🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

Find Clinical Trials for Your Rare Disease

16+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

Clinical trial records are synced from ClinicalTrials.gov.
Report missing data
Clear

Showing 16 of 16 recruiting trials for “metachromatic-leukodystrophy-adult-form

NARecruitingNCT07046338

Lentiviral Hematopoietic Stem Cell Gene Therapy for MLD

👨‍⚕️ Lung-Ji Chang, Ph.D📍 1 site📅 Started Jun 2025View details ↗
NARecruitingNCT03725670

Direct Lentiviral Injection Gene Therapy for MLD

👨‍⚕️ Lung-Ji Chang, Ph.D, Shenzhen Geno-Immune Medical Institute📍 1 site📅 Started May 2025View details ↗
Phase 1, PHASE2Enrolling by InvitationNCT06369974

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

👨‍⚕️ Florian Eichler, MD, Massachusetts General Hospital📍 1 site📅 Started Sep 2024View details ↗
NARecruitingNCT06224413

A Study of Participants With Cerebral Adrenoleukodystrophy (CALD) Treated With Elivaldogene Autotemcel

👨‍⚕️ Kiran Bhirangi, MD, bluebird bio, Inc.📍 3 sites📅 Started Mar 2024View details ↗
RecruitingNCT06190834

Relationship Between High-Density Lipoprotein Subtypes and Coronary Heart Disease Prognosis.

👨‍⚕️ Yu Geng, Beijing Tsinghua Changgeng Hospital📍 1 site📅 Started Dec 2023View details ↗
Phase 3RecruitingNCT05819866

A Clinical Study to Assess the Efficacy and Safety of Leriglitazone in Adult Male Subjects With Cerebral Adrenoleukodystrophy

🏥 Minoryx Therapeutics, S.L.📍 13 sites📅 Started Jul 2023View details ↗
Phase 1, PHASE2RecruitingNCT05757141

An Open-Label Exploratory Study of Fosigotifator in Participants With Vanishing White Matter Disease

👨‍⚕️ ABBVIE INC., AbbVie📍 5 sites📅 Started Mar 2023View details ↗
NARecruitingNCT05420064

An Intervention to Increase Genetic Testing in Families Who May Share a Gene Mutation Related to Cancer Risk and An Intervention to Help Patients and Their Primary Care Providers Stay Up-to-date About Uncertain Genetic Test Results

👨‍⚕️ Kenneth Offit, MD, MPH, Memorial Sloan Kettering Cancer Center📍 8 sites📅 Started Dec 2022View details ↗
RecruitingNCT05103969

Cohort of Tumors With POLE/D1 Mutation

👨‍⚕️ Rosine GUIMBAUD, PhD.MD, Fédération francophone de Cancérologie digestive📍 1 site📅 Started Oct 2021View details ↗
RecruitingNCT04925349

Modeling Macrophages Activation Pattern in X-linked Adrenoleukodystrophy, Metachromatic Leukodystrophy and Adult Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

👨‍⚕️ Fanny MOCHEL, MCU-PH, Institut du Cerveau et de la Moëlle épinière📍 2 sites📅 Started Aug 2021View details ↗
Enrolling by InvitationNCT04503213

A Study to Assess CSF1R-related Leukoencephalopathy After Stem Cell Transplantation

👨‍⚕️ Zbigniew K Wszolek, MD, Mayo Clinic📍 1 site📅 Started Jul 2020View details ↗
Phase 2RecruitingNCT03428802

Pembrolizumab in Treating Participants With Metastatic, Recurrent or Locally Advanced Cancer and Genomic Instability

👨‍⚕️ Eugenia Girda, MD, Rutgers Cancer Institute of New Jersey📍 2 sites📅 Started Mar 2018View details ↗
NARecruitingNCT03047369

The Myelin Disorders Biorepository Project

👨‍⚕️ Adeline Vanderver, MD, Children's Hospital of Philadelphia📍 20 sites📅 Started Dec 2016View details ↗
Phase 1RecruitingNCT02254863

UCB Transplant of Inherited Metabolic Diseases With Administration of Intrathecal UCB Derived Oligodendrocyte-Like Cells

👨‍⚕️ Joanne Kurtzberg, MD, Duke University📍 1 site📅 Started Sep 2014View details ↗
RecruitingNCT03333200

Longitudinal Study of Neurodegenerative Disorders

🏥 University of Pittsburgh📍 1 site📅 Started Jan 2012View details ↗
RecruitingNCT03639285

Natural History, Diagnosis, and Outcomes for Leukodystrophies

🏥 University of Utah📍 1 site📅 Started Jan 2007View details ↗

Get alerts when new trials open

Create a free account to follow conditions and receive trial notifications.

Create free account →