The Division of Genetics, Department of Pediatrics at the All India Institute of Medical Sciences (AIIMS), New Delhi, has announced the launch of a significant interventional clinical trial: ELEGANT.
Currently, the standard of care for Gaucher Disease Type 1 and Type 3 in India is Enzyme Replacement Therapy (ERT). While effective, ERT requires lifelong, biweekly intravenous injections, which pose significant logistical and psychological burdens on pediatric patients and their families. Also, enzymes administered do not cross the blood-brain barrier.
The ELEGANT Study (Evaluation of Eliglustat monotherapy in pediatric patients with Gaucher disease Type 1 and Type 3: A single-arm interventional trial) evaluates the safety and effectiveness of Eliglustat, an oral Substrate Reduction Therapy (SRT).
Trial Quick Facts
- Target Population: Pediatric patients (Ages 6–18 years).
- Disease Types: Gaucher Disease Type 1 and Type 3.
- Study Design: Single-Arm Interventional Trial
Trial Center & Faculty
The trial is being conducted at the Division of Genetics, Department of Pediatrics, AIIMS New Delhi, a recognized Center of Excellence (CoE) for Rare Diseases.
- Faculty In-charge: Dr. Neerja Gupta, Professor, Division of Genetics, Department of Pediatrics, AIIMS New Delhi.
Contact for Enrollment & Inquiries
Families, patient advocates, and referring physicians seeking more information about eligibility and enrollment are encouraged to contact the faculty in charge.
OPD Days: Tuesday and Friday