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Asset Brief

ATH434

ATH·Alterity Therapeutics·Multiple system atrophy (MSA) — a rare, progressive neurodegenerative disease·Phase 2 (completed; Phase 3 agreed with FDA)·NCT05109091·Primary completion: 28 Nov 2024

Plain-language Brief

The trial in plain terms

The disease: Multiple system atrophy (MSA) is a rare, rapidly progressive neurodegenerative disease affecting movement, balance, and autonomic functions such as blood pressure and bladder control. It is caused by the abnormal accumulation of a protein called alpha-synuclein inside certain brain cells, leading to their death. There are no approved disease-modifying treatments; current care is supportive only. MSA holds FDA Orphan Drug and EMA Orphan designations for ATH434, reflecting its rare-disease status.
What the asset is trying to do: ATH434 is an oral small molecule described as an iron chaperone: it binds excess loosely-bound ('labile') iron in the brain and redistributes it away from vulnerable cells. Excess labile iron is thought to promote the clumping of alpha-synuclein and to generate oxidative stress, both of which damage neurons in MSA. This mechanism differs from supportive therapies, which do not target iron or alpha-synuclein pathology directly.
What the trial is measuring, and why it matters: The lead Phase 2 trial (NCT05109091) enrolled participants with MSA and included two active dose levels of ATH434 alongside a placebo arm (per ClinicalTrials.gov NCT05109091). A separate 10-participant biomarker study (NCT05864365) with a primary completion date of 2026-02-26 assessed biological markers of drug activity (per ClinicalTrials.gov NCT05864365). Both trials are listed as completed. A post-trial access study (NCT07531433) is also registered, providing continued access to ATH434 for prior participants (per ClinicalTrials.gov NCT07531433).
Efficacy benchmarks in this setting: No therapy has been approved to slow or halt MSA progression; no established benchmark efficacy figures from approved disease-modifying agents exist for this indication. In the broader neurodegenerative disease field, trials are typically measured against placebo on validated clinical rating scales, but no directly comparable approved-therapy reference figures are available for MSA. Any early-phase figures reported for ATH434 come from small, uncontrolled or early-stage cohorts and are not statistically comparable to data from larger, later-stage trials.
Commercial context: No disease-modifying therapy is currently approved for MSA anywhere in the world, leaving an unmet medical need with no direct commercial competitor in that class. ATH434 holds FDA Fast Track, FDA Orphan Drug, and EMA Orphan designations (per Alterity Therapeutics disclosures). Alterity announced on 8 June 2026 that it and the FDA have agreed on a Phase 3 program for ATH434 in MSA (per ASX announcement 2026-06-08). ATH434 is described as the company's lead asset. No partnership or licensing deal terms have been disclosed in the available announcements.

Sources for this brief (6)

Study of ATH434 in Participants with Multiple System Atrophy · ClinicalTrials.gov · accessed 26 June 2026
A Biomarker Study of ATH434 in Participants With MSA · ClinicalTrials.gov · accessed 26 June 2026
Post-Trial Access to ATH434 for Patients With Multiple System Atrophy · ClinicalTrials.gov · accessed 26 June 2026
Alterity and FDA Agree on Phase 3 Program for ATH434 in MSA · Alterity Therapeutics / ASX · accessed 26 June 2026
Alterity Data Supports ATH434 Phase 3 Advancement in MSA · Alterity Therapeutics / ASX · accessed 26 June 2026
Multiple System Atrophy — disease background and clinical features · National Institute of Neurological Disorders and Stroke (NINDS) · accessed 26 June 2026

Trial Design

Design summary

Phase: Phase 2 (completed; Phase 3 agreed with FDA)
Design type: Randomised, placebo-controlled, multi-arm. Two active dose levels versus placebo. Open-label post-trial access study also registered.
Primary endpoint: [ TODO: fill in ]
Key secondary endpoints: [ TODO: fill in ]
Enrolment target: 77 patients (per ClinicalTrials.gov NCT05109091)
Current enrolment status: Completed (per ClinicalTrials.gov NCT05109091, primary completion 2024-11-28)
Sites: [ TODO: fill in ]
Expected readout window: Both Phase 2 trials are listed as completed (primary completions 2024-11-28 and 2025-02-26 per ClinicalTrials.gov). Alterity announced data supporting Phase 3 advancement on 19 May 2026 and FDA agreement on a Phase 3 program on 8 June 2026 (per ASX announcements). Phase 3 trial registration and timelines have not been disclosed in the available data.

Asset Track Record

Did ATH deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

9 July 2026otherATH Receives A$3.98m R&D Tax Incentivehitsrc ASX ↗
19 May 2026data readoutAlterity Data Supports ATH434 Phase 3 Advancement in MSAhit · positivesrc ASX ↗
26 Apr 2026regulatory decisionATH434 FDA — Alterity Receives Positive FDA Feedback on CMC Meetinghit · positivesrc ASX ↗
21 Apr 2026data readoutAlterity Presents New Analysis of ATH434 Phase 2 Data at AANhit · positivesrc ASX ↗
29 Mar 2026regulatory decisionATH434 FDA — Alterity Receives Positive FDA Feedback From Type C Meetinghit · positivesrc

Class Catalyst Calendar

What's landed, what's next for ATH434 and peers

ATH434's own upcoming catalysts alongside class-defining historical readouts and upcoming primary completion dates in the same mechanism classes. Each event moves the broader thesis for ATH434. Showing past 6 months and next 12 months · 4 later hidden.

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
PepGen Inc

Trial design comparison

How ATH434 reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
ATH434	Alterity Therapeutics	Small Molecule Other	Phase 2 (completed; Phase 3 agreed with FDA)[ TODO: fill in ]	Randomised, placebo-controlled, multi-arm. Two active dose levels versus placebo. Open-label post-trial access study also registered.	[ TODO: fill in ]	[ TODO: fill in ]	77 patients (per ClinicalTrials.gov NCT05109091)	77
Trial of Parkinson's And Zoledronic Acid

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 1 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Antisense oligonucleotideASO	Off-the-shelf	Synthetic	Approved (SMA/DMD)	Chronic dosing	Moderate

OtherASX peers ›

Curated Commentary

Third-party coverage(none curated yet)

expand ›

External analyst notes, journalism, and industry commentary cited with attribution.

No third-party coverage curated for ATH434 yet. Broker coverage of Alterity Therapeutics is thin; named analyst notes will appear here as they become available. For now, see the company's ASX announcements and the linked sponsor / class sources elsewhere on this page.

Asset Track Record

Did ATH deliver against its own guidance?

Past catalysts on this ticker, reconciled against the announcement parse pipeline. Hover any row for the parsed outcome detail.

9 July 2026otherATH Receives A$3.98m R&D Tax Incentivehitsrc ASX ↗
19 May 2026data readoutAlterity Data Supports ATH434 Phase 3 Advancement in MSAhit · positivesrc ASX ↗
26 Apr 2026regulatory decisionATH434 FDA — Alterity Receives Positive FDA Feedback on CMC Meetinghit · positivesrc ASX ↗
21 Apr 2026data readoutAlterity Presents New Analysis of ATH434 Phase 2 Data at AANhit · positivesrc ASX ↗
29 Mar 2026regulatory decisionATH434 FDA — Alterity Receives Positive FDA Feedback From Type C Meetinghit · positivesrc

Class Catalyst Calendar

What's landed, what's next for ATH434 and peers

ConferenceAACR Annual Meeting 2026· ChicagoApr 2026
ConferenceASCO Annual Meeting 2026· ChicagoMay 2026
Today
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
PepGen Inc

Trial design comparison

How ATH434 reads against active peer trials

Same axes, same units. Discontinued and historical pivotal trials excluded; see Competitor Landscape below for the full set.

Trial	Sponsor	Class	Phase / Line	Design	Primary endpoint	Dosing	Key eligibility	n
ATH434	Alterity Therapeutics	Small Molecule Other	Phase 2 (completed; Phase 3 agreed with FDA)[ TODO: fill in ]	Randomised, placebo-controlled, multi-arm. Two active dose levels versus placebo. Open-label post-trial access study also registered.	[ TODO: fill in ]	[ TODO: fill in ]	77 patients (per ClinicalTrials.gov NCT05109091)	77
Trial of Parkinson's And Zoledronic Acid

Competitor Landscape

Competing programmes by mechanism and phase

Read-across commentary attached per trial. Phase ordering preserved within each group.

Mechanism trade-offs· 1 classes comparedexpand ›

Mechanism	Speed to patient	Manufacturing	Best approved result	Durability	Moat
Antisense oligonucleotideASO	Off-the-shelf	Synthetic	Approved (SMA/DMD)	Chronic dosing	Moderate

OtherASX peers ›

ATH434n = 77

Alterity Therapeutics·Small Molecule Other·Phase 2 (completed; Phase 3 agreed with FDA)·[ TODO: fill in ]

Design: Randomised, placebo-controlled, multi-arm. Two active dose levels versus placebo. Open-label post-trial access study also registered.
Primary endpoint: [ TODO: fill in ]
Dosing: [ TODO: fill in ]
Eligibility: 77 patients (per ClinicalTrials.gov NCT05109091)

Trial of Parkinson's And Zoledronic Acidn = —

California Pacific Medical Center Research Institute·—·Phase 4

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophyn = —

Theravance Biopharma·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2n = —

Lupin Ltd.·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Randomized, Double-blind, Placebo-controlled, Prospective, Multi-center Trial Evaluating the Improvement of Nutritional Status and Sarcopenia With Silkworm Pupa Tablets in Patients With Malignanciesn = —

First Affiliated Hospital of Zhejiang University·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)n = —

H. Lundbeck A/S·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

MSA-01 in Multiple System Atrophyn = —

Tokyo University·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)n = —

Biogen·—·Phase 3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)n = —

ProQR Therapeutics·—·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathyn = —

Wuhan Neurophth Biotechnology Limited Company·—·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

[18F]-MFBG Versus [123I]-MIBG and [18F]-PE2I in PD vs. MSA and DLB vs. ADn = —

prof. dr. Koen Van Laere·—·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophyn = —

Hoffmann-La Roche·—·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophyn = —

Second Affiliated Hospital, School of Medicine, Zhejiang University·—·PHASE2/PHASE3

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study of Lu AF82422 in Participants With Multiple System Atrophyn = —

H. Lundbeck A/S·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

AMX0035 in Adult Patients With Wolfram Syndromen = —

Amylyx Pharmaceuticals Inc.·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndromen = —

University of Birmingham·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophyn = —

Ono Pharmaceutical Co., Ltd.·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)n = —

Benedetto Falsini·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Phase 2 Study of YA-101 in Patients With Multiple System Atrophyn = —

Dasher Neuroscience Inc.·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1n = —

PepGen Inc·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety, Tolerability, and Pharmacokinetics of Exidavnemab in Patients With Parkinson's Disease and Patients With Multiple System Atrophyn = —

BioArctic AB·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophyn = —

Mayo Clinic·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study of the Effect of (S)-Pindolol Benzoate (ACM-001.1) on Lean Body Mass (LBM) in Obese Patents During (PROACT 1), and Post-semaglutide Therapy (PROACT 2)n = —

Actimed Therapeutics Ltd·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Phase 2a Study of Foralumab Nasal in Patients With Multiple System Atrophy (MSA)n = —

Tiziana Life Sciences LTD·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study of PLH-2301 in Subjects With Sarcopenian = —

Pluto Inc.·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Trial to Test if TEV-56286 is Effective for Treatment of Participants With Multiple System Atrophyn = —

Teva Branded Pharmaceutical Products R&D LLC·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

The NADAPT Study: a Randomized Double-blind Trial of NAD Replenishment Therapy for Atypical Parkinsonismn = —

Haukeland University Hospital·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

An Extension Trial to Test if TEV-56286 is Effective in Relieving Multiple System Atrophyn = —

Teva Branded Pharmaceutical Products R&D LLC·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Autologous Stem Cell Therapy in Patients With Multiple System Atrophyn = —

Biocells Medical·—·Phase 2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety & Efficacy of Eyecyte-RPE™ in Patients With Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.n = —

Eyestem Research Pvt. Ltd.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patientsn = —

GeneCradle Inc·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Automated Abdominal Binder for Orthostatic Hypotensionn = —

Vanderbilt University Medical Center·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophyn = —

BioMarin Pharmaceutical·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Feasibility Study Assessing the Effect of Carbidopa/Levodopa Ratio on Orthostatic Hypotension in Multiple System Atrophy - Parkinsonian Type and Parkinson Disease.n = —

Julien Bally·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patientsn = —

GeneCradle Inc·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patientsn = —

GeneCradle Inc·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)n = —

Vertex Pharmaceuticals Incorporated·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Mesenchymal Stem Cell Therapy in Multiple System Atrophyn = —

Mayo Clinic·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

NS-050/NCNP-03 in Boys With DMD (Meteor50)n = —

NS Pharma, Inc.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Study of ATX-01 in Participants With DM1n = —

ARTHEx Biotech S.L.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugsn = —

AstraZeneca·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45n = —

Entrada Therapeutics, Inc.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44n = —

Entrada Therapeutics, Inc.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1n = —

Dyne Therapeutics·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skippingn = —

Dyne Therapeutics·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)n = —

Precision BioSciences, Inc.·—·PHASE1/PHASE2

Design: —
Primary endpoint: —
Dosing: —
Eligibility: —

CP
Trial of Parkinson's And Zoledronic Acid
Phase 4
California Pacific Medical Center Research Institute·Active, not recruiting·Primary completion 31 Oct 2025·NCT03924414
77 sites·5 top centres: Mayo Clinic, UCSF, +3
2721 enrolled·PI: Steve Cummings
TB
Phase 3 Efficacy and Durability of Ampreloxetine for the Treatment of Symptomatic nOH in Participants With Multiple System Atrophy
Phase 3
Theravance Biopharma·Active, not recruiting·Primary completion 1 Jan 2026·NCT05696717
79 sites·3 top centres: Stanford, Columbia / NYP, +1
L.
The Efficacy and Safety of Once Daily Mexiletine PR in Patients With Myotonic Dystrophy Type 1 and Type 2
Phase 3
Lupin Ltd.·Recruiting·Primary completion 17 Mar 2026·NCT06523400
7 sites
FA
A Randomized, Double-blind, Placebo-controlled, Prospective, Multi-center Trial Evaluating the Improvement of Nutritional Status and Sarcopenia With Silkworm Pupa Tablets in Patients With Malignancies
Phase 3
First Affiliated Hospital of Zhejiang University·Not yet recruiting·Primary completion 31 Dec 2027·NCT07554807
HL
A Trial of Amlenetug (Lu AF82422) in Participants With Multiple System Atrophy (MSA)
Phase 3
H. Lundbeck A/S·Active, not recruiting·Primary completion 17 Feb 2028·NCT06706622
74 sites·6 top centres: Mayo Clinic, UCSF, +4
401 enrolled
TU
MSA-01 in Multiple System Atrophy
Phase 3
Tokyo University·Recruiting·Primary completion 31 Mar 2028·NCT07446894
12 sites
B
A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety When Given Before Symptoms Appear in Babies With Genetically Diagnosed Spinal Muscular Atrophy (SMA)
Phase 3
Biogen·Recruiting·Primary completion 28 Nov 2028·NCT07221669
4 sites
PIs: Nancy Kuntz, Diana Castro, Crystal Proudshared KOL · Proud
P
A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)
PHASE2/PHASE3
ProQR Therapeutics·Active, not recruiting·Primary completion 31 Jan 2022·NCT03913143
14 sites
36 enrolledstalled · 1607d
WN
Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy
PHASE2/PHASE3
Wuhan Neurophth Biotechnology Limited Company·Recruiting·Primary completion 29 Feb 2024·NCT04912843
1 site
PI: Bin Li
PD
[18F]-MFBG Versus [123I]-MIBG and [18F]-PE2I in PD vs. MSA and DLB vs. AD
PHASE2/PHASE3
prof. dr. Koen Van Laere·Recruiting·Primary completion 1 July 2026·NCT06120049
2 sites
PIs: Donatienne Van Weehaeghe, Dr, Patrick Santens, Prof. dr, Tim Van Langenhove, Dr, +4
HR
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
PHASE2/PHASE3
Hoffmann-La Roche·Active, not recruiting·Primary completion 28 Oct 2026·NCT05115110
35 sites·1 top centre: Columbia / NYP
SA
Efficacy and Safety of Butylphthalide in the Treatment of Multiple System Atrophy
PHASE2/PHASE3
Second Affiliated Hospital, School of Medicine, Zhejiang University·Not yet recruiting·Primary completion 10 Apr 2027·NCT07518810
HL
A Study of Lu AF82422 in Participants With Multiple System Atrophy
Phase 2
H. Lundbeck A/S·Active, not recruiting·Primary completion 16 Nov 2023·NCT05104476
19 sites·4 top centres: UCSF, Mayo Clinic, +2
64 enrolled
AI
AMX0035 in Adult Patients With Wolfram Syndrome
Phase 2
Amylyx Pharmaceuticals Inc.·Active, not recruiting·Primary completion 23 July 2024·NCT05676034
1 site
12 enrolled·PI: Camille Bedrosian
UO
Efficacy and Safety Trial of Sodium Valproate, in Paediatric and Adult Patients With Wolfram Syndrome
Phase 2
University of Birmingham·Active, not recruiting·Primary completion 15 Nov 2024·NCT03717909
6 sites
63 enrolled·PI: Timothy Barrett, PhD, MB, BS
OP
A Phase 2 Study of ONO-2808 in Patients With Multiple System Atrophy
Phase 2
Ono Pharmaceutical Co., Ltd.·Active, not recruiting·Primary completion 15 Sept 2025·NCT05923866
35 sites·3 top centres: Stanford, Mayo Clinic, +1
92 enrolled·PI: Project Leader
BF
Safety and Efficacy of the PAINLESS Nerve Growth Factor CHF6467 in Optic Pathway Glioma (OPG)
Phase 2
Benedetto Falsini·Active, not recruiting·Primary completion 30 June 2026·NCT05733572
1 site
DN
A Phase 2 Study of YA-101 in Patients With Multiple System Atrophy
Phase 2
Dasher Neuroscience Inc.·Recruiting·Primary completion 30 June 2026·NCT06848231
9 sites·2 top centres: Mayo Clinic, Vanderbilt
PI
A Clinical Study of PGN-EDODM1 in People With Myotonic Dystrophy Type 1
Phase 2
PepGen Inc·Recruiting·Primary completion 1 Aug 2026·NCT06667453
8 sites
BA
Safety, Tolerability, and Pharmacokinetics of Exidavnemab in Patients With Parkinson's Disease and Patients With Multiple System Atrophy
Phase 2
BioArctic AB·Active, not recruiting·Primary completion 17 Aug 2026·NCT06671938
7 sites
38 enrolled
MC
Randomized Double-Blind Placebo-Controlled Adaptive Design Trial Of Intrathecally Administered Autologous Mesenchymal Stem Cells In Multiple System Atrophy
Phase 2
Mayo Clinic·Active, not recruiting·Primary completion 1 Dec 2026·NCT05167721
1 site·1 top centre: Mayo Clinic
71 enrolled·PI: Wolfgang Singer
A
A Study of the Effect of (S)-Pindolol Benzoate (ACM-001.1) on Lean Body Mass (LBM) in Obese Patents During (PROACT 1), and Post-semaglutide Therapy (PROACT 2)
Phase 2
Actimed Therapeutics Ltd·Recruiting·Primary completion 1 Mar 2027·NCT07101939
7 sites
PI: Marija Zdravkovic, Prof. Dr
TL
A Phase 2a Study of Foralumab Nasal in Patients With Multiple System Atrophy (MSA)
Phase 2
Tiziana Life Sciences LTD·Recruiting·Primary completion 1 Apr 2027·NCT06868628
1 site
PI: Vikram Khurana
PI
A Study of PLH-2301 in Subjects With Sarcopenia
Phase 2
Pluto Inc.·Not yet recruiting·Primary completion 30 June 2027·NCT07390552
5 sites
PI: Sun Ahe Lee
TB
A Trial to Test if TEV-56286 is Effective for Treatment of Participants With Multiple System Atrophy
Phase 2
Teva Branded Pharmaceutical Products R&D LLC·Recruiting·Primary completion 15 Sept 2027·NCT06568237
60 sites
HU
The NADAPT Study: a Randomized Double-blind Trial of NAD Replenishment Therapy for Atypical Parkinsonism
Phase 2
Haukeland University Hospital·Recruiting·Primary completion 1 Dec 2028·NCT06162013
3 sites
PI: Charalampos Tzoulis
TB
An Extension Trial to Test if TEV-56286 is Effective in Relieving Multiple System Atrophy
Phase 2
Teva Branded Pharmaceutical Products R&D LLC·Recruiting·Primary completion 3 May 2029·NCT07197866
27 sites
PI: Teva Medical Expert
B
Autologous Stem Cell Therapy in Patients With Multiple System Atrophy
Phase 2
Biocells Medical·Not yet recruiting·Primary completion 1 Sept 2029·NCT07465198
1 site
ER
Safety & Efficacy of Eyecyte-RPE™ in Patients With Geographic Atrophy Secondary to Dry Age-related Macular Degeneration.
PHASE1/PHASE2
Eyestem Research Pvt. Ltd.·Recruiting·Primary completion 1 Apr 2025·NCT06394232
GI
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 2 Patients
PHASE1/PHASE2
GeneCradle Inc·Active, not recruiting·Primary completion 22 Apr 2025·NCT05901987
VU
Automated Abdominal Binder for Orthostatic Hypotension
PHASE1/PHASE2
Vanderbilt University Medical Center·Recruiting·Primary completion 1 July 2026·NCT03482297
BP
A Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BMN 351 in Participants With Duchenne Muscular Dystrophy
PHASE1/PHASE2
BioMarin Pharmaceutical·Recruiting·Primary completion 30 Sept 2026·NCT06280209
JB
Feasibility Study Assessing the Effect of Carbidopa/Levodopa Ratio on Orthostatic Hypotension in Multiple System Atrophy - Parkinsonian Type and Parkinson Disease.
PHASE1/PHASE2
Julien Bally·Recruiting·Primary completion 1 Nov 2026·NCT06831500
GI
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 1 Patients
PHASE1/PHASE2
GeneCradle Inc·Recruiting·Primary completion 1 Dec 2026·NCT05824169
GI
Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type 3 Patients
PHASE1/PHASE2
GeneCradle Inc·Recruiting·Primary completion 1 Dec 2026·NCT06421831
VI
A Phase 1/2 Study of VX-670 in Adult Participants With Myotonic Dystrophy 1 (DM1)
PHASE1/PHASE2
Vertex Pharmaceuticals Incorporated·Recruiting·Primary completion 22 Dec 2026·NCT06185764
MC
Mesenchymal Stem Cell Therapy in Multiple System Atrophy
PHASE1/PHASE2
Mayo Clinic·Active, not recruiting·Primary completion 1 Mar 2027·NCT02315027
NP
NS-050/NCNP-03 in Boys With DMD (Meteor50)
PHASE1/PHASE2
NS Pharma, Inc.·Active, not recruiting·Primary completion 1 May 2027·NCT06053814
AB
Study of ATX-01 in Participants With DM1
PHASE1/PHASE2
ARTHEx Biotech S.L.·Recruiting·Primary completion 1 July 2027·NCT06300307
A
A Trial to Learn How Safe AZD9750 is and How Well it Works in People With Metastatic Prostate Cancer When Given With or Without Other Anticancer Drugs
PHASE1/PHASE2
AstraZeneca·Not yet recruiting·Primary completion 4 Jan 2029·NCT07336446
E,
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 45 Skipping to Evaluate the Safety and Efficacy of ENTR-601-45
PHASE1/PHASE2
Entrada Therapeutics, Inc.·Recruiting·Primary completion 1 Mar 2029·NCT07038824
E,
A Study in Participants With Duchenne Muscular Dystrophy Amenable to Exon 44 Skipping to Evaluate the Safety and Efficacy of ENTR-601-44
PHASE1/PHASE2
Entrada Therapeutics, Inc.·Recruiting·Primary completion 28 Mar 2029·NCT07037862
D
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-101 in Participants With Myotonic Dystrophy Type 1
PHASE1/PHASE2
Dyne Therapeutics·Recruiting·Primary completion 1 July 2029·NCT05481879
D
Safety, Tolerability, Pharmacodynamic, Efficacy, and Pharmacokinetic Study of DYNE-251 in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
PHASE1/PHASE2
Dyne Therapeutics·Active, not recruiting·Primary completion 1 Nov 2029·NCT05524883
PB
PBGENE-DMD Phase 1/2a Safety and Preliminary Efficacy Study in Duchenne Muscular Dystrophy (FUNCTION-DMD)
PHASE1/PHASE2
Precision BioSciences, Inc.·Not yet recruiting·Primary completion 1 Nov 2029·NCT07429240