About
Small molecule for rare diseases
Areas of Focus
Categories
Small Molecule Pharmaceutical
Clinical Trials (7)
View allTotal Trials
7
As Lead Sponsor
5
As Collaborator
2
Total Enrollment
2,215
By Phase
PHASE3: 2PHASE2: 4
By Status
ACTIVE_NOT_RECRUITING: 4COMPLETED: 2RECRUITING: 1
Clinical Trials Pipeline7 trials
Early Phase 1
Phase 1
Phase 2
Phase 3
Phase 4
Phase 1/2
Phase 2/3
N/A
2018
2019
2020
2021
2022
2023
2024
2025
2026
2027
2028
2029
Today
Hover over trial bars to see details
Recent Trials
NCT06673056Lead Sponsor
A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
PHASE3Active, not recruiting
60 participants
Started: Mar 18, 2025 · Completed: Jun 1, 2028
NCT06794281Collaborator
Synthetic Generation of Hematological Data Over Federated Computing Frameworks: SCD Use Case
N/AActive, not recruiting
1,500 participants
Started: Nov 1, 2022 · Completed: Nov 30, 2026
NCT05163288Lead Sponsor
A Pivotal Study of N-Acetyl-L-Leucine on Niemann-Pick Disease Type C
PHASE3Recruiting
53 participants
Started: Jun 30, 2022 · Completed: Dec 31, 2028
NCT04879563Collaborator
Artificial Intelligence Supporting CAncer Patients Across Europe - the ASCAPE Project
PHASE2Active, not recruiting
500 participants
Started: Feb 1, 2021 · Completed: Aug 30, 2025
NCT03759678Lead Sponsor
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
PHASE2Active, not recruiting
39 participants
Started: Jan 8, 2020 · Completed: Jul 1, 2025
Locations (1)
Headquarters
Oxford, UK
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