Autosomal Clinical Trials

47 recruitingLast updated: May 13, 2026

There are 47 actively recruiting autosomal clinical trials across 25 countries. Studies span Phase 2, Not Applicable, Phase 1, Phase 3. Top locations include Rochester, Minnesota, United States, London, United Kingdom, Philadelphia, Pennsylvania, United States. Updated daily from ClinicalTrials.gov.


Autosomal Trials at a Glance

47 actively recruiting trials for autosomal are listed on ClinicalTrialsFinder across 6 cities in 25 countries. The largest study group is Phase 2 with 11 trials, with the heaviest enrollment activity in Rochester, London, and Philadelphia. Lead sponsors running autosomal studies include Mayo Clinic, Inozyme Pharma, and PYC Therapeutics.

Browse autosomal trials by phase

Treatments under study

About Autosomal Clinical Trials

Looking for clinical trials for Autosomal? There are currently 1 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Autosomal trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

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 47 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

The German ADPKD Tolvaptan Treatment Registry

ADPKD (Autosomal Dominant Polycystic Kidney Disease)
University of Cologne2,000 enrolled12 locationsNCT02497521
Recruiting
Phase 2

Phase 2a Study of VX-407 in Participants With ADPKD Who Have a Subset of PKD1 Gene Variants (AGLOW)

Autosomal Dominant Polycystic Kidney Disease (ADPKD
Vertex Pharmaceuticals Incorporated24 enrolled40 locationsNCT07161037
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 enrolled6 locationsNCT07080385
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

RADIOLOGICAL AND CLINICAL EVALUATION OF RENAL EMBOLIZATION USING EVOH IN DIALYSIS PATIENTS WITH AUTOSOMAL DOMINANT POLYCYSTIC KIDNEY DISEASE: A PROSPECTIVE LONGITUDINAL OBSERVATIONAL STUDY

Autosomal Dominant Polycystic Kidney Disease (ADPKD
Fondazione IRCCS Policlinico San Matteo di Pavia30 enrolled1 locationNCT07535385
Recruiting

Long-term Prospective Study of Korean CADASIL Patients

CadasilCerebral Autosomal Dominant Arteriopatie With Subcortical Infarcts and Leukoencephalopathy
Jeju National University Hospital500 enrolled1 locationNCT07497867
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 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
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
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 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
Not Applicable

Metabolic Impacts of Ren-Nu: A Dietary Program for Polycystic Kidney Disease

Autosomal Dominant Polycystic Kidney Disease (ADPKD
Richard Fatica20 enrolled1 locationNCT07454174
Recruiting
Phase 3

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

Polycystic Kidney, Autosomal Dominant
University of Cologne420 enrolled27 locationsNCT07280585
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 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
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 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