Enriched fructose diet from day 1 to day 7.

Hereditary Fructose Intolerance

7 days (day 1 to day 7) with fructose enriched drinks 3x/d. Test meal at day 7 with fructose (0.7 g/body weight) and glucose (0.7 g/body weight)

Experimental diet Fru rich diet

7 days (day 1 to day 7) with low-fructose diet (<10g/d). Test meal at day 7 with fructose (0.7 g/body weight) and glucose (0.7 g/body weight)

Experimental low Fru diet

Guillaume Debray

Fructose Supplementation in Carriers for Hereditary Fructose Intolerance

Metabolic Effects of Short-term Dietary Supplementation With Fructose in Carriers for Hereditary Fructose Intolerance

The primary endpoint is the change of metabolite concentration after ingestion of a test meal (oral glucose and fructose load) under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in glucose concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in insulin concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in fructose concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in uric acid concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in lactate concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in urea concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in creatinine concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in phosphate concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in triglycerides concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in metabolome concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in ammonia concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in amino acid concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in alanine transminase (ALAT) concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in aspartate transaminase (ASAT) concentration after ingestion of a test meal under 2 conditions (enriched fructose diet vs poor fructose diet) according to a crossover design

Change in urine metabolome profile after low fructose diet and an enriched fructose diet according to a crossover design

Change in urine creatinine profile after low fructose diet and an enriched fructose diet according to a crossover design

Change in urine urea profile after low fructose diet and an enriched fructose diet according to a crossover design

Change in urine uric acid profile after low fructose diet and an enriched fructose diet according to a crossover design

Change of body weight after low fructose diet and an enriched fructose diet according to a crossover design

Change of systolic and diastolic pressure after low fructose diet and an enriched fructose diet according to a crossover design

Christel Tran

Principal Investigator

University of Lausanne

University of Liege

Patients with dermatomyositis and refractory skin disease on steroids and one steroid-sparing agent.

Dermatomyositis, Adult Type

Patients with refractory cutaneous dermatomyositis will be started on apremilast 30 mg twice a day in addition to a stable dose of their treatment regimen (including steroids and steroid sparing agents).

Apremilast 30mg

Tulane University

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

A Phase 2, Open Label Single Arm Study for Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

Cutaneous dermatomyositis disease area and severity index (CDASI) activity score is a validated tool to measure skin disease activity in dermatomyositis. The overall response rate (ORR) includes partial and complete responses. Complete response is defined by a CDASI activity score of zero. Partial response is defined by a decrease of CDASI activity score of at least 4 points. Calculation is performed as the CDASI activity score at 3 month(s) minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

The proportion of participants experiencing adverse events and serious adverse events will be measured over 7 months period (6 months during the study and 1 month follow up).

The durability of response will be measured using the CDASI activity score at 6 months minus CDASI activity score at 3 months. Complete response durability is defined as zero or minus difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Partial response durability is defined as >4 points difference between CDASI activity score at 6 months and CDASI activity score at 3 months. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

Complete delayed response is defined by a CDASI activity score of zero at 6 months. Partial delayed response is defined by a decrease of CDASI activity score of at least 4 points at 6 months compared to baseline. Calculation is performed as the CDASI activity score at 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
CDASI activity score: Units on a scale from 0-100. Higher scores represent worse outcome.

Dermatology Life Quality Index (DLQI) is a validated tool to measure quality of life in patients with skin disease. Complete response is defined by a DLQI of zero at 3, and 6 months. Partial response is defined by a decrease of DLQI of at least 5 points at 3, and 6 months compared to baseline. Calculation is performed as the DLQI at 3, and 6 months minus the score at baseline. Missing data will be handled using the last observation carried forward approach (LOCF).
Units : Units on a scale from 0-30, higher scores represent worse outcome.

Calculate the mean change in muscle enzymes value creatine kinase and aldolase at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: U/L, higher numbers represent worse outcomes

MMT-8 (Manual Muscle Testing-8) score is a validated tool to assess muscle strength. Calculate the mean change in MMT-8 score at 3 and 6 month(s) compared to baseline in patients with muscle disease.
Units: Units on a scale. Scale goes from 0-150. 150 is perfect strength.

Skin biopsies from lesional skin will be performed before treatment with apremilast and after 3 months of treatment to assess changes in gene expression profiling and immunohistochemistry stain. Gene expression profiling will be analyzed using inferential statistics with a False Discovery Rate (FDR) of < 0.05.

Data and research resources generated by this study will be made available to the research community and to the public at large. This strategy includes, but not limited to, presentations (poster or oral) at local, national, and international meetings; published abstracts, and journal articles (peer reviewed). Contact information will be provided in publications for direct inquiries of researchers. Data generated from sequencing and gene expression profiling will be deposited into the Gene Expression Omnibus (GEO), NCBI, to be accessible by general public. The study protocol will be shared with other researchers on the clinical trial.gov website. No identified personal patient informations will be shared.

aldolase/gyulladás

Fructose Supplementation in Carriers for Hereditary Fructose Intolerance

Evaluating Safety & Efficacy of Apremilast in the Treatment of Cutaneous Disease in Patients With Recalcitrant Dermatomyositis

A legteljesebb gyógynövény-adatbázis, amelyet a tudomány támogat