A clinical trial to learn about the effects of VHB937 in people with Alzheimer's disease

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

13 Feb 2026 → ongoing

Decision date (initial)

2025-08-04

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-516966-12-00

WHO UTN

U1111-1320-2504

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety

To evaluate the effect of VHB937 compared to placebo on cognition and function

Secondary objectives 4

1. To evaluate the safety and tolerability of VHB937 compared to placebo.
2. To evaluate the effect of VHB937 compared to placebo on cognition.
3. To evaluate the effect of VHB937 compared to placebo on activities of daily living.
4. To assess pharmacokinetics (PK) and immunogenicity (IG) of VHB937 in serum.

Conditions and MedDRA coding

Alzheimer’s disease

Regulatory references

Scientific advice from competent authorities

Food And Drug Administration

Plan to share IPD

Yes

IPD plan description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com.

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 7

1. Male and female participants 50 to 85 years of age, with a maximum body weight of 180 kg at the time of signing the informed consent.
2. Diagnosis of Mild Cognitive Impairment (MCI) due to AD or mild AD according to the NIA-AA criteria (Jack et al 2018) at Screening.
3. Clinical Dementia Rating (CDR) Global score of 0.5 or 1.0 at Screening and Baseline.
4. ADAS-Cog14 total score between CCI at Screening.
5. Biomarker-based confirmation of Alzheimer disease (AD) at Screening based on cerebral spinal fluid (CSF) biomarkers or amyloid PET imaging. Historical confirmation of amyloid positivity by CSF or PET is accepted.
6. Reliable study partner who can accompany the participant at study visits in which informant scales are administered.
7. Participants receiving an AChEI or memantine or both for AD must be on a stable dose for at least 12 weeks before Randomization. For participants who discontinued these medications before Screening, the stop date should be at least 12 weeks before Randomization. Treatment-naïve participants can be enrolled into the study.

Exclusion criteria 7

1. Dementia due to a condition other than AD including, but not limited to, frontal temporal dementia (FTD), Parkinson's disease, dementia with Lewy bodies, Huntington disease, vascular dementia.
2. Transient ischemic attacks (TIA) or stroke occurring within 12 months prior to randomization.
3. MRI evidence of more than CCI; any area of superficial siderosis; evidence of vasogenic edema; evidence of cerebral contusion, encephalomalacia, aneurysms, or infective lesions; evidence of multiple (≥ 2) lacunar infarcts irrespective of location or stroke involving a major vascular territory, severe small vessel, or white matter disease; space occupying lesions; or brain tumors (however, lesions diagnosed as meningiomas or arachnoid cysts and less than 1 cm at their greatest diameter need not be exclusionary).
4. Uncontrolled thyroid disease or uncontrolled diabetes or clinically significant laboratory abnormalities for thyroid function or fasting glucose in central laboratory results at Screening, as assessed by Investigator. The Investigator should ensure that the diabetic participants remain adequately controlled during the study.
5. Clinical evidence of liver disease or liver injury (other than hepatitis specified above) defined by any of the following results in central laboratory at Screening: · Total bilirubin > 1.5 x ULN, · Alkaline phosphatase (ALP) > 3 x ULN, · AST (SGOT) or ALT (SGPT) > 3 x ULN, and · Gamma-glutamyl-transferase (GGT) > 3 x ULN.
6. History or current diagnosis of cardiovascular conditions indicating significant risk of safety for participants in the study such as, but not limited to: · Myocardial infarction or unstable angina (within 6 months of Screening), clinically significant cardiac arrhythmia (e.g., sustained ventricular tachycardia) requiring treatment and clinically significant second- or third-degree AV block without a pacemaker. · Familial long QT syndrome or known family history of Torsade de Pointe. · Resting QTcF ≥ 450 ms (male) or ≥ 460 ms (female) at Screening or inability to determine the QTcF interval
7. Severe renal impairment (Glomerular Filtration Rate < 30 mL/min/1.73 m2) in central laboratory results at Screening.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from Baseline to Week 72 in the Clinical Dementia Rating scale – Sum of Boxes (CDR-SB)

Secondary endpoints 4

1. Incidence and severity of: ● Adverse events ● Safety findings from brain MRI (based on blinded independent central reading to assess abnormalities including ARIA events) ● Laboratory tests ● Vital signs ● ECG findings ● Suicidality assessment (Columbia Suicide Severity Rating Scale C-SSRS)
2. Changes from Baseline in CDR-SB and ADAS-Cog14 over time until Week 72
3. Changes from Baseline in instrumental activities of daily living (iADL) on the ADCS-ADL scale over time until Week 72
4. Pharmacokinetics: determination of VHB937 concentrations in serum at selected timepoints Immunogenicity: determination of anti-VHB937 antibodies in serum at selected timepoints

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 9

Locations

8 EU/EEA countries · 57 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 125 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications