotide in Patients With Xeroderma Pigmentosum C and V
The CUV152 study will evaluate the safety of afamelanotide in XP-C and XP-V patients, as well as the drug's ability to assist reparative processes following ultraviolet (UV) provoked DNA damage of the skin.
- Trial name
- NCT05370235
- Testing
- Afamelanotide
- Where
- 2 sites in Belgium, Spain
- Volunteers
- 6 people
- Sponsor
- Clinuvel Europe Limited
Huntington's Chorea
Efficacy and Safety of SOM3355 in Huntington's Disease Chorea
Phase IIa study to evaluate the efficacy and safety of SOM3355 in chorea movements associated with Huntington's disease
- Trial name
- NCT03575676
- Testing
- SOM3355 100mg BID, SOM3355 200mg BID
- Where
- 4 sites in Spain
- Volunteers
- 32 people
- Sponsor
- SOM Innovation Biotech SA
Tuberous Sclerosis Complex
Basimglurant (NOE-101) in Children, Adolescents, and Young Adults With TSC
The study intends to show that basimglurant (NOE-101) provides effective seizure control in children, adolescents and young adults with Tuberous Sclerosis Complex (TSC).
- Trial name
- NCT05059327
- Where
- 27 sites in United States, India and 6 more
- Volunteers
- 61 people
- Sponsor
- Noema Pharma AG
Growth Hormone Deficiency, Turner Syndrome
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
The purpose of this study is to evaluate the long-term safety and effectiveness of growth hormone (Eutropin Inj./Eutropin plus Inj.) treatment with GHD (Growth Hormone Deficiency), TS (Turner Syndrome),CRF (Chronic Renal…
- Conditions
- Growth Hormone Deficiency, Turner Syndrome, Chronic Renal Failure, Small for Gestational Age
- Trial name
- LGS (NCT01604395)
- Where
- South Korea
- Volunteers
- 6,000 people
- Sponsor
- LG Chem
Tuberous Sclerosis Complex Associated Neuropsychiatric Disease
A Study to Investigate Behavioral and Other Co-Occurring Outcomes With Epidiolex as Add-On Therapy in Participants Aged 1 to 65 Years of Age With Tuberous Sclerosis Complex
The purpose of this study is to investigate behavioral and other co-occurring outcomes with EPID(I/Y)OLEX as an add-on therapy in participants aged 1 to 65 years with tuberous sclerosis complex (TSC) who experience seizures.
- Trial name
- EpiCom (NCT05864846)
- Testing
- Cannabidiol Oral Solution [Epidiolex]
- Where
- 19 sites in United States, Canada and 2 more
- Volunteers
- 79 people
- Sponsor
- Jazz Pharmaceuticals
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Hydrops Fetalis, Fetal Growth Retardation
Amniotic Fluid Tandem Mass Spectrometry for Pregnancies Complicated by NIH and Severe Symmetrical IUGR
The objective of this pilot study is to prospectively evaluate amniotic fluid of pregnancies complicated by non-immune hydrops and severe symmetrical intrauterine growth restriction by tandem mass spectrometry for inborn errors…
- Trial name
- TandemMS (NCT00143039)
- Testing
- Tandem MS test for inborn errors of metabolism
- Where
- 11 sites in United States
- Volunteers
- 48 people
- Sponsor
- Obstetrix Medical Group
Alpha-1 Antitrypsin Deficiency, AATD
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD)…
- Trial name
- rhAAT-Fc (NCT03815396)
- Testing
- INBRX-101/rhAAT-Fc
- Where
- 10 sites in United States, New Zealand and 1 more
- Volunteers
- 31 people
- Sponsor
- Inhibrx Biosciences, Inc
Chronic Myeloid Leukemia
Safety and Efficacy of Nilotinib in Newly Diagnosed Chronic Myeloid Leukemia Patients
This study will further investigate the safety and efficacy of nilotinib in newly diagnosed chronic myeloid leukemia patients in the chronic phase
- Trial name
- ENESTxtnd (NCT01254188)
- Testing
- Nilotinib
- Where
- 100 sites in Algeria, Argentina and 17 more
- Volunteers
- 421 people
- Sponsor
- Novartis Pharmaceuticals
Alpha 1-Antitrypsin Deficiency
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1…
- Trial name
- ChAMP (NCT00295061)
- Testing
- Alpha-1 MP, alpha-1 proteinase inhibitor (human)
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Atopic Dermatitis
CLINICAL EFFICACY OF EMOLLIENTS IN ATOPIC DERMATITIS PATIENTS
his was a monocentric, intra-individual study, that was performed in at least 45 valid cases (50% children ≥3 YO; 50% adults). Study duration was 168 days with five (5) visits (D0, D14, D28, D84 and D168) to the research center.
- Trial name
- NCT05783453
- Testing
- Cosmetic study
- Where
- Brazil
- Volunteers
- 63 people
- Sponsor
- Cosmetique Active International
Arthritis, Osteoarthritis
Tanezumab In Osteoarthritis Of The Knee (2)
The purpose of this study is to test the efficacy and safety of 2 doses of tanezumab compared with naproxen and placebo in patients with osteoarthritis.
- Trial name
- NCT00830063
- Testing
- tanezumab 10 mg, tanezumab 5 mg, naproxen
- Where
- 94 sites in United States
- Volunteers
- 832 people
- Sponsor
- Pfizer