Ignite Creation Date:
2024-05-05 @ 9:36 AM
Last Modification Date:
2024-10-26 @ 9:02 AM
Study NCT ID:
NCT00001145
Status:
COMPLETED
Last Update Posted:
2008-03-04
First Post:
1999-11-03
Brief Title:
Study of Immune Responses and Safety of Recombinant Human CD40 Ligand in Patients With X-Linked Hyper-IgM Syndrome
Sponsor:
National Institute of Allergy and Infectious Diseases NIAID
Organization:
National Institutes of Health Clinical Center CC
Study Overview
Official Title:
Study of Immune Responses and Safety of Recombinant CD40 Ligand in Patients With X-Linked Hyper IgM Syndrome
Status:
COMPLETED
Status Verified Date:
2003-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The primary goal of this Phase III study is to assess the immune response and safety of recombinant human CD40 ligand rhuCD40L in patients with X-linked hyper IgM syndrome XHIM XHIM is a rare genetic disease caused by mutations in the gene encoding CD40 ligand Individuals with this syndrome fail to make gamma immune globulin frequently suffer from opportunistic infections and are at an increased risk of developing cancer Despite treatment with gamma globulin replacement therapy the expected survival of patients with XHIM is less than 20 percent by the age of 25
In a mouse model of this syndrome treatment with man-made CD40 ligand protein protected the mouse from opportunistic infections restored the mouses ability to make gamma globulin and improved survival We want to determine if a similar approach can work in humans with XHIM The study will be conducted at the Clinical Center of the National Institutes of Health in Bethesda Maryland
For most patients rhuCD40L will be administered by injection under the skin over a period of six months and follow-up exams are required at 2-month intervals for an additional 6 months During the study patients will be maintained on intravenous gamma globulin antibiotics to protect against opportunistic infection and if needed growth factors to control neutropenia The immune response to rhuCD40Lwill be measured by routine methods such as measuring a patients ability to synthesize gamma globulin when challenged with immunizations to keyhole limpet hemocyanin KLH and Bacteriophage Phi-X 174 Phi-X 174 Our long-term goal is to define a therapeutic regimen that will provide effective immunological reconstitution to patients with XHIM and improve their life expectancy
In a mouse model of this syndrome treatment with man-made CD40 ligand protein protected the mouse from opportunistic infections restored the mouses ability to make gamma globulin and improved survival We want to determine if a similar approach can work in humans with XHIM The study will be conducted at the Clinical Center of the National Institutes of Health in Bethesda Maryland
For most patients rhuCD40L will be administered by injection under the skin over a period of six months and follow-up exams are required at 2-month intervals for an additional 6 months During the study patients will be maintained on intravenous gamma globulin antibiotics to protect against opportunistic infection and if needed growth factors to control neutropenia The immune response to rhuCD40Lwill be measured by routine methods such as measuring a patients ability to synthesize gamma globulin when challenged with immunizations to keyhole limpet hemocyanin KLH and Bacteriophage Phi-X 174 Phi-X 174 Our long-term goal is to define a therapeutic regimen that will provide effective immunological reconstitution to patients with XHIM and improve their life expectancy
Detailed Description:
The purpose of this Phase III study is to evaluate clinical response and safety following administration of recombinant human CD40 ligand rhuCD40L in up to 5 patients with X-linked hyper IgM syndrome XHIM XHIM is a rare genetic disease caused by mutations in the gene encoding CD40 ligand CD154 and is characterized by hypogammaglobulinemia opportunistic infections and an increased risk of neoplastic disease Despite treatment with intravenous gamma globulin the expected survival of patients with XHIM is less than 20 by the age of 25 The proposed protocol is a proof of principle study designed to determine if administration of rhuCD40L can reverse the core immunologic defects of patients with XHIM To this end we will immunize patients with neo antigens specifically keyhole limpet hemocyanin KLH and Bacteriophage Phi-X 174 PhiX174 to evaluate antigen-specific B and T cell responses Clinical response and toxicity will be evaluated using routine hematological and clinical evaluation quantitation of KLH and PhiX174 specific IgG in serum measurement of proliferation and cytokine production to KLH simulation in vitro and FACS analysis to quantitate memory B and T cells Our long-term goal is to define a therapeutic regimen that will provide effective immunological reconstitution to patients with XHIM and improve life expectancy
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 00-I-0006 | None | None | None |