NCT01864655

A Phase Ib Multiple Ascending Dose Study of the Safety, Tolerability, and CNS Availability of AZD0530 in Alzheimer's Disease

Completed Phase 1 Last updated 22 April 2021

What this trial tests

Phase 1 trial testing saracatinib in Alzheimer's Disease in 24 participants. Completed in 6 November 2014.

Timeline

1 July 2013

Primary endpoint
1 March 2014

6 November 2014

Quick facts

Lead sponsor	Stephen M. Strittmatter
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	single group
Masking	quadruple
Primary purpose	treatment
Enrollment	24
Start date	1 July 2013
Primary completion	1 March 2014
Estimated completion	6 November 2014
Sites	1 location across United States

Drugs / interventions tested

• saracatinib — full drug profile →
• Placebo

Conditions studied

• Alzheimer's Disease — all drugs for Alzheimer's Disease →

Sponsor

Stephen M. Strittmatter

Who can join

Adults 50 to 90, any sex, with Alzheimer's Disease. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• Number of Participants with Adverse Events on AZD0530
  Time frame: up to 4 weeks
  Patients will be followed closely for any adverse events. These include laboratory measurements, such as complete blood cell counts, basic metabolic panel, including renal function and electrolyte levels, coagulations factors, and liver function tests. These measures will be assessed weekly. Patients will have cognitive testing weekly, to ensure the absence of a prominent decline in function while
• CNS availability of AZD0530 after oral dosing
  Time frame: 4 weeks
  Cerebrospinal fluid will be obtained at the end of the study to measure drug level. CSF will be obtained by a lumbar puncture.

Sponsor's own description

Alzheimer's disease is a devastating neurodegenerative disorder, for which there is currently no effective treatment to slow or halt progression. Beta amyloid peptide accumulates in the brains of those with Alzheimer's, and is thought to play a major role in triggering the dementia. The investigators recently characterized a molecular pathway by which ß-amyloid damages neurons, and showed that the protein termed Fyn kinase is crucial. Our data suggest that blocking Fyn will have a significant therapeutic benefit for Alzheimer's. Astra Zeneca has developed a blocker of Fyn and related kinases (AZD0530) for the treatment of cancer. Chronic AZD0530 administration is well tolerated in humans, and the investigators propose to test its potential as a novel Alzheimer's disease modifying therapy. This study will assess the safety and tolerability of AZD0530 in patients with Alzheimer's disease.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Epileptic activity in Alzheimer's disease: causes and clinical relevance.
   Vossel KA, Tartaglia MC, Nygaard HB, Zeman AZ, et al · · 2017 · cited 486× · PMID 28327340 · DOI 10.1016/s1474-4422(17)30044-3
2. Neuroinflammation in Alzheimer disease.
   Heneka MT, van der Flier WM, Jessen F, Hoozemanns J, et al · · 2025 · cited 307× · PMID 39653749 · DOI 10.1038/s41577-024-01104-7
3. Fyn inhibition rescues established memory and synapse loss in Alzheimer mice.
   Kaufman AC, Salazar SV, Haas LT, Yang J, et al · · 2015 · cited 261× · PMID 25707991 · DOI 10.1002/ana.24394
4. Advances in developing novel therapeutic strategies for Alzheimer's disease.
   Cao J, Hou J, Ping J, Cai D. · · 2018 · cited 182× · PMID 30541602 · DOI 10.1186/s13024-018-0299-8
5. Current Research Therapeutic Strategies for Alzheimer's Disease Treatment.
   Folch J, Petrov D, Ettcheto M, Abad S, et al · · 2016 · cited 173× · PMID 26881137 · DOI 10.1155/2016/8501693
6. The role of pathological tau in synaptic dysfunction in Alzheimer's diseases.
   Wu M, Zhang M, Yin X, Chen K, et al · · 2021 · cited 145× · PMID 34753506 · DOI 10.1186/s40035-021-00270-1
7. Overcoming Drug Development Bottlenecks With Repurposing: Old drugs learn new tricks.
   Strittmatter SM. · · 2014 · cited 145× · PMID 24901567 · DOI 10.1038/nm.3595
8. Fyn kinase inhibition as a novel therapy for Alzheimer's disease.
   Nygaard HB, van Dyck CH, Strittmatter SM. · · 2014 · cited 142× · PMID 24495408 · DOI 10.1186/alzrt238