Symptoms and General Pathology

All Conditions

Neoplasms

Blood and Lymph Conditions

Rare Diseases

Syndrome

Leukemia

Myeloproliferative Disorders

Preleukemia

Myelodysplastic Syndromes

Hematologic Diseases

Bone Marrow Diseases

Precancerous Conditions

Acute Graft Versus Host Disease

Chronic Myeloproliferative Disorders

Antineoplastic Agents

All Drugs and Chemicals

Anti-Inflammatory Agents

Antirheumatic Agents

Dermatologic Agents

Reproductive Control Agents

Micronutrients

Hematinics

Vitamins

Fludarabine

Fludarabine phosphate

Triamcinolone

Triamcinolone Acetonide

Triamcinolone hexacetonide

Triamcinolone diacetate

Methotrexate

Prednisone

Melphalan

Tacrolimus

Antimetabolites

Enzyme Inhibitors

Folic Acid

Vitamin B Complex

Folic Acid Antagonists

Immunosuppressive Agents

Immunologic Factors

Calcineurin Inhibitors

Folinic Acid

Folate

Vitamin B9

The primary outcome for the study is GRFS rate at one-year post transplant. GRFS will be estimated using Kaplan Meier method The reported GRFS with recent phase III trial of PTCY/tac/MMF in transplant from matched related and unrelated donors at 1 year follow up was 52%. We hypothesize that with 2 step ATG/Tac/Mini MTX regimen, we can achieve a one year GRFS of 69%.

On Day -8, you will be admitted to the hospital and receive a dose of prednisone at 1 mg/kg (ATG premedication). You will receive a steroids 3 hours before every ATG infusion. On day -7, you will receive a small dose of ATG as an intravenous (IV) infusion. ATG will be repeated on days -6, -5 and -1. Routine transplant chemotherapy agent fludarabine will be given on days -7 to -3 as daily IV infusions. Melphalan, another routine transplant chemotherapy will be given on day -4 as IV infusion. Tacrolimus (standard immune suppression agent) will start on day -3 as continuous IV infusion and switched to oral after engraftment. Methotrexate is another standard immune suppression medication which is given IV on day +1, +3, +6, and +11 post-transplant. We plan to draw blood on days -4,, -1, +3, +7, and +14 to measure ATG levels.

ATG dosing platform when combined with standard tacrolimus and mini methotrexate

Zaid S Al Kadhimi, MD

Margaret Thomas, MPH

Zaid Al Kadhimi, MD

Manuel Espinoza-Gutarra, MD

Donna Salzman, MD

Antonio Di Stasi, MD

Lauren Shea, MD

Omar Jamy, MD

In an effort to reduce graft versus host disease (GVHD) and enhance graft versus leukemia (GVL) effect post allogenic hematopoietic stem cell transplantation (AHSCT), recent research has focused on host immune cell depletion. Frame shifting anti-thymocyte globulin (ATG) backwards to earlier days before days 0 can result in deeper host and less graft T-cell depletion, leading to better immune reconstitution. Preliminary data where 80% of the ATG dose is given on days -6,-5,-4 and 20% given on day -1, showed effective prevention of severe acute GVHD, chronic GVHD and favorable early immune reconstitution. We hypothesize that our 2 step ATG dosing platform when combined with standard tacrolimus and mini methotrexate can prevent grade III-IV acute GVHD and chronic GVHD, resulting in improvement of GVHD/relapse free survival at one year post transplant.

Trial of 2 Step ATG for Prevention of Acute GVHD Post Allogeneic Stem Cell Transplant

Phase II Clinical Trial of 2 Step ATG Combined With Tacrolimus and Mini Methotrexate for Prevention of Acute GVHD Post Reduced Intensity Allogeneic Stem Cell Transplant

Rate of GRFS is defined as the time from date of first dose of fludarabine until occurrence of grade III-IV aGVHD, and or cGVHD requiring systemic immune suppression, and or disease progression or death whichever comes first.

University of Alabama at Birmingham

Professor

Heart and Blood Diseases

Thrombocytosis

Thrombocythemia, Essential

Blood Platelet Disorders

Hemostatic Disorders

Blood Coagulation Disorders

Hemorrhagic Disorders

Essential Thrombocythemia

Ropeginterferon alfa-2b-njft (P1101)

Jewell Jessup, PhD

Mitch Shea

Jaime Chang

Anne Caldwell

Jennifer Steffen

Shannon Gleason

Melissa Parker

Barbie Warner

Leta Ko

Steven Schmitt

Morgan Johnson

Karyn Gordon

Layla Tapia

Stephanie Ortiz

Jason Brecher

Noelle Townsend

Zita Makselyte

Peggy Alton

Toria Wilson

Ines Hernandez

Suzhen Gong

Mary Ann Richie

Braxton Smith

Ellie Gorham

Tathiana Ruiz

Christopher Hillis

Nina Dimson

Ray Urbanski, MD, PhD

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon alfa-2b-njft (P1101) in Adult Patients with Essential Thrombocythemia

PharmaEssentia is developing a pegylated (PEG) IFN-α product, P1101, for the treatment of Essential Thrombocythemia (ET) as lack of disease modifying therapies in essential ET constitutes a serious issue in modern hematology.

Ropeginterferon alfa-2b-njft (P1101) may represent an effective, well-tolerated treatment with the ability to provide a deeper response and superior control of important blood parameters with the potential to alter the course of the disease and prevent progression to post-ET myelofibrosis (MF) and/or secondary acute myeloid leukemia (sAML). Ropeginterferon alfa-2b-njft (P1101) is currently being evaluated in comparison to ANA in the ongoing global Phase 3 clinical study, SURPASS ET.

Enrolled patients will receive P1101 over 13 months followed by an extension period.

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET

A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of Ropeginterferon Alfa-2b-njft (P1101) in Adult Patients With Essential Thrombocythemia

Efficacy will be based on peripheral blood count remission as defined by hematocrit (HCT) \<45%, white blood cell (WBC) count ≤10 × 109/L, and platelets (PLT) ≤ 400 × 109/L in at least 80% of bi-weekly measurements for a consecutive 32-wek period during the 52-week core study treatment period.

PharmaEssentia

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Trial of 2 Step ATG for Prevention of Acute GVHD Post Allogeneic Stem Cell Transplant	NCT06265584	Acute Leukemia Myelodysplastic... Myeloproliferat...	ATG dosing plat...	18 Years - 75 Years	University of Alabama at Birmingham
A Single-arm, Multicenter Study to Assess the Efficacy, Safety, and Tolerability of P1101 in Adults With ET	NCT05482971	Essential Throm...	Ropeginterferon...	18 Years -	PharmaEssentia
Trial of 2 Step ATG for Prevention of Acute GVHD Post Allogeneic Stem Cell Transplant	NCT06265584	Acute Leukemia Myelodysplastic... Myeloproliferat...	ATG dosing plat...	18 Years - 75 Years	University of Alabama at Birmingham

Spots Global Cancer Trial Database for myeloproliferative