Active clinical trials for "Immunologic Deficiency Syndromes"

To determine the response to treatment with NVP, ZDV and 3TC in HIV positive women, who previously received NVP for the prevention of mother to child transmission.

This is a clinical trial of gene therapy for X-linked severe combined immunodeficiency (XSCID), a genetic disease caused by defects in a protein called the common gamma chain, which is normally on the surface of immune cells called lymphocytes. XSCID patients cannot make T lymphocytes, and their B lymphocytes fail to make essential antibodies for fighting infections. Without T and B lymphocytes patients develop fatal infections in infancy unless they are rescued by a bone marrow transplant from a healthy donor. However, even transplanted patients may achieve only partial immune recovery and still suffer from many infections, auto-immunity and/or and poor growth. A recent, successful trial in France used gene therapy instead of bone marrow transplantation for infants with XSCID. This experience indicates that gene therapy can provide clinical benefit to XSCID patients. We will enroll eight older XSCID patients (1.5-20 years-old), who have previously received at least one bone marrow transplant, but still have poor T and B lymphocyte function that compromises their quality of life. Before enrollment, these subjects will have had some of their own blood-forming stem cells harvested and frozen in a blood bank. These cells have a defective gene, but a correct copy of the gene will be inserted while the cells are grown in sterile conditions outside the patient's body. To do this, the cells will be unfrozen and exposed for four days in a row to growth factors and particles of a retrovirus we have constructed and tested called "GALV MFGS-gc." Retrovirus particles will attach to the patient cells and introduce a correct copy of the common gamma chain gene into cells capable of growing into all types of blood cells, including T and B lymphocytes. XSCID patients who are enrolled in the study will receive a single dose of their own cells that have been modified by the GALV MFGS-gc treatment and also will be given another drug called palifermin to help prevent side effects from the chemotherapy and possibly try to improve the development of the T cells. After this, the patients will be monitored to find out if the treatment is safe and to see if their immune function improves. Study endpoints are (1) efficient and safe clinical-scale transduction of HSC from post-BMT XSCID subjects; (2) administration of a nonmyeloablative conditioning regimen in older patients to improve engraftment; (3) administration of a transduced HSC to eight subjects; (4) administration of KGF to improve thymic function post transplant to improve T cell development; and (5) appropriate follow-up of the treated subjects to monitor vector sequence distribution, gc expression in hematopoietic lineages, and lymphoctye numbers and function as well as general health and immune status.

The purpose of this study is to determine the immunomodulatory and antiviral effects of the glutathione-restoring dithiol, alpha lipoic acid (ALA) in HIV-infected persons unresponsive to highly active antiretroviral treatment (HAART).

This study will test the safety and effectiveness of genetically altered T lymphocytes (white blood cells of the immune system) in reducing viral load in patients infected with the human immunodeficiency virus (HIV). The lymphocytes will have two genes inserted into them; a laboratory-manufactured anti-HIV gene designed to inhibit HIV reproduction (either the RevTD or Rev-TD-antiTAR gene), and a "marker" gene that will show whether or not the inserted genes have gotten into the cells. Identical twin pairs 18 years of age and older- one of whom is HIV-positive (infected with the human immunodeficiency virus) and the other HIV-negative (not infected) may be eligible for this study. All participants will have a complete medical history and physical examination, blood tests and a tetanus booster shot, if indicated. The non HIV-infected twin will then undergo lymphapheresis to collect lymphocytes. In this procedure, whole blood is collected through a needle placed in an arm vein. The blood circulates through a machine that separates it into its components. The lymphocytes are then removed, and the red cells and plasma are returned to the donor, either through the same needle or through a second needle placed in the other arm. The donor cells are grown in the laboratory for a few days, and then the new genes are inserted into them. The genetically altered cells are grown in the laboratory for several days until their numbers increase approximately a thousand-fold. They are then infused intravenously (through a vein) into the infected twin. These procedures-lymphapheresis, gene modification and infusion-will be repeated at approximately 2-month intervals up to four times. Each lymphocyte infusion takes about 60 minutes. The patient's vital signs (temperature, pulse, blood pressure and breathing) are monitored frequently during the infusion and hourly for 4 hours after the infusion. Blood samples are taken the day of the infusion, 3 days later, and then weekly to monitor the gene-modified cells, immune status, viral activity, and other factors. These tests may be done less often as the study progresses and more is learned about the safety of the infusions. The infusions are done on an outpatient basis unless side effects require that they be done in the hospital with post-infusion monitoring for at least 24 hours. Patients will be followed for long-term effects of treatment monthly for the first 3 months, once a month for the next 9 months and yearly from then on. This study will contribute information about the use and side effects of gene therapy in HIV infection that may lead to new treatment strategies. A potential direct benefit to HIV-infected individuals participating in this study is reduced viral load; in laboratory studies, the RevTD and Rev-TD-antiTAR genes have inhibited HIV spread in the test tube. However, this is an early phase of study, and the likelihood of receiving this benefit is unknown.

This study will examine the safety of giving antiviral therapy for HIV infection in a once-daily dosing schedule, and assess how well patients tolerate this regimen. A once a day dosing schedule may be easier for some people to follow than one that requires taking medicine 2 or 3 times a day. The ease of treatment is important, because not following the prescribed dosing regimen may make therapy less effective or ineffective. HIV-infected patients 18 years and older who have never been treated for their infection may be eligible for this study. Candidates will be screened with a history and physical examination, including blood tests. Participants will take the following medications once a day: 1200 mg of amprenavir (8 capsules); 300 mg of ritonavir (3 capsules); 600 mg of abacavir (2 pills); and 300 mg of lamivudine (2 pills). Patients will have routine blood tests and be seen by a nurse or doctor, or both, at follow-up visits at weeks 2, 4, 8, 12, and 16; then every 8 weeks until week 48; and then every 3 months for up to 3 years. At week 2, a special blood test will be done over the course of a day to measure blood drug levels. For this test, blood samples will be drawn 8 times over a 24-hour period. A heparin lock (a device that allows the needle to remain in the vein) will be used to avoid multiple needle sticks.

To compare the efficacy, safety, and toleration of fluconazole as a single daily oral suspension for 14 days versus nystatin oral suspension 4 times daily for 14 days in the treatment of oropharyngeal candidiasis in patients with AIDS or HIV infection.

To evaluate the efficacy, safety, and durability of response of SP-303 in decreasing stool weight in AIDS patients with diarrhea over 6 days of treatment.

To assess the tolerance and toxicity profile of deoxy-3'-fluorothymidine (FLT) after multiple oral dosing for 16 weeks. To characterize the steady-state pharmacokinetics of FLT after multiple oral doses. To assess the effect of FLT on immunologic and virologic markers of HIV infection (CD4+ lymphocyte count, p24 antigen, viremia) in patients with AIDS or AIDS related complex (ARC) after multiple oral dosing for 16 weeks.

To compare two lipid emulsions in the long-term parenteral alimentation of patients with AIDS in relation to: Clinical effectiveness. Effect on immunologic function. Effect on HIV load as measured by p24 antigen levels. Effect on relative HIV infectivity.

The purpose of this pilot study is to evaluate the efficacy of Retrovir (AZT) in the treatment of AIDS-related dementia and various neuromuscular complications. HIV is both a lymphotropic and neurotropic virus which can affect both the central and peripheral nervous systems (CNS, PNS). There is evidence that the CNS and PNS may harbor the virus in a latent state, with the potential for continuous reinfection of other body systems. Therefore, effective therapeutic efforts against HIV infection should provide effective antiviral activity within the nervous system.