EXALT-2: Comprehensive Genomic Profiling and Next Generation Functional Drug Screening for Patients With Aggressive Haematological Malignancies

• Sponsor: Medical University of Vienna (Other)
• Overall Status: Recruiting
• CT.gov ID: NCT04470947
• Collaborator: Roche Pharma AG (Industry), Allcyte GmbH (Other)
• Enrollment: 150
• Location: 1
• Anticipated Duration (Months): 54.7
• Patients Per Site Per Month: 2.7

Study Details

Study Description

Brief Summary

EXALT-2 is a prospective, randomized, three arm study for treatment decision guided either by either comprehensive genomic profiling, next generation drug screening or physician's choice

Condition or Disease	Intervention/Treatment	Phase
Advanced Lymphoma; Refractory Lymphoma; Refractory Leukemia; Refractory Acute Myeloid Leukemia; Refractory T-Cell Lymphoma	Diagnostic Test: Next generation functional drug screening; Diagnostic Test: Comprehensive genomic profiling

Study Design

• Study Type: Observational [Patient Registry]
• Anticipated Enrollment: 150 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Comprehensive Genomic Profiling and Next Generation Functional Drug Screening for Patients With Aggressive Haematological Malignancies: Next Generation Personal Hematology
• Actual Study Start Date: Jun 10, 2020
• Anticipated Primary Completion Date: Dec 31, 2024
• Anticipated Study Completion Date: Dec 31, 2024

Arms and Interventions

Arm	Intervention/Treatment
Next generation functional drug screening	Diagnostic Test: Next generation functional drug screening; High-throughput image based in-vitro drug screening on primary patient tumor cells; Diagnostic Test: Comprehensive genomic profiling; Comprehensive targeted profiling of genetic aberrations on primary patient tumor material; Other Names: FoundationOne Heme
Comprehensive genomic profiling	Diagnostic Test: Next generation functional drug screening; High-throughput image based in-vitro drug screening on primary patient tumor cells; Diagnostic Test: Comprehensive genomic profiling; Comprehensive targeted profiling of genetic aberrations on primary patient tumor material; Other Names: FoundationOne Heme
Physician's choice	Diagnostic Test: Next generation functional drug screening; High-throughput image based in-vitro drug screening on primary patient tumor cells; Diagnostic Test: Comprehensive genomic profiling; Comprehensive targeted profiling of genetic aberrations on primary patient tumor material; Other Names: FoundationOne Heme

Outcome Measures

Primary Outcome Measures

1. Percentage of patients with a ratio ≥1.3 of progression free survival (PFS) compared to most recent treatment [Through study completion, an average of 8 month]

   The study aims to identify if next-generation functional drug screening (ngFDS) and/or comprehensive genomic profiling (CGP; FoundationOne®Heme) compared to physicians' choice guided treatment will have an increased percentage of patients with a ratio ≥1.3 of progression free survival (PFS)/PFS of most recent treatment in patients with aggressive haematological malignancies

Secondary Outcome Measures

1. Average Ratio of PFS/PFS of most prior treatment [Through study completion, an average of 8 months]

   Average Ratio of PFS/PFS of most prior treatment

2. Overall response rate (ORR) [Through study completion, an average of 8 months]

   Overall response rate (ORR)

3. Number of treatable targets identified [Through study completion, an average of 8 months]

   Number of treatable targets identified

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Inclusion Criteria:

• patient is suffering from aggressive haematological disease AND has undergone at least two lines of previous therapies AND/OR has undergone at least one previous therapy and no standard treatment is available in the specific disease setting and disease specific guidelines recommend treatment in studies.

• duration of last response is less than 6 months defined as first day of last treatment to date of relapse, the response duration has to be available with dates (dd/mm/yyyy) for initiation of and relapse to previous treatment.

• best response to previous treatment has to be available.

• The patient is able to give written informed consent and wishes to undergo further therapy

• further therapy is medically feasible

• tumor cell-containing samples can be obtained

Exclusion Criteria:

• current participation in another experimental clinical trial

• performance status does not allow participation (ECOG ˃ 1)

• pregnancy, tested at screening

• patient suffers from classical or nodular, lymphocyte predominant Hodgkins lymphoma.

• other malignoma, diagnosed <1a before inclusion (except localized squamous cell carcinoma of the skin, surgically curable melanoma of the skin, basal cell carcinoma of the skin)

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Medical University of Vienna	Vienna		Austria	1090

Sponsors and Collaborators

• Medical University of Vienna
• Roche Pharma AG
• Allcyte GmbH

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

• Snijder B, Vladimer GI, Krall N, Miura K, Schmolke AS, Kornauth C, Lopez de la Fuente O, Choi HS, van der Kouwe E, Gültekin S, Kazianka L, Bigenzahn JW, Hoermann G, Prutsch N, Merkel O, Ringler A, Sabler M, Jeryczynski G, Mayerhoefer ME, Simonitsch-Klupp I, Ocko K, Felberbauer F, Müllauer L, Prager GW, Korkmaz B, Kenner L, Sperr WR, Kralovics R, Gisslinger H, Valent P, Kubicek S, Jäger U, Staber PB, Superti-Furga G. Image-based ex-vivo drug screening for patients with aggressive haematological malignancies: interim results from a single-arm, open-label, pilot study. Lancet Haematol. 2017 Dec;4(12):e595-e606. doi: 10.1016/S2352-3026(17)30208-9. Epub 2017 Nov 15.

• Responsible Party: Philipp Staber, MD, PhD, Assoc. Prof. Priv.-Doz. Dr.med.univ. Philipp Staber, Medical University of Vienna
• ClinicalTrials.gov Identifier: NCT04470947
• Other Study ID Numbers: FA711C1050
• First Posted: Jul 14, 2020
• Last Update Posted: Jul 14, 2020
• Last Verified: Jul 1, 2020

• Individual Participant Data (IPD) Sharing Statement: No
• Plan to Share IPD: No
• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: No
• Keywords provided by Philipp Staber, MD, PhD, Assoc. Prof. Priv.-Doz. Dr.med.univ. Philipp Staber, Medical University of Vienna: Personalized medicine
• Additional relevant MeSH terms: Lymphoma; Leukemia; Hematologic Neoplasms

Study Results

No Results Posted as of Jul 14, 2020