Clinical Trials for Cystinosis

Use of Cysteamine in the Treatment of Cystinosis

NCT ID: NCT00359684

This clinical trial is studying the use of a medication called cysteamine to treat cystinosis, a rare inherited disease that causes problems like poor growth and kidney failure due to the buildup o...

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford

NCT ID: NCT01793168

The Coordination of Rare Diseases at Sanford (CoRDS) is a clinical trial aimed at creating a large, centralized registry for individuals affected by rare diseases. This registry helps connect patie...

Baby Detect : Genomic Newborn Screening

NCT ID: NCT05687474

The Baby Detect trial is studying a new way to screen newborns for serious genetic conditions that can be treated early. By using a simple blood test from babies within their first 28 days of life,...

Early Check: Expanded Screening in Newborns

NCT ID: NCT03655223

European Cystinosis Cohort

NCT ID: NCT05901077

The European Cystinosis Cohort study is looking to better understand cystinosis, a rare condition that affects how the body breaks down a substance called cystine. This disease can cause serious he...

PK and PD Study of NPI-001 and Cysteamine Bitartrate

NCT ID: NCT05994534

This clinical trial is studying a new oral solution called NPI-001 as a treatment for cystinosis, a condition that affects the kidneys. Researchers want to learn about how safe this medication is a...

Development of Health-related Quality of Life Instrument for Patients With Cystinosis

NCT ID: NCT05959668

This clinical trial is focused on creating a special questionnaire to understand and measure the quality of life for patients with cystinosis, a rare inherited disorder that affects how the body pr...

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

NCT ID: NCT05146830

National Registry of Rare Kidney Diseases

NCT ID: NCT06065852

The National Registry of Rare Kidney Diseases is a research project aimed at gathering information from patients who have specific rare kidney diseases. By collecting this data, the researchers hop...

Early Trial of Allogeneic Hematopoietic Stem Cell Transplantation for Patients Who Will Receive a Kidney Transplant From the Same Donor

NCT ID: NCT05508009

This clinical trial is studying a new approach to help patients who need a kidney transplant (KT) by using a special type of stem cell transplant first. The goal is to prevent the body from rejecti...

Genetic Newborn Screening for Cystinosis and Primary Hyperoxaluria

NCT ID: NCT05843851

This clinical trial is exploring the possibility of adding genetic tests for two rare inherited diseases—cystinosis and primary hyperoxaluria—to the routine newborn screening program in Germany. Cu...