scholarly journals Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: a single-arm, open-label study

2020 ◽  
Vol 15 (1) ◽  
Author(s):  
Xuting Chang ◽  
Jie Zhang ◽  
Yuwu Jiang ◽  
Bufan Yao ◽  
Jingmin Wang ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Rating Scale ◽  
Pilot Trial ◽  
Trial Registration ◽  
Efficacy And Safety ◽  
Open Label ◽  
Pantothenate Kinase ◽  
Open Label Study ◽  
Label Study

Abstract Objective This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN). Methods A single-arm, open-label study was conducted. All subjects received pantethine during the 24-week period of treatment. The primary endpoints were change of the Unified Parkinson’s Disease Rating Scale (UPDRS) I–III and Fahn–Marsden (FM) score from baseline to week 24 after treatment. Results Fifteen children with PKAN were enrolled, and all patients completed the study. After 24 weeks of treatment with pantethine at 60 mg/kg per day, there was no difference in either UPDRS I–III (t = 0.516, P = 0.614) or FM score (t = 0.353, P = 0.729) between the baseline and W24. Whereas the rates of increase in UPDRS I-III (Z = 2.614, p = 0.009) and FM scores (Z = 2.643, p = 0.008) were slowed. Four patients (26.7%) were evaluated as “slightly improved” by doctors through blinded video assessment. Patients with lower baseline UPDRS I–III or FM scores were more likely to be improved. The quality of life of family members improved after pantethine treatment, evaluated by PedsQL TM 2.0 FIM scores, whereas the quality of life of the patients was unchanged at W24, evaluated by PedsQL TM 4.0 and PedsQL TM 3.0 NMM. Serum level of CoA was comparable between baseline and W24. There was no drug related adverse event during the study. Conclusions Pantethine could not significantly improve motor function in children with PKAN after 24 weeks treatment, but it may delay the progression of motor dysfunction in our study. Pantethine was well-tolerated at 60 mg/kg per day. Trial registration Clinical trial registration number at www.chictr.org.cn:ChiCTR1900021076, Registered 27 January2019, the first participant was enrolled 30 September 2018, and other 14 participants were enrolled after the trial was registered.

scholarly journals Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: A single-arm, open-label study.

2020 ◽  
Author(s):  
Xuting Chang ◽  
Jie Zhang ◽  
Yuwu Jiang ◽  
Bufan Yao ◽  
Jingmin Wang ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Rating Scale ◽  
Pilot Trial ◽  
Trial Registration ◽  
Efficacy And Safety ◽  
Open Label ◽  
Pantothenate Kinase ◽  
Open Label Study ◽  
Label Study

Abstract Objective This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN).Methods A single-arm, open-label study was conducted. All subjects received pantethine during the 24-week period of treatment. The primary endpoints were change of the Unified Parkinson’s Disease Rating Scale (UPDRS) I–III and Fahn–Marsden (FM) score from baseline to week 24 after treatment.Results Fifteen children with PKAN were enrolled, and all patients completed the study. After 24 weeks of treatment with pantethine at 60mg/kg per day, there was no difference in either UPDRS I–III (t=0.516, P=0.614) or FM score (t=0.353, P=0.729) between the baseline and W24. Whereas the rates of increase in UPDRS I-III (Z=2.614, p=0.009) and FM scores (Z=2.643, p=0.008) were slowed. Four patients (26.7%) were evaluated as “slightly improved” by doctors through blinded video assessment. Patients with lower baseline UPDRS I–III or FM scores were more likely to be improved. The quality of life of family members improved after pantethine treatment, evaluated by PedsQL TM 2.0 FIM scores, whereas the quality of life of the patients was unchanged at W24, evaluated by PedsQL TM 4.0 and PedsQL TM 3.0 NMM. Serum level of CoA was comparable between baseline and W24. There was no drug related adverse event during the study.Conclusions Pantethine could not significantly improve motor function in children with PKAN after 24 weeks treatment, but it may delay the progression of motor dysfunction in our study. Pantethine was well-tolerated at 60mg/kg per day.Trial registration:Clinical trial registration number at www.chictr.org.cn：ChiCTR1900021076，Registered 27 January2019，the first participant was enrolled 30 September 2018，and other 14 participants were enrolled after the trial was registered.

scholarly journals Pilot trial on the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration: A single-arm, open-label study.

2020 ◽  
Author(s):  
Xuting Chang ◽  
Jie Zhang ◽  
Yuwu Jiang ◽  
Bufan Yao ◽  
Jingmin Wang ◽  
...  
Keyword(s):  
Rating Scale ◽  
Pilot Trial ◽  
Motor Dysfunction ◽  
Efficacy And Safety ◽  
Open Label ◽  
Pantothenate Kinase ◽  
Open Label Study ◽  
Label Study ◽  
Video Assessment

Abstract Objective This study aimed to explore the efficacy and safety of pantethine in children with pantothenate kinase-associated neurodegeneration (PKAN).Methods A single-arm, open-label study was conducted. All subjects received pantethine during the 24-week period of treatment. The primary endpoints were change of the Unified Parkinson’s Disease Rating Scale (UPDRS) I–III and Fahn–Marsden (FM) score from baseline to week 24 after treatment.Results Fifteen children with PKAN were enrolled, and all patients completed the study. After 24 weeks of treatment with pantethine at 60 mg/kg per day, there was no difference in either UPDRS I–III (t = 0.516, P = 0.614) or FM score (t = 0.353, P = 0.729) between the baseline and W24. Whereas the rates of increase in UPDRS I-III (Z = 2.614, p = 0.009) and FM scores (Z = 2.643, p = 0.008) were slowed. Four patients (26.7%) were evaluated as “slightly improved” by doctors through blinded video assessment. Patients with lower baseline UPDRS I–III or FM scores were more likely to be improved. The living quality of family members improved after pantethine treatment, evaluated by PedsQL TM 2.0 FIM scores, whereas the living quality of the patients was unchanged at W24, evaluated by PedsQL TM 4.0 and PedsQL TM 3.0 NMM. Serum level of CoA was comparable between baseline and W24. There was no drug related adverse event during the study.Conclusions Pantethine could not significantly improve motor function in children with PKAN after 24 weeks treatment, but it could probably delay the progression of motor dysfunction in our study. 26.7% of patients showed slightly improved. Pantethine was well-tolerated at 60 mg/kg per day.

scholarly journals Functional impairment and disability among patients with migraine: evaluation of galcanezumab in a long-term, open-label study

2020 ◽  
Author(s):  
Janet H. Ford ◽  
Virginia L. Stauffer ◽  
Peter McAllister ◽  
Sreelatha Akkala ◽  
Matthew Sexson ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Treatment Phase ◽  
Phase Iii ◽  
Life Questionnaire ◽  
Open Label ◽  
Open Label Study ◽  
Label Study ◽  
Patient Reported ◽  
Domain Scores

Abstract Purpose Migraine can negatively impact patient functioning and quality of life. Here, we report the effects of galcanezumab (GMB), a humanized monoclonal antibody that binds to calcitonin gene-related peptide, on patient-reported outcome (PRO) measures in migraine. Methods CGAJ was a Phase III, randomized, open-label study (12-month open-label and 4-month post-treatment follow-up) in patients with episodic or chronic migraine. Patients aged 18–65 years with diagnosis of migraine (≥ 4 migraine headache days per month) as defined by International Classification of Headache Disorders (ICHD)-3 beta guidelines were included in the study. Patients were randomized 1:1 with subcutaneous GMB 120 mg (with a loading dose of 240 mg) or GMB 240 mg given once monthly for 12 months. Changes from baseline in PRO measures such as Migraine-Specific Quality of Life Questionnaire v2.1 (MSQ) and Migraine Disability Assessment (MIDAS) were assessed. Results A total of 135 patients were randomized to each galcanezumab dose group. Mean (SD) baseline MSQ total scores were 53.85 (20.34) [GMB 120 mg] and 53.69 (18.79) [GMB 240 mg]. For MIDAS, mean (SD) total scores were 45.77 (42.06) [GMB 120 mg] and 53.96 (61.24) [GMB 240 mg]. Within-group mean improvement from baseline on MSQ and MIDAS total scores and all individual item/domain scores were statistically significant for both GMB dose groups, at all-time points during the treatment phase (p < 0.001). For MSQ domain scores, greatest improvement was observed in the Role function-restrictive (RF-R) domain (overall least squares (LS) mean change ± SE: 31.55 ± 1.20 [GMB 120 mg] and 33.40 ± 1.16 [GMB 240 mg]). For MIDAS, the overall LS mean change ± SE from baseline across the entire 12-month treatment phase in total scores were: −33.58 ± 2.11 (GMB 120 mg) and −32.67 ± 2.04 (GMB 240 mg). Conclusion Galcanezumab was associated with statistically significant changes from baseline in the PRO measures across the entire 12-month treatment period. These results indicate improved health-related quality of life and decreased disability among patients treated with galcanezumab.

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