Clinical trials: Genetic Conditions
Alpha-1 Antitrypsin Deficiency (AATD)
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults…
- Trial name
- NCT07227207
- Testing
- TSRA-196
- Where
- 9 sites in United States, Australia and 2 more
- Volunteers
- 72 people
- Sponsor
- Tessera Therapeutics, Inc.
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn if Elinzanetant Affects the Ability to Drive and Brain Function in Healthy Women
Researchers are looking for a better way to treat vasomotor symptoms (VMS), also known as hot flashes. Hot flashes are intense and sudden feelings of heat along with sweating and reddening of the skin.
- Trial name
- NCT06219902
- Testing
- Elinzanetant (BAY3427080), Zopiclone
- Where
- Canada
- Volunteers
- 64 people, healthy volunteers welcome
- Sponsor
- Bayer
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
Type 2 Inflammatory Disorders
Compassionate Use or Expanded Access of Dupilumab
Provide Compassionate Use or Expanded Access of Dupilumab
- Trial name
- NCT04776694
- Testing
- Dupilumab
- Sponsor
- Regeneron Pharmaceuticals
Duchenne Muscular Dystrophy
A Study to Assess the Efficacy and Safety of Satralizumab in Duchenne Muscular Dystrophy (DMD)
The purpose of this study is to assess the efficacy, safety, pharmacokinetics (PK) and pharmacodynamics (PD) of satralizumab, a humanized anti-interleukin-6 receptor (aIL-6R) monoclonal antibody, in ambulatory and non-ambulatory…
- Trial name
- SHIELD DMD (NCT06450639)
- Testing
- Satralizumab
- Where
- 18 sites in United States, Denmark and 4 more
- Volunteers
- 30 people
- Sponsor
- Hoffmann-La Roche
Tuberous Sclerosis, Angiofibroma
Long-term Trial of Topical Sirolimus to Angiofibroma in Patient With Tuberous Sclerosis Complex
The purpose of this trial is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel (0.2% sirolimus gel) to angiofibroma and other skin lesions in patients with tuberous sclerosis complex in the open-label…
- Conditions
- Tuberous Sclerosis, Angiofibroma, Hypomelanotic Macule, Plaque
- Trial name
- NCT02634931
- Testing
- NPC-12G gel
- Where
- Japan
- Volunteers
- 94 people
- Sponsor
- Nobelpharma
Hurler Syndrome (MPS I), Hurler-Scheie Syndrome
Phase I/II Pilot Study of Mixed Chimerism to Treat Inherited Metabolic Disorders
The goal of this research study is to establish chimerism and avoid graft-versus-host-disease (GVHD) in patients with inherited metabolic disorders.
- Conditions
- Hurler Syndrome (MPS I), Hurler-Scheie Syndrome, Hunter Syndrome (MPS II), Sanfilippo Syndrome (MPS III)
- Trial name
- NCT01372228
- Testing
- hematopoietic stem cell infusion
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Talaris Therapeutics Inc.
Dermatitis, Atopic, Pruritus
The Efficacy of 3% Kanuka Oil Versus 1% Hydrocortisone Cream in Patients With Atopic Dermatitis
Atopic dermatitis is a common disease affecting millions worldwide. A common associated symptom in this population is pruritus. We seek to investigate the use of an natural, over-the-counter cosmeceutical to help this population
- Trial name
- NCT06488742
- Testing
- 3% kanuka oil
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Nexgen Dermatologics, Inc.
Cystic Fibrosis
Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis
This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and…
- Trial name
- NCT01684410
- Testing
- Alpha-1 HC 100 mg, Alpha-1 HC 200 mg
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Grifols Therapeutics LLC
Atopic Dermatitis
Efficacy and Safety Study of MSRD-100 in Subjects With Atopic Dermatitis >=3 Months of Age and Older
This is a double-blind, randomized, vehicle-controlled, multi-center, parallel group Phase 3 study of MSRD-100 in the treatment of atopic dermatitis in subjects aged 3 months and up.
- Trial name
- MSRD-100 (NCT02677610)
- Testing
- MSRD-100, Vehicle
- Sponsor
- Merz North America, Inc.
Gout, Arthritis, Gouty
Phase 2b Evaluation of Efficacy and Safety of AR882 in Gout Patients
This study will assess the serum uric acid lowering effect and safety of AR882 in gout patients at two doses compared to placebo over 12 weeks.
- Conditions
- Gout, Arthritis, Gouty, Hyperuricemia, Gout Chronic
- Trial name
- NCT05119686
- Testing
- AR882 Dose 1, AR882 Dose 2
- Where
- 23 sites in United States, Australia and 1 more
- Volunteers
- 140 people
- Sponsor
- Arthrosi Therapeutics
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.
Stent Thrombosis, Ischemic Stroke
Association Between Genetic Variant Scores and P2Y12 Inhibitor Effects
The goal of this study is to predict and prevent adverse drug events by investigating the impact of genetic variants, demographics, and environmental factors in subjects status post myocardial infarction and percutaneous coronary…
- Conditions
- Stent Thrombosis, Ischemic Stroke, Myocardial Infarction, Acute Coronary Syndrome
- Trial name
- CARES1 (NCT04580602)
- Where
- South Korea
- Volunteers
- 200 people
- Sponsor
- Cipherome, Inc.
Fragile X Syndrome, Behavior
An Investigation of Psilocybin on Behavioural and Cognitive Symptoms of Adults With Fragile X Syndrome
Diverse symptomatology makes Fragile X Syndrome (FXS) difficult to treat, and currently there are no approved prevention or treatment methods for FXS.
- Conditions
- Fragile X Syndrome, Behavior, Cognitive Dysfunction
- Trial name
- NCT05832255
- Testing
- Psilocybin, 1.5 mg
- Where
- Canada
- Volunteers
- 10 people
- Sponsor
- Nova Mentis Life Science Corp
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
Eczema, Atopic Dermatitis
NPQ Plus Supplementation for Skin Health and Antioxidant Status
This study evaluated the effects of NPQ Plus supplementation on skin health, antioxidant status, fatigue, and self-perceived well-being in healthy adults with skin concerns.
- Conditions
- Eczema, Atopic Dermatitis, Skin Health, Fatigue Symptom
- Trial name
- NCT07703215
- Testing
- NPQ Plus
- Where
- Malaysia
- Volunteers
- 55 people
- Sponsor
- N REMEDIER (M) SDN BHD
Atopic Dermatitis
Trial to Determine the Therapeutic Benefit of an OTC Cream on Dry, Itchy Skin of Adults and Children With Eczema
The objective of this 1 week trial is to evaluate the efficacy of an over-the-counter (OTC) 1% colloidal oatmeal skin protectant cream in adults and children with mild to moderate AD.
- Trial name
- NCT02691507
- Testing
- EpiCeram Skin Barrier Emulsion, Experimental Product 1% Colloidal Oatmeal Balm
- Where
- 2 sites in United States
- Volunteers
- 53 people, healthy volunteers welcome
- Sponsor
- Johnson & Johnson Consumer Inc. (J&JCI)
Onychomycosis of Toenails
Efficacy and Safety Evaluation of AN2690 Topical Solution to Treat Onychomycosis of the Toenail
The purpose of this study is to determine whether AN2690 topical solution is a safe and effective treatment for onychomycosis of the toenail.
- Trial name
- NCT01270971
- Testing
- AN2690 Topical Solution, 5%, Solution Vehicle
- Where
- 27 sites in United States
- Volunteers
- 594 people
- Sponsor
- Pfizer
Usher's Syndrome
A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B
Primary Objective: To evaluate the long-term safety and tolerability of SAR421869 in patients with Usher syndrome Type 1B Secondary Objective: To assess long-term safety and biological activity of SAR421869
- Trial name
- NCT02065011
- Testing
- Blood draw for the laboratory assessment
- Where
- 2 sites in United States, France
- Volunteers
- 9 people
- Sponsor
- Sanofi
Respiratory Tract Diseases, Abnormal Mucus Secretions
A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions
This is a phase 3, multicenter, randomized, rater- and patient-blind, placebo- and active-controlled, 3-arm parallel group clinical trial.
- Trial name
- NCT03843541
- Testing
- N-acetylcysteine (NAC) 600 mg, Ambroxol hydrochloride 30 mg
- Where
- 32 sites in China
- Volunteers
- 333 people
- Sponsor
- Zambon SpA