Study to Assess the Efficacy and Safety of Umbralisib in Participants With Non-Follicular Indolent Non-Hodgkin's Lymphoma

NCT03364231 | Completed Phase 2 Results FDA Drug

• 1 other identifier: TGR-1202-202
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 4

Brief Summary

This research study will evaluate the safety and efficacy of a study drug called Umbralisib (also known as TGR-1202) alone as a possible treatment for Waldenstrom's Macroglobulinemia that has come back or that has not responded to standard treatment.

Conditions

Marginal Zone Lymphoma; Waldenstrom Macroglobulinemia; Non Follicular Indolent Non-Hodgkin Lymphoma

Outcome Measures

Primary Outcomes (2)

• Overall Response Rate (ORR) as Assessed by Revised Response Criteria for Non- Hodgkin's Lymphoma (Lugano Classification) and Consensus-Based 6th International Workshop on Waldenstrom's Macroglobulinemia (IWWM)

  ORR for MZL=percentage of participants with complete response (CR)/partial response (PR). ORR for WM=CR/PR/very good partial response (VGPR)/minor response (MR). Response assessed per revised Lugano Classification for MZL \&per IWWM for WM participants. Per Lugano criteria CR=complete disappearance of all evidence of disease \& disease-related symptoms. PR=regression of measurable disease \& no new disease sites. Regression=≥50% decrease in the sum of the products of the diameters (SPD) of index lesions, with no increase in size of other lymph nodes/liver/spleen.Per IWWM criteria CR=disappearance of serum monoclonal immunoglobulin M (IgM) protein by immunofixation with a normal serum IgM level. VGPR=reduction of monoclonal IgM protein \>90% from baseline. PR=reduction of monoclonal IgM protein between 50-90% from baseline with regression of measurable disease. Regression defined in similar manner as Lugano Classification. MR=reduction of monoclonal IgM protein \>25% but \<50% from baseline.

  Every 3 cycles (1 Cycle = 28 days) from Day 1 Cycle 1 up to approximately 4.2 years

• Duration of Response (DOR)

  DOR is defined as the time from documentation of a response to treatment to the first documentation of tumor progression or death due to any cause, whichever comes first.

  From the first demonstration of response to umbralisib till disease progression/death (up to approximately 4.2 years)

Secondary Outcomes (4)

• Complete Response (CR) Rate

  Every 3 cycles (1 Cycle = 28 days) from Day 1 Cycle 1 up to approximately 4.2 years

• Progression-Free Survival (PFS)

  From date of randomization until the date of first documented progression (up to approximately 4.2 years)

• Time to Treatment Failure (TTF)

  From first dose on Day 1 of Cycle 1 (28 days = 1 cycle) up to discontinuation of treatment (up to approximately 4.2 years)

• Number of Participants With at Least One Adverse Event (AE)

  From first dose of study treatment up to end of study (up to approximately 4.2 years)

Study Arms (2)

Marginal Zone Lymphoma (MZL): Umbralisib

EXPERIMENTAL

Participants with non-follicular indolent non-Hodgkin's lymphoma (iNHL) with MZL as the histology type received umbralisib, 800 milligrams (mg), orally, once daily (QD), until disease progression, unacceptable toxicity, or withdrawal from the study whichever occurred first.

Drug: Umbralisib

Waldenstrom's Macroglobulinemia (WM): Umbralisib

EXPERIMENTAL

Participants with non-follicular iNHL with WM as the histology type received umbralisib, 800 mg, orally, QD, until disease progression, unacceptable toxicity, or withdrawal from the study whichever occurred first.

Drug: Umbralisib

Interventions

Umbralisib DRUG

Oral Daily Dose

Also known as: TGR-1202

Marginal Zone Lymphoma (MZL): Umbralisib; Waldenstrom's Macroglobulinemia (WM): Umbralisib

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Confirmed diagnosis of Waldenstroms Macroglobulinemia
• Relapsed or refractory after at least one prior treatment regimen
• Eastern Cooperative Oncology Group (ECOG) score of 0 to 2

You may not qualify if:

• Any major surgery, chemotherapy or immunotherapy within the last 21 days
• Evidence of hepatitis B virus, hepatitis C virus or known HIV infection
• Prior autologous stem cell transplant within 6 months of study entry

Sponsors & Collaborators

• TG Therapeutics, Inc.: lead

Study Sites (4)

TG Therapeutics Investigational Trial Site

Washington D.C., District of Columbia, 20007, United States

Location

TG Therapeutics Investigational Trial Site

Boston, Massachusetts, 02215, United States

Location

TG Therapeutics Investigational Trial Site

Hackensack, New Jersey, 07601, United States

Location

TG Therapeutics Investigational Trial Site

New York, New York, 10065, United States

Location

MeSH Terms

Conditions

Lymphoma, B-Cell, Marginal Zone; Waldenstrom Macroglobulinemia

Interventions

umbralisib

Condition Hierarchy (Ancestors)

Lymphoma, B-Cell; Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Neoplasms, Plasma Cell; Hemostatic Disorders; Vascular Diseases; Cardiovascular Diseases; Paraproteinemias; Blood Protein Disorders; Hematologic Diseases; Hemorrhagic Disorders

Results Point of Contact

• Title: TG Therapeutics Clinical Support Team
• Organization: TG Therapeutics

clinicalsupport@tgtxinc.com

1-877-575-8489

Study Officials

• Bruce Cheson, MD

  Lombardi Comprehensive Cancer Center

  STUDY CHAIR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: November 30, 2017
• First Posted: December 6, 2017
• Study Start: November 30, 2017
• Primary Completion: February 15, 2022
• Study Completion: February 15, 2022
• Last Updated: June 23, 2023
• Results First Posted: June 23, 2023

Record last verified: 2023-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (4)