Autosomal Clinical Trials

1 recruiting

Frequently Asked Questions

Common questions about Autosomal clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 120 of 40 trials

Recruiting
Phase 3

Implementation of Metformin theraPy to Ease Decline of Kidney Function in Polycystic Kidney Disease (IMPEDE-PKD)

Autosomal Dominant Polycystic Kidney Disease
The University of Queensland1,174 enrolled49 locationsNCT04939935
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
Not Applicable

Developing a Pipeline to Employ RNA-Seq as a Complementary Diagnostic Tool in Rare Diseases

HealthyAtypical Hemolytic Uremic SyndromeMembranoproliferative Glomerulonephritis+1 more
Mario Negri Institute for Pharmacological Research105 enrolled1 locationNCT05996731
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 3

ENERGY 2: Evaluation of the Efficacy and Safety of INZ-701 in Infants With ENPP1 Deficiency

Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy 1
Inozyme Pharma12 enrolled8 locationsNCT07473973
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 2Phase 3

Pharmacokinetics, Efficacy, and Safety of Encaleret in Pediatric Participants With Autosomal Dominant Hypocalcemia Type 1 (ADH1)

Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Calcilytix Therapeutics, Inc., a BridgeBio company28 enrolled5 locationsNCT07080385
Recruiting
Phase 3

STOP-PKD: SGLT2-inhibition to Improve Prognosis in Polycystic Kidney Disease

Polycystic Kidney, Autosomal Dominant
University of Cologne420 enrolled27 locationsNCT07280585
Recruiting
Not Applicable

Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias

Autosomal Recessive Spastic Ataxia of Charlevoix-SaguenaySpastic Paraplegia 7
Laval University84 enrolled5 locationsNCT06261424
Recruiting
Phase 3

A Study to See Iftolvaptan is Safe in Infants and Children Who at Enrollment Are 28 Days to Less Than 18 Years Old withAutosomal Recessive Polycystic Kidney Disease (ARPKD)

Autosomal Recessive Polycystic Kidney (ARPKD)
Otsuka Pharmaceutical Development & Commercialization, Inc.20 enrolled23 locationsNCT04782258
Recruiting
Phase 2

A Study to Assess Adverse Events and Effectiveness of IntraVenous Infusions of ABBV-CLS-628 in Adult Participants With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal Dominant Polycystic Kidney Disease
AbbVie240 enrolled75 locationsNCT06902558
Recruiting
Not Applicable

Autosomal Dominant Spinocerebellar Ataxias and Social Cognition

Autosomal Dominant Spinocerebellar Ataxia (SCA1, 2,3,6,7,27B)
University Hospital, Angers160 enrolled1 locationNCT07099651
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

A Prospective Observational Study of Foam Sclerotherapy .

Autosomal Dominant Polycystic KidneyRenal CystAutosomal Dominant Polycystic Liver Disease+1 more
Mayo Clinic100 enrolled1 locationNCT04111692
Recruiting
Phase 1Phase 2

Safety of Single and Repeat Dose of PYC-001 Eye Injections in People With Autosomal Dominant Optic Atrophy

Autosomal Dominant Optic AtrophyOPA1 Gene MutationHereditary Optic Atrophies+1 more
PYC Therapeutics21 enrolled3 locationsNCT06970106
Recruiting

Taiwan Associated Genetic and Nongenetic Small Vessel Disease

StrokeMagnetic Resonance ImagingFabry Disease+5 more
National Taiwan University Hospital500 enrolled1 locationNCT05473637
Recruiting

Characterization of the Nrf2 Response in Patients With Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Autosomal Dominant Polycystic Kidney Disease
Mayo Clinic40 enrolled1 locationNCT04344769
Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting

NOX4 and Related Biomarkers in ADPKD

Autosomal Dominant Polycystic Kidney Disease
Mayo Clinic60 enrolled1 locationNCT04630613
Recruiting

Hypertension in Children and Young People at Risk of Autosomal Dominant Polycystic Kidney Disease

Autosomal Dominant Polycystic Kidney Disease (ADPKD
King's College London200 enrolled1 locationNCT07260071