Clinical trials: Primary Immune Deficiency Disorders
Hereditary Angioedema
Efficacy and Safety of GNR-038 vs Berinert® in Patients With Hereditary Angioedema
It is a placebo-controlled randomized trial to evaluate the efficacy and safety of GNR-038 in comparison with Berinert® in patients with hereditary angioedema
- Trial name
- NCT04898309
- Testing
- GNR-038, 50 МЕ/ kg, GNR-038, 100 МЕ/ kg, Berinert®, 20 МЕ/ kg
- Where
- 5 sites in Russia
- Sponsor
- AO GENERIUM
C1 Esterase Inhibitor Deficiency, Angio Edema
Biomarker for Hereditary AngioEdema Disease
International, multicenter, observational, longitudinal monitoring study to identify, validate and/or monitor Mass Spectrometry (MS)-based biomarker/s for Hereditary Angioedeme (HAE) disease and to test the clinical robustness…
- Conditions
- C1 Esterase Inhibitor Deficiency, Angio Edema, C4 Deficiency, HAE
- Trial name
- BioHAE (NCT03029728)
- Where
- 7 sites in Armenia, Georgia and 5 more
- Volunteers
- 42 people
- Sponsor
- CENTOGENE GmbH Rostock
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
Hereditary Angioedema, HAE
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
This is a Phase 1/2, single-arm, open-label, dose-escalation and dose-expansion study of BMN 331 for the treatment of hereditary angioedema (HAE) due to C1 Esterase Inhibitor (C1-INH) protein deficiency.
- Trial name
- HAErmony-1 (NCT05121376)
- Testing
- Dose 1 of BMN 331, Dose 2 of BMN 331, Dose 3 of BMN 331
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 44 people
- Sponsor
- BioMarin Pharmaceutical
Hereditary Angioedema
A Study of Icatibant in Patients With Acute Attacks of Hereditary Angioedema (FAST-3)
This study is being conducted to evaluate the efficacy and safety of icatibant compared to placebo in patients experiencing acute attacks of hereditary angioedema (HAE).
- Trial name
- NCT00912093
- Testing
- Icatibant
- Where
- 64 sites in United States, Australia and 7 more
- Volunteers
- 98 people
- Sponsor
- Shire
Primary Immune Deficiency
Retrospective Chart Review of Subcutaneous IgG Use in Infants
This study is a retrospective chart review of the use of subcutaneous IgG (SCIG) in infants less than two years old.
- Trial name
- NCT01166074
- Testing
- Subcutaneous IgG (SCIG)
- Where
- 5 sites in United States
- Volunteers
- 27 people
- Sponsor
- CSL Behring
Primary Immunodeficiency Diseases (PID)
Non-Interventional Post-Marketing Safety Study on the Long-Term Safety of HYQVIA (Global)
The purpose of the proposed study is to acquire additional data (including the assessment of anti-rHuPH20 antibodies) on the long-term safety of HYQVIA and to assess the prescribed treatment regimens and treatment administration…
- Trial name
- NCT02593188
- Testing
- HYQVIA
- Where
- 30 sites in United States
- Volunteers
- 264 people
- Sponsor
- Baxalta now part of Shire
Hereditary Angioedema
C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks
This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and…
- Trial name
- NCT01397864
- Testing
- rhC1INH or pdC1INH
- Where
- 17 sites in Bulgaria, Croatia and 11 more
- Volunteers
- 181 people
- Sponsor
- Pharming Technologies B.V.
Hereditary Angioedema (HAE)
Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers.
- Trial name
- NCT02663687
- Testing
- Recombinant human C1 esterase inhibitor, SHP623
- Where
- United States
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Shire
Hereditary Angioedema (HAE)
EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
- Trial name
- NCT01826916
- Testing
- DX-88 (ecallantide)
- Volunteers
- 77 people
- Sponsor
- Shire
Primary Immune Deficiency Disorder
Follow-up Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy (Japan Study)
The objective of this study is to assess the long-term safety, tolerability, and efficacy of IgPro20 in subjects with primary immunodeficiency (PID) as a follow-up to the pivotal study ZLB06_002CR (NCT01199705).
- Trial name
- NCT01458171
- Testing
- Immune globulin subcutaneous (Human)
- Where
- 9 sites in Japan
- Volunteers
- 23 people
- Sponsor
- CSL Behring
Primary Immunodeficiencies, Inborn Errors of Metabolism
Treosulfan-based Versus Busulfan-based Conditioning in Paediatric Patients With Non-malignant Diseases
The aim of the trial is to describe the safety and efficacy of intravenous (i.v.) Treosulfan compared to the conventional (myeloablative) dose of i.v.
- Conditions
- Primary Immunodeficiencies, Inborn Errors of Metabolism, Haemoglobinopathies, Bone Marrow Failure Syndromes
- Trial name
- NCT02349906
- Testing
- Treosulfan, Busilvex
- Where
- 21 sites in Czechia, Germany and 2 more
- Volunteers
- 106 people
- Sponsor
- medac GmbH
Hereditary Angioedema (HAE)
FIRAZYR General Drug Use-Results Survey (Japan)
The objectives of this survey are to collect data to report the safety and efficacy of Firazyr (Icatibant acetate) in the post-marketing phase in participants diagnosed with Hereditary Angioedema (HAE).
- Trial name
- NCT04057131
- Testing
- Firazyr
- Where
- 18 sites in Japan
- Volunteers
- 179 people
- Sponsor
- Shire
Chronic Lymphocytic Leukemia, Hypogammaglobulinemia
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)
Study Evaluating Efficacy and Safety of Panzyga in Primary Infection Prophylaxis in Patients with Chronic Lymphocytic Leukemia
- Trial name
- NCT04502030
- Testing
- Panzyga
- Where
- 84 sites in United States, Czechia and 10 more
- Volunteers
- 247 people
- Sponsor
- Octapharma
Cancer, Metastatic, Cancer
SYNERGY-AI: Artificial Intelligence Based Precision Oncology Clinical Trial Matching and Registry
International registry for cancer patients evaluating the feasibility and clinical utility of an Artificial Intelligence-based precision oncology clinical trial matching tool, powered by a virtual tumor boards (VTB) program, and…
- Conditions
- Cancer, Metastatic, Cancer, Cancer of Pancreas, Cancer of Liver
- Trial name
- NCT03452774
- Testing
- Clinical Trial Matching
- Where
- 68 sites in United States, Puerto Rico
- Volunteers
- 50,000 people
- Sponsor
- Massive Bio, Inc.
Hereditary Angioedema
NTLA-2002 in Adults With Hereditary Angioedema (HAE)
This study will be conducted to evaluate the safety, tolerability, activity, pharmacokinetics, and pharmacodynamics of NTLA-2002 in adults with Hereditary Angioedema (HAE).
- Trial name
- NTLA-2002 (NCT05120830)
- Testing
- Biological NTLA-2002, Normal Saline IV Administration
- Where
- 9 sites in Australia, France and 4 more
- Volunteers
- 37 people
- Sponsor
- Intellia Therapeutics
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
Hereditary Angioedema (HAE)
A Study of Lanadelumab (Takhzyro) and Icatibant (Firazyr®) in Persons With HAE in China
The Chinese health authority has approved lanadelumab to prevent Hereditary Angioedema (HAE) attacks in persons of 12 years and older. It has also approved icatibant to treat acute HAE attacks in persons 2 years and older.
- Trial name
- NCT06346899
- Testing
- No intervention
- Where
- 13 sites in China
- Volunteers
- 115 people
- Sponsor
- Takeda
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
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.