Studies
| Study First Submitted Date | 2020-12-23 |
| Study First Posted Date | 2020-12-30 |
| Last Update Posted Date | 2023-03-28 |
| Verification Month Year | March 2023 |
| Verification Date | 2023-03-31 |
| Last Update Posted Date | 2023-03-28 |
Conditions
| Sequence: | 52041440 | Sequence: | 52041439 |
| Name | Barth Syndrome | Name | Mitochondrial Diseases |
| Downcase Name | barth syndrome | Downcase Name | mitochondrial diseases |
Id Information
| Sequence: | 40056252 | Sequence: | 40056253 |
| Id Source | org_study_id | Id Source | secondary_id |
| Id Value | SPIES-006 | Id Value | SPIES-007 |
| Id Type | Other Identifier | ||
| Id Type Description | StealthBiotherapeutics | ||
Interventions
| Sequence: | 52353100 |
| Intervention Type | Drug |
| Name | elamipretide |
| Description | Elamipretide is an aromatic-cationic tetrapeptide that readily penetrates cell membranes and transiently localizes to the inner mitochondrial membrane. |
Keywords
| Sequence: | 79657420 |
| Name | Barth |
| Downcase Name | barth |
Browse Conditions
| Sequence: | 192968857 | Sequence: | 192968858 | Sequence: | 192968859 | Sequence: | 192968860 | Sequence: | 192968861 | Sequence: | 192968862 | Sequence: | 192968863 | Sequence: | 192968864 | Sequence: | 192968865 | Sequence: | 192968866 | Sequence: | 192968867 | Sequence: | 192968868 | Sequence: | 192968869 | Sequence: | 192968870 |
| Mesh Term | Barth Syndrome | Mesh Term | Mitochondrial Diseases | Mesh Term | Metabolic Diseases | Mesh Term | Heart Defects, Congenital | Mesh Term | Cardiovascular Abnormalities | Mesh Term | Cardiovascular Diseases | Mesh Term | Heart Diseases | Mesh Term | Abnormalities, Multiple | Mesh Term | Congenital Abnormalities | Mesh Term | Genetic Diseases, X-Linked | Mesh Term | Genetic Diseases, Inborn | Mesh Term | Lipid Metabolism, Inborn Errors | Mesh Term | Metabolism, Inborn Errors | Mesh Term | Lipid Metabolism Disorders |
| Downcase Mesh Term | barth syndrome | Downcase Mesh Term | mitochondrial diseases | Downcase Mesh Term | metabolic diseases | Downcase Mesh Term | heart defects, congenital | Downcase Mesh Term | cardiovascular abnormalities | Downcase Mesh Term | cardiovascular diseases | Downcase Mesh Term | heart diseases | Downcase Mesh Term | abnormalities, multiple | Downcase Mesh Term | congenital abnormalities | Downcase Mesh Term | genetic diseases, x-linked | Downcase Mesh Term | genetic diseases, inborn | Downcase Mesh Term | lipid metabolism, inborn errors | Downcase Mesh Term | metabolism, inborn errors | Downcase Mesh Term | lipid metabolism disorders |
| Mesh Type | mesh-list | Mesh Type | mesh-list | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor | Mesh Type | mesh-ancestor |
Sponsors
| Sequence: | 48197383 |
| Agency Class | INDUSTRY |
| Lead Or Collaborator | lead |
| Name | Stealth BioTherapeutics Inc. |
Central Contacts
| Sequence: | 11981218 | Sequence: | 11981219 |
| Contact Type | primary | Contact Type | backup |
| Name | Donna Cowan | Name | Donna Cowan |
| Phone | 617.600.6888 | ||
| donna.cowan@stealthbt.com | access@stealthbt.com | ||
| Role | Contact | Role | Contact |
Eligibilities
| Sequence: | 30688856 |
| Gender | All |
| Minimum Age | 1 Year |
| Maximum Age | 80 Years |
| Criteria | Key Inclusion Criteria: ≥1 year and ≤ 80 years of age or ≥12 years for Barth Syndrome in SPIES-007 Patients with genetically confirmed rare primary mitochondrial diseases including Barth Syndrome observed cardiomyopathy, renal impairment, neuropathic, or ophthalmic manifestation where such disease is serious or life-threatening and no comparable or satisfactory alternative therapy options available. Patients without genetic confirmation of a rare disease with known mitochondrial dysfunction but do exhibit serious or life-threatening clinical manifestations of mitochondrial dysfunction. Is self-able or has caregiver willing and able to administer SC injection. Would potentially benefit from treatment with elamipretide and cannot be treated satisfactorily with any approved medicinal product in the opinion of the treating physician. Key Exclusion Criteria: Known hypersensitivity to elamipretide or any excipients. Women who are pregnant, are planning on becoming pregnant, or are breast-feeding. Patients receiving any other investigational agent within 30 days of dosing. Any active, serious psychiatric, medical, or other conditions/situations which, in the treating physician's opinion, could compromise the patient's safety. |
| Adult | True |
| Child | True |
| Older Adult | True |
Calculated Values
| Sequence: | 253897014 |
| Registered In Calendar Year | 2020 |
| Were Results Reported | False |
| Has Single Facility | False |
| Minimum Age Num | 1 |
| Maximum Age Num | 80 |
| Minimum Age Unit | Year |
| Maximum Age Unit | Years |
Intervention Other Names
| Sequence: | 26603451 |
| Intervention Id | 52353100 |
| Name | MTP-131 |
Responsible Parties
| Sequence: | 28802073 |
| Responsible Party Type | Sponsor |