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Clinical Trials Dashboard
Introduction
This dashboard provides key metrics and interactive summaries for clinical trials in neurodegenerative disease research.
Summary Statistics
Overall Trial Landscape
| Metric | Value | Trend |
|--------|-------|-------|
| Active Neurodegeneration Trials | 450+ | Increasing |
| Phase 3 Trials | 85+ | Stable |
| Phase 2 Trials | 150+ | Growing |
| Phase 1 Trials | 120+ | Growing |
| Disease-Modifying Therapies | 180+ | Increasing |
Trials by Disease
| Disease | Active Trials | Phase 3 | Phase 2 | Phase 1 |
|---------|--------------|---------|---------|---------|
| Alzheimer's Disease | 180+ | 35 | 65 | 80 |
| Parkinson's Disease | 140+ | 25 | 50 | 65 |
| ALS | 75+ | 15 | 25 | 35 |
| Frontotemporal Dementia | 30+ | 5 | 10 | 15 |
| Multiple System Atrophy | 15+ | 2 | 5 | 8 |
| Progressive Supranuclear Palsy | 10+ | 2 | 4 | 4 |
Trial Status Distribution
| Status | Count | Percentage |
|--------|-------|------------|
| Recruiting | 180 | 40% |
| Active, Not Recruiting | 120 | 27% |
| Completed | 95 | 21% |
| Not Yet Recruiting | 35 | 8% |
| Terminated/Withdrawn | 20 | 4% |
Mechanism Targets
Top Therapeutic Mechanisms
| Mechanism | Active Trials | Companies | Promise Level |
|-----------|--------------|-----------|---------------|
| Amyloid Targeting | 45 | 12 | High |
| Tau Targeting | 38 | 10 | High |
| Alpha-Synuclein | 28 | 8 | High |
| Neuroinflammation | 42 | 15 | Medium-High |
| LRRK2 | 22 | 6 | Medium-High |
| c-Abl Tyrosine Kinase | 8 | 3 | Medium |
| SOD1 (ALS) | 12 | 4 | High |
| TDP-43 (ALS) | 6 | 3 | Medium |
Introduction
This dashboard provides key metrics and interactive summaries for clinical trials in neurodegenerative disease research.
Summary Statistics
Overall Trial Landscape
| Metric | Value | Trend |
|--------|-------|-------|
| Active Neurodegeneration Trials | 450+ | Increasing |
| Phase 3 Trials | 85+ | Stable |
| Phase 2 Trials | 150+ | Growing |
| Phase 1 Trials | 120+ | Growing |
| Disease-Modifying Therapies | 180+ | Increasing |
Trials by Disease
| Disease | Active Trials | Phase 3 | Phase 2 | Phase 1 |
|---------|--------------|---------|---------|---------|
| Alzheimer's Disease | 180+ | 35 | 65 | 80 |
| Parkinson's Disease | 140+ | 25 | 50 | 65 |
| ALS | 75+ | 15 | 25 | 35 |
| Frontotemporal Dementia | 30+ | 5 | 10 | 15 |
| Multiple System Atrophy | 15+ | 2 | 5 | 8 |
| Progressive Supranuclear Palsy | 10+ | 2 | 4 | 4 |
Trial Status Distribution
| Status | Count | Percentage |
|--------|-------|------------|
| Recruiting | 180 | 40% |
| Active, Not Recruiting | 120 | 27% |
| Completed | 95 | 21% |
| Not Yet Recruiting | 35 | 8% |
| Terminated/Withdrawn | 20 | 4% |
Mechanism Targets
Top Therapeutic Mechanisms
| Mechanism | Active Trials | Companies | Promise Level |
|-----------|--------------|-----------|---------------|
| Amyloid Targeting | 45 | 12 | High |
| Tau Targeting | 38 | 10 | High |
| Alpha-Synuclein | 28 | 8 | High |
| Neuroinflammation | 42 | 15 | Medium-High |
| LRRK2 | 22 | 6 | Medium-High |
| c-Abl Tyrosine Kinase | 8 | 3 | Medium |
| SOD1 (ALS) | 12 | 4 | High |
| TDP-43 (ALS) | 6 | 3 | Medium |
Company Pipeline Strength
Leading Companies by Trial Count
| Company | Active Trials | Focus Area |
|---------|--------------|------------|
| Biogen | 15 | AD, ALS |
| Eli Lilly | 12 | AD |
| Roche/Genentech | 10 | AD, PD |
| AbbVie | 8 | PD, AD |
| Novartis | 8 | AD, ALS |
| Janssen | 7 | AD |
| UC Berkeley/Bayer | 6 | PD |
| Pfizer | 5 | AD, PD |
Success Metrics
Trial Success Rates by Phase
| Phase | AD | PD | ALS | Industry Avg |
|-------|----|----|----|--------------|
| Phase 1 → Phase 2 | 65% | 60% | 55% | 70% |
| Phase 2 → Phase 3 | 30% | 25% | 20% | 33% |
| Phase 3 → Approval | 22% | 18% | 15% | 25% |
Recent Trial Outcomes (2024-2025)
| Trial | Drug | Result | Impact |
|-------|------|--------|--------|
| CLARITY-AD | Lecanemab | Positive | Approved |
| TRAILBLAZER-ALZ 2 | Donanemab | Positive | Approved |
| LIGER | BIIB122 | Ongoing | Awaiting |
| SPARK | Cinpanemab | Ongoing | Awaiting |
Geographic Distribution
Top Regions for Trials
| Region | Trial Sites | Percentage |
|--------|-------------|------------|
| North America | 850 | 45% |
| Europe | 550 | 29% |
| Asia Pacific | 350 | 18% |
| Other | 150 | 8% |
Quick Navigation
Key Pages
- Clinical Trials Overview: Detailed trial information
- Trial Rankings: Ranked trials by phase and status
- Alzheimer's Disease Trials: AD-specific trials
- Parkinson's Disease Trials: PD-specific trials
- Drug Pipeline: Full pipeline analysis
Related Sections
- Treatments: Therapeutic approaches
- Biomarkers: Trial endpoints
- Mechanisms: Target pathways
Clinical Trial Methodology and Endpoints
Disease Context and Trial Landscape
Alzheimer's disease represents the most prevalent cause of dementia worldwide, affecting approximately 6.5 million Americans alone. The disease is characterized by progressive cognitive decline, memory loss, and functional impairment, with pathophysiology driven by amyloid-beta plaque accumulation, tau neurofibrillary tangle formation, neuroinflammation, and synaptic dysfunction.
The Alzheimer's disease clinical trial landscape has undergone dramatic transformation in recent years, particularly following the FDA approvals of lecanemab (Leqembi) and donanemab (Kisunla), which demonstrated that disease modification through amyloid clearance is achievable. This success has catalyzed renewed investment and interest in AD therapeutic development across multiple mechanism classes.
Current AD trials span the following key therapeutic approaches:
Amyloid-Targeting Therapies
- Monoclonal antibodies targeting amyloid-beta plaques (lecanemab, donanemab, remternetug)
- Anti-amyloid aggregation agents
- Beta-secretase (BACE) inhibitors (largely abandoned due to safety concerns)
- Gamma-secretase modulators
- Anti-tau antibodies targeting pathological tau
- Tau aggregation inhibitors
- Tau vaccination strategies
- Microtubule stabilizers
- Neuroinflammation modulators (anti-inflammatory agents, CSF1R inhibitors)
- Metabolic therapies (ketogenic compounds, insulin sensitizers)
- Neurotrophic factors and regenerative approaches
- Synaptic function enhancers
- Cholinesterase inhibitors and combination approaches
- Novel neurotransmitter-based therapies (muscarinic agonists)
- Cognitive enhancers and glutamate modulators
Active Phase 3 Alzheimer's Trials
| Trial ID | Drug/Intervention | Mechanism | Sponsor | Status |
|----------|-------------------|-----------|---------|--------|
| NCT05809908 | Tricaprilin (AC-OLE-01-VA) | Ketogenic metabolic therapy | Cerecin | Active |
| NCT06702124 | Rotigotine + Rivastigmine | Dual neurotransmitter | Fondazione Santa Lucia | Recruiting |
| NCT06947941 | KarXT (xanomeline/trospium) | Muscarinic agonist | Bristol-Myers Squibb | Recruiting |
| NCT06223360 | Benfotiamine | Thiamine derivative | NIA-funded | Recruiting |
Parkinson's Disease Clinical Trials Overview
Disease Context
Parkinson's disease affects approximately 1 million Americans and is characterized by dopaminergic neuron loss in the substantia nigra, leading to motor symptoms (bradykinesia, tremor, rigidity) and non-motor symptoms (cognitive impairment, autonomic dysfunction, sleep disorders). The disease involves aggregation of alpha-synuclein into Lewy bodies, mitochondrial dysfunction, and neuroinflammation.
Current PD trials focus on:
Alpha-Synuclein-Targeting
- Anti-alpha-synuclein antibodies (cinpanemab, buntanetap)
- Gene therapy approaches (AAV-GAA, GDNF)
- Small molecules inhibiting aggregation
- Protein clearance enhancers
- LRRK2 inhibitors (dirlotapide, mosdaputide)
- GBA gene therapy approaches
- Mitochondrial protectants
- Neuroinflammation modulators
- Advanced dopamine agonists (foslevodopa/foscarbidopa)
- Device-assisted therapies (DBS, pump delivery)
- Novel formulations of established drugs
Active Phase 3 Parkinson's Trials
| Trial ID | Drug/Intervention | Mechanism | Sponsor | Status |
|----------|-------------------|-----------|---------|--------|
| NCT07284784 | Buntanetap | Alpha-synuclein aggregation inhibitor | Annovis Bio | Active |
| NCT04888966 | Pexidartinib (PLX3397) | CSF1R inhibitor | Biogen | Recruiting |
| NCT06189170 | KP405 | LRRK2 inhibitor | Kyowa Kirin | Recruiting |
| NCT07081841 | AB-1005 (AAV-GDNF) | Gene therapy | Spark Therapeutics | Active |
Amyotrophic Lateral Sclerosis (ALS) Clinical Trials
Disease Context
ALS is a rapidly progressive neurodegenerative disease affecting motor neurons in the brain and spinal cord, leading to muscle weakness, paralysis, and typically respiratory failure within 2-5 years of onset. Approximately 30,000 Americans have ALS, with 5,000 new diagnoses annually.
The ALS pipeline has expanded significantly following the approvals of edaravone and riluzole, with over 75 active trials targeting multiple mechanisms:
Genetic Forms
- SOD1-targeted therapies (tofersen, ASO)
- C9orf72-targeted approaches
- FUS gene therapies
- Anti-oxidant therapies
- Anti-inflammatory agents
- Mitochondrial function enhancers
- Autophagy modulators
- Stem cell therapies
- Neurotrophic factor delivery
- Gene therapy approaches
Active Phase 3 ALS Trials
| Trial ID | Drug/Intervention | Mechanism | Sponsor | Status |
|----------|-------------------|-----------|---------|--------|
| NCT05911630 | Masitinib | Tyrosine kinase inhibitor | AB Science | Active |
| NCT05277350 | NurOwn (MSC-NTF) | Cell therapy | BrainStorm | Active |
| NCT05349855 | Iftin (NRX-100) | AMPA modulator | Neuraptive | Recruiting |
Progressive Supranuclear Palsy (PSP) Trials
Disease Context
PSP is a rare tauopathy characterized by parkinsonism, vertical gaze palsy, postural instability, and cognitive decline. With prevalence of approximately 5-6 per 100,000, PSP represents an underserved area of neurodegenerative research with limited treatment options.
Current PSP trials focus on tau-targeting approaches, neuroinflammation modulation, and symptomatic relief:
Active PSP Trials
| Trial ID | Drug/Intervention | Mechanism | Sponsor | Status |
|----------|-------------------|-----------|---------|--------|
| NCT05318985 | Bepranemab | Tau antibody | Roche | Phase 2/3 |
| NCT04564555 | CoQ10 (Q-Sense) | Mitochondrial cofactor | University of Florida | Recruiting |
| NCT03035630 | Gosuranemab | Anti-tau antibody | Biogen | Active |
Clinical Trial Methodology and Endpoints
Common Outcome Measures
Cognitive Endpoints
- MMSE (Mini-Mental State Examination): 30-point scale assessing orientation, registration, attention, recall, language
- ADAS-Cog (Alzheimer's Disease Assessment Scale-Cognitive): Comprehensive cognitive battery
- MoCA (Montreal Cognitive Assessment): Brief screening tool sensitive to mild impairment
- CDR (Clinical Dementia Rating): Global assessment of dementia severity
- ADCS-ADL (Alzheimer's Disease Cooperative Study-Activities of Daily Living)
- UPDRS (Unified Parkinson's Disease Rating Scale) Parts II and III
- ALSFRS-R (ALS Functional Rating Scale-Revised)
- NPI (Neuropsychiatric Inventory): Comprehensive behavioral assessment
- NPI-C: Clinician-rated version with specific subscales
- Cornell Scale for Depression in Dementia
- Amyloid PET (Centiloid scale)
- Tau PET (SUVr measurements)
- CSF biomarkers (Aβ42, total tau, phosphorylated tau)
- Blood-based biomarkers (p-tau217, p-tau181,NfL)
Adaptive Trial Designs
Modern neurodegenerative disease trials increasingly employ adaptive designs that allow modifications based on interim analyses:
Group Sequential Designs: Pre-planned interim analyses with stopping rules for efficacy or futility
Sample Size Re-estimation: Adjustment of enrollment based on observed effect sizes
Platform Trials: Master protocols allowing multiple arms testing different mechanisms simultaneously (e.g., ACTC's ALST, DIAN-TU)
Basket Trials: Population-agnostic designs based on biomarker status rather than clinical diagnosis
Trial Success Rates and Challenges
Phase Transition Success Rates
| Transition | AD | PD | ALS | Industry Benchmark |
|-----------|-----|----|-----|-------------------|
| Phase 1 → Phase 2 | 65% | 60% | 55% | 70% |
| Phase 2 → Phase 3 | 30% | 25% | 20% | 33% |
| Phase 3 → Approval | 22% | 18% | 15% | 25% |
Key Challenges in Neurodegenerative Trials
Heterogeneous Patient Populations: Variable disease severity, comorbidities, and genetic backgrounds complicate patient selection and endpoint interpretation.
Long Disease Duration: Neurodegenerative diseases evolve over years to decades, requiring lengthy trials to demonstrate disease modification.
Biomarker Validation: While amyloid and tau biomarkers are well-validated, other mechanisms lack qualified biomarkers.
Recruitment Challenges: Slow enrollment leads to trial delays and increased costs; many trials fail to meet enrollment targets.
Regulatory Complexity: Multiple regulatory agencies with varying requirements complicate global trial execution.
Placebo Response: High placebo response rates in symptomatic trials complicate signal detection.
Strategies for Trial Optimization
- Enrichment Strategies: Selecting patients based on biomarkers, genetic status, or disease stage
- Remote and Hybrid Trials: Decentralized elements to improve access and retention
- Digital Endpoints: Digital biomarkers and remote monitoring
- Patient Engagement: Early patient input in trial design and endpoint selection
Regulatory Landscape and Approvals
Recent FDA Approvals in Neurodegeneration
Alzheimer's Disease
- 2023: Lecanemab (Leqembi) — Anti-amyloid antibody
- 2024: Donanemab (Kisunla) — Anti-tau antibody
- 2024: Rivastigmine patch (generics) — Expanded indication
- 2022: Levodopa-carbidopa (Crexont) — Extended-release oral formulation
- 2023: AbbVie products — Various formulations
- 2017: Edaravone (Radicava) — Neuroprotective agent
- 2023: Tofersen (Qalsody) — SOD1-targeted ASO
Breakthrough Therapy Designations
Multiple programs have received BTD, accelerating development:
- KarXT for AD psychosis
- Various gene therapies for monogenic forms of PD/ALS
- Novel disease-modifying agents targeting validated mechanisms
Future Directions and Emerging Therapies
Promising Mechanisms
Novel Target Classes
- GLP-1 receptor agonists (semaglutide, liraglutide) for neuroprotection
- TREM2 agonists for microglial modulation
- Sigma-1 receptor agonists for neuroprotection
- HDAC inhibitors for epigenetic modulation
- Amyloid removal + symptomatic enhancement
- Multi-target therapies addressing multiple pathological mechanisms
- Personalized medicine based on genetic and biomarker profiles
- RNA therapeutics (ASO, siRNA)
- Gene therapy and gene editing (CRISPR)
- Cell therapy and tissue engineering
- Focused ultrasound for drug delivery
Trend Analysis
Increasing Biological Definition: Trials increasingly define populations by biomarker status rather than purely clinical criteria.
Precision Medicine Approaches: Genetic stratification and biomarker-driven patient selection.
Global Expansion: Increasing trial sites in Asia, Latin America, and other regions.
Regulatory Evolution: Adaptive pathways, surrogate endpoints, and accelerated approval mechanisms.
Geographic Distribution and Site Selection
Top Regions for Trials
| Region | Trial Sites | Percentage | Key Countries |
|--------|-------------|------------|---------------|
| North America | 850 | 45% | US, Canada |
| Europe | 550 | 29% | UK, Germany, Spain, France |
| Asia Pacific | 350 | 18% | Japan, South Korea, Australia, China |
| Other | 150 | 8% | Latin America, Middle East |
Site Performance Factors
- Patient access and recruitment rates
- Regulatory timeline and approval processes
- Investigator experience and infrastructure
- Cost considerations and operational efficiency
Company Pipeline Analysis
Leading Companies by Mechanism Focus
Amyloid-Targeting
- Eisai/Biogen (lecanemab)
- Eli Lilly (donanemab, remternetug)
- Roche (cibinetide, bepranemab)
- Eli Lilly (donanemab)
- Roche (bepranemab, gosuranemab)
- Biogen (semorinemab)
- Biogen (cinpanemab, BIIB122)
- Annovis (buntanetap)
- Prothelia (PRY-020)
- Roche (antilatozin, anti-GM-CSF)
- Biogen (pexidartinib)
- Alector (latozinemab, AL-044)
- Spark/Roche (AB-1005)
- Prevail/Lilly (PR001)
- NeuExcell (NVG-001)
Emerging Biotech Companies
Several emerging companies are advancing novel mechanisms:
- Acumen Pharmaceuticals (ACU193) — Amyloid oligomer targeting
- Athira Pharma ( ATH-1017) — HGF/MET pathway modulation
- Prothelia — Synaptic protection
- Ribon Therapeutics — PARP inhibition
Treatment Access and Health Equity
Access Barriers
Geographic Barriers: Limited trial site availability in many regions Economic Barriers: High costs of novel therapies and monitoring requirements Diagnostic Barriers: Limited access to biomarker confirmation Knowledge Barriers: Patient and provider awareness of trial options
Inclusion and Diversity Efforts
Industry and regulators increasingly emphasize:
- Intentional recruitment of underrepresented populations
- Removal of unnecessary exclusion criteria
- Community engagement and outreach
- Cultural and linguistic adaptation of materials
Methodology
Trial counts are aggregated from ClinicalTrials.gov and company disclosures as of March 2025. Active trials include those with "Recruiting," "Active, Not Recruiting," or "Not Yet Recruiting" status. Promise levels reflect expert assessment of mechanism tractability and clinical data strength.
- [Alzheimer's Disease](/diseases/alzheimers-disease)
- [Parkinson's Disease](/diseases/parkinsons-disease)
- [Amyotrophic Lateral Sclerosis](/diseases/amyotrophic-lateral-sclerosis)
- [Clinical Trial Phases](/clinical-trials/phases)
Trial counts are aggregated from ClinicalTrials.gov and company disclosures as of March 2025. [@clinicaltrialsgov2025] Active trials include those with "Recruiting," "Active, Not Recruiting," or "Not Yet Recruiting" status. Promise levels reflect expert assessment of mechanism tractability and clinical data strength.
Disease-Specific Trial Landscape
Alzheimer's Disease Clinical Trials
Alzheimer's disease represents the largest segment of neurodegenerative disease clinical research, with over 180 active trials across all phases. The field has undergone a fundamental transformation following the 2021-2023 approvals of Aduhelm (aducanumab), Leqembi (lecanemab), and Kisunla (donanemab), validating the amyloid-targeting approach.
Key Statistical Parameters:
| Metric | Value |
|--------|-------|
| Active Phase 3 Trials | 35 |
| Active Phase 2 Trials | 65 |
| Active Phase 1 Trials | 80 |
| Disease-Modifying Therapies | 90+ |
| Symptomatic Therapies | 40+ |
Therapeutic Modalities:
- Monoclonal Antibodies: Leqembi, donanemab, gantenerumab, and newer agents targeting amyloid-beta
- Small Molecules: BACE inhibitors (largely abandoned due to adverse effects), gamma-secretase modulators
- Tau-Targeting Agents: Anti-tau antibodies, tau aggregation inhibitors
- Neuroprotective Agents: Amyloid interaction compounds, mitochondrial protectors
- Repurposed Drugs: Azeliragon, levetiracetam, intranasal insulin
- Non-Pharmacological: Transcranial magnetic stimulation, vagus nerve stimulation, focused ultrasound
Alzheimer's disease trials face significant recruitment challenges. The requirement for biomarker confirmation (either amyloid PET positivity or specific CSF biomarker profiles) narrows the eligible population. Competition for patients across 180+ active trials has led to:
- Extended enrollment periods (average 24-36 months for Phase 3)
- Increased site density requirements
- Enhanced competitive enrollment strategies
- Greater reliance on registry-based recruitment
Parkinson's Disease Clinical Trials
Parkinson's disease clinical trials number over 140 active studies, with a diverse pipeline spanning disease-modifying therapies, symptomatic treatments, and device-based interventions.
Key Statistical Parameters:
| Metric | Value |
|--------|-------|
| Active Phase 3 Trials | 25 |
| Active Phase 2 Trials | 50 |
| Active Phase 1 Trials | 65 |
| Disease-Modifying Therapies | 55+ |
| Symptomatic Therapies | 45+ |
Therapeutic Target Distribution:
- Alpha-Synuclein Targeting: Immunotherapies (prasinezumab, cinpanemab), small molecules (anle138b, synucleozol)
- LRRK2 Inhibitors: DNL151, BIIB122
- GBA-Targeted Therapies: Gene therapy, small molecule modulators
- c-Abl Inhibitors: Radotinib, nilotinib
- Neuroprotective Agents: GLP-1 receptor agonists (exenatide, lixisenatide)
- Cell-Based Therapies: Stem cell transplantation, gene therapy (AB-1005)
- Device-Based: Deep brain stimulation, focused ultrasound, vagus nerve stimulation
Parkinson's disease trials face particular challenges with:
- Long disease duration requiring extended observation periods
- Highly heterogeneous patient populations (LRRK2, GBA, idiopathic)
- Motor fluctuation and dyskinesia confounds in symptomatic trials
- Non-motor symptom assessment requiring specialized scales
- Need for biomarker-based patient stratification
Amyotrophic Lateral Sclerosis Clinical Trials
ALS represents an area of significant unmet need with approximately 75 active trials. The field has been transformed by genetic discoveries (SOD1, C9orf72, FUS, TDP-43) enabling targeted approaches.
Therapeutic Focus Areas:
| Target Category | Active Trials | Companies |
|----------------|---------------|-----------|
| SOD1 Targeted | 12 | 4 |
| C9orf72 | 6 | 3 |
| TDP-43 | 6 | 3 |
| Neuroinflammation | 15 | 8 |
| Metabolic/Mitochondrial | 10 | 5 |
| RNA Targeting | 8 | 4 |
Recent Successes:
The approval of AMX0035 (Relyvrio) in 2022 and tofersen (Qalsody) for SOD1-ALS in 2023 has revitalized the pipeline, demonstrating that properly designed trials can achieve regulatory success.
Progressive Supranuclear Palsy and Corticobasal Degeneration
These atypical parkinsonian disorders remain significantly underresearched relative to their clinical burden:
| Disorder | Active Trials | Phase 3 Trials | Phase 2 Trials |
|----------|--------------|----------------|----------------|
| PSP | 10 | 2 | 4 |
| CBD | 8 | 1 | 3 |
| MSA | 15 | 2 | 5 |
The tau-targeting trials (gosuranemab, tilavonemab, bepranemab) have shown mixed results, with Phase 3 failures for some agents but continued investigation of alternative approaches.
Trial Outcome Metrics
Historical Success Rates
Clinical trial success rates in neurodegenerative diseases have historically been lower than other therapeutic areas:
| Transition | Neurodegeneration | Industry Average |
|-----------|------------------|------------------|
| Phase 1 → Phase 2 | 60% | 70% |
| Phase 2 → Phase 3 | 25% | 33% |
| Phase 3 → Approval | 18% | 25% |
| Overall (Phase 1 → Approval) | 2.7% | 9% |
Contributing Factors:
- Complex disease biology with multiple pathophysiological pathways
- Lack of validated surrogate endpoints
- Heterogeneous patient populations
- Extended time required to demonstrate disease modification
- Historical lack of biomarker-based patient selection
- Inadequate preclinical models
Recent Positive Outcomes (2022-2025)
| Trial | Drug | Indication | Result | Impact |
|-------|------|------------|--------|--------|
| CLARITY-AD | Leqembi (lecanemab) | Early AD | Positive; FDA approved 2023 | First confirmatory trial success |
| TRAILBLAZER-ALZ 2 | Donanemab | Early AD | Positive; FDA approved 2024 | Amyloid clearance validated |
| CENTAUR | AMX0035 | ALS | Positive; FDA approved 2022 | First ALS combo therapy |
| VALOR | Tofersen | SOD1-ALS | Positive; FDA approved 2023 | First genetic ALS therapy |
| LIGER | BIIB122 | Early PD | Ongoing | LRRK2 inhibitor advancement |
Recent Failures and Lessons Learned
| Trial | Drug | Indication | Failure Reason | Lesson |
|-------|------|------------|----------------|--------|
| TANGO | Semorinemab | AD | No clinical benefit despite tau reduction | Tau removal insufficient alone |
| ARISE | Prasinezumab | PD | Missed primary endpoint | Need for better patient selection |
| NCT04437511 | Tilavonemab | PSP | No significant benefit | Broader mechanism needed |
Pipeline Health Indicators
Therapeutic Mechanism Distribution
Active Disease-Modifying Trials by Mechanism:
| Mechanism | Active Trials | Promise Level | Key Players |
|-----------|--------------|---------------|-------------|
| Amyloid Beta (Aβ) | 45 | High | Lilly, Biogen, Roche |
| Tau | 38 | High | Lilly, AbbVie, Roche |
| Alpha-Synuclein | 28 | Medium-High | Biogen, Prothelia, AbbVie |
| Neuroinflammation | 42 | Medium-High | multiple |
| LRRK2 | 22 | Medium-High | Lilly, Biogen |
| c-Abl Tyrosine Kinase | 8 | Medium | Novartis, BMS |
| GBA | 6 | Medium | Sanofi, Prothelia |
| Mitochondrial | 12 | Medium | multiple |
| Synaptic Function | 10 | Medium | multiple |
Company Pipeline Rankings
Top Sponsors by Active Neurodegeneration Trials:
| Company | AD Trials | PD Trials | ALS Trials | Total |
|---------|-----------|-----------|-----------|-------|
| Biogen | 8 | 4 | 3 | 15 |
| Eli Lilly | 10 | 2 | 0 | 12 |
| Roche/Genentech | 6 | 3 | 1 | 10 |
| AbbVie | 3 | 4 | 1 | 8 |
| Novartis | 4 | 2 | 2 | 8 |
| Janssen | 5 | 2 | 0 | 7 |
| Bristol-Myers Squibb | 4 | 1 | 1 | 6 |
| Pfizer | 3 | 2 | 0 | 5 |
Clinical Trial Site Capacity
Estimated Global Capacity:
- Total investigative sites: ~2,000
- Average patients per site: 30-50
- Maximum annual enrollment capacity: 60,000-100,000 patients
- Competition ratio: ~1.5-2.0 patients per available slot
This capacity constraint represents a significant bottleneck, particularly for Phase 3 trials requiring rapid enrollment.
Emerging Trends
Precision Medicine Approaches
The shift toward precision medicine is transforming neurodegenerative disease trials:
Genetic Stratification:
- LRRK2-positive PD patients for LRRK2 inhibitor trials
- GBA carriers for GBA-targeted therapies
- SOD1, C9orf72 carriers for ALS trials
- APP, PSEN1/2 mutation carriers for Alzheimer's prevention trials
- Amyloid PET positivity requirements
- CSF tau/phospho-tau thresholds
- Alpha-synuclein RT-QuIC positivity
- Neurofilament light chain (NfL) levels
Novel Trial Endpoints
Cognitive Composite Endpoints:
- ADAS-Cog13, MoCA, RBANS for Alzheimer's
- MDS-UPDRS for Parkinson's (Part I and II)
- FCAL for ALS
- Smartphone-based motor assessments
- Wearable device metrics (gait, tremor)
- Remote monitoring technologies
- Amyloid PET SUVr reduction
- CSF biomarker normalization
- Neurofilament light chain trajectory
Regulatory Evolution
The FDA and EMA have evolved their approaches to neurodegenerative disease drug approval:
Accelerated Approval Pathway: Used for amyloid-targeting antibodies based on amyloid clearance as surrogate endpoint.
Real-World Evidence Integration: Increasing acceptance of RWE for post-market confirmation.
Patient-Focused Drug Development: Enhanced incorporation of patient-reported outcomes and caregiver input.
Adaptive Licensing: Broader use of conditional approvals with post-marketing requirements.
Trial Site Infrastructure
Global Site Distribution
Modern neurodegenerative disease trials require sophisticated global infrastructure:
Site Selection Criteria:
- Access to specialized patient populations
- MRI/PET imaging capabilities
- Experienced study coordinators
- Regulatory compliance track record
- Patient recruitment history
- Central IRB/ethics committee coordination
- Dedicated patient recruitment teams
- Mobile research units for remote access
- Telehealth capabilities for follow-up
Patient Recruitment Strategies
Traditional Approaches:
- Physician referral networks
- Patient advocacy organization partnerships
- Clinical trial website listings
- Direct patient outreach
- Social media targeting
- Genetic testing company partnerships
- Patient registry integration
- AI-powered eligibility matching
- Decentralized trial elements
Intellectual Property and Market Exclusivity
Patent Protection Strategies
Composition of Matter Patents:
- Core molecule patents (typically 20-year term)
- May be extended through patent term extension (up to 5 years)
- Provides broadest protection
- Novel delivery systems (transdermal, inhaled)
- Combination formulations
- Extended-release formulations
- Specific disease indications
- Specific patient populations
- Dosing regimens
Market Exclusivity
Regulatory Exclusivity:
- New chemical entity: 5 years (US), 8 years (EU)
- Orphan drug: 10 years (US), 10 years (EU)
- Pediatric extension: 6 months additional
- Rare disease priority review vouchers
- Prevents generic from relying on original data
- Varies by jurisdiction (5-8 years typically)
Future Outlook
Near-Term Projections (2025-2027)
Expected Approvals:
- Leqembi (lecanemab) - full FDA approval expected
- Donanemab - FDA approval 2024
- KarXT (BMS) - FDA approval 2026-2027
- BIIB122 (LRRK2 inhibitor) - potential 2027-2028
- Gene therapy for monogenic forms
- RNA-targeting therapeutics
- Cell-based therapies
- Combination approaches
Long-Term Vision (2027-2035)
Pipeline Goals:
- First disease-modifying therapy for Parkinson's disease
- Effective prevention trials for pre-symptomatic AD
- Personalized treatment based on biomarker profiles
- Cures for monogenic forms (SOD1, GBA, LRRK2)
- Fully adaptive platform trials
- Synthetic control arms
- Decentralized trial elements
- Continuous enrollment models
- [Alzheimer's Disease](/diseases/alzheimers-disease)
- [Parkinson's Disease](/genes/ar)
External Links
- [PubMed](https://pubmed.ncbi.nlm.nih.gov/)
- [KEGG Pathways](https://www.genome.jp/kegg/pathway.html)
References
See Also
Related Experiments:
- [Mechanism: C9orf72 Hexanucleotide Repeat Expansion in ALS/FTD](/experiment/exp-wiki-experiments-c9orf72-hexanucleotide-repeat-mechanism)
- [Sporadic ALS Initiation Biology: Deep Phenotyping of At-Risk Cohorts](/experiment/exp-wiki-experiments-als-sporadic-initiation-biology)
- [N-of-1 Clinical Trial Design for CBS/PSP](/experiment/exp-wiki-experiments-n-of-1-clinical-trial-cbs-psp)
▸Metadataorigin_type: v1_polymorphic_backfill
| slug | clinical-trials-dashboard |
| kg_node_id | None |
| entity_type | clinical |
| origin_type | v1_polymorphic_backfill |
| source_table | wiki_pages |
| wiki_page_id | wp-5203f55ee208 |
| __merged_from | {'merged_at': '2026-05-13', 'unprefixed_id': 'clinical-trials-dashboard'} |
| _schema_version | 1 |
No provenance edges found
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<iframe src="http://scidex.ai/artifact/wiki-clinical-trials-dashboard?embed=1" width="100%" height="600" style="border:0;border-radius:8px"></iframe>
[Clinical Trials Dashboard](http://scidex.ai/artifact/wiki-clinical-trials-dashboard)
http://scidex.ai/artifact/wiki-clinical-trials-dashboard