Parkinson's DiseasePhase 2SKYNCT02728843
Study of Parkinson's Early Stage With Deferiprone
A Phase 2 study testing Deferiprone in people with Parkinson's Disease, run by ApoPharma at 20 sites in 4 countries.
Trial summary
The goal of this study is to evaluate the effects of deferiprone, an iron-chelating drug, in patients with Parkinson's disease. Participants will be randomized to receive one of four different dosages of deferiprone or placebo, and will take the assigned study product twice a day for nine months. Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Parkinson's Disease
- What is being tested?
- Deferiprone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 140 participants
- Where
- 20 sitesin 4 countries
- Study dates
- 12 Oct 2016to 4 Sep 2019
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Male or female aged ≥18 to < 80 years
- Body weight ≥60 kg but ≤100 kg
- Parkinson's disease diagnosed
- Absolute neutrophil count (ANC) ≥1.5 x 10^9/L (≥1.0 x 10^9/L for Black population) at screening
- On a stable dose for at least 3 months prior to the screening visit of any of the following treatments at an L-dopa equivalent daily dose of up to 600 mg:
- Dopaminergic agonist alone
Show all 10Show fewer
- L-dopa alone
- Combination therapy with dopaminergic agonist and L-dopa
- Rasagiline
- At an early stage of the disease, without motor fluctuations and/or L-dopa-induced dyskinesia
Who cannot join
- Diagnosis of Parkinson's disease more than 3 years prior to screening visit
- Hoehn and Yahr stage ≥ 3
- Atypical or secondary Parkinsonism without dopa-sensitivity (e.g., vascular parkinsonism, supranuclear palsy, multisystem atrophy)
- Progressing Axis I psychiatric disorders (psychosis, hallucinations, compulsive disorders, substance addiction, bipolar disorder, severe depression, anxiety) as assessed in a semi-structured interview in accordance with the Diagnostic and Statistical Manual of Mental Disorders
- Not stabilized in terms of the current antiparkinsonian therapeutic regimen: already requires dose adaptation and/or is likely to require any change in dopamine therapy over the duration of the trial
- Current treatment with bromocriptine
Show all 9Show fewer
- Current treatment with any antiparkinsonian drug other than those listed in the inclusion criteria
- Current treatment with coenzyme Q10 or idebenone. (Patients who are on these medications but stop taking them at least 2 weeks prior to baseline may be enrolled.)
- Current use of a Deep Brain Stimulation (DBS) system. (Patients who previously had a DBS system but have had it removed may be enrolled.)
Trial locations and contacts
20 sites in 4 countries.
20 sites in 4 countries
France10 sites
CHU de Bordeaux, Centre Expert Parkinson
Bordeaux
Hôpital Henri Mondor
Créteil
Centre Hospitalier Régional Universitaire de Lille, Hôpital Roger Salengro
Lille
CHU Dupuytren
Limoges
Hôpital Neurologique Pierre Wertheimer
Lyon
CHRU de Montpellier - Hôpital Gui de Chauliac
Montpellier
CHU Pontchaillou
Rennes
CHU Charles Nicoll - Rouen
Rouen
Hôpitaux Universitaires de Strasbourg, Hôpital de Hautepierre
Strasbourg
CHU Purpan, Hôpital Pierre Paul Riquet
Toulouse
United Kingdom5 sites
Fairfield General Hospital
Bury
Royal Devon & Exeter Hospital
Exeter, Devon
Charing Cross Hospital
London
Newcastle Clinical Ageing Research Unit
Newcastle upon Tyne
Derriford Hospital
Plymouth
Germany4 sites
Heinriche-Heine Universität Düsseldorf
Düsseldorf
UKSH Campus Kiel, Neurologie
Kiel
Universitätsklinikum Gießen und Marburg GmbH
Marburg
Klinikum rechts der Isar
Munich
About this study
Official title: A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease
The goal of this study is to evaluate the effects of deferiprone, an iron-chelating drug, in patients with Parkinson's disease. Participants will be randomized to receive one of four different dosages of deferiprone or placebo, and will take the assigned study product twice a day for nine months.
Read the detailed description
This study will enroll 140 patients who have been diagnosed with Parkinson's disease within the last 3 years and are currently taking antiparkinsonian medication. There are four dosage cohorts, with patients in each cohort receiving either deferiprone tablets or placebo. At the baseline visit, participants will be randomized to a dosage cohort and to either active product or placebo within that cohort, and will take the assigned study product twice-daily for 9 months. They will come back to the study site for assessments at Months 1, 2, 3, 4, 5, 6, and 9, and will need to have their blood count checked weekly, at either the study site or a local laboratory.
- Conditions
- Parkinson's Disease
- Being tested
- Deferiprone, Placebo
- Keywords
- Parkinson's disease, Deferiprone
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DeferiproneDrug
Also called DFP
600 mg tablets
PlaceboDrug
Tablets that match the deferiprone tablets in appearance
| Study group | Role | What they receive |
|---|---|---|
| Deferiprone 300 mg | Experimental | One-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg Receives: Drug: Deferiprone |
| Deferiprone 600 mg | Experimental | One 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg Receives: Drug: Deferiprone |
| Deferiprone 900 mg | Experimental | One and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg Receives: Drug: Deferiprone |
| Deferiprone 1200 mg | Experimental | Two 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg Receives: Drug: Deferiprone |
| Placebo | Placebo comparator | Depending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Change from baseline to Month 9 in the score for the Part III subscale (motor examination) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 132 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
Measured: Nine months
Secondary outcomes (10)
Change in Total Score on the MDS-UPDRS
Change from baseline to Month 9 in total score on the MDS-UPDRS. The total score can range from 0 (best) to 260 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
Measured: Nine months
Change in Score on the Part I Subscale of the MDS-UPDRS
Change from baseline to Month 9 in the score for the Part I subscale (mentation, behavior, and mood) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 52 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
Measured: Nine months
Change in Score on the Part II Subscale of the MDS-UPDRS
Change from baseline to Month 9 in the score for the Part II subscale (activities of daily living) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 52 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
Measured: Nine months
Change in Score in the Part IV Subscale of the MDS-UPDRS
Change from baseline to Month 9 in the score for the Part IV subscale (motor complications) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 24 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
Measured: Nine months
Change in the Combined Scores From Parts II and III of the MDS-UPDRS
Change from baseline to Month 9 in the combined score for the Part II subscale (motor experiences of daily living) and the Part III subscale (motor examination) of the MDS-UPDRS. The scales for these two parts range from 0 (best) to 52 (worst) and from 0 (best) to 132 (worst), respectively. Hence, the combined score can range from 0 (best) to 184 (worst), and an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score is presented as least square means.
Measured: Nine months
Change in Score on the Montreal Cognitive Assessment (MoCA) Test
Change from baseline to Month 9 in the score for overall cognitive function as assessed by the MoCA. The total score on the MoCA can range from 0 (worst) to 30 (best). Hence, a decrease in score would indicate progression of the disease and an increase would indicate improvement. The change in score is presented as least square means.
Measured: Nine months
Safety of Deferiprone
Number of subjects with adverse events
Measured: Nine months
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The maximum measured serum concentration (Cmax) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.
Measured: 4 hours
Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The time to maximum observed serum concentration (Tmax) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.
Measured: 4 hours
Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide
Blood samples for pharmacokinetics assessments were collected at baseline, and at pre-dose and specified time points up to 12 hours post-dose at the Month 3 visit. The total drug exposure, AUC0-∞ (area under the serum concentration time curve extrapolated to infinity) at the Month 3 visit was determined for deferiprone and its main metabolite, deferiprone 3-O-glucuronide.
Measured: 4 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Apr 2016 (estimated)
- Study start12 Oct 2016
- Primary completion2 Aug 2019
- Study completion4 Sep 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Parkinson's DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 140 participants (actual)
Sponsor and investigators
- Lead sponsor
- ApoPharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- David Devos, MD, Principal investigator, Hospitalier Régional Universitaire de Lille
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02728843
- Sponsor's protocol ID
- LA48-0215
- Acronym
- SKY
- Status last verified
- Mar 2024
- First submitted
- 31 Mar 2016
- Last updated
- 10 Apr 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Devos D, Rascol O, Meissner WG, Foubert-Samier A, Lewis S, Tranchant C, Anheim M, Maltete D, Remy P, Eggert K, Pape H, Geny C, Couratier P, Carroll C, Sheridan R, Burn D, Pavese N, Raw J, Berg D, Suchowersky O, Kalia LV, Evans A, Drapier S, Danaila T, Schnitzler A, Corvol JC, Defer G, Temin NT, Fradette C, Tricta F, Moreau C; a Parkinson's disease study group. Therapeutic modalities of deferiprone in Parkinson's disease: SKY and EMBARK studies. J Parkinsons Dis. 2025 Feb;15(1):72-86. doi: 10.1177/1877718X241300295. Epub 2024 Dec 27.
PubMed 39973479
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose-Ranging Study of the Efficacy, Safety, and Pharmacokinetics of Deferiprone Delayed Release Tablets in Patients With Parkinson's Disease
- ClinicalTrials.gov ID
- NCT02728843
- Acronym / sponsor ID
- SKY / LA48-0215
- Conditions
- Parkinson's Disease
- Keywords
- Parkinson's disease, Deferiprone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 140 participants (actual)
- Lead sponsor
- ApoPharmaIndustry (pharma, biotech, devices)
- Study start
- 12 Oct 2016 (actual)
- Primary completion
- 2 Aug 2019 (actual)
- Study completion
- 4 Sep 2019 (actual)
- First posted
- 5 Apr 2016 (estimated)
- Results
- Posted 10 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The goal of this study is to evaluate the effects of deferiprone, an iron-chelating drug, in patients with Parkinson's disease. Participants will be randomized to receive one of four different dosages of deferiprone or placebo, and will take the assigned study product twice a day for nine months.
Detailed description
This study will enroll 140 patients who have been diagnosed with Parkinson's disease within the last 3 years and are currently taking antiparkinsonian medication. There are four dosage cohorts, with patients in each cohort receiving either deferiprone tablets or placebo. At the baseline visit, participants will be randomized to a dosage cohort and to either active product or placebo within that cohort, and will take the assigned study product twice-daily for 9 months. They will come back to the study site for assessments at Months 1, 2, 3, 4, 5, 6, and 9, and will need to have their blood count checked weekly, at either the study site or a local laboratory.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| Deferiprone 300 mg | Experimental | Drug: DeferiproneOne-half of a 600 mg tablet of deferiprone twice a day, for a total daily dosage of 600 mg |
| Deferiprone 600 mg | Experimental | Drug: DeferiproneOne 600 mg tablet of deferiprone twice a day, for a total daily dosage of 1200 mg |
| Deferiprone 900 mg | Experimental | Drug: DeferiproneOne and a half 600 mg tablets of deferiprone twice a day, for a total daily dosage of 1800 mg |
| Deferiprone 1200 mg | Experimental | Drug: DeferiproneTwo 600 mg tablets of deferiprone twice a day, for a total daily dosage of 2400 mg |
| Placebo | Placebo comparator | Drug: PlaceboDepending on dosage cohort, either one half-tablet, one tablet, one and a half tablets, or two tablets of placebo, twice a day |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Male or female aged ≥18 to < 80 years
- Body weight ≥60 kg but ≤100 kg
- Parkinson's disease diagnosed
- Absolute neutrophil count (ANC) ≥1.5 x 10^9/L (≥1.0 x 10^9/L for Black population) at screening
- On a stable dose for at least 3 months prior to the screening visit of any of the following treatments at an L-dopa equivalent daily dose of up to 600 mg:
Key exclusion criteria (9 listed)
- Diagnosis of Parkinson's disease more than 3 years prior to screening visit
- Hoehn and Yahr stage ≥ 3
- Atypical or secondary Parkinsonism without dopa-sensitivity (e.g., vascular parkinsonism, supranuclear palsy, multisystem atrophy)
- Progressing Axis I psychiatric disorders (psychosis, hallucinations, compulsive disorders, substance addiction, bipolar disorder, severe depression, anxiety) as assessed in a semi-structured interview in accordance with the Diagnostic and Statistical Manual of Mental Disorders
- Not stabilized in terms of the current antiparkinsonian therapeutic regimen: already requires dose adaptation and/or is likely to require any change in dopamine therapy over the duration of the trial
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DeferiproneAlso: DFP600 mg tablets | Drug | Deferiprone 1200 mg, Deferiprone 300 mg, Deferiprone 600 mg, Deferiprone 900 mg |
| PlaceboTablets that match the deferiprone tablets in appearance | Drug | Placebo |
Primary outcome
Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS)
Time frame: Nine months
Change from baseline to Month 9 in the score for the Part III subscale (motor examination) of the MDS-UPDRS. Scores on this subscale can range from 0 (best) to 132 (worst); hence, an increase would indicate progression of the disease and a decrease would indicate improvement. The change in score was calculated as least square means.
10 secondary outcomes
Change in Total Score on the MDS-UPDRS
Time frame: Nine months
Change in Score on the Part I Subscale of the MDS-UPDRS
Time frame: Nine months
Change in Score on the Part II Subscale of the MDS-UPDRS
Time frame: Nine months
Change in Score in the Part IV Subscale of the MDS-UPDRS
Time frame: Nine months
Change in the Combined Scores From Parts II and III of the MDS-UPDRS
Time frame: Nine months
Change in Score on the Montreal Cognitive Assessment (MoCA) Test
Time frame: Nine months
Safety of Deferiprone
Time frame: Nine months
Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Time frame: 4 hours
Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide
Time frame: 4 hours
Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide
Time frame: 4 hours
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Apr 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39973479Devos D, Rascol O, Meissner WG, Foubert-Samier A, Lewis S, Tranchant C, Anheim M, Maltete D, Remy P, Eggert K, Pape H, Geny C, Couratier P, Carroll C, Sheridan R, Burn D, Pavese N, Raw J, Berg D, Suchowersky O, Kalia LV, Evans A, Drapier S, Danaila T, Schnitzler A, Corvol JC, Defer G, Temin NT, Fradette C, Tricta F, Moreau C; a Parkinson's disease study group. Therapeutic modalities of deferiprone in Parkinson's disease: SKY and EMBARK studies. J Parkinsons Dis. 2025 Feb;15(1):72-86. doi: 10.1177/1877718X241300295. Epub 2024 Dec 27.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David Devos, MD principal investigator
Hospitalier Régional Universitaire de Lille
20 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Deferiprone in people with Parkinson's Disease, run by ApoPharma at 20 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 10 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in France, United Kingdom, Germany, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) (time frame: Nine months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02728843, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 10 Apr 2024.