Clinical trials: Inherited Genetic Conditions
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.
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
Growth Disorders
Treatment of Children and Adolescents With Growth Failure Associated With Primary IGF-1 Deficiency
This is an extension study to Tercica study MS301 (NCT00125164) and is intended to collect long term safety and efficacy data on the continued use of recombinant human insulin-like growth factor-1 (rh IGF-1) in children and…
- Trial name
- NCT00330668
- Testing
- rh IGF-1 (mecasermin)
- Where
- France
- Volunteers
- 114 people
- Sponsor
- Ipsen
Gout, Acute Gout Flare
A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of Oral ABP-745 Administration in Healthy Volunteers
A randomized, double-blind, placebo-controlled, dose-escalation study will be conducted in healthy volunteers. This study will evaluate the safety, tolerability, and pharmacokinetics (PK) of ABP-745 following single (Part 1) and…
- Conditions
- Gout, Acute Gout Flare, Gout Flares
- Trial name
- ABP-745 (NCT06258213)
- Testing
- ABP-745
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- Atom Therapeutics Co., Ltd
Breast Cancer, Benign Breast Condition
ATUSA Ultrasound Diagnostic Imaging for Breast Lesions Evaluation (AUDIBLE) - iSono Health
AUDIBLE is a multi-site, clinical study conducted in the United States aimed at gathering data on 3D Automated Breast Ultrasound imaging using the iSono Health ATUSA system.
- Conditions
- Breast Cancer, Benign Breast Condition, Breast Fibroadenoma, Breast Neoplasms
- Trial name
- AUDIBLE (NCT07505797)
- Testing
- ATUSA 3D Automated Breast Ultrasound System
- Where
- 2 sites in United States
- Volunteers
- 800 people
- Sponsor
- iSono Health, Inc.
Photoaging, Weight Loss
Evaluation of the Effects of a Biostimulator and Dermal Fillers for Cheek Augmentation and Contour Deficiencies
Adult subjects with a history of or currently taking glucagon-like peptide-1 (GLP-1) receptor agonist medication and moderate-to-severe cheek wrinkles and midface contour deficiencies will be treated with Sculptra correction of…
- Conditions
- Photoaging, Weight Loss, Skin Laxity, Photodamaged Skin
- Trial name
- NCT06351358
- Testing
- Sculptra treatment, Restylane Treatment
- Where
- 2 sites in United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Galderma R&D
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Chronic Granulomatous Disease (CGD)
Prevalence of Antibodies and Cytokines in Participants With Chronic Granulomatous Disease
This research study is designed to assess the prevalence of specific antibodies and inflammatory cytokines in adult and pediatric participants with CGD.
- Trial name
- NCT06605378
- Where
- 52 sites in United States, United Kingdom
- Volunteers
- 60 people
- Sponsor
- Ensoma
Atopic Dermatitis
A Pilot Study of Lactofiltrum to Treat Atopic Dermatitis in Adults
The purpose is to study efficacy of Lactofiltrum in adult patients with atopic dermatitis. Lactofiltrum consists of 85% of hidrolytic lignin and 15% of lactulose and acts as an enterosorbent, which enables to enhance metabolism…
- Trial name
- LF-AD-09 (NCT01124318)
- Testing
- Lactofiltrum
- Where
- 2 sites in Russia
- Volunteers
- 90 people
- Sponsor
- Avva Rus, JSC
Lymphocytic Leukemia, Chronic, Lymphoma, Small Lymphocytic
Duvelisib With Obinutuzumab in Patients With CLL/SLL Previously Treated With a BTKi (SYNCHRONY)
To evaluate the safety, pharmacokinetics, and pharmacodynamics of Duvelisib (IPI-145) in combination with obinutuzumab in patients with Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma previously treated with a BTKi.
- Trial name
- NCT02292225
- Testing
- IPI-145 (duvelisib), Obinutuzumab
- Where
- 7 sites in United States, Canada
- Volunteers
- 3 people
- Sponsor
- SecuraBio
Fragile X Syndrome (FXS), Fragile X Syndrome
Safety, Tolerability, and Preliminary Effectiveness of CTH120 in Fragile X Syndrome
The purpose of this Phase IIa study is to evaluate the safety, tolerability, and effectiveness of CTH120 in adult males with Fragile X syndrome.
- Trial name
- FXS-CTH120-01 (NCT07654114)
- Testing
- CTH120
- Where
- 2 sites in Spain
- Volunteers
- 30 people
- Sponsor
- Connecta Therapeutics, S.L.
Fed
Galantamine Bioequivalence Study of Dr. Reddy's Under Fed Condition
Galantamine Hydrobromide Tablets, Bioequivalence study of Dr. Reddy's under Fed condition.
- Trial name
- NCT01372306
- Testing
- Reminyl
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Dr. Reddy's Laboratories Limited
Fabry Disease
A Study to Evaluate Migalastat in Fabry Subjects With Amenable GLA Variant and Renal Disease
An Open-label Study to Evaluate the Safety and Pharmacokinetics of Migalastat HCl in Subjects with Fabry Disease and Amenable GLA Variants and Severe Renal Impairment (SRI) or End Stage Renal Disease (ESRD)
- Trial name
- NCT04020055
- Testing
- migalastat HCl 150 mg
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 14 people
- Sponsor
- Amicus Therapeutics
Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes
A Study to Learn How the Study Drug Elinzanetant (BAY 3427080) Affects the Way the Drug Dabigatran Moves Into, Through and Out of the Body in Healthy Male and Female Participants
Researchers are looking for a better way to treat men and women with vasomotor symptoms, a condition of having hot flashes caused by hormonal changes.
- Conditions
- Vasomotor Symptoms as a Sex Hormone-dependent Disorder in Women and Men, Hot Flashes, Healthy Volunteers
- Trial name
- NCT05471817
- Testing
- Elinzanetant (BAY3427080), Dabigatran etexilate
- Where
- Germany
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Bayer
Osteoarthritis
Efficacy of Proprietary Cherry Juice Blend in Osteoarthritis of the Knee
The primary purpose of this research study is to test the ability of a proprietary cherry juice blend to be helpful in the treatment of osteoarthritis (OA) of the knee.
- Trial name
- NCT00443092
- Testing
- proprietary cherry juice blend (food), control juice (kool aid blend)
- Where
- United States
- Volunteers
- 59 people
- Sponsor
- CherryPharm
Atopic Dermatitis
A Study to Learn How Different Forms of the Study Medicine Called PF 08049820 Are Taken up Into the Bloodstream in Healthy Adults
The purpose of this study is to see how different forms of a medicine called PF-08049820 move through the body when taken by mouth.
- Trial name
- NCT07172321
- Testing
- PF-08049820 MR1, PF-08049820 MR2, PF-08049820 IR
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- 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
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
Huntington's Disease
Randomized, Placebo Controlled Study Of The Efficacy And Safety Of PF-02545920 In Subjects With Huntington's Disease
This study is a 26 week, randomized, parallel group, double blind comparison of PF-02545920 5 mg, PF-02545920 20 mg, and placebo dosed BID in the treatment of motor impairment of subjects with Huntington's Disease.
- Trial name
- NCT02197130
- Testing
- PF-02545920
- Where
- 57 sites in United States, Canada and 3 more
- Volunteers
- 272 people
- Sponsor
- Pfizer
Alpha1-Antitrypsin Deficiency
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran…
- Trial name
- NCT05677971
- Testing
- Fazirsiran Injection
- Where
- 89 sites in United States, Australia and 17 more
- Volunteers
- 160 people
- Sponsor
- Takeda