Clinical trials: Rare Disorders
- Condition Rare Disorders
- Funder type Industry (pharma, biotech, devices)
- Accepts healthy volunteers yes
Rare Diseases
A Study to Evaluate Emapalumab in Japanese Healthy Volunteers.
This is a randomized, placebo controlled and double-blinded study to evaluate the pharmacokinetics (PK), pharmacodynamics (PD) and safety of a single dose (1 mg/kg) of emapalumab in adult healthy Japanese subjects.
- Trial name
- NCT04765553
- Testing
- NI-0501, Saline
- Where
- Japan
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Swedish Orphan Biovitrum
Anemia
A Safety and Tolerability Study of AG-519 in Healthy Subjects
The purpose of the study is to investigate a drug called AG-519, which is being developed for the treatment of a disease called pyruvate kinase deficiency (also known as PK deficiency) and other forms of anemia.
- Trial name
- NCT02630927
- Testing
- AG-519
- Where
- United Kingdom
- Volunteers
- 108 people, healthy volunteers welcome
- Sponsor
- Agios Pharmaceuticals, Inc.
Fragile X Syndrome
CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction
The purpose of this First-in-Human Phase I study is to investigate the safety, tolerability and pharmacokinetics of CTH120 in adult healthy volunteers.
- Trial name
- FIH-CTH120 (NCT06480968)
- Testing
- CTH120
- Where
- Spain
- Volunteers
- 76 people, healthy volunteers welcome
- Sponsor
- Connecta Therapeutics, S.L.
Sleep Disorders, Circadian Rhythm, Cognitive Dysfunction
Feasibility Study on the Use of Redormin®500 on Day-time Cognition
Randomized, double-blind, parallel groups, placebo-controlled, baseline/run-in period of 21 days followed by trial period of 21 days, digital phenotyping (sleep, cognitive/psychological parameters and HRV).
- Trial name
- NCT05684523
- Testing
- Valerian-Hop Extract
- Where
- Switzerland
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Max Zeller Soehne AG
Primary Mitochondrial Diseases
Collection of Blood and Skin Samples From Patients With Primary Mitochondrial Diseases and Healthy Volunteers
Mitochondrial diseases are a genetically diverse group of disorders, some of which are caused by mutations or deletions in the mitochondrial DNA (mtDNA) and which display a wide range of severity and phenotypes.
- Trial name
- NCT06474104
- Where
- Israel
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Minovia Therapeutics Ltd.
Tay-Sachs Disease, Sandhoff Disease
A Multinational, Randomized, Double-blind, Placebo-controlled Study to Assess the Efficacy, Pharmacodynamics, Pharmacokinetics, and Safety of Venglustat in Late-onset GM2
Primary Objectives: Primary population (adult participants with late-onset GM2 gangliosidosis): To assess the efficacy and pharmacodynamics (PD) of daily oral dosing of venglustat when administered over a 104-week period…
- Trial name
- AMETHIST (NCT04221451)
- Testing
- venglustat GZ402671
- Where
- 23 sites in United States, Argentina and 11 more
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- Genzyme, a Sanofi Company
Occasional Hair Loss
Evaluation of the Dietary Supplement Effect on Improvement of Hair Condition in Subjects With Occasional Hair Loss.
The goal of this study is to learn if dietary supplement works to help with improving hair condition in subjects with occasional hair loss. It will also learn about its safety.
- Trial name
- NCT07835646
- Testing
- Dietary supplement for hair loss
- Where
- Spain
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Lacer S.A.
Low Back Pain
Internet App to Self-Manage Occasional Low Back Pain
Nonspecific low back pain (NLBP) is the diagnosis for 85% of low back pain sufferers with no underlying medical cause (e.g., herniated disc, spinal stenosis) that requires physician care.
- Trial name
- BackPain2 (NCT01950091)
- Testing
- FitBack on-line intervention, Alternative Website Control
- Volunteers
- 606 people, healthy volunteers welcome
- Sponsor
- Oregon Center for Applied Science, Inc.
Erectile Dysfunction
The Effects of a Nitrate Supplementation on Erectile Function
Twenty (20) individuals to be recruited from Ballantyne Medical Associates in Charlotte, NC, without any significant chronic medical history. These patients are general patients for primary care that meet inclusion criteria.
- Trial name
- NCT06213077
- Testing
- Nitric Oxide, Tadalafil
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- RHA Investments Ltd
Healthy, Lupus
Direct to Patient Minimal Risk Biospecimen and Data Collection Research
This study aims to help researchers better understand health conditions and develop improved tests, treatments, and cures for diseases.
- Conditions
- Healthy, Lupus, Celiac, Kidney Disease
- Trial name
- JBMR (NCT07128966)
- Testing
- Observation
- Where
- United States
- Volunteers
- 100,000 people, healthy volunteers welcome
- Sponsor
- Joined Bio
Rare Diseases
A Multi-Site Leukopak Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
To collect, preserve, and distribute annotated leukopak biospecimens and associated medical data to institutionally approved, investigator-directed biomedical research to discover and develop new treatments, diagnostics, and…
- Trial name
- NCT05794217
- Testing
- This is a non-interventional study
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Sanguine Biosciences
Fragile X Syndrome (FXS), Neurotypical Adults
Neural Biomarkers in Fragile X Syndrome
The purpose of this non-interventional test /re-test study is to assess neural biomarkers in adult subjects with Fragile X syndrome compared to those measured in a population of typically developing adults
- Trial name
- EXP-CTH120 (NCT06957054)
- Testing
- Any
- Where
- 2 sites in Spain
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Connecta Therapeutics, S.L.
Insomnia, Sleeplessness
Evaluation of Vestibular Stimulation to Help Occasional Sleeplessness
The purpose of this study is to determine whether a small electrical current to the vestibular nerve (balance organ) will decrease the time it take for participants to fall asleep.
- Conditions
- Insomnia, Sleeplessness, Transient Insomnia
- Trial name
- VSOM (NCT00594022)
- Testing
- Electric stimulation of the Vestibular Nerve - "VirtuSom"
- Where
- 6 sites in United States
- Volunteers
- 349 people, healthy volunteers welcome
- Sponsor
- Philips Respironics
Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder
Phase 1 Single Dose Study of ALXN1101 in Healthy Volunteers
Phase 1 single dose study of ALXN1101 in healthy volunteers.
- Conditions
- Molybdenum Cofactor Deficiency (MoCD), Rare Autosomal Recessive Disorder, Deficiency of Activity of Molybdenum-dependent Enzymes (Sulfite Oxidase [SOX], Xanthine Dehydrogenase, and Aldehyde Oxidase)
- Trial name
- NCT01894165
- Testing
- ALXN1101
- Where
- United States
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Origin Biosciences