NCT04825119 - Clinical Trial References

Phase 1: Researchers test a new drug or treatment in a small group of people for the first time to evaluate its safety, determine a safe dosage range, and identify side effects.
Phase 2: The drug or treatment is given to a larger group of people to see if it is effective and to further evaluate its safety.
Phase 3: The drug or treatment is given to large groups of people to confirm its effectiveness, monitor side effects, compare it to commonly used treatments, and collect information that will allow the drug or treatment to be used safely.
Phase 4: Studies are done after the drug or treatment has been marketed to gather information on the drug's effect in various populations and any side effects associated with long-term use.

Citation(s)

Brooks BR, Miller RG, Swash M, Munsat TL; World Federation of Neurology Research Group on Motor Neuron Diseases
El Escorial revisited: revised criteria for the diagnosis of amyotrophic lateral sclerosis. Amyotroph Lateral Scler Other Motor Neuron Disord. 2000 Dec;1(5):293-9. Review.
Dawood AA, Moosa A
Hand and ECG tremor in spinal muscular atrophy. Arch Dis Child. 1983 May;58(5):376-8.
Pupillo E, Bianchi E, Messina P, Chiveri L, Lunetta C, Corbo M, Filosto M, Lorusso L, Marin B, Mandrioli J, Riva N, Sasanelli F, Tremolizzo L, Beghi E; Eurals Consortium
Extrapyramidal and cognitive signs in amyotrophic lateral sclerosis: A population based cross-sectional study. Amyotroph Lateral Scler Frontotemporal Degener. 2015;16(5-6):324-30. doi: 10.3109/21678421.2015.1040028. Epub 2015 May 12.
Wurster CD, Ludolph AC
Nusinersen for spinal muscular atrophy. Ther Adv Neurol Disord. 2018 Mar 13;11:1756285618754459. doi: 10.1177/1756285618754459. eCollection 2018.

Hyperkinetic Movements in Patients With Motor Neuron Disease and in Patients With Spinal Muscular Atrophy and Their Response to Treatment With Nusinersen

Details for clinical trial NCT04825119

Clinical trial NCT04825119 - References