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IRCT20210413050958N1 IRCT 2021-10-27 Mashhad University of Medical Sciences Effect of N-Acetyl-l-Leucine in treatment of ataxia-telangiectasia The effects of N-Acetyl-l-Leucine on the improvement of symptoms in patients with ataxia-telangiectasia: A double-blind crossover trial 2021-12-06 anticipated 16 Complete https://irct.ir/trial/56693 interventional Randomization: Randomized, Blinding: Triple blinded, Placebo: Used, Assignment: Crossover, Purpose: Treatment, Randomization description: Regarding the presence of the gender as a confounder, we run the block randomization method within each category (male and female) separately. For this reason, inside each category, blocks with size 4 and 2 of the combination of letters A and B (ABBA, ABAB, AABB, BAAB, BBAA, BABA), (AB and BA) are selected to the required number using a table of random numbers and individuals are assigned to groups according to the created sequence, Blinding description: The random allocation sequence is made using the table of random numbers. Sequentially numbered sealed envelopes are used to implement the random allocation sequence which opened by a person not involved in the project. The participants, care providers and statistician are blinded after assignment to intervention. So that, the caplet bottles are coded by a non-researcher person and remain confidential until data analysis. Moreover, In addition, placebo caplet is similar to supplement ones in shape, weight and color. 2 Ataxia-Telangiectasia. Intervention 1: Intervention group: Subjects in the intervention group receive N-Acetyl-L-Leucine caplets (daily intake of 1-4 gr depending on the subjects’ weight) for 6 weeks (n=8) and then after a 4-weeks wash-out period, they were crossed over to the alternate regimen. The participants take the supplement every day, which was contained in an unlabeled bottle. Supplements are from Hubei ipure Biotech co., ltd (Shenzhen, China). Intervention 2: Control group: The control group received the placebo (daily consumption between 1 to 4 grams depending on the subject's weight) for 6 weeks (n=8) and then after a 4-weeks wash-out period, they were crossed over to the alternate regimen. Participants take a placebo every day orally in an unlabeled bottle. The placebo is prepared by from faculty of pharmacy (Mashhad, Iran) company. No - There is not a plan to make this available Justification or reason for not sharing IPD is Raw data will be shared upon a reasonable request from the corresponding author.
public Maryam Saberi-Karimian
Ghaem Hospital, Ahmadabad Ave.
Mashhad Iran (Islamic Republic of) 99199-91766 +98 51 3840 0001 maryamsabery2012@gmail.com Mashhad University of Medical Sciences scientific Maryam Saberi-Karimian
Ghaem Hospital, Ahmadabad Ave.
Mashhad Iran (Islamic Republic of) 99199-91766 +98 51 3840 0001 maryamsabery2012@gmail.com Mashhad University of Medical Sciences Iran (Islamic Republic of) Signed informed consent form by the subjects or their parents after explaining the study objectives by the research team Patients with a definitive diagnosis of AT Having clinical signs Not addiction to Drugs and alcohol If the patient is receiving concomitant speech therapy or physiotherapy, he/she has been on a stable dose/duration and type of therapy for at least 4 weeks before visit 1 and throughout the duration of the study If the patient is taking any medication, he/she should maintain a constant dose/not change his/her treatment during the study period. no limit no limit Both Have not taken any forbidden drugs (including any variant of N-acetyl-DL-leucine, aminopyridines, Riluzole, gabapentin, Varenicline, Chlorzoxazone, sulfasalazine, Rosuvastatin at least 4 weeks before visit 1 and throughout the duration of the study Asymptomatic patients Patient who have clinical signs of A-T, but do not have a confirmed genetic test for A-T Patients who have any of the following: Chronic diarrhea, Unexplained visual loss, Malignancies, Insulin-dependent diabetes mellitus, Known history of hypersensitivity to the N-Acetyl-Leucine (DL-, L-, D-) or derivatives Having severe vision or hearing impairment that interferes with their ability to complete study assessments Having a definite diagnosis of arthritis or other musculoskeletal disorders that affects patient's mobility and interferes with their ability to complete study assessments G11.3 Cerebellar ataxia with defective DNA repair Treatment - Drugs Placebo Intervention group: Subjects in the intervention group receive N-Acetyl-L-Leucine caplets (daily intake of 1-4 gr depending on the subjects’ weight) for 6 weeks (n=8) and then after a 4-weeks wash-out period, they were crossed over to the alternate regimen. The participants take the supplement every day, which was contained in an unlabeled bottle. Supplements are from Hubei ipure Biotech co., ltd (Shenzhen, China). Control group: The control group received the placebo (daily consumption between 1 to 4 grams depending on the subject's weight) for 6 weeks (n=8) and then after a 4-weeks wash-out period, they were crossed over to the alternate regimen. Participants take a placebo every day orally in an unlabeled bottle. The placebo is prepared by from faculty of pharmacy (Mashhad, Iran) company. Movement signs. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Using the Scale for Assessment and Rating of Ataxia (SARA) score and Spinocerebellar Ataxia Functional Index (SCAFI). The quality of life. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Using PedsQL questionnaire. Cell blood count. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Sysmex Cell Counter. Lactate dehydrogenase. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Aspartate aminotransferase. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Alanine aminotransferase. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Urea. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Creatinine. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Alkaline phosphatase. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Na. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. K. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Total bilirubin. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Direct bilirubin. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: Auto analyzer instrument. Food recall. Timepoint: Before the intervention and 6 weeks after taking supplement or placebo in every study stage. Method of measurement: 24-hour food recall tool. Mashhad University of Medical Sciences Approved 2021-08-24 Ethics committee of Mashhad University of Medical Sciences Daneshgah Ave., Ghoreishi buildings Mashhad Razavi Khorasan Iran (Islamic Republic of)