SNPMiner Trials by Shray Alag


SNPMiner Trials: Clinical Trial Report


Report for Clinical Trial NCT02590588

Developed by Shray Alag, 2019.
SNP Clinical Trial Gene

Study of Phosphatidylinositol-3-kinase (PI3K) Inhibitor, Idelalisib (GS-1101), in IgM-Associated AL Amyloid

The investigators expect to enroll 15 participants with relapsed or refractory IgM-associated AL amyloidosis onto this Phase II clinical trial. Idelalisib will be self-administered orally at a dose of 100 mg twice daily (may be increased to 150 mg (one tablet) twice daily after 3 months at investigator discretion). Participants will be treated until disease progression, unacceptable toxicity, or decision to withdraw from the trial. Disease evaluations will be performed every three months until disease progression.

NCT02590588 Amyloidosis
MeSH: Amyloidosis
HPO: Amyloidosis

1 Interventions

Name: Idelalisib

Description: Idelalisib daily until unacceptable toxicity or disease progression.

Type: Drug

Idelalisib


Primary Outcomes

Description: Evaluate hematologic response according to standard criteria

Measure: Overall Response

Time: 3 months

Secondary Outcomes

Description: Evaluate time to progression

Measure: Progression Free Survival

Time: 1 year

Description: Number of patients with organ response using standard AL amyloidosis criteria.

Measure: Organ Response

Time: 3 months

Description: Number of Participants With Treatment-Related Adverse Events as Assessed by Common Toxicity Criteria for Adverse Effects (CTCAE) v4.0

Measure: Evaluate Safety and Tolerability of Agent

Time: 3 months

Description: Evaluate quality of life according to Functional Assessment of Cancer Therapy Lymphoma Subscale (FACT-Lym) assessment tool

Measure: Quality of Life

Time: 3 months

Purpose: Treatment

Single Group Assignment


There is one SNP

SNPs


1 L265P

IgM paraprotein identified on serum immunofixation electrophoresis OR light chain-restricted CD20+ lymphoplasmacytic population on biopsy of bone marrow or lymph node (identified by H&E/immunohistochemistry or flow cytometry) OR positive myeloid differentiation primary response gene 88 (MYD88-L265P) OR CXCR4WHIM mutation (CXCR4 mutation - warts, hypogammaglobulinemia, infections, myelokathexis) on submitted samples 3.1.2 --- L265P ---



HPO Nodes


HPO:
Amyloidosis
Genes 22
SLC7A7 PSEN2 FGA ITM2B LYZ TTR NLRP1 POLA1 APOA1 SAA1 MEFV PRNP NLRP3 IL31RA GSN B2M RET APOE OSMR TNFRSF1A APP CST3