Experimental spinal injection aims to keep kids with rare disease moving
NCT ID NCT03771898
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a drug called SHP611, given as a spinal injection, in 36 children with late infantile metachromatic leukodystrophy (MLD), a rare and severe brain disease. The main goal is to see if the treatment helps children keep their ability to walk and move for as long as possible. The study compares their progress to similar children who did not receive the drug.
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Attikon University General Hospital
Chaïdári, Attica, 124 62, Greece
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Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B4 6NH, United Kingdom
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British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4, Canada
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CHU Lenval
Nice, 06200, France
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Children's Hospital of Pittsburgh
Pittsburgh, Pennsylvania, 15224, United States
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Childrens Hospital Colorado
Aurora, Colorado, 80045, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Hospital Universitario Austral - PIN
Ciudad Autónoma Buenos Aires, Buenos Aires, B1629AHJ, Argentina
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Hospital Universitario Cruces
Barakaldo, Vizcaya, 48903, Spain
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Hospital Vall d'Hebrón
Barcelona, 8035, Spain
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Hospital de Clínicas de Porto Alegre
Porto Alegre, 90035-903, Brazil
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Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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Hôpital Bicêtre - Paris Sud
Le Kremlin-Bicêtre, 94275, France
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IRCCS Ospedale Pediatrico Bambino Gesù - INCIPIT - PIN
Roma, 165, Italy
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Kanazawa University Hospital
Kanazawa, 920-8641, Japan
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Los Angeles Biomedical Research Institute at Harbor-UCLA
Torrance, California, 90502, United States
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Mayo Clinic - PPDS
Rochester, Minnesota, 55905, United States
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Montreal Children's Hospital
Montreal, Quebec, H3H 1P3, Canada
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New York University Langone Medical Center
New York, New York, 10016, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30303, United States
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Stollery Children's Hospital University of Alberta
Edmonton, Alberta, T6G 2R7, Canada
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Tel Aviv Sourasky Medical Center
Tel Aviv, 64239, Israel
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UZ Antwerpen
Edegem, 2650, Belgium
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
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University of Utah
Salt Lake City, Utah, 84108, United States
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Universitätsklinikum Hamburg Eppendorf
Hamburg, 20246, Germany
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Universitätsklinikum Tübingen
Tübingen, 72076, Germany
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VU Medisch Centrum
Amsterdam, 1081 HV, Netherlands