A Phase I/II Trial of MB-dNPM1-TCR.1 in HLA-A*02:01-positive Patients With Relapsed or Refractory NPM1-mutated AML to Determine Safety and Obtain First Data on Efficacy

NCT06424340 Phase 1/Phase 2 RECRUITING

The goal of this Phase I/II, single arm, prospective, open label, dose escalation trial is to assess safety, feasibility and efficacy of ex vivo expanded autologous T cells genetically modified to express a T cell receptor (TCR) specific for dNPM1 peptides restricted to human leukocyte antigen (HLA) A\*02:01 in patients with relapsed or refractory AML.

Details

Lead sponsor	Miltenyi Biomedicine GmbH
Phase	Phase 1/Phase 2
Status	RECRUITING
Enrolment	29
Start date	2024-08-01
Completion	2028-06

Conditions

• Leukemia, Myeloid, Acute

Interventions

• MB-dNPM1-TCR.1

Primary outcomes

• Primary endpoint Phase I — day 28
  Maximum tolerated dose (MTD), as identified by a Bayesian Optimal Interval (BOIN) design at a target toxicity rate of 30%, with toxicity defined as patients experiencing dose limiting toxicity (DLT) until day 28 (week 4) after infusion of MB-dNPM1-TCR.1.
• Primary endpoint Phase II — week 12
  BOR rate to the treatment with MB-dNPM1-TCR.1 assessed at any time within the first 3 months (12 weeks) after infusion as described above.

Countries

Netherlands