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Clinical trials for Metabolic Disease

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

  • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).


    The EU Clinical Trials Register currently displays   44381   clinical trials with a EudraCT protocol, of which   7393   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
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    Query did not match any clinical trials.
    These are paediatric studies completed by 26 January 2007, in respect of products authorised in the Community (Article 45 of the Paediatric Regulation (EC) No 1901/2006).
    Note: Advanced search filters for country, trial phase, gender, rare disease, IMP with orphan designation in the indication and age ranges of in-utero, adult or elderly do not apply to these records.
    Study title: ACARBOSE (BAY G 5421) IN INSULIN-DEPENDENT DIABETES - THE EFFECT ON THE DAILY BLOOD-GLUCOSE PROFILE AND ON THE INSULINCONSUMPTION BY AN ARTIFICIAL PANCREAS (STUDY 0160)(PH-9873)
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0160_study_synopsis.pdf
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    Document reference: 46154
    Study title: Assessment of efficacy and tolerability of Acarbose in children under 16 years
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_9981_study_synopsis.pdf
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    Document reference: 46160
    Study title: Clinical Study on the Efficacy and Tolerability of Acarbose (BAY g 5421) Compared with Placeboin Diabetic Children on Conventional Insulin Treatment, in Particular with Observation of the Glycemic Control Index ( PH-24820 )
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0665_study_synopsis.pdf
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    Document reference: 46158
    Study title: Clinical Study to Investigate the Efficacy and Tolerability of Acarbose (BAY g 5421) Versus Placebo in Diabetic Children (PH 23433)
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0634_study_synopsis.pdf
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    Document reference: 46155
    Study title: Double-Blind, Cross-Over Clinical Study at a Single Centre to Compare Acarbose (BAY g 5421) with Placebo in Respect of Efficacy and Tolerability in Patients with Cystic Fibrosis and Pathological Glucose Tolerance (Pilot Study)(PH-26068)
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0696_study_synopsis.pdf
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    Document reference: 46159
    Study title: NON RANDOMISED FOLLW-UP STUDY WITH ACARBOSE-TREATMENT AND PRE- AND AFTER-TREATMENT WITH PLACEBO IN DIABETIC CHILDREN (TYPE I) STUDY 0428 (PH 14352)
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0428_study_synopsis.pdf
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    Document reference: 46156
    Study title: The effect of acarbose (Bay G 5421) on the insulin consumption of children treated with insulin.
    Active substance: ACARBOSE
    Study summary document link (including results): Bayer Pharma_Acarbose_0221_study_synopsis.pdf
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    Document reference: 46157
    Study title: VIALLATE J. Appréciation clinique du rôle de l'Arginine sur la nutrition et la croissance (Clinical estimate of the part played by arginine on nutrition and growth). Rev. Pédiatrie, VIII, 7, 419-423, 1972
    Active substance: ARGININE DIHYDRATE
    Study summary document link (including results): Arginine Option1.xls
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    Document reference: 47170
    Study title: Higuchi-R, Mizukoshi-M, Koyama-H, Kitano-N, Koike-M. Intractable diaper dermatitis as an early sign of biotin deficiency. Intractable diaper dermatitis as an early sign of biotin deficiency. Acta paediatrica (Oslo Norway : 1992), {Acta-Paediatr}, Feb 1998, vol. 87, no. 2, p. 228-9, ISSN: 0803-5253.Higuchi-R, Mizukoshi-M, Koyama-H, Kitano-N, Koike-M. Intractable diaper dermatitis as an early sign of biotin deficiency. Intractable diaper dermatitis as an early sign of biotin deficiency. Acta paediatrica (Oslo Norway : 1992), {Acta-Paediatr}, Feb 1998, vol. 87, no. 2, p. 228-9, ISSN: 0803-5253.
    Active substance: BIOTIN
    Study summary document link (including results):
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    Document reference: 22558
    Study title: Higuchi-R, Noda-E, Koyama-Y, Shirai-T, Horino-A, Juri-T, Koike-M. Department of Pediatrics, Wakayama Medical College, Japan. Biotin deficiency in an infant fed with amino acid formula and hypoallergenic rice. Acta paediatrica (Oslo Norway : 1992), {Acta-Paediatr}, Jul 1996, vol. 85, no. 7, p. 872-4, ISSN: 0803-5253.Higuchi-R, Noda-E, Koyama-Y, Shirai-T, Horino-A, Juri-T, Koike-M. Department of Pediatrics, Wakayama Medical College, Japan. Biotin deficiency in an infant fed with amino acid formula and hypoallergenic rice. Acta paediatrica (Oslo Norway : 1992), {Acta-Paediatr}, Jul 1996, vol. 85, no. 7, p. 872-4, ISSN: 0803-5253.
    Active substance: BIOTIN
    Study summary document link (including results):
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    Document reference: 22560
    Study title: Hoffman-Trevor-L, Simon-Erin-M, Ficicioglu-Can. Section of Metabolism, The Children's Hospital of Philadelphia, 34th and Civic Center Boulevard, Philadelphia, PA 19104-4399, USA. Biotinidase deficiency: the importance of adequate follow-up for an inconclusive newborn screening result. European journal of pediatrics, {Eur-J-Pediatr}, May 2005 (epub: 15 Feb 2005), vol. 164, no. 5, p. 298-301, ISSN: 0340-6199.Hoffman-Trevor-L, Simon-Erin-M, Ficicioglu-Can. Section of Metabolism, The Children's Hospital of Philadelphia, 34th and Civic Center Boulevard, Philadelphia, PA 19104-4399, USA. Biotinidase deficiency: the importance of adequate follow-up for an inconclusive newborn screening result. European journal of pediatrics, {Eur-J-Pediatr}, May 2005 (epub: 15 Feb 2005), vol. 164, no. 5, p. 29...
    Active substance: BIOTIN
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    Document reference: 22597
    Study title: Mardach-R, Zempleni-J, Wolf-B, Cannon-M-J, Jennings-M-L, Cress-S, Boylan-J, Roth-S, Cederbaum-S, Mock-D-M. Department of Biochemistry, Univ. of Arkansas for Med. Sciences, 4301 West Markham Street, Little Rock, AR 72205, United States. Biotin dependency due to a defect in biotin transport. Journal of Clinical Investigation {J-CLIN-INVEST}, 2002, Vol/Iss/Pg. 109/12 (1617-1623), ISSN: 0021-9738.Mardach-R, Zempleni-J, Wolf-B, Cannon-M-J, Jennings-M-L, Cress-S, Boylan-J, Roth-S, Cederbaum-S, Mock-D-M. Department of Biochemistry, Univ. of Arkansas for Med. Sciences, 4301 West Markham Street, Little Rock, AR 72205, United States. Biotin dependency due to a defect in biotin transport. Journal of Clinical Investigation {J-CLIN-INVEST}, 2002, Vol/Iss/Pg. 109/12 (1617-1623), ISSN: 0021-9738.
    Active substance: BIOTIN
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    Document reference: 22605
    Study title: Martin-C. Biotin (vitamin H); use in the treatment of infant dermatitis. Le Nourrisson, {Nourrisson}, May-Jun 1951, vol. 39, no. 3, p. 111-9.Martin-C. Biotin (vitamin H); use in the treatment of infant dermatitis. Le Nourrisson, {Nourrisson}, May-Jun 1951, vol. 39, no. 3, p. 111-9.
    Active substance: BIOTIN
    Study summary document link (including results):
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    Document reference: 22593
    Study title: McVoy-J-R, Levy-H-L, Lawler-M, Schmidt-M-A, Ebers-D-D, Hart-P-S, Pettit-D-D, Blitzer-M-G, Wolf-B. Department of Human Genetics, Medical College of Virginia, Richmond 23298-0033. Partial biotinidase deficiency: clinical and biochemical features. The Journal of pediatrics, {J-Pediatr}, Jan 1990, vol. 116, no. 1, p. 78-83, ISSN: 0022-3476.McVoy-J-R, Levy-H-L, Lawler-M, Schmidt-M-A, Ebers-D-D, Hart-P-S, Pettit-D-D, Blitzer-M-G, Wolf-B. Department of Human Genetics, Medical College of Virginia, Richmond 23298-0033. Partial biotinidase deficiency: clinical and biochemical features. The Journal of pediatrics, {J-Pediatr}, Jan 1990, vol. 116, no. 1, p. 78-83, ISSN: 0022-3476.
    Active substance: BIOTIN
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    Document reference: 22630
    Study title: Messaritakis-J, Kattamis-C, Karabula-C, Matsaniotis-N. Generalized seborrhoeic dermatitis. Clinical and therapeutic data of 25 patients. Archives of disease in childhood, {Arch-Dis-Child}, Nov 1975, vol. 50, no. 11, p. 871-4, ISSN: 1468-2044.Messaritakis-J, Kattamis-C, Karabula-C, Matsaniotis-N. Generalized seborrhoeic dermatitis. Clinical and therapeutic data of 25 patients. Archives of disease in childhood, {Arch-Dis-Child}, Nov 1975, vol. 50, no. 11, p. 871-4, ISSN: 1468-2044.
    Active substance: BIOTIN
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    Document reference: 22586
    Study title: Michalski-A-J, Berry-G-T, Segal-S. Division of Biochemical Development and Molecular Diseases, Children's Hospital of Philadelphia, PA. Holocarboxylase synthetase deficiency: 9-year follow-up of a patient on chronic biotin therapy and a review of the literature. Journal of inherited metabolic disease, {J-Inherit-Metab-Dis}, 1989, vol. 12, no. 3, p. 312-6, 19 refs, ISSN: 0141-8955.Michalski-A-J, Berry-G-T, Segal-S. Division of Biochemical Development and Molecular Diseases, Children's Hospital of Philadelphia, PA. Holocarboxylase synthetase deficiency: 9-year follow-up of a patient on chronic biotin therapy and a review of the literature. Journal of inherited metabolic disease, {J-Inherit-Metab-Dis}, 1989, vol. 12, no. 3, p. 312-6, 19 refs, ISSN: 0141-8955.
    Active substance: BIOTIN
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    Document reference: 22633
    Study title: Mock-D-M, Baswell-D-L, Baker-H, Holman-R-T, Sweetman-L. Biotin deficiency complicating parenteral alimentation: diagnosis, metabolic repercussions, and treatment. Annals of the New York Academy of Sciences, {Ann-N-Y-Acad-Sci}, 1985, vol. 447, p. 314-34, ISSN: 0077-8923. Mock-D-M, Baswell-D-L, Baker-H, Holman-R-T, Sweetman-L. Biotin deficiency complicating parenteral alimentation: diagnosis, metabolic repercussions, and treatment. Annals of the New York Academy of Sciences, {Ann-N-Y-Acad-Sci}, 1985, vol. 447, p. 314-34, ISSN: 0077-8923.
    Active substance: BIOTIN
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    Document reference: 22643
    Study title: Mock-D-M, deLorimer-A-A, Liebman-W-M, Sweetman-L, Baker-H. Biotin deficiency: an unusual complication of parenteral alimentation. The New England journal of medicine, {N-Engl-J-Med}, 2 Apr 1981, vol. 304, no. 14, p. 820-3, ISSN: 0028-4793.Mock-D-M, deLorimer-A-A, Liebman-W-M, Sweetman-L, Baker-H. Biotin deficiency: an unusual complication of parenteral alimentation. The New England journal of medicine, {N-Engl-J-Med}, 2 Apr 1981, vol. 304, no. 14, p. 820-3, ISSN: 0028-4793.
    Active substance: BIOTIN
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    Document reference: 22654
    Study title: Moeslinger-D, Plecko-B, Stoeckler-Ipsiroglu-S. Universitaets-Kinderklinik, Waehringer Guertel 18-20, A-1090 Wien, Austria. Biotinidase deficiency - A treatable genetic disorder of biotin (vitamin H) metabolism. Symptoms and results of the Australian neonatal screening programme. Padiatrische Praxis {PADIATR-PRAX}, 2003, Vol/Iss/Pg. 63/2 (265-271), ISSN: 0030-9346.Moeslinger-D, Plecko-B, Stoeckler-Ipsiroglu-S. Universitaets-Kinderklinik, Waehringer Guertel 18-20, A-1090 Wien, Austria. Biotinidase deficiency - A treatable genetic disorder of biotin (vitamin H) metabolism. Symptoms and results of the Australian neonatal screening programme. Padiatrische Praxis {PADIATR-PRAX}, 2003, Vol/Iss/Pg. 63/2 (265-271), ISSN: 0030-9346.
    Active substance: BIOTIN
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    Document reference: 22602
    Study title: Honke-K, Hasui-M, Takano-N. Department of Pediatrics, National Sanatorium Io Hospital, Ishikawa. Abnormal metabolism of fatty acids and ketone bodies in Duchenne muscular dystrophy, and the effect of biotin on these abnormalities. No to hattatsu. Brain and development, {No-To-Hattatsu}, Jan 1997, vol. 29, no. 1, p. 13-8, ISSN: 0029-0831.Honke-K, Hasui-M, Takano-N. Department of Pediatrics, National Sanatorium Io Hospital, Ishikawa. Abnormal metabolism of fatty acids and ketone bodies in Duchenne muscular dystrophy, and the effect of biotin on these abnormalities. No to hattatsu. Brain and development, {No-To-Hattatsu}, Jan 1997, vol. 29, no. 1, p. 13-8, ISSN: 0029-0831.
    Active substance: BIOTIN
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    Document reference: 22615
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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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