Clinical Trials

Now that insight into the disease mechanisms and clinical disease course of Vanishing White Matter (VWM) have improved over the past decade, worldwide efforts focus on treatment opportunities for VWM. This page lists the current clinical trials for VWM.

Current Trial

Calico starts phase 1b study evaluating safety, tolerability and pharmacokinetics of eIF2B activator.

A clinical trial evaluating the safety, tolerability and pharmacokinetics of ABBV-CLS-7262, an investigational drug, in people with Vanishing White Matter has been initiated and posted on ClinicalTrials.gov, the U.S. NIH registry of clinical trials “Phase 1b Study to Investigate ABBV-CLS-7262’s Safety, Tolerability & Pharmacokinetics in Vanishing White Matter Patients.” ABBV-CLS-7262 is being developed by Calico Life Sciences (Calico) in collaboration with AbbVie. Calico will continue to partner with the VWM Consortium on researching ABBV-CLS-7262 for VWM disease and will provide periodic updates on the status of this clinical research via the News & Updates section of the VWM Consortium website.

The most up to date information in relation to the above mentioned clinical trial can be found by going to www.clinicaltrials.gov and entering “NCT05757141” in the “Other terms” field and then clicking the blue “Search” button at the bottom of the page. Clicking on the link shown in the results table takes you to the study record detail page where you can see currently recruiting sites or find information under ‘Contacts’ if you have questions about this trial and your potential participation.

Current Trial

Guanabenz trial for VWM

The Amsterdam Leukodystrophy Center at the Amsterdam University Medical Centers (Amsterdam UMC) is conducting a clinical trial in children with Vanishing White Matter (VWM). In this trial, they want to evaluate whether a drug called Guanabenz is effective in slowing progression, stabilizing, or even improving the brain white matter abnormalities in VWM. Guanabenz is an old and well-known medicine that has been used for decades for the treatment of high blood pressure. It has been approved by the FDA, the American medicines agency.

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More is coming soon…

This overview will continuously be updated with the latest clinical trials. As soon as clinical trials with new treatment options for VWM are initiated, the studies will be listed on this page.