Clinical trials: Primary Immune Deficiency Disorder
DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
Thymus Transplantation in DiGeorge Syndrome #668
The study purpose is to determine whether cultured thymus tissue implantation (CTTI) is effective in treating typical complete DiGeorge syndrome.
- Trial name
- NCT00576407
- Testing
- Cultured Thymus Tissue for Implantation (CTTI)
- Where
- United States
- Volunteers
- 26 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Bronchiectasis, Cystic Fibrosis
Evaluating Bronchodilator Response in Patients With Bronchiectasis
Although patients with bronchiectasis tend to have non reversible obstructive patterns on pulmonary function tests (PFTs), reversible obstruction is not uncommon.
- Conditions
- Bronchiectasis, Cystic Fibrosis, Primary Ciliary Dyskinesia, Primary Immune Deficiency
- Trial name
- NCT05932316
- Testing
- spirometry, Salbutamol
- Where
- Israel
- Volunteers
- 96 people, healthy volunteers welcome
- Sponsor
- Rambam Health Care Campus
Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation
A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients
Treatment Study to assess of safety and efficiency of conditioning with Plerixafor and G-CSF as additional agents for prevention of graft failure after transplantation with TCR alpha/beta grafts depletion in patient with…
- Conditions
- Wiskott-Aldrich Syndrome, Hematopoietic Stem Cell Transplantation, Graft Failure
- Trial name
- NCT03019809
- Testing
- G-CSF for Conditioning before HSCT., Plerixafor for Conditioning before HSCT.
- Where
- Russia
- Volunteers
- 30 people
- Sponsor
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
Hereditary Angioedema
Patient Registry to Evaluate the Real-world Safety of Ruconest®
This is a prospective, real-world, observational patient registry for patients with HAE who are receiving treatment with Ruconest for HAE.
- Trial name
- NCT03697187
- Testing
- rhC1INH
- Where
- United States
- Volunteers
- 152 people
- Sponsor
- Pharming Technologies B.V.
Severe Combined Immunodeficiency
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study
This study will monitor the long-term effects of gene therapy in patients with severe combined immunodeficiency disease (SCID) due to a deficiency in an enzyme called adenosine deaminase (ADA).
- Trial name
- NCT00001255
- Testing
- ADA PBSC, ADA Umbilical Cord Blood Cells, Transduced Lymphocytes
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Hereditary Angioedema (HAE)
A Study of Takhzyro in Teenagers and Adults With Hereditary Angioedema (HAE) in South Korea
Takhzyro is an approved treatment for hereditary angioedema (HAE) in South Korea. HAE is a rare condition which causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals.
- Trial name
- NCT07445087
- Testing
- No intervention
- Where
- 3 sites in South Korea
- Volunteers
- 35 people
- Sponsor
- Takeda
Quality of Health Care, HIV/AIDS
Using Electronic Medical Record Data to Improve HIV Primary Care
This cluster randomized control trial examines whether an audit and feedback study improves care of patients living with HIV/AIDS in a family health team setting.
- Conditions
- Quality of Health Care, HIV/AIDS, Electronic Health Records
- Trial name
- NCT04107233
- Testing
- Audit and Feedback
- Where
- Canada
- Volunteers
- 13 people, healthy volunteers welcome
- Sponsor
- Unity Health Toronto
Ataxia Telangiectasia
International Ataxia Rating Scale in Younger Patients
The project will collect information on the mapping of clinical ratings on a number of scales that are used in the assessment of patients with ataxias.
- Trial name
- NCT01942850
- Where
- 5 sites in United States, India and 2 more
- Volunteers
- 63 people
- Sponsor
- Quince Therapeutics S.p.A.
Hereditary Angioedema (HAE)
A Pharmacokinetic, Tolerability and Safety Study of Icatibant in Children and Adolescents With Hereditary Angioedema
HGT-FIR-086 is a multicenter, open-label, non-randomized, single-arm study to evaluate the Pharmacokinetics, tolerability,safety, and efficacy on reproductive hormones, of a single subcutaneous (SC) administration of icatibant in…
- Trial name
- NCT01386658
- Testing
- icatibant
- Where
- 26 sites in United States, Australia and 8 more
- Volunteers
- 32 people
- Sponsor
- Shire
Primary Immune Deficiencies, Autoimmune Diseases
Investigation of Immune Disorders and Deficiencies
The immune system is an intricate system comprised of specialized cells, proteins, tissues and organs. Proper functioning is critical to the body's ability to defend itself against harmful pathogens.
- Conditions
- Primary Immune Deficiencies, Autoimmune Diseases, Inflammatory Diseases, Common Variable Immune Deficiencies
- Trial name
- NCT01981785
- Where
- United States
- Volunteers
- 343 people, healthy volunteers welcome
- Sponsor
- O & O Alpan LLC