Clinical trials: Deficiency Disease
Hereditary Angioedema
A Study to Evaluate the Safety and Effect of Escalating Doses of CINRYZE
The objectives of the study were: 1. To assess the safety and tolerability of escalating doses of CINRYZE. 2. To assess the effect of an escalating dose algorithm for CINRYZE on hereditary angioedema (HAE) attack rates. 3.
- Trial name
- NCT00914966
- Testing
- C1 inhibitor (human) [C1 INH]
- Where
- 11 sites in United States
- Volunteers
- 20 people
- Sponsor
- Shire
Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy
Open-Label Clinical Study of Droxidopa in Patients With Neurogenic Orthostatic Hypotension (NOH)
The purpose of this study is to assess the durability of effect of Droxidopa in treating symptoms of neurogenic orthostatic hypotension in patients with Primary Autonomic Failure (Pure Autonomic Failure, Multiple System Atrophy…
- Conditions
- Neurogenic Orthostatic Hypotension, Non-Diabetic Autonomic Neuropathy, Multiple System Atrophy, Dopamine Beta Hydroxylase Deficiency
- Trial name
- NOH303 (NCT00738062)
- Testing
- Droxidopa
- Where
- 54 sites in United States, Australia and 2 more
- Volunteers
- 103 people
- Sponsor
- Chelsea Therapeutics
HIV Infections
Cabenuva Injection Tracking in CHORUS
This is a cluster randomized trial in which clinics will be randomized to the intervention or the control arm. The purpose of this study is to assess if receiving alerts can help providers manage the scheduling of monthly…
- Trial name
- NCT04863261
- Testing
- Cabenuva Scheduling Alerts in the Retention & Huddle modules of the CHORUS App
- Where
- United States
- Volunteers
- 37 people
- Sponsor
- Epividian
Cytomegalovirus Retinitis, HIV Infections
The Safety and Effectiveness of ISIS 2922 in Patients With AIDS Who Have Cytomegalovirus (CMV) of the Eyes
To evaluate the efficacy and safety of ISIS 2922 in AIDS patients with Cytomegalovirus ( CMV ) retinitis who are unresponsive or intolerant to ganciclovir and/or foscarnet but are otherwise ineligible for ISIS Pharmaceuticals'…
- Trial name
- NCT00002356
- Testing
- Fomivirsen sodium
- Where
- 25 sites in United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
HIV, HIV Infections
Open-label Safety Study of E/C/F/TAF (Genvoya®) in HIV-1 Positive Patients With Mild to Moderate Renal Impairment
The primary objective of this study is to evaluate the effect of elvitegravir/cobicistat/emtricitabine/tenofovir alafenamide (E/C/F/TAF) fixed-dose combination (FDC) tablet on renal parameters at Week 24 in treatment-naive and…
- Trial name
- NCT01818596
- Testing
- E/C/F/TAF
- Where
- 70 sites in United States, Australia and 7 more
- Volunteers
- 252 people
- Sponsor
- Gilead Sciences
HIV Infections, Cytopenias
A Double-Blind, Placebo-Controlled Study With Open-Label Follow-Up To Determine the Safety and Efficacy of Subcutaneous Doses of r-HuEPO in AIDS Patients With Anemia Induced by Their Disease and AZT Therapy
To determine the safety and efficacy of erythropoietin administered subcutaneously to AIDS patients with anemia secondary to their disease and/or concomitant zidovudine (AZT) therapy.
- Trial name
- NCT00002073
- Testing
- Epoetin alfa
- Where
- United States
- Sponsor
- Ortho Pharmaceuticals
Primary Immunodeficiency Diseases
Study of BP-SCIG 20% in Patients With Primary Immunodeficiency (PID)
All patients will receive the investigational medicinal product BP-SCIG 20%, solution for subcutaneous administration, manufactured by BIOPHARMA PLASMA LLC.
- Trial name
- NCT07346859
- Testing
- BP-SCIG 20%
- Where
- 12 sites in Ukraine
- Volunteers
- 56 people
- Sponsor
- Biopharma Plasma LLC
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study
The purpose of this study is to evaluate the efficacy of ARALAST NP A1PI augmentation therapy 120 milligrams per kilogram (mg/kg) body weight (BW)/week compared with an external placebo comparator on the loss of emphysematous…
- Trial name
- NCT04440488
- Testing
- ARALAST NP
- Sponsor
- Shire
HIV-1
A Rollover Study For Subjects Discontinuing From UK-453,061 Studies For The Treatment Of HIV-1
The purpose of the protocol is to assess long-term safety and tolerability of subjects who discontinue for any reason from UK-453,061 qualifying studies.
- Trial name
- NCT00824369
- Testing
- No drug will be administered
- Where
- 27 sites in United States, Argentina and 10 more
- Volunteers
- 52 people
- Sponsor
- Pfizer
HIV Infections, Peripheral Neuropathy
Study of Pregabalin Versus Placebo in the Treatment of Nerve Pain Associated With HIV Neuropathy
Study to determine if pregabalin is more effective than placebo in treating subjects with nerve pain associated with HIV neuropathy.
- Trial name
- NCT00232141
- Testing
- pregabalin
- Where
- 44 sites in United States, Puerto Rico
- Volunteers
- 302 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Agammaglobulinemia, IgG Deficiency
Efficacy and Safety of Intravenous Immunoglobulin IVIG-F10 in Patients With Primary Immunodeficiencies (PID)
The purpose of this study is to demonstrate the effect of IVIG-F10 on the prevention of serious bacterial infections in patients with primary immunodeficiency.
- Conditions
- Agammaglobulinemia, IgG Deficiency, Common Variable Immunodeficiency
- Trial name
- NCT00168012
- Testing
- Immunoglobulins Intravenous (Human)
- Volunteers
- 42 people
- Sponsor
- CSL Behring
Alpha-Mannosidosis
Lamazym Aftercare Study FR Designed to Provide Treatment for French Patients
The overall objective of this trial is to provide aftercare treatment with Lamazym and to evaluate the safety of repeated Lamzede i.v. treatment of subjects with alpha-Mannosidosis.
- Trial name
- rhLAMAN-07 (NCT01908712)
- Testing
- Lamazym
- Where
- France
- Volunteers
- 13 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Human Immunodeficiency Virus Type 1
Study to Evaluate Efficacy and Safety of Darunavir/Cobicistat/Emtricitabine/Tenofovir Alafenamide (D/C/F/TAF) Regimen Versus Boosted Protease Inhibitor (bPI) Along With Emtricitabine/Tenofovir Disoproxil Fumarate (FTC/TDF) Regimen in Virologically-Suppressed, HIV-1 Infected Participants
The purpose of this study is to demonstrate non-inferiority in efficacy while switching to a once-daily single-tablet regimen containing darunavir (DRV)/ cobicistat (COBI)/ emtricitabine (FTC)/ tenofovir alafenamide (TAF)…
- Trial name
- NCT02269917
- Testing
- D/C/F/TAF, Boosted Protease Inhibitor (bPI), FTC/TDF
- Where
- 79 sites in United States, Belgium and 8 more
- Volunteers
- 1,149 people
- Sponsor
- Janssen R&D Ireland
HIV Infections
Phase II Study of AGS-004 as an Immunotherapeutic in Antiretroviral Therapy (ART)-Treated Subjects Infected With HIV
The purpose of this study is to examine the ability of AGS-004 to control HIV-1 replication and to determine if HIV-1 immunotherapy made with dendritic cells is safe and well tolerated, to determine if immunotherapy increases the…
- Trial name
- NCT00672191
- Testing
- AGS-004
- Where
- 12 sites in United States, Canada
- Volunteers
- 59 people
- Sponsor
- Argos Therapeutics
Bioequivalence
Bioequivalence Study of Metformin 1000 mg Tablets Immediate Release (IR) Versus Glucophage® 1000 mg Tablets IR In Healthy Subjects
Bioequivalence Study of 2 formulation of metformin (Metformin GEROPHARM vers. Glucophage® Merck )
- Trial name
- NCT03458208
- Testing
- Metformin, Glucophage®
- Where
- Russia
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Geropharm
HIV Infections
Effectiveness and Safety of Epivir/Ziagen/Zerit (3TC/ABC/d4T) Versus Epivir/Ziagen/Sustiva (3TC/ABC/EFV) Versus Epivir/Ziagen/Agenerase/Norvir (3TC/ABC/APV/RTV) in HIV Patients Who Have Never Received Treatment
The purpose of this study is to see how effective and safe it is to give 1 of the 3 following treatments to patients who may not have received anti-HIV treatment: 1) lamivudine (3TC)/abacavir (ABC)/stavudine (d4T); 2)…
- Trial name
- NCT00005017
- Testing
- Ritonavir, Abacavir sulfate, Amprenavir
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- Glaxo Wellcome
Cytomegalovirus Retinitis, HIV Infections
A Randomized Controlled Study of the Efficacy and Safety of Maintenance Treatment With Oral Ganciclovir for Newly Diagnosed Cytomegalovirus Retinitis in People With AIDS
To compare the time to progression of CMV retinitis between oral ganciclovir and IV ganciclovir during 20 weeks of maintenance treatment.
- Trial name
- NCT00002257
- Testing
- Ganciclovir
- Where
- 13 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- Hoffmann-La Roche
Idiopathic Pulmonary Fibrosis
23andMe IPF Research Study
The long term goal of this study is to increase genetic understanding of IPF to enable the development of an effective drug for IPF that can improve the lives of those living with the condition.
- Trial name
- IPF (NCT05028894)
- Testing
- No intervention
- Where
- United States
- Volunteers
- 690 people
- Sponsor
- 23andMe, Inc.
Pediatric Pyruvate Kinase Deficiency, Pediatric Hemolytic Anemia
A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period
Study ACTIVATE-Kids (AG348-C-023) will evaluate the efficacy and safety of orally administered mitapivat as compared with placebo in pediatric participants with pyruvate kinase deficiency (PKD) who are not regularly receiving…
- Trial name
- ACTIVATE-Kids (NCT05175105)
- Testing
- Mitapivat
- Where
- 19 sites in United States, Canada and 5 more
- Volunteers
- 30 people
- Sponsor
- Agios Pharmaceuticals, Inc.
15q Duplication Syndrome, CDKL5 Deficiency Disease
A Multicenter, Open-label, Pilot Study of Soticlestat (TAK-935/OV935) in Participants With 15Q Duplication Syndrome (Dup 15q) or Cyclin-Dependent Kinase-Like 5 (CDKL5) Deficiency Disorder (ARCADE STUDY)
The purpose of this study is to investigate the effect of soticlestat on the frequency of motor seizures for participants with Dup15q or CDD during the Maintenance Period.
- Trial name
- NCT03694275
- Testing
- Soticlestat
- Where
- 8 sites in United States
- Volunteers
- 20 people
- Sponsor
- Takeda
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.