Phase 2 Autosomal Clinical Trials

11 recruitingPhase 2

What is a Phase 2 trial?

Phase 2 trials evaluate whether a treatment works for a specific condition and further assess its safety. These studies typically enroll several hundred participants and provide preliminary data on effectiveness.

Showing 111 of 11 trials

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
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 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
Phase 2

Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease

Autosomal Dominant Polycystic Kidney Disease
Cantonal Hospital Graubuenden40 enrolled2 locationsNCT06435858
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
Recruiting
Phase 2

Trial of JMKX003142 in Participants With Rapidly Progressive Autosomal Dominant Polycystic Kidney Disease (ADPKD)

ADPKD (Autosomal Dominant Polycystic Kidney Disease)
Jemincare135 enrolled1 locationNCT06800651
Recruiting
Phase 2

Glucagon-Like Peptide-1 Receptor Agonist in ADPKD

ObesityAutosomal Dominant Polycystic Kidney
University of Colorado, Denver126 enrolled1 locationNCT06582875
Recruiting
Phase 2

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Arterial calcificationPseudoxanthoma ElasticumEctonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency+2 more
Inozyme Pharma200 enrolled5 locationsNCT06462547
Recruiting
Phase 2

A GnRH Agonist IN Pre-menopausal Women STudy to Treat Severe Polycystic Liver Disease

Autosomal Dominant Polycystic KidneyPolycystic Liver Disease
University Medical Center Groningen36 enrolled2 locationsNCT05478083