A New Approach to Treating Parkinson’s Disease


What is the SKY Study?

SKY (Study of Parkinson’s Early Stage) is looking to enroll patients who were diagnosed with Parkinson’s disease within the last three years.  

Existing treatments for Parkinson’s involve giving drugs that increase dopamine or that copy its action.  

Four things to know about dopamine:

  1. Dopamine is a chemical found in the brain.
  2. It carries signals between nerve cells.
  3. These signals are involved in the control of our movements.
  4. Lack of dopamine causes most motor symptoms of Parkinson’s disease.

SKY takes a different approach by looking at the build-up of iron that is observed in certain regions of the brain among people with Parkinson’s disease.

It is not known whether this build-up of iron is causing harm to the brain cells, or whether removing it may improve the motor symptoms of the disease or slow its progression. The SKY clinical study aims to address these questions. 



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What is the reason for this study?

Parkinson’s disease is associated with excess iron accumulation in certain regions of the brain. It is important to look at whether brain iron accumulation is causing some of the symptoms of Parkinson’s disease and if eliminating the excess iron will improve motor symptoms or slow progression of the disease.

How will iron be removed from brain cells?

The study will use an investigational form of the drug deferiprone. Deferiprone is a drug that attaches itself to iron. The deferiprone-iron combination is then moved away from the brain and naturally eliminated from the body in the urine.

Are there published studies on iron damage on the brain?

It has not yet proven whether removing the excess of iron from the brain will modify the course of Parkinson’s disease but the following publications support the idea that this approach should be further studied.

  • Munoz et al, Parkinson's Disease: The Mitochondria-Iron Link. Parkinsons Dis Epub 2016 May 17
  • Belaidi and Bush, Iron neurochemistry in Alzheimer's disease and Parkinson's disease: targets for therapeutics. J Neurochem. 2015 Nov 6. doi: 10.1111/jnc.13425. [Epub ahead of print]
  • Isaya. Mitochondrial iron-sulfur cluster dysfunction in neurodegenerative disease. Front Pharmacol. 2014 Mar 3;5:29
  • Kumar et al. The role of free radicals in the aging brain and Parkinson's Disease: convergence and parallelism. Int J Mol Sci. 2012;13(8):10478-504
  • Devos et al: D. Devos, C. Moreau, J. C. Devedjian, J. Kluza, M. Petrault, C. Laloux, A. Jonneaux, G. Ryckewaert, G. Garcon, N. Rouaix, A. Duhamel, P. Jissendi, K. Dujardin, F. Auger, L. Ravasi, L. Hopes, G. Grolez, W. Firdaus, B. Sablonniere, I. Strubi-Vuillaume, N. Zahr, A. Destee, J. C. Corvol, D. Poltl, M. Leist, C. Rose, L. Defebvre, P. Marchetti, Z. I. Cabantchik, and R. Bordet. Targeting Chelatable Iron as a Therapeutic Modality in Parkinson's Disease. Antioxid.Redox.Signal., 2014.

Has deferiprone been used in patients before?

Tablet and liquid formulations of deferiprone are approved to treat iron overload in patients with thalassemia, a blood disorder in which patients require frequent blood transfusions, which contributes to an excessive amount of iron in their bodies. Deferiprone has been used for many years to help such patients get rid of extra iron and protect their organs from iron toxicity.

What form of deferiprone is being used in this study?

The drug being used in this study is called deferiprone delayed-release tablets. It is investigational product, meaning that it is not licensed or approved anywhere. So far, it has been tested only in healthy people. This study will look at the safety and effectiveness of this new form of deferiprone in the treatment of Parkinson’s disease.

What is the goal of this study?

The goal of this study is to evaluate a dose of deferiprone that could improve motor symptoms or slow the progression of the disease.

What will happen in the study?

If you are found to be eligible and decide to enroll, you will be assigned randomly (by chance) to take either deferiprone tablets or a placebo. Placebo pills are inactive. They look the same as the pills with active medication but placebos have no therapeutic expectation.

Neither you nor anyone on the study staff will know which of these products you are getting until the study is over.

Four different doses of deferiprone will be tested. Over the 9-month study period, you will be assigned to take one of the following:

  • a half-tablet
  • one tablet
  • one and a half tablets
  • two tablets

What are some basic things to know?

  • You will take your medication at home.
  • Nine (9) visits to the study center are required.
  • Weekly blood tests need to be taken.
  • You can take the blood tests anywhere.
  • Other tests, some more challenging, will be required.

Will it cost me anything to be in the study? Or will I be paid to be in it?

Enrolling in this study is free. There will be no charge to you for any of the study visits, medications, tests, and procedures.

You will not be paid to be in the study, but you may be reimbursed for expenses such as parking, or bus fare.

Who can be in the SKY study?

Participants in this study must:

  • Have received a diagnosis of Parkinson’s disease within the last three years.
  • Have been on a stable dose of certain Parkinson’s medicines for at least the last three months.
  • Be between the ages of 18 and 80 years.
For more details regarding inclusion in this study please refer to:

May I take other medications while I am in this study?

You must continue taking the exact type and dosage of your Parkinson’s medication for the entire time that you are in the study.

If the study doctor feels that you must change anything about your Parkinson’s treatments, you will need to drop out of the study.

Some drugs to treat other medical problems will be allowed during the study and some not. All medications that you are taking must be discussed with the study doctor. If during the study you require any new medications for other conditions, you may have to discontinue participation in the study.

Why should I participate if I may not get the active medication?

  • To learn if a medication is effective and safe, it must be compared against a placebo.
  • Even if you are randomly assigned a placebo, your participation in the trial is important to this trial and future investigations of new treatments.
  • Everyone participating in a clinical trial has an important role in advancing the treatment and cure of diseases, even those who may be assigned a dose that is later determined to be too low or too high.
  • In the SKY study you will have a 4:1 chance of getting active drug. This means you have 4 times the chance of taking a dose of deferiprone verses 1 chance of getting placebo.

Visit for comprehensive information about enrolling in a clinical trial.

Where will the study be done, and how many people will be in it?

The SKY study will be conducted at sites in North America and Europe. It is planned that a total of 140 people will take part.

I’ve never been involved in a clinical study. How can I learn more?

Visit for comprehensive information about enrolling in a clinical trial.

How do I enroll? 

The locations of the study centers can be found through either of the two links below:

Participants in any clinical trial will be asked to sign an informed consent document, which explains the study in detail and describes its risks and benefits.  The study leaders will spend time reviewing this document with you.  It is important that you take the time to carefully consider every aspect of the study before you decide to enroll.  You should have all your questions addressed before you sign the document. 

Learn more about Informed Consent at:


How Will the Safety of Patients in This Study Be Protected?

The independent safety board watches over the safety of the study, from before it is approved until the last patient has completed it. 

The safety board members have the authority to put a study on hold or stop it entirely if they have serious safety concerns.

In addition, if the study doctor has concerns about a particular patient’s safety, that patient may be taken out of the study even if he or she wishes to remain.