Clinical trials: Genetic
Cystic Fibrosis
Ketone Supplementation in Cystic Fibrosis
This study will examine the effects of a novel ketone monester supplement on patients with cystic fibrosis experiencing an acute pulmonary exacerbation requiring hospitalization.
- Trial name
- NCT04938726
- Testing
- Ketone Monoester
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- University of Alabama at Birmingham
Anemia, Sickle Cell
Effectiveness of Arginine as a Treatment for Sickle Cell Anemia
Sickle cell disease (SCD), also known as sickle cell anemia, is an inherited genetic disease that can cause intense pain episodes.
- Trial name
- Arginine (NCT00513617)
- Testing
- Arginine
- Where
- 17 sites in United States
- Volunteers
- 128 people
- Sponsor
- UCSF Benioff Children's Hospital Oakland
Cervical Cancer
Bintrafusp Alfa Combination Therapy in Participants With Cervical Cancer (INTR@PID 046)
This study was to evaluate the safety and tolerability of bintrafusp alfa in combination with other anti-cancer therapies in participants with locally advanced or advanced cervical cancer.
- Trial name
- NCT04551950
- Testing
- M7824, Carboplatin, Paclitaxel
- Where
- 13 sites in United States, Japan and 1 more
- Volunteers
- 25 people
- Sponsor
- EMD Serono Research & Development Institute, Inc.
Atopic Dermatitis
Phase 2a Study of Efficacy and Safety of OpSCF in Moderate to Severe AD (Atopic Dermatitis)
The purpose of this study is to determine the efficacy and safety of a monoclonal antibody, OpSCF, in the treatment of adults with moderate to severe Atopic Dermatitis (Eczema). OpSCF will be compared to a placebo.
- Trial name
- NCT06101823
- Testing
- OpSCF
- Where
- 22 sites in United States, Canada
- Volunteers
- 50 people
- Sponsor
- Insmed Incorporated
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Myotonic Dystrophy 1, Excessive Daytime Sleepiness
Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1
The primary objective of this study is to evaluate the safety and efficacy of pitolisant compared with placebo in treating excessive daytime sleepiness (EDS) in patients with Myotonic Dystrophy Type 1 ages 18 to 65 years.
- Trial name
- NCT04886518
- Testing
- Pitolisant Oral Tablet
- Where
- 13 sites in United States, Canada
- Volunteers
- 30 people
- Sponsor
- Harmony Biosciences Management, Inc.
Respiratory Syncytial Virus Infection
Study on a Live-attenuated Respiratory Syncytial Virus Vaccine for Assessment of Safety, Transmissibility, and Genetic Stability of the Vaccine Virus Among Close Contacts in Infants and Toddlers 6 to < 24 Months of Age in Puerto Rico (USA)
The primary purpose of the study is to assess the shedding, transmission, and genetic stability of the live-attenuated RSVt vaccine after each intranasal vaccination (56 days apart) in infants and toddlers 6 to < 24 months of age.
- Trial name
- NCT05687279
- Testing
- RSVt Vaccine, Control Group
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Sanofi Pasteur, a Sanofi Company
Atopic Dermatitis
JAK1 Inhibitor With Medicated Topical Therapy in Adolescents With Atopic Dermatitis
This is a randomized, double blind, placebo controlled, parallel group, Phase 3 study to evaluate the efficacy and safety of PF 04965842 in adolescent participants 12 to <18 years of age with moderate to severe AD.
- Trial name
- JADE TEEN (NCT03796676)
- Testing
- PF-04965842, PF04965842
- Where
- 136 sites in United States, Australia and 12 more
- Volunteers
- 287 people
- Sponsor
- Pfizer
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of Ciprofloxacin (Inhaled) in Patients With Cystic Fibrosis
To evaluate the change in forced expiratory volume (FEV1) from baseline to Day 28-30 between Cipro Inhale-treated and placebo-treated subjects after a 4-week treatment period.
- Trial name
- NCT00645788
- Testing
- Ciprofloxacin (Cipro Inhale, BAYQ3939)
- Where
- 86 sites in United States, Australia and 7 more
- Volunteers
- 288 people
- Sponsor
- Bayer
Spinal Muscular Atrophy Type II, Spinal Muscular Atrophy Type III
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
The purpose of this study is to identify the maximum tolerated dosage of sodium phenylbutyrate in children with spinal muscular atrophy types II or III; and to determine if the drug has an effect on SMN mRNA and protein levels.
- Trial name
- NPTUNE01 (NCT00439569)
- Testing
- sodium phenylbutyrate
- Where
- 5 sites in United States
- Volunteers
- 9 people
- Sponsor
- Westat
Colorectal Cancer, Hereditary Non-polyposis Colon Cancer
Educational CD-ROM Compared With Standard Informed Consent for Patients With Colorectal Cancer or a Family History of Colorectal Cancer
RATIONALE: The use of a CD-ROM may help patients with colorectal cancer or a family history of colorectal cancer make informed decisions about undergoing microsatellite instability (MSI) testing.
- Trial name
- NCT00450424
- Testing
- counseling intervention, CD-ROM intervention
- Where
- 3 sites in United States
- Volunteers
- 239 people
- Sponsor
- Fox Chase Cancer Center
Atopic Dermatitis Eczema, Atopic Dermatitis
Trends in the Epidemiology and Treatment of Atopic Dermatitis in the United Kingdom (UK)
Atopic dermatitis (AD), commonly called eczema, is one of the most frequently occurring skin conditions. It is estimated to affect around one fifth of children in developed countries and is also becoming increasingly common in…
- Conditions
- Atopic Dermatitis Eczema, Atopic Dermatitis, Eczema
- Trial name
- NCT03823794
- Testing
- No intervention
- Where
- United Kingdom
- Volunteers
- 148,166 people, healthy volunteers welcome
- Sponsor
- Momentum Data
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study B
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD B (NCT01885559)
- Testing
- Lisinopril, Telmisartan
- Where
- 7 sites in United States
- Volunteers
- 486 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Duchenne Muscular Dystrophy
Phase 3 Trial of Pamrevlumab or Placebo With Systemic Corticosteroids in Participants With Non-ambulatory Duchenne Muscular Dystrophy (DMD)
To evaluate the efficacy and safety of pamrevlumab versus placebo in combination with systemic corticosteroids in participants with non-ambulatory Duchenne muscular dystrophy (age 12 years and older).
- Trial name
- LELANTOS-1 (NCT04371666)
- Testing
- Pamrevlumab, Corticosteroids
- Where
- 53 sites in United States, Australia and 12 more
- Volunteers
- 98 people
- Sponsor
- Kyntra Bio
CAH - Congenital Adrenal Hyperplasia
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NBI-74788 (Crinecerfont) in Pediatric Participants With Congenital Adrenal Hyperplasia
This is a Phase 2, open-label, multiple-dose, study to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of NBI-74788 (crinecerfont) in pediatric participants (14 to 17 years of age) with a…
- Trial name
- NCT04045145
- Testing
- Crinecerfont
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Neurocrine Biosciences
Primary Immunodeficiency Diseases (PID)
A Study to Evaluate the Long-term Safety of TAK-771 in Japanese Primary Immunodeficiency Disease (PID) Participants
The main aim of the study is to check side effect from the study treatment with TAK-771 in long term. Participants can have taken part in the previous study TAK-771-3004 (NCT05150340).
- Trial name
- NCT05513586
- Testing
- TAK-771
- Where
- 9 sites in Japan
- Volunteers
- 15 people
- Sponsor
- Takeda
Down Syndrome
Efficacy Assessment of Systematic Treatment With Folinic Acid and Thyroid Hormone on Psychomotor Development of Down Syndrome Young Children
Evaluation of the following in very young children with Down syndrome: the efficacy of systematic treatment with L-thyroxine at controlled doses (clinically and by ultrasensitive thyreostimulating hormone (TSH) assay), the…
- Trial name
- ACTHYF (NCT01576705)
- Testing
- thyroid hormone and folinic acid
- Where
- France
- Volunteers
- 175 people
- Sponsor
- Institut Jerome Lejeune
Sickle Cell Anemia, Severe Acute Malnutrition
Management of Severe Acute Malnutrition in SCD, in Northern Nigeria
Except for children with HIV, all recommendations for treatment of childhood malnutrition are for children < 5 years of age. The overall goal of this randomized controlled nutrition feasibility trial is to identify whether…
- Trial name
- NCT03634488
- Testing
- hydroxyurea (20mg/kg/day), Ready-to-use therapeutic food
- Where
- 3 sites in United States, Nigeria
- Volunteers
- 132 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University Medical Center
Atopic Dermatitis
Evaluating the Effects of Tradipitant vs. Placebo in Atopic Dermatitis (EPIONE)
This is a multicenter, randomized, double-blind, placebo-controlled study to be conducted in the United States in subjects with atopic dermatitis.
- Trial name
- NCT03568331
- Testing
- Tradipitant
- Where
- 70 sites in United States
- Volunteers
- 375 people
- Sponsor
- Vanda Pharmaceuticals
Age-Related Macular Degeneration, Macular Degeneration
Repeated Eye Injections of Aflibercept for Treatment of Wet Age Related Macular Degeneration
To evaluate the visual outcome and number of injections required during an optical coherence tomography (OCT)-guided treat and extend regimen with intravitreal aflibercept for treatment of subfoveal neovascular age-related…
- Trial name
- ATLAS (NCT01773954)
- Testing
- Intravitreal Aflibercept Injection
- Where
- 3 sites in United States
- Volunteers
- 40 people
- Sponsor
- MidAtlantic Retina