Clinical trials: Safety and Tolerability
Mucopolysaccharidosis Type II (MPS II)
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)
RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system.
- Trial name
- NCT04571970
- Testing
- RGX-121
- Where
- 2 sites in United States, Canada
- Volunteers
- 6 people
- Sponsor
- REGENXBIO Inc.
Autoimmune Encephalitis (AE)
A Study to Learn About How Well the Medicine Efgartigimod Works to Treat Autoimmune Encephalitis In Children 12 Years or Older and Adults
The POLARIS study is designed to evaluate how well efgartigimod PH20 SC may work (called "efficacy") and how safe it is for people diagnosed with Autoimmune Encephalitis (AIE).
- Trial name
- Polaris (NCT07583641)
- Testing
- Efgartigimod PH20 (ARGX-113) SC
- Where
- 2 sites in United States
- Volunteers
- 170 people
- Sponsor
- argenx
Human Immunodeficiency Virus 1
A Safety Study to Evaluate the Antiviral Activity of Darunavir in Combination With Ritonavir in HIV 1 Infected Children
The purpose of this study is to evaluate the pharmacokinetics (what the body does to the medication), safety and antiviral activity to support dose recommendations by body weight of darunavir with low-dose ritonavir (DRV/rtv), in…
- Trial name
- NCT00919854
- Testing
- Darunavir, Ritonavir
- Where
- 8 sites in Argentina, Brazil and 3 more
- Volunteers
- 27 people
- Sponsor
- Tibotec Pharmaceuticals, Ireland
Healthy Participants
A Study to Assess the Safety, Tolerability, and Pharmacokinetics of a Single Ascending Dose of AZD0292 in Healthy Japanese Participants
The purpose of the study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single ascending doses of AZD0292 administered via intravenous (IV) infusion in healthy Japanese participants.
- Trial name
- NCT07222254
- Testing
- AZD0292
- Where
- Japan
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- AstraZeneca
HIV, AIDS
TMC278-TiDP6-C130: Pharmacokinetics, Safety and Tolerability of TMC278 in Subjects With Mildly or Moderately Impaired Hepatic Function.
The purpose of this study is to investigate the pharmacokinetics (how the drug is absorbed into the bloodstream, distributed in the body and eliminated from the body) after a single dose and after repeated administration of…
- Conditions
- HIV, AIDS, Hepatic Impairment
- Trial name
- NCT00736905
- Testing
- TMC278
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Tibotec Pharmaceuticals, Ireland
Varicella Zoster Virus Infection, Herpes Zoster
Clinical Trial to Evaluate EuHZV in Healthy Adults Aged 50 to 69 Years
Phase 1 clinical trial to evaluate the safety of HZV-1 and HZV-2 vaccines in healthy adults aged 50 to 69 years who have voluntarily given written consent to participate in this study.
- Trial name
- NCT06409494
- Testing
- EuHZV, Shingrix
- Where
- South Korea
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- EuBiologics Co.,Ltd
Solid Tumor
Study of PYX-106 in Solid Tumors
The primary objective of this study is to determine the recommended dose(s) of PYX-106 in participants with relapsed/refractory solid tumors.
- Trial name
- NCT05718557
- Testing
- PYX-106
- Where
- 20 sites in United States, Belgium and 1 more
- Volunteers
- 47 people
- Sponsor
- Pyxis Oncology, Inc
Fabry Disease
A Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) in participants with Fabry disease.
- Trial name
- NCT00214500
- Testing
- migalastat HCl
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Amicus Therapeutics
Seasonal Allergic Rhinitis, Asthma
Study of Grass-SPIRE in Subjects With Grass Allergies and Asthma
The purpose of this study is to determine whether it is safe to administer Grass-SPIRE to subjects suffering from both grass allergy and asthma
- Trial name
- NCT02161107
- Testing
- Grass-SPIRE
- Where
- 5 sites in Canada
- Volunteers
- 54 people
- Sponsor
- Circassia Limited
Stargardt Disease, Stargardt Macular Dystrophy
A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye Conditions
Macular dystrophies are a group of inherited eye conditions that affect the macula. The macula is in the center of the retina, the light sensitive part at the back of the eye.
- Conditions
- Stargardt Disease, Stargardt Macular Dystrophy, Stargardt-like Macular Dystrophy
- Trial name
- DEVOTE (NCT07734064)
- Testing
- ASP2020
- Where
- 4 sites in United States
- Volunteers
- 30 people
- Sponsor
- Astellas Institute for Regenerative Medicine
Autism Spectrum Disorder
A Study to Investigate the Efficacy, Safety, and Tolerability of JNJ-42165279 in Adolescent and Adult Participants With Autism Spectrum Disorder
The purpose of this study is to evaluate the efficacy of JNJ-42165279 compared with placebo in the improvement of symptoms of Autism Spectrum Disorder (ASD) during 12 weeks of treatment using the Autism Behavior Inventory (ABI).
- Trial name
- Envision (NCT03664232)
- Testing
- JNJ-42165279
- Where
- 9 sites in United States
- Volunteers
- 78 people
- Sponsor
- Janssen Research & Development, LLC
Acute Ischemic Stroke
A Phase 2a Study to Evaluate the Dose-response, Safety, Tolerability, Efficacy, and Population Pharmacokinetics of Intravenous NTS-521 Infusions in the Early Recovery Treatment From Acute Ischemic Stroke
This is a trial to evaluate the safety and effect of three intravenous (IV) doses of NTS-521 for the early recovery treatment of acute ischemic stroke (AIS). Participants will be followed for 90 days.
- Trial name
- ACT-IVATE (NCT07742098)
- Testing
- NTS-521
- Volunteers
- 140 people
- Sponsor
- NeuroTrauma Sciences, LLC
Healthy Subjects
A Safety, Tolerability, and Pharmacokinetics Study of AP303 in Healthy Subjects
This is a single-center, randomized, double-blind, placebo-controlled, first-in-human study in which the safety, tolerability, and pharmacokinetics of orally administered AP303 will be assessed in healthy adult subjects.
- Trial name
- NCT05503693
- Testing
- AP303 50 μg, AP303 150 μg, AP303 300 μg
- Where
- Australia
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Alebund Pty Ltd
Overactive Bladder (OAB)
Study to Evaluate the Efficacy, Safety, and Tolerability of Mirabegron in Older Adult Subjects With Overactive Bladder (OAB)
The purpose of this study was to assess the efficacy, safety and tolerability of mirabegron versus placebo in the treatment of older adult subjects with OAB.
- Trial name
- PILLAR (NCT02216214)
- Testing
- Mirabegron
- Where
- 120 sites in United States, Canada
- Volunteers
- 888 people
- Sponsor
- Astellas Pharma Global Development, Inc.
Paroxysmal Nocturnal Hemoglobinuria
A Study to Test if SAR443809 is Tolerated and Safe When Taken as a Single Dose in Healthy Adults
Primary objective The tolerability and safety of SAR443809 Secondary The PK parameters of SAR443809 The PD activity of SAR443809 The immunogenicity of SAR443809
- Trial name
- NCT06326814
- Testing
- Humanized anti-Factor Bb monoclonal antibody
- Where
- 2 sites in United States
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- Sanofi
Major Depressive Disorder
A Study to Evaluate SAGE-217 in Adult Participants With Major Depressive Disorder (MDD)
This is a Phase 3, open-label, 1-year study of the safety, tolerability, and need for re-treatment with SAGE-217 in adult participants with MDD.
- Trial name
- NCT03864614
- Testing
- SAGE-217
- Where
- 52 sites in United States
- Volunteers
- 1,515 people
- Sponsor
- Biogen
Alzheimer Disease, Healthy Subjects
Multiple Dose Study Evaluating the Safety and Tolerability of NSA-789
This is a multiple dose study of NSA-789, the purpose of which is to assess the safety and tolerability in healthy subjects and subjects with Alzheimer's Disease
- Trial name
- NCT00795730
- Testing
- NSA-789
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Wyeth is now a wholly owned subsidiary of Pfizer
Healthy Volunteer - Complete, Cystic Fibrosis - Complete
Study Designed to Assess the Safety, Tolerability and PK of PTI-808 in Healthy Volunteers and in Adults With Cystic Fibrosis
Part 1 of this trial will enroll healthy volunteers into a single ascending dose (SAD), multiple ascending dose (MAD), and Food Effect (FE) treatment groups.
- Trial name
- NCT03251092
- Testing
- PTI-808, PTI-428, PTI-801
- Where
- 48 sites in United States, Australia and 7 more
- Volunteers
- 179 people, healthy volunteers welcome
- Sponsor
- Proteostasis Therapeutics, Inc.
Ulcerative Colitis, Crohn Disease
HMPL004-6599 Phase I Dose-escalating Study
To assess the safety and tolerability of single and multiple doses of HMPL004-6599 in healthy male volunteers
- Trial name
- NCT03597971
- Testing
- HMPL004-6599
- Where
- Australia
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Nutrition Science Partners Limited
Type 2 Diabetes Mellitus
Tofogliflozin GLP-1 Analogue Combination Trial
An open-label, multicenter study to evaluate 52-week long-term safety, tolerability and efficacy of Tofogliflozin with GLP-1 analogue treatment in type 2 diabetes mellitus.
- Trial name
- NCT02537834
- Testing
- Tofogliflozin, GLP-1 analogue
- Where
- 11 sites in Japan
- Volunteers
- 65 people
- Sponsor
- Kowa Company, Ltd.