Amyotrophic Lateral Sclerosis Clinical Trial
Official title:
A Second Intermediate Expanded Access Protocol for Amyotrophic Lateral Sclerosis With CNM-Au8
The primary objective of the intermediate expanded access protocol is to provide access to the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS). No formal clinical hypotheses are being evaluated with concurrent controls. Secondary objectives include assessment of the safety of CNM-Au8 treatment in pALS. Safety will be assessed through the frequency of serious adverse events (SAEs), treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due to TEAEs, and laboratory abnormalities assessed as clinically significant during routine clinical monitoring (as applicable).
This is a multi-center intermediate expanded access program to provide access to the investigational product, CNM-Au8, up to 300 participants diagnosed with ALS. The safety of CNM-Au8 treatment in ALS participants will be evaluated. Visits will occur at a clinic or remotely via telephone or video-visit. Visits may be conducted remotely due to COVID-19-related pandemic concerns, or if due to ALS disease progression. Participants who meet the inclusion criteria and none of the exclusionary criteria may be enrolled into the EAP. There will be three study periods: 1. A treatment period of forty-eight (48) weeks (Treatment Period 1); 2. Additional optional follow-on treatment period(s) of up to forty-eight (48) weeks duration may be added at the discretion of the Sponsor and Site Investigator (e.g., Treatment Period 2, Treatment Period 3); 3. A four (4) week safety follow-up period (End-of-Study [EOS]Assessment). All participants will receive open-label oral treatment daily up to 48 weeks during Treatment Period 1. Additional 48-week treatment periods may be approved at the discretion of the Sponsor. The EAP may be discontinued at any time at the Sponsor's discretion. At treatment discontinuation or following the end of the participant's final Treatment Period, participants will complete an end-of-study (EOS) assessment 4 weeks following discontinuation of the investigational drug product. Visit assessments may be collected remotely, via tele-visit with study site staff. Investigational product may be shipped by the site to participants who do attend in-clinic visits. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Terminated |
NCT04428775 -
A Safety and Biomarker Study of ALZT-OP1a in Subjects With Mild-Moderate ALS Disease
|
Phase 2 | |
Recruiting |
NCT04998305 -
TJ-68 Clinical Trial in Patients With Amyotrophic Lateral Sclerosis (ALS) and Muscle Cramps
|
Phase 1/Phase 2 | |
Recruiting |
NCT05951556 -
Telehealth Implementation of Brain-Computer Interface
|
N/A | |
Terminated |
NCT04579666 -
MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)
|
Phase 2 | |
Recruiting |
NCT04082832 -
CuATSM Compared With Placebo for Treatment of ALS/MND
|
Phase 2/Phase 3 | |
Completed |
NCT01925196 -
Natural History and Biomarkers of Amyotrophic Lateral Sclerosis and Frontotemporal Dementia Caused by the C9ORF72 Gene Mutation
|
||
Completed |
NCT02496767 -
Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year
|
Phase 3 | |
Recruiting |
NCT04816227 -
Expression Profile Study of Macrophages From Patients Affected by ALS or Other Related Motor Impairments
|
||
Active, not recruiting |
NCT04494256 -
A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
|
Phase 1/Phase 2 | |
Completed |
NCT03706391 -
Study of ALS Reversals 4: LifeTime Exposures
|
||
Recruiting |
NCT04882904 -
Continuous Measurement of Activity in Patients With Muscle Pathology and in Control Subjects. ActiSLA Part.
|
N/A | |
Completed |
NCT04557410 -
Open Label Study: Treatment of ALS Fatigue With PolyMVA
|
Phase 1 | |
Active, not recruiting |
NCT04948645 -
A Phase 1 Study to Investigate the Safety and Pharmacokinetics of ABBV-CLS-7262 in Patients With Amyotrophic Lateral Sclerosis
|
Phase 1 | |
Not yet recruiting |
NCT04089696 -
Validation of the "ExSpiron©" in Patients With ALS
|
N/A | |
Not yet recruiting |
NCT06450691 -
Modeling Amyotrophic Lateral Sclerosis With Fibroblasts
|
N/A | |
Not yet recruiting |
NCT04220190 -
RAPA-501 Therapy for ALS
|
Phase 2/Phase 3 | |
Not yet recruiting |
NCT05860244 -
Effect of Salbutamol on Walking Capacity in Ambulatory ALS Patients
|
Phase 2 | |
Recruiting |
NCT02917681 -
Study of Two Intrathecal Doses of Autologous Mesenchymal Stem Cells for Amyotrophic Lateral Sclerosis
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT03067857 -
Autologous Bone Marrow-Derived Stem Cell Therapy for Motor Neuron Disease
|
Phase 1/Phase 2 | |
Recruiting |
NCT02874209 -
Noninvasive Assessment of Neuronal Damage by MRI Sodium ( 23Na ) in Amyotrophic Lateral Sclerosis
|
N/A |