Clinical trials: Genetic Conditions
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
Healthy Volunteers, Cryopyrin Associated Periodic Syndrome
Safety and Tolerability, Pharmacokinetic and Pharmacodynamic Study With IZD334
This is a first in human (FIH), single-centre, double -blind, randomised, cross-over, SAD followed by a MAD study of IZD334 conducted in healthy adult participants as well as an open-label cohort in adult patients with CAPS.
- Trial name
- NCT04086602
- Testing
- IZD334
- Where
- Australia
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Inflazome UK Ltd
Gout, Hyperuricemia
A Dose Finding Study to Assess Efficacy and Safety of IBI128 in Chinese Gout Subjects
IBI128 (Tigulixostat) is a novel non-purine selective inhibitor of xanthine oxidase (XO). The XO inhibitors lower uric acid concentrations in serum by inhibiting the production of uric acid.
- Trial name
- NCT06501534
- Testing
- Febuxostat, IBI128
- Where
- China
- Volunteers
- 84 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Obesity Associated With Defects in Leptin-melanocortin Pathway
Long Term Extension Trial of Setmelanotide
This was a long-term extension trial to study the safety and tolerability of continued setmelanotide treatment in participants who had completed a previous clinical trial on treatment with setmelanotide for obesity associated…
- Trial name
- NCT03651765
- Testing
- Setmelanotide
- Where
- 27 sites in United States, Canada and 6 more
- Volunteers
- 205 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Emphysema, Alpha 1-proteinase Inhibitor Deficiency
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v).
- Trial name
- NCT00670007
- Testing
- Alpha1- proteinase inhibitor [human]
- Where
- 21 sites in Australia, Canada and 9 more
- Volunteers
- 140 people
- Sponsor
- CSL Behring
Pulmonary Fibrosis, Thyroid Eye Disease
A Study to Evaluate the Safety, Preliminary Efficacy, and Pharmacokinetic Properties of LASN01 in Healthy Subjects and in Patients With Pulmonary Fibrosis or Thyroid Eye Disease
LASN01 is a novel, fully human antibody directed against the human IL-11 receptor that is being developed to address the fibro-inflammatory pathology of pulmonary fibrosis and TED.
- Trial name
- NCT05331300
- Testing
- LASN01
- Where
- 4 sites in Australia, Hong Kong
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Lassen Therapeutics 1 PTY LTD
Gout and Hyperuricemia
Clinical Trial of BR2251 Tablets for Patients With Primary Gout and Hyperuricemia
This study is a randomized, double-blind, non-befloxacin-controlled, multicenter, phase II clinical trial, evaluating the efficacy, safety, and pharmacokinetic characteristics of BR2251 tablets when administered multiple times in…
- Trial name
- BR2251-201 (NCT07498647)
- Testing
- BR2251, Febuxostat
- Volunteers
- 160 people
- Sponsor
- BioRay Pharmaceutical Co., Ltd.
Fed
Bioavailability Study of Desloratadine 5 mg Tablet of Dr. Reddy's Under Fed Conditions
The purpose of this study is to compare the rate and extent of absorption of Dr. Reddy's Desloratadine 5 mg tablet to that of Clarinex® 5 mg tablet in healthy subjects under fed conditions.
- Trial name
- NCT01314352
- Testing
- Desloratadine
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Pediatric Growth Hormone Deficiency
Versartis Long-Term Safety Study of Somavaratan
Protocol 13VR3 is is a multi-center, open-label study assessing long-term somavaratan administration. Patients will be monitored for safety throughout their participation in the study.
- Trial name
- VISTA (NCT02068521)
- Testing
- somavaratan
- Volunteers
- 385 people
- Sponsor
- Versartis Inc.
Tuberous Sclerosis Complex
Roll-over Study to Collect and Assess Long-term Safety of Everolimus in Patients With TSC and Refractory Seizures Who Have Completed the EXIST-3 Study [CRAD001M2304] and Who Are Benefitting From Continued Treatment
The purpose of this study is to evaluate the long-term safety in patients with TSC and refractory seizures who are currently receiving everolimus treatment in the Novartis-sponsored EXIST-3 study and who are determined to be…
- Trial name
- NCT02962414
- Testing
- everolimus
- Where
- 67 sites in United States, Australia and 16 more
- Volunteers
- 206 people
- Sponsor
- Novartis Pharmaceuticals
Chronic Myeloid Leukemia (CML) Philadelphia Chromosome Positive
A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP
The study is designed to compare the efficacy of asciminib 80 mg QD versus Investigator selected Tyrosine Kinase Inhibitor (TKI) for the treatment of newly diagnosed, previously untreated patients with Ph+ CML-CP.
- Trial name
- NCT04971226
- Testing
- Imatinib, Nilotinib, Bosutinib
- Where
- 110 sites in United States, Australia and 27 more
- Volunteers
- 405 people
- Sponsor
- Novartis Pharmaceuticals
Cardiac Diseases, Muscular Weakness
Biomarker for Pompe Disease (BioPompe)
Development of a new MS-based biomarker for the early and sensitive diagnosis of Pompe disease from blood (plasma)
- Conditions
- Cardiac Diseases, Muscular Weakness, Hepato-splenomegaly, Lung Disease
- Trial name
- BioPompe (NCT01457443)
- Where
- 4 sites in Germany, India and 1 more
- Sponsor
- CENTOGENE GmbH Rostock
Genetic Obesity, Obesity
Setmelanotide Phase 2 Treatment Trial in Participants With Rare Genetic Disorders of Obesity
The purpose of the study was to determine the effect of setmelanotide (RM-493) on weight, hunger assessments, and other factors in participants with rare genetic disorders of obesity.
- Conditions
- Genetic Obesity, Obesity, Obesity Due to Melanocortin 4 Receptor Deficiency
- Trial name
- NCT03013543
- Testing
- Setmelanotide
- Where
- 58 sites in United States, Canada and 7 more
- Volunteers
- 213 people
- Sponsor
- Rhythm Pharmaceuticals, Inc.
Breast Neoplasms, Prostate Neoplasms
PF-06952229 Treatment in Adult Patients With Advanced Solid Tumors
A Phase 1 dose escalation and expansion study evaluating safety, tolerability and pharmacokinetics of PF-06952229 in adult patients with advanced solid tumors.
- Conditions
- Breast Neoplasms, Prostate Neoplasms, Neoplasms, Squamous Cell, Melanoma
- Trial name
- NCT03685591
- Testing
- PF-06952229, Enzalutamide
- Where
- 15 sites in United States
- Volunteers
- 49 people
- Sponsor
- Pfizer
Bioequivalence
Bioequivalence Study of Metformin 750 mg Tablets XR Versus Glucophage® Long 750 mg Tablets XR In Normal Healthy Subjects Under Fasting and Fed Conditions
Bioequivalence Study of 2 formulation of metformin (Metformin GEROPHARM vers. Glucophage® Long Merck )
- Trial name
- NCT03452306
- Testing
- Metformine, Glucophage® Long, Metformin
- Where
- Russia
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Geropharm
Systemic Juvenile Idiopathic Arthritis, Hereditary Periodic Fevers
A Study of Canakinumab in Patients With Systemic Juvenile Idiopathic Arthritis or Hereditary Periodic Fevers Who Participated in the CACZ885G2301E1, CACZ885G2306 or CACZ885N2301 Studies
The objective of this extension protocol is to collect safety data (serious and non-serious adverse events) and to provide continuous canakinumab to patients in France who completed study CACZ885G2301E1(NCT00891046), CACZ885G2306…
- Trial name
- NCT02334748
- Testing
- canakinumab
- Where
- 3 sites in France
- Volunteers
- 31 people
- Sponsor
- Novartis Pharmaceuticals
Gout
Efficacy and Safety of Oral Febuxostat in Participants With Gout
The purpose of this study is to compare the efficacy and safety of febuxostat, once Daily (QD), to allopurinol in subjects with hyperuricemia and gout.
- Trial name
- CONFIRMS (NCT00430248)
- Testing
- Febuxostat, Allopurinol
- Where
- 258 sites in United States
- Volunteers
- 2,269 people
- Sponsor
- Takeda
Migraine
Biomarker and Genetic Predictors of Erenumab Treatment Response
To explore the relationship between clinical response to erenumab and genetic biomarkers
- Trial name
- INTERROGATE (NCT04265755)
- Testing
- Erenumab
- Where
- 2 sites in Denmark, Iceland
- Volunteers
- 1,406 people
- Sponsor
- Amgen
Mucopolysaccharidosis II
Mucopolysaccharidosis Type II Natural History
Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome, is caused by a deficiency of iduronate-2-sulfatase (IDS) leading to an accumulation of glycosaminoglycans (GAGs) in tissues of MPS II patients, resulting in…
- Trial name
- NCT03529786
- Where
- 3 sites in United States, Brazil and 1 more
- Volunteers
- 36 people
- Sponsor
- REGENXBIO Inc.
Alzheimer Disease, Alzheimer Dementia
Alzheimer's Autism and Cognitive Impairment Stem Cell Treatment Study
The purpose of the study is to evaluate the use of autologous Bone Marrow Derived Stem Cells (BMSC) as a means to improve cognitive impairment as occurs in Alzheimer's Disease and other dementias and to improve behavior and…
- Conditions
- Alzheimer Disease, Alzheimer Dementia, Vascular Dementia, Lewy Body Disease
- Trial name
- ACIST (NCT03724136)
- Testing
- Intravenous Bone Marrow Stem Cell (BMSC) Fraction, Intranasal Topical Bone Marrow Stem Cell (BMSC) Fraction, Near Infrared Light
- Where
- 3 sites in United States, United Arab Emirates
- Volunteers
- 100 people
- Sponsor
- MD Stem Cells