Clinical trials: Primary Immune Deficiency Disorders
- Condition Primary Immune Deficiency Disorders
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
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
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
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
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
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
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
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
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
Hereditary Angioedema, HAE
12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks
This study will evaluate the safety and efficacy of an oral treatment, BCX4161, in preventing acute attacks in participants with hereditary angioedema (HAE).
- Trial name
- OPuS-2 (NCT02303626)
- Testing
- BCX4161
- Where
- 40 sites in United States, Belgium and 6 more
- Volunteers
- 110 people
- Sponsor
- BioCryst Pharmaceuticals
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
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- CHANGE 3 (NCT00462709)
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 48 sites in United States
- Volunteers
- 146 people
- Sponsor
- Shire
Primary Immunodeficiency Diseases (PID)
A Study of TAK-771 in Japanese People With Primary Immunodeficiency Diseases (PID)
The main aim of the study is to check how much TAK-771 stays in their blood over time, side effect from the study treatment or TAK-771, how much TAK-771 participants can receive without getting side effects from it, and if…
- Trial name
- NCT05150340
- Testing
- TAK-771
- Where
- 12 sites in Japan
- Volunteers
- 16 people
- Sponsor
- Takeda
Primary Immune Deficiency Disorders (PIDD)
Open Label, Phase III Study of NABI-IGIV 10% [Immune Globulin Intravenous(Human), 10%] In Subjects With Primary Immune Deficiency Disorders (PIDD)
The purpose of this study is to determine if NABI-IGIV (10%) [Immune Globulin Intravenous (Human), 10%] is safe and effective in preventing serious bacterial infections (SBIs) in the treatment of patients with primary immune…
- Trial name
- NCT00538915
- Testing
- Nabi-IGIV 10% [Immune Globulin Intravenous (Human). 10%]
- Where
- 17 sites in United States
- Volunteers
- 63 people
- Sponsor
- ADMA Biologics, Inc.
Hereditary Angioedema, Hereditary Angioedema Type I
Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II
This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE).
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- HAE CHAPTER-1 (NCT05047185)
- Testing
- Deucrictibant low dose, Deucrictibant high dose
- Where
- 19 sites in United States, Austria and 7 more
- Volunteers
- 34 people
- Sponsor
- Pharvaris Netherlands B.V.
Hereditary Angioedema (HAE)
Study of C1 Inhibitor (Human) for the Prevention of Angioedema Attacks and Treatment of Breakthrough Attacks in Japanese Subjects With Hereditary Angioedema (HAE)
The purpose of this study is to determine if an investigational treatment is safe and well tolerated when administered by intravenous (IV) infusion in Japanese subjects with HAE.
- Trial name
- NCT02865720
- Testing
- CINRYZE 500 U, CINRYZE 1000 U
- Where
- 10 sites in Japan
- Volunteers
- 8 people
- Sponsor
- Shire
Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child
Long Term Follow-up Study for Patients Enrolled on the BP-004 Clinical Study
This is a long-term follow up study evaluating the safety of BPX-501 T cells (rivogenlecleucel) and infused in pediatric patients previously enrolled on the BP-004 study.
- Conditions
- Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child, Lymphoma, Non-Hodgkin, Myelodysplastic Syndromes
- Trial name
- NCT03733249
- Testing
- Rimiducid, rivogenlecleucel
- Where
- Italy
- Volunteers
- 187 people
- Sponsor
- Bellicum Pharmaceuticals
Meningococcal Disease
Safety and Immunogenicity of Novartis Meningococcal B Vaccine When Administered to Immunocompromised Children and Adolescents Compared to Healthy Subjects.
The study aims at evaluating the safety and immunogenicity of rMenB+OMV NZ when administered to subjects from 2 to 17 years of age with increased risk of meningococcal disease because either of primary or secondary complement…
- Trial name
- NCT02141516
- Testing
- rMenB+OMV
- Where
- 20 sites in Italy, Poland and 3 more
- Volunteers
- 239 people, healthy volunteers welcome
- Sponsor
- Novartis
Hereditary Angioedema
A Phase III, Crossover Trial Evaluating the Efficacy and Safety of KVD900 (Sebetralstat) for On-Demand Treatment of Angioedema Attacks in Adolescent and Adult Patients With Hereditary Angioedema (HAE)
This study is a randomized, double-blind, placebo-controlled, phase III, three-way crossover clinical trial evaluating the efficacy and safety of KVD900, in the treatment of hereditary angioedema attacks in adolescent and adult…
- Trial name
- NCT05259917
- Testing
- KVD900 600 mg, KVD900 300 mg
- Where
- 66 sites in United States, Australia and 19 more
- Volunteers
- 136 people
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Primary Immune Deficiency Disease
Safety and Efficacy Study of Flebogamma 5% DIF IGIV in Pediatric Subjects
This is a multi-center, open-label study to assess the efficacy and safety of Flebogamma 5% DIF in the pediatric population.
- Trial name
- NCT00634569
- Testing
- Flebogamma 5% DIF
- Where
- 7 sites in United States
- Volunteers
- 24 people
- Sponsor
- Instituto Grifols, S.A.