Phase 2
N=140
Study of Parkinson's Early Stage With Deferiprone
Parkinson's Disease
Bottom Line
View on ClinicalTrials.gov: NCT02728843 ↗Enrolled (actual)
140
Serious AEs
7.9%
Results posted
Apr 2024
Primary outcome: Primary: Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) — 2.1; 4.2; -0.8; 3.9 Score on a scale — p=>0.05
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Deferiprone (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- ApoPharma
- Primary completion
- Aug 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Score on the Part III Subscale of the Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) |
2.1; 4.2; -0.8; 3.9; 2.2 | >0.05 |
| SECONDARY Change in Total Score on the MDS-UPDRS |
4.1; 7.8; 0.5; 6.6; 5.9 | >0.05 |
| SECONDARY Change in Score on the Part I Subscale of the MDS-UPDRS |
0.8; 1.0; 0.3; 0.3; 1.1 | >0.05 |
| SECONDARY Change in Score on the Part II Subscale of the MDS-UPDRS |
1.1; 2.2; 0.6; 2.1; 1.8 | >0.05 |
| SECONDARY Change in Score in the Part IV Subscale of the MDS-UPDRS |
-0.1; 0.2; 0.3; 0.2; 0.4 | >0.05 |
| SECONDARY Change in the Combined Scores From Parts II and III of the MDS-UPDRS |
3.3; 6.5; -0.3; 6.0; 4.3 | >0.05 |
| SECONDARY Change in Score on the Montreal Cognitive Assessment (MoCA) Test |
1.1; 1.0; 0.9; 1.1; 1.4 | >0.05 |
| SECONDARY Safety of Deferiprone |
26; 25; 23; 25; 25 | — |
| SECONDARY Cmax for Serum Deferiprone and Deferiprone 3-O-glucuronide |
2.748; 3.459; 5.080; 7.942; 7.844; 12.419 | — |
| SECONDARY Tmax for Serum Deferiprone and Deferiprone 3-O-glucuronide |
2.00; 2.17; 2.00; 1.67; 2.84; 3.25 | — |
| SECONDARY Area Under the Curve for Serum Deferiprone and Deferiprone 3-O-glucuronide |
8.358; 16.292; 20.606; 26.328; 39.057; 62.679 | — |
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.
Eligibility Criteria
Inclusion Criteria
- 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
- 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
Exclusion Criteria
- 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
- 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.)
Data sourced from ClinicalTrials.gov (NCT02728843). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.