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Stopped: IND withdrawn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 range2 Years – 17 Years

SexALL

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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.
✕. 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.
✕. Symptomatic congestive heart failure (CHF), unstable angina pectoris, cardiac arrhythmia, uncontrolled hypertension, or unstable coronary artery disease.
✕. Prior exposure to ABI-009, sirolimus, everolimus, or any other known rapamycin derivative, or previous treatment with any known mTOR inhibitor.
✕. Patients who are breast feeding or have a confirmed or suspected pregnancy.

What they're measuring

Incidence of treatment emergent adverse events in pediatric patients with genetically-confirmed Leigh or Leigh-like syndrome administered ABI-009 intravenously (IV)

Timeframe: Up to 24 weeks

Trial details

NCT IDNCT03747328

SponsorAadi Bioscience, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-12-31

View on ClinicalTrials.gov More Leigh Syndrome trials