This dashboard provides key metrics and interactive summaries for clinical trials in neurodegenerative disease research.
| 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 |
| 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 |
| Status | Count | Percentage |
|---|---|---|
| Recruiting | 180 | 40% |
| Active, Not Recruiting | 120 | 27% |
| Completed | 95 | 21% |
| Not Yet Recruiting | 35 | 8% |
| Terminated/Withdrawn | 20 | 4% |
| 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 | 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 |
| 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% |
| Trial | Drug | Result | Impact |
|---|---|---|---|
| CLARITY-AD | Lecanemab | Positive | Approved |
| TRAILBLAZER-ALZ 2 | Donanemab | Positive | Approved |
| LIGER | BIIB122 | Ongoing | Awaiting |
| SPARK | Cinpanemab | Ongoing | Awaiting |
| Region | Trial Sites | Percentage |
|---|---|---|
| North America | 850 | 45% |
| Europe | 550 | 29% |
| Asia Pacific | 350 | 18% |
| Other | 150 | 8% |
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
Tau-Targeted Approaches
Neuroprotective and Disease-Modifying Approaches
Symptomatic Treatments
| 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 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
Disease-Modifying Approaches
Motor Symptom Treatments
| 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 |
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
Neuroprotective Approaches
Cellular and Regenerative
| 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 |
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:
| 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 |
Cognitive Endpoints
Functional Endpoints
Behavioral Endpoints
Biomarker Endpoints
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
| 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% |
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.
Alzheimer's Disease
Parkinson's Disease
ALS
Multiple programs have received BTD, accelerating development:
Novel Target Classes
Combination Approaches
Emerging Modalities
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.
| 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 |
Amyloid-Targeting
Tau-Targeting
Alpha-Synuclein
Neuroinflammation
Gene Therapy
Several emerging companies are advancing novel mechanisms:
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
Industry and regulators increasingly emphasize:
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.
Trial counts are aggregated from ClinicalTrials.gov and company disclosures as of March 2025. [1] 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 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:
Enrollment Challenges:
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:
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:
Unique Considerations:
Parkinson's disease trials face particular challenges with:
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.
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.
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:
| 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 |
| 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 |
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 |
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 |
Estimated Global Capacity:
This capacity constraint represents a significant bottleneck, particularly for Phase 3 trials requiring rapid enrollment.
The shift toward precision medicine is transforming neurodegenerative disease trials:
Genetic Stratification:
Biomarker-Based Enrichment:
Cognitive Composite Endpoints:
Digital Endpoints:
Biomarker Surrogates:
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.
Modern neurodegenerative disease trials require sophisticated global infrastructure:
Site Selection Criteria:
Site Support Infrastructure:
Traditional Approaches:
Modern Approaches:
Composition of Matter Patents:
Formulation Patents:
Method of Use Patents:
Regulatory Exclusivity:
Data Exclusivity:
Expected Approvals:
Emerging Modalities:
Pipeline Goals:
Trial Design Evolution:
Fully adaptive platform trials
Synthetic control arms
Decentralized trial elements
Continuous enrollment models