An Intermediate Size Expanded Access Protocol of Elamipretide


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


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


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.


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


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
Email Email
Role Contact Role Contact


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