Back to search
Inherited Metabolic Diseases
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Metabolic Diseases trials may be worth asking aboutClinical trial pipeline · Data from ClinicalTrials.gov
See which Inherited Metabolic Diseases trials may be worth asking aboutNorth America
Europe
Asia-Pacific
Rest of World
Trials may have sites in multiple countries. Filtering shows any trial with at least one site in the selected country.
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).
The iCaRe2 is a multi-institutional resource created and maintained by the Fred \& Pamela Buffett Cancer Center to collect and manage standardized, multi-dimens…
This study aims to gather safety data and determine the optimal dosing regimen for PYC-001 in participants with confirmed OPA1 mutation-associated ADOA. Approxi…
RATIONALE: Gathering information about older patients with cancer may help the study of cancer in the future. PURPOSE: This research study is gathering informa…
VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial…
This is a single-site Phase 1/2 open-label umbrella clinical trial designed to evaluate the safety, tolerability, and efficacy of a single intravenous dose of L…
Background: Clinical Genetics Branch (CGB) researchers study individuals and populations at high genetic risk of cancer in order to improve our understanding o…