Participants are now being recruited for several clinical trials testing investigational treatments for Parkinson’s disease, The Michael J. Fox Foundation (MJFF) announced.
Clinical trials are well-designed experiments conducted on human participants. Since such trials actually test a treatment in people, these studies are essential for understanding whether a given therapy is effective — and, regulatory agencies typically require robust clinical trial data before a treatment can be approved for widespread use.
Two of the trials are testing ways to slow the progression of Parkinson’s. One, an investigator-sponsored Phase 2a trial being conducted at the University of Texas Health Science Center at Houston (NCT04506073), is testing mesenchymal stem cells (MSCs) as a way to slow the progression of the disease.
Like other stem cells, MSCs are able to differentiate into multiple other cell types. In this trial, MSCs are taken from the bone marrow of living donors and administered to participants via intravenous (into-the-bloodstream) infusion. Researchers believe that this treatment could lessen abnormal inflammation, which is characteristic of Parkinson’s. The study aims to recruit 45 people with Parkinson’s, ages 50 to 79. Study contacts are available here.
Another trial (NCT04370665) is evaluating imiglucerase as a potential Parkinson’s treatment. This medication replaces beta-glucocerebrosidase (GCase), a protein thought to play a principal role in Parkinson’s. Raising the levels of this protein may help cells stay healthy and slow disease progression.
The trial will utilize MRI-guided focused ultrasound to get the medication into the brain.
Imiglucerase is approved, under the brand name Cerezyme (by Sanofi Genzyme), to treat Gaucher disease. Mutations in GBA — the gene that encodes GCase — are associated with both Gaucher and Parkinson’s.
Sponsored by InSightec, the trial aims to recruit six people, with ages between 35 and 75, who have been diagnosed with Parkinson’s in the past two years. The Sunnybrook Health Sciences Centre, in Toronto, will host this study; contacts are available here.
The remaining three trials are testing treatments aimed at symptom relief. One Phase 2 study (NCT04334317) is testing TAK-071, an investigational therapy that aims to ease cognitive impairment and reduce falls in people with Parkinson’s. Scientists believe these two symptoms may share an underlying biological cause, which the therapy is designed to target.
Takeda, which is developing TAK-071, is sponsoring this trial in collaboration with the MJFF. It aims to recruit 64 people with Parkinson’s who are experiencing mild cognitive impairment and have had at least two falls within the last six months. Recruitment is currently ongoing at sites in California, Florida, and Michigan, and more locations will be opening in the coming months. More information on study locations is available here.
A Phase 4 clinical trial (NCT03924414) is testing the effect of zoledronic acid on falls in people with Parkinson’s. The medication is currently approved to treat bone damage.
Called TOPAZ, or the Trial of Parkinson’s And Zoledronic Acid, the study is enrolling about 3,500 people. It will take place remotely: interested participants complete an online consent form and video screening visit, and a nurse visits eligible participants’ homes to administer the study medication. More information is available here.
TEMPO-2 (NCT04223193), a Phase 3 study now recruiting, is investigating a medication called tavapadon. This medication mimics the activity of dopamine, a neurotransmitter that is depleted in people with Parkinson’s. The therapy’s goal is to ease Parkinson’s motor symptoms, such as tremors, slowness, and stiffness.
The trial aims to recruit 296 participants who have been diagnosed with Parkinson’s in the last three years and will be held at 12 sites across the U.S. More information on trial sites is available here. Cerevel Therapeutics, which is developing tavapadon, is sponsoring the TEMPO-2 trial.
“We have a responsibility as patients to share our experience — what works for us, what we respond to, what we can contribute to research,” Foundation founder Michael J. Fox says on the organization’s website.
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