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Results for “Barth Syndrome”

11 trials

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Showing 11 of 11 results

Large-scale testing (Phase 3)Study completedNCT03098797
What this trial is testing

A Trial to Evaluate Safety, Tolerability and Efficacy of Elamipretide in Subjects With Barth Syndrome

Who this might be right for
Barth Syndrome
Stealth BioTherapeutics Inc. 12
Not applicableStudy completedNCT01625663
What this trial is testing

Heart and Muscle Metabolism in Barth Syndrome

Who this might be right for
Barth Syndrome
Duke University 64
Not applicableStudy completedNCT01194141
What this trial is testing

Exercise Training in Barth Syndrome

Who this might be right for
Barth Syndrome
Washington University School of Medicine 4
Not applicableStudy completedNCT01629459
What this trial is testing

Resistance Exercise in Barth Syndrome

Who this might be right for
Barth Syndrome
Washington University School of Medicine 12
Large-scale testing (Phase 3)Looking for participantsNCT06056297
What this trial is testing

Mavorixafor in Participants With Congenital and Acquired Primary Autoimmune and Idiopathic Chronic Neutropenic Disorders Who Are Experiencing Recurrent and/or Serious Infections

Who this might be right for
Neutropenia
X4 Pharmaceuticals 176
Not applicableLooking for participantsNCT05554835
What this trial is testing

Global Registry and Natural History Study for Mitochondrial Disorders

Who this might be right for
Mitochondrial DiseasesKearns-Sayre SyndromeMIDD+15 more
LMU Klinikum 6,000
Not applicableLooking for participantsNCT01694940
What this trial is testing

North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC)

Who this might be right for
Mitochondrial DisordersMitochondrial Genetic DisordersMitochondrial Diseases+2 more
Columbia University 1,000
Not applicableEnrolling By InvitationNCT03655223
What this trial is testing

Early Check: Expanded Screening in Newborns

Who this might be right for
Spinal Muscular AtrophyFragile X SyndromeFragile X - Premutation+182 more
RTI International 30,000
Not applicableNo Longer AvailableNCT01461304
What this trial is testing

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

Who this might be right for
Very Long-chain acylCoA Dehydrogenase (VLCAD) DeficiencyCarnitine Palmitoyltransferase Deficiencies (CPT1, CPT2)Mitochondrial Trifunctional Protein Deficiency+5 more
Jerry Vockley, MD, PhD
Not applicableAvailableNCT04689360
What this trial is testing

An Intermediate Size Expanded Access Protocol of Elamipretide

Who this might be right for
Mitochondrial DiseasesBarth Syndrome
Stealth BioTherapeutics Inc.
Post-approval studies (Phase 4)Not Yet RecruitingNCT07531251
What this trial is testing

Ph3b/4 Trial in Patients With Barth Syndrome

Who this might be right for
Barth Syndrome
Stealth BioTherapeutics Inc. 48