Creatine ( DrugBank: Creatine )

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16 diseases

ID	Disease name (Link within this page)	Number of trials
1	Spinal and bulbar muscular atrophy	1
2	Amyotrophic lateral sclerosis	6
3	Spinal muscular atrophy	1
5	Progressive supranuclear palsy	1
6	Parkinson disease	2
8	Huntington disease	6
13	Multiple sclerosis/Neuromyelitis optica	1
15	Inclusion body myositis	1
21	Mitochondrial disease	1
46	Malignant rheumatoid arthritis	1
49	Systemic lupus erythematosus	1
50	Dermatomyositis	2
113	Muscular dystrophy	3
156	Rett syndrome	1
201	Angelman syndrome	1
256	Muscle glycogenosis	1

1. Spinal and bulbar muscular atrophy

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Clinical trials : 17 /  Drugs : 16 - (DrugBank : 8) / Drug target genes : 10 - Drug target pathways : 17

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	JPRN-UMIN000012503	2014/06/01	25/12/2013	Effect of creatine in SBMA patients: A double-blind, placebo-controlled clinical study		Spinal and Bulbar Muscular Atrophy	Creatine monohydrate 10 g, daily for 8 weeks Creatine monohydrate 15 g, daily for 8 weeks placebo for 8 weeks	Department of Neurology, Nagoya University Graduate School of Medicine	NULL	Complete: follow-up continuing	20years-old	80years-old	Male	45	Not applicable	Japan

2. Amyotrophic lateral sclerosis

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Clinical trials : 624 /  Drugs : 611 - (DrugBank : 160) / Drug target genes : 172 - Drug target pathways : 225

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01257581 (ClinicalTrials.gov)	March 2011	8/12/2010	Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS)	Phase 2 Selection Trial of High Dosage Creatine and Two Dosages of Tamoxifen in Amyotrophic Lateral Sclerosis (ALS)	Amyotrophic Lateral Sclerosis	Drug: creatine;Drug: tamoxifen	Nazem Atassi	ALS Therapy Alliance;State University of New York - Upstate Medical University	Completed	18 Years	N/A	All	60	Phase 2	United States
2	NCT00355576 (ClinicalTrials.gov)	July 2006	21/7/2006	Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis	Phase II Combination Therapy Selection Trial in Amyotrophic Lateral Sclerosis	Amyotrophic Lateral Sclerosis	Drug: Celecoxib;Drug: Creatine;Drug: Minocycline	Columbia University	ALS Association;Pfizer	Completed	21 Years	85 Years	Both	86	Phase 2	United States
3	NCT00069186 (ClinicalTrials.gov)	June 2003	17/9/2003	Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis	A Phase III, Multi-Center, Double-Blind, Placebo Controlled, Randomized Study of Creatine Monohydrate in Patients With Amyotrophic Lateral Sclerosis	Amyotrophic Lateral Sclerosis (ALS)	Drug: Creatine Monohydrate	The Avicena Group	NULL	Active, not recruiting	21 Years	80 Years	Both	107	Phase 3	United States
4	NCT00070993 (ClinicalTrials.gov)	December 2002	9/10/2003	Creatine for the Treatment of Amyotrophic Lateral Sclerosis	Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis	Amyotrophic Lateral Sclerosis	Drug: creatine monohydrate	National Center for Complementary and Integrative Health (NCCIH)	Office of Dietary Supplements (ODS)	Completed	21 Years	80 Years	Both	110	Phase 2	United States
5	NCT00005674 (ClinicalTrials.gov)	May 2000	26/5/2000	Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis [ALS]		Amyotrophic Lateral Sclerosis	Drug: Creatine	National Center for Research Resources (NCRR)	Muscular Dystrophy Association	Completed	18 Years	80 Years	Both		Phase 2	United States
6	NCT00005766 (ClinicalTrials.gov)	May 2000	1/6/2000	Clinical Trial of Creatine in Amyotrophic Lateral Sclerosis		Amyotrophic Lateral Sclerosis	Drug: Creatinine	National Center for Research Resources (NCRR)	NULL	Completed	18 Years	80 Years	Both		Phase 2	United States

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3. Spinal muscular atrophy

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Clinical trials : 217 /  Drugs : 149 - (DrugBank : 33) / Drug target genes : 54 - Drug target pathways : 80

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05272969 (ClinicalTrials.gov)	March 31, 2022	28/1/2022	Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease	Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)	Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHD	Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencing	LMU Klinikum	NULL	Not yet recruiting	18 Years	N/A	All	95		Germany

5. Progressive supranuclear palsy

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Clinical trials : 95 /  Drugs : 119 - (DrugBank : 40) / Drug target genes : 65 - Drug target pathways : 108

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00605930 (ClinicalTrials.gov)	April 2004	14/1/2008	A Pilot Clinical Trial of Pyruvate, Creatine, and Niacinamide in Progressive Supranuclear Palsy.	Energy Metabolism in Neurodegenerative Diseases: A Randomized, Double Blind, Placebo-Controlled Clinical Pilot Trial of Pyruvate, Creatine, and Niacinamide in Progressive Supranuclear Palsy.	Progressive Supranuclear Palsy	Dietary Supplement: Pyruvate, creatine, niacinamide;Dietary Supplement: Placebo	University of Louisville	NULL	Completed	N/A	N/A	All	20	N/A	United States

6. Parkinson disease

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Clinical trials : 2,298 /  Drugs : 2,202 - (DrugBank : 350) / Drug target genes : 188 - Drug target pathways : 202

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00449865 (ClinicalTrials.gov)	March 2007	20/3/2007	NET-PD LS-1 Creatine in Parkinson's Disease	A Multicenter, Double-Blind, Parallel Group, Placebo Controlled Study of Creatine in Subjects With Treated Parkinson's Disease (PD) Long Term Study (LS-1)	Parkinson's Disease	Drug: creatine;Other: placebo	University of Rochester	National Institute of Neurological Disorders and Stroke (NINDS)	Terminated	N/A	N/A	All	1741	Phase 3	United States;Canada
2	NCT00063193 (ClinicalTrials.gov)	May 2003	23/6/2003	National Institute of Neurological Disorders and Stroke (NINDS) Parkinson's Disease Neuroprotection Trial	A Multi-center, Double-blind, Pilot Study of Minocycline and Creatine in Subjects With Early Untreated Parkinson's Disease	Parkinson's Disease	Drug: minocycline;Drug: creatine	University of Rochester	National Institute of Neurological Disorders and Stroke (NINDS)	Completed	30 Years	N/A	Both	195	Phase 2	United States

8. Huntington disease

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Clinical trials : 229 /  Drugs : 193 - (DrugBank : 60) / Drug target genes : 84 - Drug target pathways : 158

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01411163 (ClinicalTrials.gov)	April 2010	4/8/2011	Premanifest Huntington's Disease Extension Study II: Creatine Safety & Tolerability	Premanifest Huntington's Disease Extension Study II: Creatine Safety & Tolerability	Huntington's Disease	Drug: Creatine monohydrate	Massachusetts General Hospital	National Institutes of Health (NIH)	Completed	26 Years	N/A	Both	24	Phase 2	United States
2	NCT00712426 (ClinicalTrials.gov)	September 2009	8/7/2008	Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)	Creatine Safety, Tolerability, & Efficacy in Huntington's Disease (CREST-E)	Huntington's Disease	Drug: Creatine Monohydrate;Drug: Placebo	Massachusetts General Hospital	University of Rochester;National Center for Complementary and Integrative Health (NCCIH)	Terminated	18 Years	N/A	Both	553	Phase 3	United States;Australia;Canada;New Zealand
3	NCT01411150 (ClinicalTrials.gov)	May 2009	4/8/2011	Premanifest Huntington's Disease: Creatine Safety & Tolerability Extension Study	Premanifest Huntington's Disease: Creatine Safety & Tolerability Extension Study	Huntington's Disease	Drug: Creatine Monohydrate	Massachusetts General Hospital	National Institutes of Health (NIH)	Completed	26 Years	N/A	Both	38	Phase 2	United States
4	NCT00592995 (ClinicalTrials.gov)	December 2007	28/12/2007	Creatine Safety and Tolerability in Premanifest HD: PRECREST	Creatine Safety and Tolerability in Premanifest HD: PRECREST	Huntington Disease	Drug: Creatine monohydrate;Drug: Placebo	Massachusetts General Hospital	NULL	Completed	26 Years	N/A	Both	64	Phase 2	United States
5	NCT01412151 (ClinicalTrials.gov)	April 2005	5/8/2011	Creatine Safety & Tolerability in Huntington's Disease	Creatine Safety & Tolerability in Huntington's Disease (CREST-X): A Single-Center, Open-Label, Long-Term Safety & Tolerability Extension Study of Creatine in Subjects With HD	Huntington's Disease (HD)	Drug: Creatine monohydrate	Massachusetts General Hospital	NULL	Completed	18 Years	N/A	All	10	Phase 2	United States
6	NCT00026988 (ClinicalTrials.gov)	October 2001	15/11/2001	Creatine Therapy for Huntington's Disease	Creatine Therapy for Huntington's Disease	Huntington's Disease	Drug: Creatine	National Center for Complementary and Integrative Health (NCCIH)	NULL	Completed	18 Years	N/A	Both	64	Phase 1/Phase 2	United States

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13. Multiple sclerosis/Neuromyelitis optica

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Clinical trials : 3,342 /  Drugs : 2,355 - (DrugBank : 406) / Drug target genes : 269 - Drug target pathways : 241

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01381354 (ClinicalTrials.gov)	October 2010	26/5/2011	Nutrition, Neuromuscular Electrical Stimulation (NMES) and Secondary Progressive Multiple Sclerosis (SPMS)	Nutrition, Neuromuscular Electrical Stimulation (NMES) and Secondary Progressive Multiple Sclerosis (SPMS)	Multiple Sclerosis	Other: Progressive exercise;Device: Neuromuscular electrical stimulation delivered using the electrical therapy device EMPI 300 PV;Other: Modified paleolithic diet;Dietary Supplement: Omega 3 fatty acids;Dietary Supplement: Full Spectrum vitamin;Dietary Supplement: Essential - hydroxytyrosol;Dietary Supplement: Maltodextrin fiber supplement;Dietary Supplement: Mineral boost (magnesium);Dietary Supplement: Niacinamide;Dietary Supplement: Methyl B12;Dietary Supplement: Taurine;Dietary Supplement: creatine;Dietary Supplement: thiamine;Dietary Supplement: riboflavin;Dietary Supplement: N acetylcysteine;Dietary Supplement: alpha lipoic acid;Dietary Supplement: L acetyl carnitine;Dietary Supplement: methyl folate;Dietary Supplement: coenzyme Q;Behavioral: meditation;Behavioral: self massage;Behavioral: learning;Dietary Supplement: Coconut oil	University of Iowa	Direct MS Canada;DJO Incorporated;Pinnaclife Inc.;TZ Press, LLC	Completed	18 Years	65 Years	All	38	Phase 1	United States

15. Inclusion body myositis

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Clinical trials : 42 /  Drugs : 60 - (DrugBank : 16) / Drug target genes : 12 - Drug target pathways : 123

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05272969 (ClinicalTrials.gov)	March 31, 2022	28/1/2022	Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease	Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)	Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHD	Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencing	LMU Klinikum	NULL	Not yet recruiting	18 Years	N/A	All	95		Germany

21. Mitochondrial disease

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Clinical trials : 33 /  Drugs : 42 - (DrugBank : 32) / Drug target genes : 47 - Drug target pathways : 67

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04734626 (ClinicalTrials.gov)	May 25, 2021	25/1/2021	Muscle CrCest Muscle Phenotyping in Primary Mitochondrial Disease	Magnetic Resonance Imaging (MRI) Muscle Phenotyping in Mitochondrial Disease	Mitochondrial Diseases	Diagnostic Test: Creatine Chemical Exchange Saturation Transfer (CrCEST) Imaging Sequence	Children's Hospital of Philadelphia	NULL	Recruiting	7 Years	75 Years	All	40		United States

46. Malignant rheumatoid arthritis

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Clinical trials : 4,325 /  Drugs : 2,671 - (DrugBank : 417) / Drug target genes : 187 - Drug target pathways : 224

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01767844 (ClinicalTrials.gov)	January 2013	8/1/2013	Can Creatine Supplementation Improve Body Composition and Physical Function in Rheumatoid Arthritis Patients?	Can Creatine Supplementation Improve Body Composition and Physical Function in Rheumatoid Arthritis Patients? A Randomised Controlled Pilot Trial	Rheumatoid Arthritis	Dietary Supplement: Creatine;Dietary Supplement: Placebo	Bangor University	NULL	Active, not recruiting	18 Years	N/A	Both	43	N/A	United Kingdom

49. Systemic lupus erythematosus

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Clinical trials : 946 /  Drugs : 722 - (DrugBank : 186) / Drug target genes : 117 - Drug target pathways : 199

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01217320 (ClinicalTrials.gov)	January 2011	6/10/2010	Creatine Supplementation in Pediatric Rheumatology	Efficacy and Safety of Creatine Supplementation in Patients With Juvenile Systemic Lupus Erythematosus and Juvenile Dermatomyositis	Juvenile Systemic Lupus Erythematosus;Juvenile Dermatomyositis	Dietary Supplement: creatine;Dietary Supplement: placebo (dextrose)	University of Sao Paulo	NULL	Recruiting	6 Years	18 Years	Both	40	N/A	Brazil

50. Dermatomyositis

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Clinical trials : 182 /  Drugs : 229 - (DrugBank : 88) / Drug target genes : 48 - Drug target pathways : 147

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT02267005 (ClinicalTrials.gov)	March 2015	1/10/2014	The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis	The Effect of Creatine Supplementation on Muscle Function in Childhood Myositis	Juvenile Dermatomyositis	Dietary Supplement: Creapure;Dietary Supplement: Glucose Tablet	The Hospital for Sick Children	NULL	Completed	7 Years	18 Years	All	13	N/A	Canada
2	NCT01217320 (ClinicalTrials.gov)	January 2011	6/10/2010	Creatine Supplementation in Pediatric Rheumatology	Efficacy and Safety of Creatine Supplementation in Patients With Juvenile Systemic Lupus Erythematosus and Juvenile Dermatomyositis	Juvenile Systemic Lupus Erythematosus;Juvenile Dermatomyositis	Dietary Supplement: creatine;Dietary Supplement: placebo (dextrose)	University of Sao Paulo	NULL	Recruiting	6 Years	18 Years	Both	40	N/A	Brazil

113. Muscular dystrophy

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Clinical trials : 622 /  Drugs : 485 - (DrugBank : 99) / Drug target genes : 59 - Drug target pathways : 168

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2006-006776-37-NL (EUCTR)	14/05/2007	22/12/2006	Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD	Towards the assessment of metabolic biomarkers and creatine uptake and turnover in skeletal muscles of patients with Facioscapulohumeral muscular dystrophy using MR spectroscopy - Assessment of biomarkers in FSHD	Healthy volunteers. Later the protocol will be applied to Facioscapular humeral dystrophy and/or other muscular dystrophies in general. MedDRA version: 8.1;Level: LLT;Classification code 10011328;Term: Creatine	Product Name: creatine-13C Product Code: Cr13C	Radboud University Nijmegen Medical Center	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes			Netherlands
2	NCT00018109 (ClinicalTrials.gov)	June 2001	3/7/2001	A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)	A Multicenter Randomized Placebo-Controlled Double-Blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy (DMD)	Muscular Dystrophy, Duchenne	Drug: glutamine;Drug: creatine monohydrate	National Center for Research Resources (NCRR)	Children's Research Institute	Completed	5 Years	10 Years	Male		Phase 3	United States
3	NCT00016653 (ClinicalTrials.gov)	June 2000	21/5/2001	Creatine and Glutamine in Steroid-Naive Duchenne Muscular Dystrophy	A Multicenter Randomized Placebo-controlled Double-blind Study to Assess Efficacy and Safety of Glutamine and Creatine Monohydrate in Duchenne Muscular Dystrophy	Muscular Dystrophy, Duchenne	Drug: Creatine Monohydrate;Drug: Glutamine	Cooperative International Neuromuscular Research Group	NULL	Completed	5 Years	9 Years	Male	48	Phase 2/Phase 3	United States;Belgium;Israel;Puerto Rico

156. Rett syndrome

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Clinical trials : 40 /  Drugs : 53 - (DrugBank : 19) / Drug target genes : 77 - Drug target pathways : 113

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01147575 (ClinicalTrials.gov)	January 2005	17/6/2010	Effects of Creatine Supplementation in Rett Syndrome	Effects of Creatine Supplementation in Rett Syndrome: A Randomized, Placebo-controlled Trial	Rett Syndrome	Dietary Supplement: Creatine monohydrate;Dietary Supplement: Placebo	Medical University of Vienna	NULL	Completed	3 Years	24 Years	Female	21	N/A	Austria

201. Angelman syndrome

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Clinical trials : 24 /  Drugs : 35 - (DrugBank : 9) / Drug target genes : 22 - Drug target pathways : 20

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT00348933 (ClinicalTrials.gov)	July 2006	3/7/2006	Dietary Supplements for the Treatment of Angelman Syndrome	Efficacy of a Therapeutic Treatment Trial in Angelman Syndrome	Angelman Syndrome;Nervous System Diseases	Drug: Betaine;Drug: Creatine;Drug: Metafolin;Drug: Vitamin B12	University of California, San Diego	Baylor College of Medicine;Rady Children's Hospital, San Diego;Boston Children’s Hospital;Greenwood Genetic Center;Rare Diseases Clinical Research Network	Completed	N/A	5 Years	All	90	N/A	United States

256. Muscle glycogenosis

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Clinical trials : 180 /  Drugs : 153 - (DrugBank : 30) / Drug target genes : 31 - Drug target pathways : 134

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT05272969 (ClinicalTrials.gov)	March 31, 2022	28/1/2022	Pompe & Pain - Study to Assess Nociceptive Pain in Adult Patients With Pompe Disease	Pompe & Pain - Observational Study to Assess Musculoskeletal Pain in Late-onset Pompe Disease (LOPD)	Pompe Disease (Late-onset);Inclusion Body Myositis;Spinal Muscular Atrophy Type 3;FSHD	Diagnostic Test: Beck depression inventory fast screen (Questionnaire);Diagnostic Test: Brief Pain Inventory (BPI) (Questionnaire);Diagnostic Test: German Pain Inventory (Questionnaire);Diagnostic Test: Fatigue Severity and Disability Scale (FSS) (Questionnaire);Diagnostic Test: Rotterdam Handicap Scale (RHS) (Questionnaire);Diagnostic Test: R-PAct (Questionnaire);Diagnostic Test: Quick Motor Function Test;Diagnostic Test: Handheld Dynamometry (HHD);Diagnostic Test: Six-minute walk test (6MWT);Diagnostic Test: Pressure pain threshold;Diagnostic Test: Muscle ultrasound;Diagnostic Test: Vital signs;Diagnostic Test: Borg Scale;Diagnostic Test: Laboratory assessment: Creatine kinase;Diagnostic Test: Laboratory assessment: Vitamin D Level;Diagnostic Test: Laboratory assessment: calcium;Diagnostic Test: Laboratory assessment: magnesium;Diagnostic Test: Laboratory assessment: phosphate;Genetic: Genetic test: ACE polymorphism;Genetic: Genetic test: ACTN3 polymorphism;Genetic: Blood draw for optional genetic exome sequencing	LMU Klinikum	NULL	Not yet recruiting	18 Years	N/A	All	95		Germany

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