Double-blind Placebo Surgery Trial for Treatment of Parkinson's Disease

NCT03347955 | Completed

• 1 other identifier: R29NS032009-05
• Study Type: interventional
• Target: 40
• Locations: 1 country
• Sites: 1

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.

Conditions

Parkinson Disease; Surgery

Keywords

placebo effect

Outcome Measures

Primary Outcomes (1)

• Global Rating Scale

  one item scale where participants marked their answer on a scale ranging from -3 (much worse since surgery) to 0 (no change) to +3 (much improved since surgery)

  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.

Study Arms (2)

Neural implantation group

EXPERIMENTAL

Human embryonic dopamine neurons were implanted into brains of half the randomized participants (n = 20). Participants were evaluated at baseline, 4, 8, and 12 months after surgery.

Procedure: Neural implantation group

Sham Surgery group

SHAM COMPARATOR

This group (n = 20) received sham surgery with a steel frame affixed to their heads and four burr holes drilled into their foreheads without crossing the blood/brain barrier. Participants were assessed at baseline, 4, 8, and 12 months after surgery.

Procedure: Sham surgery

Interventions

Neural implantation group PROCEDURE

Cultured mesencephalic tissue from four embryos was implanted into the putamen bilaterally for those receiving the implant

Neural implantation group

Sham surgery PROCEDURE

Steel frame affixed to head and four burr holes drilled into forehead of sham surgery patients

Sham Surgery group

Eligibility Criteria

• Age: 30 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Denver: lead

Study Sites (1)

Morgridge College of Education, University of Denver

Denver, Colorado, 80208, United States

Location

Related Publications (2)

• 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.

  PMID: 15066900 BACKGROUND

• 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.

  PMID: 11236774 RESULT

Related Links

• Quality of life report on double-blind fetal transplant surgery for treatment of Parkinsons disease
• Neurological and medical results of double-blind fetal transplant surgery as treatment for Parkinson's disease

MeSH Terms

Conditions

Parkinson Disease

Condition Hierarchy (Ancestors)

Parkinsonian Disorders; Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Movement Disorders; Synucleinopathies; Neurodegenerative Diseases

Study Officials

• Cynthia McRae, PhD

  University of Denver

  PRINCIPAL INVESTIGATOR

• Stanley Fahn, MD

  Neurological Institute, Columbia University

  STUDY CHAIR

• Curt Freed, MD

  University of Colorado Health Sciences

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Masking Details: All of the above were "masked." Only the neurosurgeon (Dr. Breeze) who did the implant and the person in charge of random assignment were aware of which participant received which treatment.
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Research Professor

Model Details: 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.

Study Record Dates

• First Submitted: November 13, 2017
• First Posted: November 20, 2017
• Study Start: November 1, 1994
• Primary Completion: February 1, 1999
• Study Completion: February 1, 1999
• Last Updated: November 20, 2017

Record last verified: 2017-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)