Meta AnalysisID 7616
评估弗里德赖希共济失调的药物治疗：随机试验的网状Meta分析
CRD42023481130
Primary question - To compare the clinical efficacy of different pharmacological agents in Friedreich Ataxia. Secondary question - To compare the safety profile of different pharmacological agents in Friedreich Ataxia.
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Record Fields
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- Meta Analysis Id
- 7616
- Evidence Id
- 16174
- Core Evidence Id
- 16174
- Source Meta Analysis Id
- 7610
- Herb2 Meta Analysis Id
- HBMA007610
- Crd Id
- CRD42023481130
- Title
- Evaluating Pharmacological Interventions for Friedreich Ataxia: A Network Meta-Analysis of Randomised Trials
- Review Question
- Primary question - To compare the clinical efficacy of different pharmacological agents in Friedreich Ataxia. Secondary question - To compare the safety profile of different pharmacological agents in Friedreich Ataxia.
- Study Type Included
- We will include only Randomised Controlled Trials that utilize drugs under consideration as the intervention and report the specific outcome measures of interest.
- Condition Being Studied
- Pharmacological interventions used to manage Friedreich Ataxia. Friedreich ataxia is a rare genetic disorder that primarily affects the nervous system. It is characterized by progressive damage to the nervous tissue in the spinal cord and specific areas of the brain, leading to symptoms such as muscle weakness, difficulty with coordination and balance, and speech problems. The condition is typically caused by mutations in the FXN gene, which leads to a deficiency of a protein called frataxin. This deficiency results in an accumulation of iron in the mitochondria of cells, leading to oxidative stress and damage to nerve cells.
- Participant
- Patients diagnosed with Friedreich Ataxia
- Animal
- Human Disease Modelled
- Intervention
- Linoleic Acid Triterpenes - Omaveloxolone Luvadaxistat Interferon-gamma (interferon- γ 1b) Idebenone EPI-743 Deferiprone Epoetin alfa Riluzole Carbamylated erythropoietin A0001 (α-tocopheryl quinone) Erythropoietin L-carnitine creatine 5-Hydroxytryptophan Amantadine Hydrochloride Coenzyme Q10 Vitamin E Bupropion Citalopram VP 20629 Epicatechin Riboflavin CTI-1601 Leriglitazone Lecithin Vigabatrin Resveratrol EGb 761 Dimethyl fumarate TAK-831 IGF-1 After a thorough evaluation of the available trials and interim analysis published in the literature, relevant trials will be incorporated for meta-analysis.
- Comparator Control
- Placebo
- Main Outcome
- Compare the clinical efficacy and safety of different pharmacological agents in Friedreich Ataxia. For assessing the efficacy of the drugs: 1. Modified Friedreich's Ataxia Rating Scale (mFARS) assesses declining domains (speech, limb function, standing, gait) as the disease progresses. The maximum possible score is 93, with higher scores indicating more severe impairment. Friedreich's Ataxia Rating Scale (FARS) scores will be transformed to mFARS on a 125-point scale. 2. The International Cooperative Ataxia Rating Scale (ICARS) quantifies symptoms grouped into domains of posture and gait, kinetic function, speech, and oculomotor aspects. 3. Scale for the Assessment and Rating of Ataxia (SARA) scores assess clinical progression across domains including gait, stance, sitting ability, speech, and coordination in the upper limbs. The maximum score is 40, indicating the most severe form of ataxia. 4. 9-Hole Peg Test (9-HPT) scores measure finger dexterity and arm-hand coordination. The score is represented by the time taken to complete the task, with shorter times reflecting better function. 5. Timed 25-Foot Walk (T25-FW) scores reflect lower-limb functional status. The averaged time to complete two trials is recorded as the score, providing insight into the individual's mobility. For assessing the safety/tolerability of the drug: 1. Any AE 2. Serious AE 3. Any other side effects specific to the drug Measures of effect Proportion variable: Risk ratio/Hazard ratio will be calculated Continuous variable: Mean difference between populations will be calculated
- Outcome Measure
- Additional Outcome
- Alongside the prespecified research questions, other clinically relevant and clinically and statistically homogenous retrievable outcomes will also be synthesised.
- Study Method
- Intervention, Meta-analysis, Network meta-analysis, Systematic review
- Keyword
- MeSH headings have not been applied to this record
- Contact
- Tirth Dave [email protected]
- Organisational Affiliation
- Bukovinian State Medical University
- Funding Source
- Other Selection Criteria
- Final Publication
- Same Topic Review
- Published Protocol
- Review Type
- Language
- English
- Country
- India, Nepal, Ukraine
- Review Stage
- Review Ongoing
- First Submission Date
- 2023-11-08
- Registration Date
- 2023-11-20
- Anticipated Start Date
- 2023-11-03
- Anticipated Completion Date
- 2024-03-03
- Title Cn
- 评估弗里德赖希共济失调的药物治疗：随机试验的网状Meta分析
- Title En
- Evaluating Pharmacological Interventions for Friedreich Ataxia: A Network Meta-Analysis of Randomised Trials
- Bilingual Status
- complete