NCT00638547

Intrathecal Enzyme Replacement for Hurler Syndrome

Completed Phase 1 Last updated 3 December 2018

What this trial tests

Phase 1 trial testing IRT Laronidase in Hurler Syndrome in 26 participants. Completed in 18 November 2018.

Timeline

2 January 2008

Primary endpoint
18 February 2017

18 November 2018

Quick facts

Lead sponsor	Masonic Cancer Center, University of Minnesota
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	26
Start date	2 January 2008
Primary completion	18 February 2017
Estimated completion	18 November 2018
Sites	1 location across United States

Drugs / interventions tested

• IRT Laronidase — full drug profile →

Conditions studied

• Hurler Syndrome — all drugs for Hurler Syndrome →

Sponsor

Masonic Cancer Center, University of Minnesota

Who can join

Adults 6 Months to 3, any sex, with Hurler Syndrome. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Mucopolysaccharidosis type III (Sanfilippo syndrome) and misdiagnosis of idiopathic developmental delay, attention deficit/hyperactivity disorder or autism spectrum disorder.
   Wijburg FA, Węgrzyn G, Burton BK, Tylki-Szymańska A. · · 2013 · cited 98× · PMID 23336697 · DOI 10.1111/apa.12169
2. Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.
   Hampe CS, Wesley J, Lund TC, Orchard PJ, et al · · 2021 · cited 59× · PMID 33572941 · DOI 10.3390/biom11020189
3. Open issues in Mucopolysaccharidosis type I-Hurler.
   Parini R, Deodato F, Di Rocco M, Lanino E, et al · · 2017 · cited 53× · PMID 28619065 · DOI 10.1186/s13023-017-0662-9
4. Mucopolysaccharidosis Type I.
   Kubaski F, de Oliveira Poswar F, Michelin-Tirelli K, Matte UDS, et al · · 2020 · cited 47× · PMID 32188113 · DOI 10.3390/diagnostics10030161
5. Mucopolysaccharidoses I and II: Brief Review of Therapeutic Options and Supportive/Palliative Therapies.
   Nan H, Park C, Maeng S. · · 2020 · cited 27× · PMID 33344633 · DOI 10.1155/2020/2408402
6. Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes.
   Eisengart JB, Pierpont EI, Kaizer AM, Rudser KD, et al · · 2019 · cited 23× · PMID 31019279 · DOI 10.1038/s41436-019-0522-1
7. Research challenges in central nervous system manifestations of inborn errors of metabolism.
   Dickson PI, Pariser AR, Groft SC, Ishihara RW, et al · · 2011 · cited 18× · PMID 21176882 · DOI 10.1016/j.ymgme.2010.11.164
8. Targeted enzyme delivery systems in lysosomal disorders: an innovative form of therapy for mucopolysaccharidosis.
   Safary A, Akbarzadeh Khiavi M, Omidi Y, Rafi MA. · · 2019 · cited 17× · PMID 31101939 · DOI 10.1007/s00018-019-03135-z