Newborn Diseases

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Newborn Diseases trials you may qualify for

100

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 47 trials

RecruitingNot Applicable

NCT02069756

The Duchenne Registry

The Duchenne Registry is an online, patient-report registry for individuals with Duchenne and Becker muscular dystrophy and carrier females. The purpose of the…

United States10,000 participantsStarted 2007-10

RecruitingNot Applicable

NCT07576504

Sensorimotor Technique on Navicular Drop in School Going Children

The goal of the study is to determine effects of sensorimotor technique (SMT) program with short foot exercises on navicular drop and foot posture in school goi…

Pakistan22 participantsStarted 2026-04-14

Treatment: sensorimotor technique, short foot exercise

RecruitingPhase 2

NCT06747858

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

ARCT-032-02 is a Phase 2, open-label, multicenter, multiple-ascending dose study of ARCT-032 in adults with CF who are not eligible for CFTR modulator therapy o…

United States33 participantsStarted 2024-12-12

Treatment: ARCT-032

RecruitingPhase 3

NCT06247085

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Ag…

The purpose of this study is to measure efficacy and safety of pegtibatinase treatment compared with placebo in participants with classical HCU receiving standa…

United States70 participantsStarted 2023-12-28

Treatment: Pegtibatinase, Placebo

RecruitingNot Applicable

NCT05612256

Electrical Impedance Tomography for Identification of Optimal Positive End-expiratory Pressure in Newborn Infants

Electrical impedance tomography (EIT) enables assessment of regional lung ventilation at the bedside. EIT has been safely used in newborn infants to image intra…

Switzerland86 participantsStarted 2022-12-10

Treatment: Electrical impedance tomography (EIT), Forced oscillation technique (FOT)

RecruitingPhase 2

NCT07450547

Phase 2 Study to Assess the Safety and Efficacy of ANG003

In this study, ANG003, a pancreatic enzyme replacement therapy (PERT; commonly called "enzymes"), is being investigated as a potential treatment for exocrine pa…

United States113 participantsStarted 2026-04-24

Treatment: ANG003 Dose A, ANG003 Dose B

All recent trials (100 shown)

Active — Not RecruitingPhase 3

NCT05926583

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

The purpose of the study is to assess the safety and tolerability of bilateral subretinal delivery of adeno-associated virus vector with a serotype 5 capsid hum…

Japan4 participantsStarted 2023-09-12

Treatment: AAV5-hRKp.RPGR, AAV5-hRKp.RPGR

CompletedPhase 2

NCT05059041

Dilated Versus Non-Dilated Wavefront Corrections for Patients With Down Syndrome

Individuals with Down syndrome (DS) live with visual deficits due, in part, to elevated levels of higher-order optical aberrations (HOA). HOAs are distortions/a…

United States42 participantsStarted 2022-05-06

Treatment: Dilated Refraction, Non-Dilated Refraction

Newborn Diseases

Trial phase breakdown

Trial status

Actively recruiting — 47 trials

The Duchenne Registry

Sensorimotor Technique on Navicular Drop in School Going Children

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Ag…

Electrical Impedance Tomography for Identification of Optimal Positive End-expiratory Pressure in Newborn Infants

Phase 2 Study to Assess the Safety and Efficacy of ANG003

All recent trials (100 shown)

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Dilated Versus Non-Dilated Wavefront Corrections for Patients With Down Syndrome

Trial phase breakdown

Trial status

Actively recruiting — 47 trials

The Duchenne Registry

Sensorimotor Technique on Navicular Drop in School Going Children

Safety, Tolerability and Efficacy Study of ARCT-032 in People With Cystic Fibrosis

A Study to Investigate Efficacy and Safety of Pegtibatinase Compared With Placebo in Participants ≥12 to ≤65 Years of Ag…

Electrical Impedance Tomography for Identification of Optimal Positive End-expiratory Pressure in Newborn Infants

Phase 2 Study to Assess the Safety and Efficacy of ANG003

All recent trials (100 shown)

A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa

Dilated Versus Non-Dilated Wavefront Corrections for Patients With Down Syndrome

Alveolar Cleft Repair Using Osteoinductive Ceramics

The Effect of Music and Musical Mobile Interventions on Pain and Physiological Parameters

The Use of Wireless Sensors in Neonatal Intensive Care

Human Milk Concentrating Device to Optimize Mother's Own Milk

Ultrasound-Guided Pudendal vs Dorsal Penile Nerve Block for Pediatric Hypospadias Surgery

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

Prognostic Value of Myocardial Fibrosis Quantified Using CMR in Patient With Dilated Cardiomyopathy

Pharmacokinetics of Omadacycline in Cystic Fibrosis

Observational Study of Subcutaneous Immunoglobulin (Cutaquig) in Patients With Primary and Secondary Immunodeficiency.

A Study of Navenibart (STAR-0215) in Participants With Hereditary Angioedema

A Study Evaluating APG777 in Atopic Dermatitis

A Study of Erdafitinib Intravesical Delivery System in Japanese Participants With Bladder Cancer

Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis

Estimation of Fetal Weight by Measuring Abdominal Circumference, Abdominal Subcutaneous Soft Tissue Thickness, Femur Len…

Individual Patient Compassionate Use of Mirdametinib

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Gait in Young Children With NF1

INcreased Sun Exposure Without Pain In Research Participants With EPP or XLP