ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome (NCT03747328) | Clinical Trial Compass
WithdrawnPhase 2
ABI-009 (Nab-sirolimus) in Patients With Genetically-confirmed Leigh or Leigh-like Syndrome
Stopped: IND withdrawn
0Started 2022-04-30
Plain-language summary
A phase 2a, open-label study to evaluate the safety, tolerability, and clinical activity of ABI-009 (nab-sirolimus) in patients with genetically-confirmed Leigh or Leigh-like syndrome
Who can participate
Age range
2 Years – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Diagnosis of genetically-confirmed Leigh or Leigh-like syndrome, as well as documented clinical evidence (including demonstrated neurologic manifestations) of the syndrome, as confirmed by the investigator.
. Moderate disease severity based on NPMDS score of \>15 on Sections I through III, inclusive.
. Male or female patients, ≥2 and ≤17 years of age at the time of enrollment.
. Body weight ≥5 kg (11 lbs) at the time of enrollment.
. Chronic, stable disease, as determined by the investigator, for a minimum of 3 months prior to enrollment. This includes, but is not limited to, patients without hospitalizations or emergency room visits, or fever \<101 F, and/or acute illness.
. Adequate liver function:
. Total bilirubin ≤1.5 x upper limit of normal (ULN) mg/dL
. Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) \<3 x ULN.
Exclusion criteria
. Renal insufficiency that, in the opinion of the investigator, requires or may require dialysis during the treatment and follow-up periods.
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Incidence of treatment emergent adverse events in pediatric patients with genetically-confirmed Leigh or Leigh-like syndrome administered ABI-009 intravenously (IV)
. Cardiac ejection fraction/shortening fraction ≤50 % and/or severe end-organ hypo-perfusion syndrome (secondary to cardiac failure) resulting in lactic acidosis.
. Patients with implanted cardiac assist/medical devices (including pacemakers), unless device was implanted prophylactically, and the patient is clinically asymptomatic.
. In the opinion of the investigator, clinically significant ECG and/or ECHO findings at the time of screening.
. Myocardial infarction within 6 months prior to enrollment.