Clinical trials: Primary Immunodeficiencies
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Primary Immune Deficiency Disorder
Study to Evaluate the Efficacy, Tolerability and Safety of Octanorm in Patients With Primary Immunodeficiency Diseases
Clinical phase 3 study to evaluate the efficacy, tolerability and safety of subcutaneous human immunoglobulin (octanorm) in patients with primary immunodeficiency diseases.
- Trial name
- NCT03988426
- Testing
- Octanorm
- Where
- 5 sites in Russia
- Volunteers
- 25 people
- Sponsor
- Octapharma
HIV Infections
Anti-HIV Medications for People Recently Infected With HIV
It is not known if anti-HIV treatment for recently infected patients improves long-term patient prognosis. The purpose of this study is to determine if a one year course of anti-HIV medications slows progression of HIV disease in…
- Trial name
- NCT00106171
- Testing
- Highly active antiretroviral therapy (HAART)
- Where
- 5 sites in United States, Canada
- Volunteers
- 113 people
- Sponsor
- Johns Hopkins Bloomberg School of Public Health
Primary Immune Deficiency
Study of Subcutaneous Immunoglobulin in Patients With PID Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Trial name
- NCT00419341
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration
- Where
- 13 sites in United States
- Volunteers
- 49 people
- Sponsor
- CSL Behring
Opioid Use Disorder, HIV Infections
Combination Primary Care and Prevention Services for Women Who Inject Drugs and Exchange Sex in Seattle, Washington
The investigators will implement a non-randomized observational clinical trial that will include a pop-up clinic for women who inject drugs (WWID) near venues for exchange sex and drug use in north Seattle.
- Conditions
- Opioid Use Disorder, HIV Infections, Chlamydia, Gonorrhea
- Trial name
- NCT06187064
- Testing
- Mobile Van Clinic
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- University of Washington
Ataxia Telangiectasia
Evaluate the Neurological Effects of EryDex on Subjects With A-T
This is an international, multi-center, randomized, prospective, double-blind, placebo-controlled, Phase 3 study, designed to assess the effect of EryDex (dexamethasone sodium phosphate [DSP] in autologous erythrocytes)…
- Trial name
- NEAT (NCT06193200)
- Testing
- Dexamethasone sodium phosphate
- Where
- 19 sites in United States, Denmark and 7 more
- Volunteers
- 105 people
- Sponsor
- Quince Therapeutics S.p.A.
Primary and Secondary Immunodeficiency and Other Conditions Requiring Regular Administration of Octagam 5% or 10% IVIG
Non-Interventional Study on the Tolerability and Efficacy of IVIG
Non-Interventional Study on the Tolerability and Efficacy of octagam® 10%
- Trial name
- GAM 10-06 (NCT02303093)
- Testing
- Octagam IVIG 5% or 10%, Panzyga
- Where
- 38 sites in Austria, Canada and 3 more
- Volunteers
- 344 people
- Sponsor
- Octapharma
Agammaglobulinemia, IgG Deficiency
Safety and Efficacy of Intravenous Immunoglobulin IgPro10 in Patients With Primary Immunodeficiencies (PID)
The objectives of this trial are the assessment of safety and efficacy of IgPro10 in patients with PID, and the assessment of tolerability of high infusion rates.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00322556
- Testing
- Immunoglobulins Intravenous (Human)
- Where
- 10 sites in United States
- Volunteers
- 55 people
- Sponsor
- CSL Behring
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant
This study will evaluate pediatric patients with malignant or non-malignant blood cell disorders who are having a blood stem cell transplant depleted of T cell receptor (TCR) alfa and beta cells that comes from a partially…
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndrome
- Trial name
- NCT02065869
- Testing
- BPX-501 T cells, Rimiducid
- Where
- 4 sites in Italy, United Kingdom
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Primary Immunodeficiency, PID
Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals…
- Trial name
- BOLT-BMT (NCT03330795)
- Testing
- CD3/CD19 neg allogeneic BMT
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
HIV Infections
Anti-HIV Drugs for Treating Infants Who Acquired HIV Infection at Birth
The purpose of this study is to compare the effects of anti-HIV drug courses of different lengths in infants who became HIV infected at birth.
- Trial name
- NCT00102960
- Testing
- Abacavir sulfate, Didanosine, Efavirenz
- Volunteers
- 377 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
HIV Infection
The Effect of Raltegravir on HIV Decay During Primary and Chronic Infection
The purpose of this study is to measure the decay characteristics of HIV in the blood of patients after taking a combination of anti-HIV drugs, which includes a new class of anti-HIV drug, an integrase inhibitor.
- Trial name
- PINT (NCT00641641)
- Testing
- Tenofovir + emtricitabine + raltegravir.
- Where
- 4 sites in Australia
- Volunteers
- 16 people
- Sponsor
- Kirby Institute
Primary Immune Deficiency Disorder
Study of Octanorm Subcutaneous IG in Patients With PID
This is an open-label phase III study with a 12-week wash-in/wash-out period followed by a 12-month efficacy period. The main goals of the study are to assess the efficacy of octanorm in preventing serious bacterial infections…
- Trial name
- NCT01888484
- Testing
- octanorm 16.5%
- Where
- 24 sites in United States, Canada and 5 more
- Volunteers
- 75 people
- Sponsor
- Octapharma
Hereditary Angioedema
A Phase II, Cross-over Clinical Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) in the On-demand Treatment of Angioedema Attacks in Adult Subjects With Hereditary Angioedema Type I or II
This study is a randomized, double-blind, placebo-controlled, phase II, cross-over clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adult subjects.
- Trial name
- NCT04208412
- Testing
- KVD900
- Where
- 25 sites in United States, Austria and 8 more
- Volunteers
- 84 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Hereditary Angioedema
A Study of Lanadelumab to Prevent Hereditary Angioedema (HAE) Attacks in Children
The main aims of this study are to learn how lanadelumab moves through a child's body and if the children have any medical problems from lanadelumab.
- Trial name
- SPRING (NCT04070326)
- Testing
- Lanadelumab
- Where
- 17 sites in United States, Canada and 3 more
- Volunteers
- 21 people
- Sponsor
- Shire
Hereditary Angioedema, HAE
A Long Term Safety Study of BCX7353 in Hereditary Angioedema
This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II Hereditary Angioedema (HAE).
- Conditions
- Hereditary Angioedema, HAE, Prophylaxis
- Trial name
- APeX-S (NCT03472040)
- Testing
- BCX7353
- Where
- 87 sites in United States, Australia and 18 more
- Volunteers
- 387 people
- Sponsor
- BioCryst Pharmaceuticals
Primary Immunodeficiency Diseases
A Study of NewGam, Human Immunoglobulin 10%, in Patients With Primary Immunodeficiency Diseases
The purpose of this study was to determine the efficacy of NewGam in preventing serious bacterial infections and to determine the pharmacokinetic profile of NewGam.
- Trial name
- NCT01012323
- Testing
- NewGam
- Where
- 7 sites in United States
- Volunteers
- 51 people
- Sponsor
- Octapharma
Hereditary Angioedema (HAE)
A Study of Icatibant for Acute Attacks of Hereditary Angioedema in Japanese Participants
The objective of this study is to evaluate the efficacy, pharmacokinetics (PK), and safety of icatibant for the treatment of acute attacks in Japanese participants with type I or type II hereditary angioedema (HAE).
- Trial name
- NCT03888755
- Testing
- Icatibant
- Where
- 8 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire