Infections

All Conditions

Symptoms and General Pathology

Skin and Connective Tissue Diseases

Immune System Diseases

Blood and Lymph Conditions

Diseases and Abnormalities at or Before Birth

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Rare Diseases

Syndrome

Communicable Diseases

Herpes Simplex

Immunologic Deficiency Syndromes

Neutropenia

Primary Immunodeficiency Diseases

Warts

Agammaglobulinemia

Papillomavirus Infections

Virus Diseases

DNA Virus Infections

Skin Diseases

Skin Diseases, Viral

Tumor Virus Infections

Skin Diseases, Infectious

Granulocytopenia

WHIM Syndrome

No intervention

Sudha Parasuraman, MD

This natural history study is a prospective and retrospective, observational study of WHIM patients. WHIM syndrome is a rare, genetic, primary immunodeficiency disorder (a disorder in which the body's immune system does not function properly). WHIM is an acronym for some of the symptoms of the disorder - Warts, Hypogammaglobulinemia (low levels of certain antibodies), Infections and Myelokathexis (too many white blood cells in the bone marrow).This study includes 10-year retrospective (Retrospective Phase) and up to 5-year prospective (Prospective Phase) components.

Given the rarity of patients with WHIM syndrome, this study is being conducted to better understand the clinical course of untreated patients with WHIM syndrome.

The goals of this Natural History Study are to define both the frequency and diversity of WHIM syndrome by specific genetic mutation, as well as to understand the clinical course and phenotype of untreated WHIM patients.

Blood or buccal swab for the purposes of identifying a CXCR4 mutation.

A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome

Infections assessed by hospitalizations (including intensive care), antibiotic use, outpatient medical appointments and missed days of school/work.

Warts assessed by number and size of lesions, need for surgical, systemic or topical treatment and complications.

Quality of life as assessed by the quality of life instrument the 36-Item Short Form Survey (SF-36)

Quality of life as assessed by the quality of life instrument the Pediatric Quality of Life Inventory (Peds-QL)

Quality of life as assessed by the quality of life instrument the Life Quality Index (LQI)

Quality of life as assessed by the quality of life instrument the HPV Impact Profile (HIP)

X4 Pharmaceuticals

Neoplasms

Leukemia

Leukemia, Lymphoid

Leukemia, Lymphocytic, Chronic, B-Cell

Neoplasms by Histologic Type

Hematologic Diseases

Lymphoproliferative Disorders

Lymphatic Diseases

Immunoproliferative Disorders

Leukemia, B-Cell

Chronic Disease

Disease Attributes

Blood Protein Disorders

Chronic Graft Versus Host Disease

Chronic Lymphocytic Leukemia

Panzyga is a 10% IVIG produced from a pool of human fresh frozen plasma donations

Panzyga

Placebo

Patrick M Murphy

Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia

Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)

Major infection for this trial is defined as:

* Bacterial and/or viral infections resulting in death
* Bacterial and/or viral infections, which are microbiologically documented (MDI) or clinically documented (CDI) requiring treatment with anti-infectives; upper respiratory tract infections, bronchitis, lower urinary tract infections, localized skin infections and stomatitis (MDI or CDI) are considered major only if they require treatment with antiinfectives AND hospitalization or hospitalization prolongation.
* Fever of unknown origin (FUO) requiring hospitalization or hospitalization prolongation

Inclusive of antibacterials and antivirals

Octapharma

Bacterial Infections

Recurrence

Mycoses

Bacterial Infections and Mycoses

All Drugs and Chemicals

Antibodies

Immunoglobulins

Immunoglobulins, Intravenous

gamma-Globulins

Rho(D) Immune Globulin

Immunologic Factors

Participants will receive a loading dose of 150 milligrams per kilograms per day (mg/kg/day) (Week 1, Days 1 to 5) subcutaneously (SC) for 5 consecutive daily doses followed by weekly infusions of 150 mg/kg starting Week 2 (Day 8) through Week 53 (end of Treatment Phase).

The SMT will include the antileukemic treatments and the other supportive treatments that the participants will need during their participation.

Participants will receive sterile 0.9 percent Sodium Chloride Injection, United States Pharmacopeia (USP) or equivalent starting at Week 1 (Days 1 to 5) SC for 5 consecutive daily doses followed by weekly infusions starting at Week 2 (Day 8) through Week 53.

The SMT will include the antileukemic treatments and the other supportive treatments that the participants will need during their participation.

Xembify

Terra Stockwell

Marina Acosta Enslen

The primary purpose of the study is to evaluate whether weekly administered XEMBIFY® plus Standard Medical Treatment (SMT) over a one-year period will reduce the rate of major bacterial infections per participant per year in participants with hypogammaglobulinemia (HGG) associated with B-cell chronic lymphocytic leukemia (CLL) in comparison to the Placebo plus SMT group.

A Study to Evaluate Efficacy, Safety, and Pharmacokinetics of XEMBIFY® Plus Standard Medical Treatment (SMT) Compared to Placebo Plus SMT to Prevent Infections in Participants With Hypogammaglobulinemia and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia

A Multi-Center, Parallel, Double-Blinded, Placebo-Controlled Clinical Trial to Evaluate Efficacy, Safety, and Pharmacokinetics of XEMBIFY® Plus Standard Medical Treatment Compared to Placebo Plus Standard Medical Treatment to Prevent Infections in Patients With Hypogammaglobulinemia and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia

Grifols Therapeutics LLC

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
A Retrospective and Prospective Natural History Study of Patients With WHIM Syndrome	NCT03087370	WHIM Syndrome	No intervention	-	X4 Pharmaceuticals
Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)	NCT04502030	Chronic Lymphoc... Hypogammaglobul...	Panzyga Placebo	18 Years -	Octapharma
Double-blind, Randomized, Placebo-controlled, Prospective Phase III Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients With Chronic Lymphocytic Leukemia ("PRO-SID" Study)	NCT04502030	Chronic Lymphoc... Hypogammaglobul...	Panzyga Placebo	18 Years -	Octapharma
A Study to Evaluate Efficacy, Safety, and Pharmacokinetics of XEMBIFY® Plus Standard Medical Treatment (SMT) Compared to Placebo Plus SMT to Prevent Infections in Participants With Hypogammaglobulinemia and Recurrent or Severe Infections Associated With B-cell Chronic Lymphocytic Leukemia	NCT05645107	Hypogammaglobul... Bacterial Infec... B-cell Chronic ...	Xembify Placebo	18 Years -	Grifols Therapeutics LLC

Spots Global Cancer Trial Database for hypogammaglobulinemia