Inherited Genetic Conditions

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Inherited Genetic Conditions trials you may qualify for

100

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 53 trials

RecruitingNot Applicable

NCT06191640

Sinus Disease in Young Children With Cystic Fibrosis

This is a prospective, observational study examining the impact of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators on chr…

United States80 participantsStarted 2023-04-12

Treatment: Ivacaftor or elexacaftor/tezacaftor/ivacaftor

RecruitingNot Applicable

NCT05644522

Nomad P-KAFO Study

The goal of this clinical trial is to evaluate the impact of using the Nomad powered KAFO in people who have had a musculoskeletal or neurological injury that h…

United States36 participantsStarted 2024-03-01

Treatment: Indego Nomad® Powered Knee-Ankle-Foot Orthosis (P-KAFO) (Parker Hannifin Corp., Cleveland, OH)

RecruitingNot Applicable

NCT07444008

Randomized, Blinded, Placebo-Controlled Evaluation of BioAmicus Complete

This is a randomized, single-blind, placebo-controlled study to evaluate the efficacy of the dietary supplement BioAmicus Complete (oral drops; a mixture of 10…

Russia140 participantsStarted 2026-03-16

Treatment: BioAmicus Complete, Placebo

RecruitingPhase 2

NCT05010031

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

This study will test whether reduced-dose radiotherapy is an effective treatment for metastatic tumors with an ATM mutation. The researchers want to find the lo…

United States45 participantsStarted 2021-08-11

Treatment: Palliative radiotherapy

RecruitingNot Applicable

NCT07091617

Testing an Enhanced Digital Delivery Model for Inherited Cancer Genetic Testing in Young Adults With Cancer

This phase III trial compares the use of a digital chatbot enabled intervention to standard remote genetic services for increasing uptake of genetic counseling…

United States535 participantsStarted 2025-12-10

Treatment: Telemedicine, Genetic Testing

RecruitingNot Applicable

NCT06998134

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

People with cerebral palsy (CP), muscular dystrophy (MD), spina bifida, or spinal cord injury often have muscle weakness, and problems moving their arms and leg…

United States23 participantsStarted 2026-05-14

Treatment: Extension assist knee ankle foot orthosis (EA-KAFO)

All recent trials (100 shown)

Not Yet RecruitingNot Applicable

NCT07577466

Screening, Characterization, and Longitudinal Follow-up of Patients With Cardiac Amyloidosis

Cardiac amyloidosis is a progressive disorder caused by extracellular deposition of amyloid fibrils in the heart, leading to heart failure and impaired cardiac…

Germany200 participantsStarted 2026-06-15

Not Yet RecruitingPhase 1

NCT07531940

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

Down syndrome (DS) is typically caused by an extra chromosome 21 in the cell nucleus (trisomy 21, or T21). T21 is both the most common cause of genetically defi…

United States25 participantsStarted 2026-06

Treatment: Memantine

Inherited Genetic Conditions

Trial phase breakdown

Trial status

Actively recruiting — 53 trials

Sinus Disease in Young Children With Cystic Fibrosis

Nomad P-KAFO Study

Randomized, Blinded, Placebo-Controlled Evaluation of BioAmicus Complete

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

Testing an Enhanced Digital Delivery Model for Inherited Cancer Genetic Testing in Young Adults With Cancer

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

All recent trials (100 shown)

Screening, Characterization, and Longitudinal Follow-up of Patients With Cardiac Amyloidosis

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

Trial phase breakdown

Trial status

Actively recruiting — 53 trials

Sinus Disease in Young Children With Cystic Fibrosis

Nomad P-KAFO Study

Randomized, Blinded, Placebo-Controlled Evaluation of BioAmicus Complete

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

Testing an Enhanced Digital Delivery Model for Inherited Cancer Genetic Testing in Young Adults With Cancer

Toward Ubiquitous Lower Limb Exoskeleton Use in Children and Young Adults

All recent trials (100 shown)

Screening, Characterization, and Longitudinal Follow-up of Patients With Cardiac Amyloidosis

Escalating Doses of Memantine in Down Syndrome (MEDS-123)

Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)

Quality of Life in Adults With Untreated Fabry Disease in Sweden (QoLUF)

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Safety and Efficacy of Tofacitinib for Chronic Granulomatous Disease With Inflammatory Complications

Natural History Study of Smith-Magenis Syndrome

Use of a New Smartphone Application to Determine Changes in Eyeblink Conditioning From Home Training in Individuals With…

Study of Brain and Spinal Cord Tumor Growth and Cyst Development in Patients With Von Hippel Lindau Disease

Genetic and Physical Study of Childhood Nerve and Muscle Disorders

AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors

Analysis of Specimens From Individuals With Pulmonary Fibrosis

Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995

Hormonal Regulation of Puberty and Fertility

Examination of Clinical and Laboratory Abnormalities in Patients With Defective DNA Repair: Xeroderma Pigmentosum, Cocka…

Prospective Phenotyping for Genetic Subtypes of Early-onset Atrial Fibrillation

Classification, Functional Stratification and Biomarkers in Ciliopathy (CILLICORIRCM)

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

Early Detection of Concealed Cardiac Amyloidosis Using AI-ECG and CT-Derived Extracellular Volume in Patients With Atria…

A Study To Evaluate The Safety Of CMTX-101 In People With Cystic Fibrosis