A Phase 3 interventional study of Deferiprone oral solution in Pantothenate Kinase-Associated Neurodegeneration, sponsored by ApoPharma. Completed at 4 sites in 4 countries. Open to participants aged 5 Years and older. Per ClinicalTrials.gov, last updated 2020-08-25.
Sponsored by ApoPharma (part of Chiesi Farmaceutici S.p.A.) · Phase 3, Interventional, and Treatment
Patients with PKAN will be treated with the iron chelator deferiprone for 18 months. Only patients who have completed the earlier study TIRCON2012V1 (NCT01741532), a double-blind placebo-controlled trial in which participants were randomized to receive either deferiprone or placebo for 18 months, are eligible to enroll.
TIRCON2012V1-EXT is a multi-center, single-arm, open-label study. All patients who completed the earlier study TIRCON2012V1 (NCT01741532) are eligible to take part. In the initial study, patients were randomized in a 2:1 ratio to receive 18 months of treatment with either the iron chelator deferiprone or placebo, respectively. In this extension study, all participants will receive deferiprone for 18 months. Thus, depending on which product was received earlier, patients will be on deferiprone for a total of either 1.5 years or 3 years. As in the earlier study, assessments will be carried out every six months to look at the safety of the drug and to see if patients are showing any improvement in dystonia and other symptoms of PKAN.
ApoPharma is the lead sponsor of 23 studies on the registry; none are open to participants now.
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Exclusion Criteria:
All patients will receive deferiprone oral solution.
Drug: Deferiprone oral solution
Deferiprone oral solution at a dosage of up to 15 mg per kilogram of body weight, twice a day
Also known as: DFP
Number of Participants With Adverse Events
Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product.
Time frame: 18 months
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of both the initial study (during which one group received placebo and the other received deferiprone) and the extension study (during which both groups received deferiprone).
Time frame: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of each study.
Time frame: Baseline and Month 18 of each study
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of the study.
Time frame: Baseline and Month 18 of each study
Proportion of Patients With Improved or Unchanged BAD Score
Patients were deemed to be responders if their BAD total score either improved or remained unchanged from baseline, with baseline being the start of each study for the placebo-DFP group and the start of the initial study for the DFP-DFP group
Time frame: Month 18 of each study
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies
The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.
Time frame: Month 18 of each study
| Milestone | Placebo-DFP | DFP-DFP |
|---|---|---|
| Started | 24 | 44 |
| Provided post-baseline efficacy data | 19 | 43 |
| Completed | 17 | 38 |
| Not completed | 7 | 6 |
| Withdrew: Adverse event | 2 | 1 |
| Withdrew: Withdrawal by subject | 3 | 3 |
| Withdrew: Lost to follow-up | 0 | 1 |
| Withdrew: Worsening of disease | 1 | 1 |
| Withdrew: Unable to comply with study requirements | 1 | 0 |
Safety and tolerability were assessed based on changes in: frequency of adverse events (AEs), frequency of serious adverse events (SAEs), and discontinuation due to AEs. No statistical comparison between the groups was conducted as all participants received the same study product.
| Participants | Placebo-DFP | DFP-DFP |
|---|---|---|
| Number of patients with at least one AE | 22 | 42 |
| Number of patients with at least one SAE | 12 | 14 |
| Number of patients who withdrew due to an AE | 2 | 1 |
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of both the initial study (during which one group received placebo and the other received deferiprone) and the extension study (during which both groups received deferiprone).
| score on a scale | Placebo-DFP | DFP-DFP |
|---|---|---|
| Change in BAD score over initial study | 4.4 ± 4.8 | 1.9 ± 3.2 |
| Change in BAD score over extension study | 1.4 ± 3.7 | 1.4 ± 2.4 |
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of each study.
| score on a scale | Placebo-DFP in Initial Study | Placebo-DFP in Extension Study |
|---|---|---|
| Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies | 4.4 ± 4.8 | 1.4 ± 3.7 |
The Barry-Albright Dystonia (BAD) scale is an instrument for rating the severity of dystonia in eight body regions. The individual scores are summed to provide a total score that ranges from 0 to 32; the higher the score, the more severe the dystonia. Patients were assessed for the change in total BAD score over the course of the study.
| score on a scale | DFP-DFP Group in Initial Study | DFP-DFP Group in Extension Study |
|---|---|---|
| Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies | 1.9 ± 3.2 | 1.4 ± 2.4 |
Patients were deemed to be responders if their BAD total score either improved or remained unchanged from baseline, with baseline being the start of each study for the placebo-DFP group and the start of the initial study for the DFP-DFP group
| Participants | Placebo-DFP | DFP-DFP |
|---|---|---|
| Completion of initial study | 3 | 17 |
| Completion of 18 months of deferiprone treatment | 9 | 17 |
The Patient Global Impression of Improvement (PGI-I) is a global index used to rate the response of a condition to a therapy. Patients were asked at each post-baseline visit to rate their overall condition since the start of the extension study on a 7-point rating scale: 1 = very much improved, 2 = much improved, 3 = minimally improved, 4 = no change, 5 = minimally worse, 6 = much worse, and 7 = very much worse.
| score on a scale | Placebo-DFP in Initial Study | Placebo-DFP in Extension Study |
|---|---|---|
| Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies | 4.4 ± 1.5 | 4.7 ± 1.4 |
Collected over Safety data were collected from the time of first dose until the end of the study (up to 18 months). Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Patients | 2/68 (2.9%) | 33/68 (48.5%) | 66/68 (97.1%) |
| Event | All Patients |
|---|---|
| DystoniaNervous system disorders | 8/68 |
| NeutropeniaBlood and lymphatic system disorders | 4/68 |
| GastrostomySurgical and medical procedures | 4/68 |
| PyrexiaGeneral disorders | 3/68 |
| Medical device implantationSurgical and medical procedures | 3/68 |
| BronchitisInfections and infestations | 2/68 |
| PneumoniaInfections and infestations | 2/68 |
| Wound infectionInfections and infestations | 2/68 |
| Medical device battery replacementSurgical and medical procedures | 2/68 |
| Medical device changeSurgical and medical procedures | 2/68 |
| Event | All Patients |
|---|---|
| DystoniaNervous system disorders | 32/68 |
| PyrexiaGeneral disorders | 20/68 |
| HeadacheNervous system disorders | 19/68 |
| Serum ferritin decreasedInvestigations | 18/68 |
| Condition aggravatedGeneral disorders | 17/68 |
| Pain in extremityMetabolism and nutrition disorders | 14/68 |
| Upper respiratory tract infectionInfections and infestations | 13/68 |
| AnemiasBlood and lymphatic system disorders | 12/68 |
| CoughRespiratory, thoracic and mediastinal disorders | 12/68 |
| NasopharyngitisInfections and infestations | 11/68 |
| Age, Continuous(years) | Placebo-DFP | DFP-DFP | Total |
|---|---|---|---|
| Mean | 19.9 ± 13.0 | 22.4 ± 9.6 | 21.5 ± 10.9 |
| Sex: Female, Male(Participants) | Placebo-DFP | DFP-DFP | Total |
|---|---|---|---|
| Female | 14 | 16 | 30 |
| Male | 10 | 28 | 38 |
| Race (NIH/OMB)(Participants) | Placebo-DFP | DFP-DFP | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 0 | 0 |
| Asian | 1 | 3 | 4 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 0 | 0 | 0 |
| White | 22 | 41 | 63 |
| More than one race | 1 | 0 | 1 |
| Unknown or Not Reported | 0 | 0 | 0 |
| BAD score at baseline(units on a scale) | Placebo-DFP | DFP-DFP | Total |
|---|---|---|---|
| BAD score at baseline of initial study | 16.0 ± 8.0 | 19.4 ± 8.1 | 18.3 ± 8.2 |
| BAD score at baseline of extension study | 20.4 ± 8.2 | 21.3 ± 7.6 | 21.0 ± 7.7 |
PDFs hosted by the registry, ClinicalTrials.gov.
The record gives a start of Jun 2014, a primary completion of Mar 2018 and a completion of Mar 2018. It does not say whether these dates are actual or estimated.
Results were first posted in Jul 2019, 1 year 4 months after primary completion.
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This study is completed, as verified in Aug 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Deferiprone
Pantothenate Kinase-Associated Neurodegeneration