NCT03652363

GDNF in ideopathicParkinsons Disease

Completed Phase 2 Last updated 29 August 2018

What this trial tests

Phase 2 trial testing glial cell line-derived neurotrophic factor in Idiopathic Parkinson Disease in 42 participants. Completed in 1 April 2016.

Timeline

25 October 2012

Primary endpoint
13 March 2015

1 April 2016

Quick facts

Lead sponsor	North Bristol NHS Trust
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	quadruple
Primary purpose	treatment
Enrollment	42
Start date	25 October 2012
Primary completion	13 March 2015
Estimated completion	1 April 2016

Drugs / interventions tested

• glial cell line-derived neurotrophic factor — full drug profile →

Conditions studied

• Idiopathic Parkinson Disease — all drugs for Idiopathic Parkinson Disease →

Sponsor

North Bristol NHS Trust — full company profile →

Who can join

Adults 35 to 75, any sex, with Idiopathic Parkinson Disease. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

A Placebo Controlled Randomised Trial of GDNF vs placebo

The study will require patients to undergo surgery to implant microcatheters precisely into the brain. Patients will then attend clinic on a 2 weekly basis for infusions of a nerve growth factor called GDNF or placebo. Specific tests will also be carried out at regular intervals to assess your symptoms. All participants will undergo radio-isotope brain imaging at the beginning and end of the study. Periodically patients will also be required to undergo an MRI scan to assess the delivery of the study drug or placebo.

Publications & conference data

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

1. GDNF and Parkinson's Disease: Where Next? A Summary from a Recent Workshop.
   Barker RA, Björklund A, Gash DM, Whone A, et al · · 2020 · cited 100× · PMID 32508331 · DOI 10.3233/jpd-202004
2. Netrin-1 and its receptor DCC modulate survival and death of dopamine neurons and Parkinson's disease features.
   Jasmin M, Ahn EH, Voutilainen MH, Fombonne J, et al · · 2021 · cited 63× · PMID 33351190 · DOI 10.15252/embj.2020105537
3. CDNF Protein Therapy in Parkinson's Disease.
   Huttunen HJ, Saarma M. · · 2019 · cited 60× · PMID 30947516 · DOI 10.1177/0963689719840290
4. Neurotrophic factors for disease-modifying treatments of Parkinson's disease: gaps between basic science and clinical studies.
   Chmielarz P, Saarma M. · · 2020 · cited 40× · PMID 32700249 · DOI 10.1007/s43440-020-00120-3
5. On the Right Track to Treat Movement Disorders: Promising Therapeutic Approaches for Parkinson's and Huntington's Disease.
   Troncoso-Escudero P, Sepulveda D, Pérez-Arancibia R, Parra AV, et al · · 2020 · cited 22× · PMID 33101007 · DOI 10.3389/fnagi.2020.571185
6. Mechanistic Insight from Preclinical Models of Parkinson's Disease Could Help Redirect Clinical Trial Efforts in GDNF Therapy.
   Delgado-Minjares KM, Martinez-Fong D, Martínez-Dávila IA, Bañuelos C, et al · · 2021 · cited 16× · PMID 34769132 · DOI 10.3390/ijms222111702
7. Overcoming the Blood-Brain Barrier for Drug Delivery to the Brain.
   Katz JS, Slika H, Sattari SA, Malla AP, et al · · 2025 · cited 4× · PMID 40787406 · DOI 10.1021/acsomega.5c00364
8. Redefining Parkinson's Disease management: the synergistic role of neurotrophic factors and mitochondria.
   Chang J, Li Z, Liu T, Cheng X, et al · · 2026 · PMID 41814010 · DOI 10.1007/s00018-026-06152-x