Clinical trials: Primary Immune Deficiency
Hereditary Angioedema
Treatment of Angioedema Attacks in Adolescent and Adult Patients 12 Years and Older With HAE Type I or II With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Type I or II Hereditary Angioedema (HAE) adolescent and adult post-trial and…
- Trial name
- NCT06628713
- Testing
- Sebetralstat
- Where
- United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
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)
Efficacy and Safety of Lanadelumab (SHP643) in Japanese Participants With Hereditary Angioedema (HAE)
The purpose of this phase 3, open-label, multi-center study is to evaluate the safety and efficacy of lanadelumab in Japanese participants with HAE Type I or II.
- Trial name
- NCT04180163
- Testing
- Lanadelumab
- Where
- 12 sites in Japan
- Volunteers
- 12 people
- Sponsor
- Shire
Hereditary Angioedema (HAE)
Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.
- Trial name
- NCT00457015
- Testing
- ecallantide, Phosphate Buffer Saline (PBS), pH 7.0
- Where
- 45 sites in United States, Canada and 1 more
- Volunteers
- 96 people
- Sponsor
- Shire
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
Hereditary Angioedema
A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema
The purpose of this study was to evaluate the clinical efficacy, safety, and tolerability of donidalorsen in participants with hereditary angioedema (HAE) type 1 (HAE-1), HAE type 2 (HAE-2), or HAE with normal C1-inhibitor…
- Trial name
- NCT04030598
- Testing
- Donidalorsen
- Where
- 8 sites in United States, Netherlands
- Volunteers
- 23 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Hereditary Angioedema (HAE)
A Study About the Diagnosis and Management of Hereditary Angioedema (HAE) in Egypt
Hereditary angioedema (HAE) is a rare condition. It causes sudden swelling under the skin and inside the body, like in the belly, throat, or genitals. This swelling happens because of a temporary leak in blood vessels.
- Trial name
- EXPLORHAE (NCT07218393)
- Testing
- No intervention
- Where
- 7 sites in Egypt
- Volunteers
- 100 people
- Sponsor
- Takeda
Primary Immunodeficiency, Agammaglobulinemia
Efficacy and Safety Study of Kedrion IVIG 10% to Treat Subjects With Primary Immunodeficiency (PID)
The purpose of this study is to determine whether Kedrion IVIG 10% (an immunoglobulin solution) is effective in treating Primary Immunodeficiency (PID).
- Conditions
- Primary Immunodeficiency, Agammaglobulinemia, Hypogammaglobulinemia, Antibody Deficiency
- Trial name
- NCT01581593
- Testing
- Kedrion IVIG 10%
- Where
- 15 sites in United States, Canada
- Volunteers
- 45 people
- Sponsor
- Kedrion S.p.A.
Hereditary Angioedema, HAE
Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of oral BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- APeX-2 (NCT03485911)
- Testing
- BCX7353 capsules
- Where
- 47 sites in United States, Austria and 9 more
- Volunteers
- 121 people
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema (HAE)
CSL312_3003 Safety and Pharmacokinetic Study in Subjects 2 to 11 Years of Age With Hereditary Angioedema
The purpose of this study is to investigate the safety, PK / PD, and efficacy of SC CSL312 for prophylactic treatment of pediatric subjects with HAE.
- Trial name
- NCT05819775
- Testing
- CSL312
- Where
- 13 sites in United States, Australia and 3 more
- Volunteers
- 22 people
- Sponsor
- CSL Behring
Primary Immunodeficiency Disorders (PIDs)
Leniolisib for Immune Dysregulation in PIDs
This study is an exploratory, non-randomized, open-label, within-patient dose escalation study. The primary objective is to assess safety and tolerability of leniolisib.
- Trial name
- NCT06549114
- Testing
- Leniolisib
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Pharming Technologies B.V.
Hereditary Angioedema
Safety Study in Subjects ≥ 12 Years of Age With Hereditary Angioedema Switching to Garadacimab
This study is designed to evaluate the safety after switching to garadacimab from another prophylactic hereditary angioedema (HAE) treatment (marketed kallikrein [KK] inhibitor or plasma-derived C1-esterase inhibitor…
- Trial name
- NCT06806657
- Testing
- Garadacimab
- Where
- 11 sites in United States, Canada and 1 more
- Volunteers
- 18 people
- Sponsor
- CSL Behring
Primary Immunodeficiency Disease
High Infusion Rate Study of Immunoglobulin Intravenous (Human) 10% (NewGam)
This was a prospective, open-label, non-controlled, non-randomized multicenter Phase III study of 2 multiple-dose intravenous NewGam regimens (every 3 weeks or every 4 weeks, continuing the patient's infusion interval in the main…
- Trial name
- NGAM-05 (NCT01313507)
- Testing
- NewGam
- Where
- 6 sites in United States
- Volunteers
- 21 people
- Sponsor
- Octapharma
Primary Immune Deficiency Disorder
Octagam 5% Versus Comparator Post Marketing Trial
Study Design: This is a 2 arm non-interventional trial that will compare the occurrence of adverse drug reactions between Octagam 5% and other marketed IVIG infusion treatments.
- Trial name
- NCT01859754
- Testing
- Octagam 5%, Other marketed IVIG product
- Where
- 27 sites in United States
- Volunteers
- 623 people
- Sponsor
- Octapharma
Hereditary Angioedema
A Drug-Drug Interaction Study to Evaluate Drug Transporter Interactions
This is an open-label, three part study to evaluate the effect of BCX7353 on drug transporters as well as the effect of an inhibitor of drug transport on BCX7353.
- Trial name
- NCT03136237
- Testing
- BCX7353, Digoxin, BCX7353 + digoxin
- Where
- United Kingdom
- Volunteers
- 54 people, healthy volunteers welcome
- Sponsor
- BioCryst Pharmaceuticals
Hereditary Angioedema
Treatment of Angioedema Attacks in Pediatric (Ages 2-11) Post-Trial and Naive Patients With HAE With Sebetralstat
The sebetralstat Early Access Program (EAP) provides early access to the investigational medicinal product (IMP) sebetralstat to eligible and approved Hereditary Angioedema (HAE) pediatric (ages 2-11) post-trial and naïve…
- Trial name
- NCT07216378
- Testing
- Sebetralstat
- Where
- 6 sites in United States
- Sponsor
- KalVista Pharmaceuticals, Ltd.
Pain
Evaluation of the Tolerability of Switching Subjects on Chronic ATC Opioid Therapy to Buprenorphine HCl Buccal Film
The primary aim of this study is to determine if chronic pain subjects on around-the-clock opioids who are receiving 100 to 220 mg oral morphine sulfate equivalent (MSE) can be safely transitioned on to buprenorphine…
- Trial name
- NCT01871285
- Testing
- Buprenorphine, Oxycodone, Morphine sulfate
- Where
- 3 sites in United States
- Volunteers
- 39 people
- Sponsor
- BioDelivery Sciences International