NCT05048498

Pharmacokinetics, Tolerability and Safety of NEX-18a

An Open Pilot Study to Assess the Pharmacokinetics, Tolerability, and Safety of NEX-18a Given as a Subcutaneous Injection for the Treatment of Intermediate 2 or Higher-risk MDS, CMML or AML

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 2 (actual)
• Sponsor: Nanexa AB · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The study will evaluate the safety, tolerability and pharmacokinetics of NEX-18a, a long-acting injectable azacitidine, in patients diagnosed with intermediate 2 or higher-risk MDS, CMML, or AML and already on treatment with azacitidine.

Detailed description

Since 2010, subcutaneous treatment with azacitidine has been the first-line treatment for patients with high-risk MDS. Azacitidine has been established as a standard of care and is described in the National Comprehensive Cancer Network (NCCN) guidelines as a core component of optimal treatment of MDS. However, mainly due to its short half-life (41 minutes) when administered subcutaneously azacitidine should, according to the approved label, be administered for seven consecutive days at a dose of 75 mg/m2 body surface area (BSA) each 28-day cycle. In the Nordic Guidelines, two alternative dosing schedules may also be considered: 100 mg/m2 BSA sc day 1-5 or 75 mg/m2 BSA sc day 1-5 + 8-9 (to avoid injection during weekends). Nanexa AB has developed NEX-18a, a subcutaneous injection of azacitidine with extended-release based on the drug delivery system, PharmaShell®. Drug particles are enclosed in a coating with controlled solubility, and as the coating dissolves over time the drug is released in a predefined manner. This technique provides a way to create drugs with a prolonged release for parenteral administration. The technology used by Nanexa to manufacture the coating is via Atomic Layer Deposition (ALD). In ALD, reactive gases are used which build up a surface coating with high precision, atomic layer by atomic layer. NEX-18a will offer a benefit to current azacitidine treatment with a reduction of subcutaneous administrations, decreased need for pre-medication, and will reduce the time each patient has to spend in the hospital in order to receive the treatment in each cycle. In addition, the patients will spend less time traveling to and from the hospital and from a health care perspective, one injection instead of seven per cycle will reduce the time and the resources the health care provider dedicates to treating the patients.

Conditions

• Myelodysplastic Syndromes (MDS)
• Chronic Myelomonocytic Leukemia (CMML)
• Acute Myeloid Leukemia (AML)

Interventions

Type	Name	Description
DRUG	NEX-18a injection	In Treatment phase 1, NEX-18a will be given as a single subcutaneous injection. In Treatment phase 2, NEX-18a will be given as a single subcutaneous injection.
DRUG	Azacitidine Injection	In Treatment phase 1, azacitidine will be administered once daily for four days. In Treatment phase 2, azacitidine will be administered once daily for three days.

Timeline

• Start date: 2021-04-27
• Primary completion: 2022-02-10
• Completion: 2022-02-10
• First posted: 2021-09-17
• Last updated: 2023-10-17

Locations

2 sites across 1 country: Sweden