Phase 1 Autosomal Clinical Trials
7 recruitingPhase 1
What is a Phase 1 trial?
Phase 1 trials test a new treatment in a small group of people for the first time. The primary goal is to evaluate safety, determine a safe dosage range, and identify side effects. These studies typically enroll 20 to 80 participants.
Showing 1–7 of 7 trials
Recruiting
Phase 1
Safety, Tolerability and Pharmacokinetics of AZD1613 in Adults With Autosomal Dominant Polycystic Kidney Disease
Autosomal Dominant Polycystic Kidney Disease
AstraZeneca40 enrolled15 locationsNCT07228364
Recruiting
Phase 1Phase 2
Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy (Myrtle)
Autosomal Dominant Optic AtrophyOPA1 Gene MutationHereditary Optic Atrophies+1 more
PYC Therapeutics18 enrolled3 locationsNCT06970106
Recruiting
Phase 1Phase 2
Safety and Tolerability of Subretinally Injected OPGx-BEST1 in Patients With Best Vitelliform Macular Dystrophy (BVMD) or Autosomal-Recessive Bestrophinopathy (ARB)
ARBBVMDAutosomal-Dominant Bestrophinopathy+1 more
Opus Genetics, Inc10 enrolled4 locationsNCT07185256
Recruiting
Phase 1
The ENERGY Study: Evaluation of Safety and Tolerability of INZ-701 in Infants With ENPP1 Deficiency or ABCC6 Deficiency
Pseudoxanthoma ElasticumEctonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic Rickets+2 more
Inozyme Pharma16 enrolled7 locationsNCT05734196
Recruiting
Phase 1
Phase 1 Study to Evaluate the Safety and Tolerability of Intravenously Administered PYC-003
Autosomal Dominant Polycystic Kidney Disease (ADPKD
PYC Therapeutics116 enrolled9 locationsNCT06714006
Recruiting
Phase 1
A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK
Alzheimer DiseaseAutosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder)Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder)+1 more
University College, London10 enrolled2 locationsNCT06372821
Recruiting
Phase 1Phase 2
Retina is a Marker for Cerebrovascular Heath
Cerebral Small Vessel DiseasesCerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and LeukoencephalopathyCerebral Microbleeding+1 more
Mayo Clinic100 enrolled1 locationNCT04753970