Trial Outcomes & Findings for Long-term Deferiprone Treatment in Patients With Pantothenate Kinase-Associated Neurodegeneration (NCT NCT02174848)

Last Updated: 2020-08-25

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

68 participants

Primary outcome timeframe

18 months

Results posted on

2020-08-25

Participant Flow

Participant milestones

Participant milestones
Measure	Placebo-DFP Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment over the duration of the two studies.	DFP-DFP Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment over the duration of the two studies.
Overall Study STARTED	24	44
Overall Study Provided Post-baseline Efficacy Data	19	43
Overall Study COMPLETED	17	38
Overall Study NOT COMPLETED	7	6

Reasons for withdrawal

Reasons for withdrawal
Measure	Placebo-DFP Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment over the duration of the two studies.	DFP-DFP Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment over the duration of the two studies.
Overall Study Adverse Event	2	1
Overall Study Withdrawal by Subject	3	3
Overall Study Lost to Follow-up	0	1
Overall Study Worsening of disease	1	1
Overall Study Unable to comply with study requirements	1	0

Baseline Characteristics

The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Placebo-DFP n=24 Participants Patients who received 18 months of placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study, so received up to 18 months of deferiprone treatment.	DFP-DFP n=44 Participants Patients who received 18 months of deferiprone treatment in the TIRCON2012V1 study and continued to receive it in the extension study, so received up to 36 months of deferiprone treatment.	Total n=68 Participants Total of all reporting groups
Age, Continuous	19.9 years STANDARD_DEVIATION 13.0 • n=24 Participants	22.4 years STANDARD_DEVIATION 9.6 • n=44 Participants	21.5 years STANDARD_DEVIATION 10.9 • n=68 Participants
Sex: Female, Male Female	14 Participants n=24 Participants	16 Participants n=44 Participants	30 Participants n=68 Participants
Sex: Female, Male Male	10 Participants n=24 Participants	28 Participants n=44 Participants	38 Participants n=68 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=24 Participants	0 Participants n=44 Participants	0 Participants n=68 Participants
Race (NIH/OMB) Asian	1 Participants n=24 Participants	3 Participants n=44 Participants	4 Participants n=68 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=24 Participants	0 Participants n=44 Participants	0 Participants n=68 Participants
Race (NIH/OMB) Black or African American	0 Participants n=24 Participants	0 Participants n=44 Participants	0 Participants n=68 Participants
Race (NIH/OMB) White	22 Participants n=24 Participants	41 Participants n=44 Participants	63 Participants n=68 Participants
Race (NIH/OMB) More than one race	1 Participants n=24 Participants	0 Participants n=44 Participants	1 Participants n=68 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=24 Participants	0 Participants n=44 Participants	0 Participants n=68 Participants
BAD score at baseline BAD score at baseline of initial study	16.0 units on a scale STANDARD_DEVIATION 8.0 • n=19 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.	19.4 units on a scale STANDARD_DEVIATION 8.1 • n=43 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.	18.3 units on a scale STANDARD_DEVIATION 8.2 • n=62 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.
BAD score at baseline BAD score at baseline of extension study	20.4 units on a scale STANDARD_DEVIATION 8.2 • n=19 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.	21.3 units on a scale STANDARD_DEVIATION 7.6 • n=43 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.	21.0 units on a scale STANDARD_DEVIATION 7.7 • n=62 Participants • The baseline efficacy measurements are provided only for the 62 patients who provided at least one post-baseline efficacy assessment and were included in the efficacy analyses of the extension study.

PRIMARY outcome

Timeframe: 18 months

Population: Safety population

Outcome measures

Outcome measures
Measure	Placebo-DFP n=24 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=44 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Number of Participants With Adverse Events Number of patients with at least one AE	22 Participants	42 Participants
Number of Participants With Adverse Events Number of patients with at least one SAE	12 Participants	14 Participants
Number of Participants With Adverse Events Number of patients who withdrew due to an AE	2 Participants	1 Participants

SECONDARY outcome

Timeframe: Baseline and Month 18 of 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).

Outcome measures

Outcome measures
Measure	Placebo-DFP n=19 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=43 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study Change in BAD score over initial study	4.4 score on a scale Standard Deviation 4.8	1.9 score on a scale Standard Deviation 3.2
Change in Score on the BAD Scale -- Comparison of Treatment Groups Over Each Study Change in BAD score over extension study	1.4 score on a scale Standard Deviation 3.7	1.4 score on a scale Standard Deviation 2.4

SECONDARY outcome

Timeframe: Baseline and Month 18 of each study

Outcome measures

Outcome measures
Measure	Placebo-DFP n=19 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=19 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Change in Score on the BAD Scale -- Comparison of Placebo-DFP Patients Across Studies	4.4 score on a scale Standard Deviation 4.8	1.4 score on a scale Standard Deviation 3.7

SECONDARY outcome

Timeframe: Baseline and Month 18 of each study

Outcome measures

Outcome measures
Measure	Placebo-DFP n=43 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=43 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Change in Score on the BAD Scale -- Comparison of DFP-DFP Patients Across Studies	1.9 score on a scale Standard Deviation 3.2	1.4 score on a scale Standard Deviation 2.4

SECONDARY outcome

Timeframe: Month 18 of each study

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

Outcome measures

Outcome measures
Measure	Placebo-DFP n=19 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=43 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Proportion of Patients With Improved or Unchanged BAD Score Completion of initial study	3 Participants	17 Participants
Proportion of Patients With Improved or Unchanged BAD Score Completion of 18 months of deferiprone treatment	9 Participants	17 Participants

SECONDARY outcome

Timeframe: Month 18 of each study

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.

Outcome measures

Outcome measures
Measure	Placebo-DFP n=19 Participants Patients in this group had been randomized to placebo treatment in the TIRCON2012V1 study and were then switched to deferiprone in the extension study. Accordingly, they received up to 18 months of deferiprone treatment.	DFP-DFP n=19 Participants Patients in this group had been randomized to deferiprone treatment in the TIRCON2012V1 study and then continued on deferiprone in the extension study. Accordingly, they received up to 36 months of deferiprone treatment.
Patient Global Impression of Improvement (PGI-I) Comparison of Placebo-DFP Patients Across Studies	4.4 score on a scale Standard Deviation 1.5	4.7 score on a scale Standard Deviation 1.4

Adverse Events

All Patients

Serious events: 33 serious events

Other events: 66 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	All Patients n=68 participants at risk All participants received deferiprone during the extension study
Blood and lymphatic system disorders Neutropenia	5.9% 4/68 • Number of events 7 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Cardiac disorders Cyanosis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Cardiac disorders Tachycardia	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Constipation	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Gastritis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Intestinal dilatation	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Intestinal obstruction	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Oesophagitis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Salivary hypersecretion	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Vomiting	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Condition aggravated	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Medical device site inflammation	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Multiple organ dysfunction syndrome	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Obstruction	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Pyrexia	4.4% 3/68 • Number of events 3 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Bacterial disease carrier	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Bronchitis	2.9% 2/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Device related infection	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Infective glossitis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Peritonitis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Pneumonia	2.9% 2/68 • Number of events 3 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Respiratory tract infection	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Urinary tract infection	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Viral infection	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Wound infection	2.9% 2/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Chemical eye injury	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Clavicle fracture	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Toxicity to various agents	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Unintentional medical device removal	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Wound	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Device function test	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Physical examination	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Weight decreased	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Musculoskeletal and connective tissue disorders Bursitis	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Dystonia	11.8% 8/68 • Number of events 8 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Headache	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Hyporesponsive to stimuli	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Loss of consciousness	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Oromandibular dystonia	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Somnolence	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Syncope	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Product Issues Device dislocation	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Product Issues Device malfunction	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Product Issues Device stimulation issue	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Psychiatric disorders Agitation	1.5% 1/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Renal and urinary disorders Urinary bladder rupture	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Aspiration	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Choking	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Cough	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Pneumonia aspiration	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Pneumothorax	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Respiratory disorder	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Respiratory failure	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Colostomy closure	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Dental operation	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Gastrointestinal tube insertion	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Gastrostomy	5.9% 4/68 • Number of events 4 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Hip surgery	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Intrathecal pump insertion	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Jejunostomy	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Medical device battery replacement	2.9% 2/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Medical device change	2.9% 2/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Medical device implantation	4.4% 3/68 • Number of events 3 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Medical device removal	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Tracheostomy	1.5% 1/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Tracheostomy tube removal	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Surgical and medical procedures Wound treatment	1.5% 1/68 • Number of events 1 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Vascular disorders Thrombosis	1.5% 1/68 • Number of events 2 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)

Other adverse events

Other adverse events
Measure	All Patients n=68 participants at risk All participants received deferiprone during the extension study
Blood and lymphatic system disorders Anemias	17.6% 12/68 • Number of events 27 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Abdominal pain upper	11.8% 8/68 • Number of events 16 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Diarrhoea	5.9% 4/68 • Number of events 6 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Dysphagia	11.8% 8/68 • Number of events 8 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Nausea	7.4% 5/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Gastrointestinal disorders Vomiting	14.7% 10/68 • Number of events 13 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Condition aggravated	25.0% 17/68 • Number of events 25 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Pain	7.4% 5/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
General disorders Pyrexia	29.4% 20/68 • Number of events 64 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Bronchitis	8.8% 6/68 • Number of events 30 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Influenza	5.9% 4/68 • Number of events 4 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Nasopharyngitis	16.2% 11/68 • Number of events 22 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Upper respiratory tract infection	19.1% 13/68 • Number of events 19 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Infections and infestations Urinary tract infection	8.8% 6/68 • Number of events 8 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Contusion	7.4% 5/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Injury, poisoning and procedural complications Laceration	11.8% 8/68 • Number of events 20 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Body temperature increased	5.9% 4/68 • Number of events 4 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Neutrophil count decreased	14.7% 10/68 • Number of events 26 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Investigations Serum ferritin decreased	26.5% 18/68 • Number of events 25 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Metabolism and nutrition disorders Iron deficiency	16.2% 11/68 • Number of events 11 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Musculoskeletal and connective tissue disorders Arthralgia	16.2% 11/68 • Number of events 12 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Musculoskeletal and connective tissue disorders Back pain	5.9% 4/68 • Number of events 4 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Musculoskeletal and connective tissue disorders Muscle spasms	11.8% 8/68 • Number of events 11 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Metabolism and nutrition disorders Pain in extremity	20.6% 14/68 • Number of events 26 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Aphasia	7.4% 5/68 • Number of events 6 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Ataxia	5.9% 4/68 • Number of events 4 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Balance disorder	7.4% 5/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Dystonia	47.1% 32/68 • Number of events 74 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Headache	27.9% 19/68 • Number of events 64 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Somnolence	8.8% 6/68 • Number of events 8 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Nervous system disorders Tremor	5.9% 4/68 • Number of events 7 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Psychiatric disorders Agitation	7.4% 5/68 • Number of events 13 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Cough	17.6% 12/68 • Number of events 19 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Oropharyngeal pain	14.7% 10/68 • Number of events 11 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Respiratory, thoracic and mediastinal disorders Rhinorrhoea	8.8% 6/68 • Number of events 8 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)
Skin and subcutaneous tissue disorders Rash	7.4% 5/68 • Number of events 5 • Safety data were collected from the time of first dose until the end of the study (up to 18 months)

Additional Information

Fernando Tricta, MD

Chiesi Canada Corp.

Phone: 416--558-6342

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Sponsor retained title to and the right to publish all documentation, records, raw data, specimens or other work product generated in connection with the trial. Such publications shall not be made without the prior written consent of Sponsor. Neither Party will use the other Party's name in connection with any publication or promotion without the other Party's prior written consent. However, Sponsor has the right to publish appropriate information in order to satisfy regulatory requirements.
Publication restrictions are in place

Restriction type: OTHER