Menkes Disease

Clinical trial pipeline · Data from ClinicalTrials.gov

See which Menkes Disease trials you may qualify for

Total trials

Actively recruiting

Phase 3 trials

Completed

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

RecruitingPhase 1/2

NCT07398508

Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.

This clinical trial will evaluate the safety, tolerability, dosing, and efficacy of Northera (Droxidopa) in children with Menkes disease aged 7 to 17 years who…

United States6 participantsStarted 2026-06-01

Treatment: Droxidopa Oral Product, Placebo Control

All recent trials (9 shown)

CompletedPhase 1/2

NCT04977388

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

The purpose of this study is to evaluate whether Northera (Droxidopa) is safe and effective in young adults with Menkes disease who survived the most severe com…

United States3 participantsStarted 2021-07-12

Treatment: Droxidopa, Placebo

APPROVED_FOR_MARKETINGNot Applicable

NCT04074512

Copper Histidinate Treatment for Menkes Disease

This expanded access protocol provides subcutaneous copper histidinate for Menkes disease patients under 6 years of age.

United States

Treatment: copper histidinate

Menkes Disease

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.

All recent trials (9 shown)

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

Copper Histidinate Treatment for Menkes Disease

Trial phase breakdown

Trial status

Actively recruiting — 1 trial

Phase I/II Study of NORTHERA (DROXIDOPA) for Dysautonomia in Pediatric Survivors of Menkes Disease.

All recent trials (9 shown)

NORTHERA (DROXIDOPA) for Dysautonomia in Adult Survivors of Menkes Disease and Occipital Horn Syndrome

Copper Histidinate Treatment for Menkes Disease

Long Term Follow-up on Menkes Disease Patients

Baby Detect : Genomic Newborn Screening

Early Check: Expanded Screening in Newborns

Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome

Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency

Copper Histidine Therapy for Menkes Diseases