Clinical trial NCT03347955: Double-blind Placebo Surgery Trial for Treatment of Parkinson's Disease
Official title: Transplantation of Embryonic Dopamine Neurons for Treatment of Parkinson's Disease: Double-blind Placebo Surgery Trial
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: NA
Conditions: Parkinson Disease
Surgery
Keywords: placebo effect
Lead sponsor: University of Denver
Sponsor class: OTHER
First posted: 2017-11-20
Enrollment: 40
Sex: ALL
Minimum age (years): 30
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: In this double-blind study, 20 participants were randomly assigned to the treatment group and 20 to the sham surgery group with the blind being maintained for 12 months.
Primary purpose: TREATMENT

Brief summary: A double-blind sham surgery-controlled trial was developed to determine the effectiveness of implantation of human embryonic dopamine neurons into the putamen of patients with advanced Parkinson's disease (PD), with half the patients receiving the implant (n = 20) and half receiving sham surgery (n = 20). The blind was maintained for 12 months before participants were told which surgery they received, at which time those receiving sham surgery could request the implant surgery.

Detailed description: A double-blind sham surgery-controlled trial was conducted to determine the effectiveness of implantation of human embryonic dopamine neurons into the putamen of patients with advanced Parkinson's disease (PD), with half the patients receiving the implant (n = 20) and half receiving sham surgery (n = 20). The goals were to determine whether the cells survived and led to improvement of symptoms, and to examine the effect of age on results of the implantation. Quality of life of participants and care partners was also examined at each of the assessment periods (baseline, 4, 8 and 12 months). The blind was maintained for 12 months.

Eligibility criteria: Inclusion Criteria:

* Diagnosed with Parkinson's disease (PD) for more than 7 years, improvement in response to levodopa, Positive Emission Tomography scan compatible with presence of PD, improvement of at least 33% on UPDRS after first morning dose of levodopa

Exclusion Criteria:

* Mini-mental status exam score of less than 24, hallucinations during levodopa therapy, epilepsy, previous brain surgery, severe depression, another neurologic disorder, cardiovascular disease, and medical contraindication of surgery

Primary outcomes:
measure: Global Rating Scale
timeFrame: Same scale was administered 7 days after surgery and prior to follow-up visits at Columbia at 4, 8, and 12 months after surgery and before the blind was lifted.
Secondary outcomes:
Other outcomes:
Locations:
city: Denver
country: United States
facility: Morgridge College of Education, University of Denver
state: Colorado
Study officials:
affiliation: University of Denver
name: Cynthia McRae, PhD
role: PRINCIPAL_INVESTIGATOR
affiliation: Neurological Institute, Columbia University
name: Stanley Fahn, MD
role: STUDY_CHAIR
affiliation: University of Colorado Health Sciences
name: Curt Freed, MD
role: STUDY_DIRECTOR
References:
citation: McRae C, Cherin E, Yamazaki TG, Diem G, Vo AH, Russell D, Ellgring JH, Fahn S, Greene P, Dillon S, Winfield H, Bjugstad KB, Freed CR. Effects of perceived treatment on quality of life and medical outcomes in a double-blind placebo surgery trial. Arch Gen Psychiatry. 2004 Apr;61(4):412-20. doi: 10.1001/archpsyc.61.4.412.
citation: Freed CR, Greene PE, Breeze RE, Tsai WY, DuMouchel W, Kao R, Dillon S, Winfield H, Culver S, Trojanowski JQ, Eidelberg D, Fahn S. Transplantation of embryonic dopamine neurons for severe Parkinson's disease. N Engl J Med. 2001 Mar 8;344(10):710-9. doi: 10.1056/NEJM200103083441002.