A Study of Intrathecal SHP611 in Children With Metachromatic Leukodystrophy
Completed · Phase 2 · NCT03771898
Conditions: Metachromatic Leukodystrophy (MLD)
At a glance
- Research payments reported, 2025
- $2.3M
- U.S. investigators named
- 9
- Sites paid
- 14
- Planned or actual enrollment
- 36
About the trial
- Sponsor
- Shire (with Takeda Development Center Americas, Inc.)
- Tested
- SHP611 (drug)
- Dates
- Started 2019-05-13; completion 2026-05-06
- Results posted
- Yes
The main aim of the study is to determine if SHP611 given by injection into the spinal fluid that surrounds the brain and spinal cord (intrathecal; IT) prolongs the time for children with Metachromatic Leukodystrophy (MLD) to retain the ability to move from place to place. Other aims of the study are to determine the effects of intrathecal administration of SHP611 on movement and speech functions and to learn how well SHP611 injected in the spinal fluid that surrounds the brain and spinal cord is tolerated. Study participants will receive SHP611 for about 2 years with the possibility of an extended treatment period.
From ClinicalTrials.gov, last updated 2026-07-10. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Takeda Pharmaceuticals U.S.A., Inc. reported $2.3M in research payments for this study in 2025 (118 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Agnes Chen, MD Child Neurology · Torrance, CA
- Scott Demarest, MD Neurology · Aurora, CO
- Han C. Phan, MD Child Neurology · Atlanta, GA
- John A. Bernat, MD,PHD Medical Genetics · Iowa City, IA
- Deepa Soundara Rajan Pediatrics · Detroit, MI
- Marc C. Patterson, MD Neurology · Rochester, MN
- Devorah Segal, M.D., PH.D. Child Neurology · New York, NY
- Adeline Vanderver, MD Child Neurology · Philadelphia, PA
- Joshua L. Bonkowsky, MD, PHD Child Neurology · Salt Lake City, UT
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- Children's Hospital of Philadelphia, PA $1.0M
- Rare Disease Research LLC $322,838
- NYU Langone Hospitals, NY $281,785
- University of Iowa Hospital & Clinics, IA $220,498
- University of Pittsburgh Physicians $134,274
- Univ of Pittsburgh Physicians $100,488
- Lundquist Institute for Biomedical Innovation at Harbor UCLA Medical Center $51,257
- New York University NYUlmc $49,697
- Lundquist Institute for Biomed $43,821
- Lac/harbor-UCLA Med Center, CA $39,089
- Children's Hospital Colorado, CO $11,340
- University of Utah Hospital and Clinics, UT $7,040
- Los Angeles Biomedical $1,375
- Mayo Clinic Hospital Rochester, MN $700
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.