Clinical trials: Deficiency Disease
Hereditary Angioedema (HAE)
Efficacy and Safety Study of DX-2930 to Prevent Acute Angioedema Attacks in Patients With Type I and Type II HAE
This is a phase 3, multicenter, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of DX-2930 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
- Trial name
- NCT02586805
- Testing
- DX-2930 - 300mg/2wk, DX-2930 - 300mg/4wk, DX-2930 - 150mg/4wk
- Where
- 41 sites in United States, Canada and 5 more
- Volunteers
- 125 people
- Sponsor
- Shire
Vaccine Response
Probiotics and Influenza Vaccination Response
Assess efficacy of oral ingestion of a probiotic product on immune function in a population of healthy adult men and women in a clinical vaccination study.
- Trial name
- VERB (NCT05690373)
- Testing
- Probiotic effect on influenza vaccine response
- Where
- Netherlands
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- The Archer-Daniels-Midland Company
AIDS
Evaluation of the Immune Response of a HIV Candidate Vaccine After Administration of One Chloroquine Dose
The purpose of this study is to evaluate the safety and reactogenicity of one booster dose of a HIV candidate vaccine after administration of one oral dose of chloroquine.
- Trial name
- NCT00972725
- Testing
- GSK Biologicals' HIV vaccine (732461), Chloroquine
- Where
- Belgium
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Limbal Stem Cell Deficiency (LSCD)
BRM421 Ophthalmic Solution in Patients With Limbal Stem Cell Deficiency
The goal of this program is to provide patients with Limbal Stem Cell Deficiency access to the BRM421 eye drops. Participants will: Use BRM421 two times a day for at least 12 weeks Visit the clinic monthly for checkups and tests
- Trial name
- NCT07422389
- Testing
- BRM421 Ophthalmic Solution, 0.06%
- Where
- United States
- Sponsor
- BRIM Biotechnology Inc.
Relapsed, Diffuse Large B-cell Lymphoma, Refractory Diffuse Large B-Cell Lymphoma
Study of Betalutin for Treatment of Relapsed or Refractory Non-Hodgkin Lymphoma (LYMRIT-37-05)
This study is a phase 1, dose finding, open-label study in patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL).
- Trial name
- NCT02658968
- Testing
- Betalutin
- Where
- 10 sites in United States, Germany and 3 more
- Volunteers
- 18 people
- Sponsor
- Nordic Nanovector
Healthy Volunteers, Growth Hormone Deficiency
A Study to Compare the Uptake Into the Blood of Two Strengths of Somapacitan After Injection Under the Skin in Healthy Subjects
This study will compare two strengths of the new long-acting growth hormone somapacitan. The aim of this study is to test if both strengths are taken up in the blood in the same way.
- Trial name
- NCT03905850
- Testing
- somapacitan
- Where
- Germany
- Volunteers
- 33 people, healthy volunteers welcome
- Sponsor
- Novo Nordisk A/S
NLRC4-GOF, AIFEC (Autoinflammation With Infantile Enterocolitis), XIAP Deficiency, CDC42 Mutations
Study to Evaluate the Efficacy, Safety and Tolerability of MAS825 in Patients With Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations
This study is a Phase 2 trial designed to evaluate the clinical efficacy, safety, and tolerability of MAS825 in patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations.
- Trial name
- MASter-1 (NCT04641442)
- Testing
- MAS825
- Where
- 12 sites in United States, Czechia and 5 more
- Volunteers
- 17 people
- Sponsor
- Novartis Pharmaceuticals
HIV Infections
Study to Evaluate Pharmacokinetic (PK), Safety and Tolerability of Cabotegravir (CAB) 400 Milligrams Per Milliliter (mg/mL) Formulation in Healthy Adult Participants
This is an active control, randomized study to investigate the safety, tolerability and PK of repeat dose administration of long-acting CAB 400 mg/mL formulation intramuscular (IM) (gluteus medius and vastus lateralis) and…
- Trial name
- NCT04484337
- Testing
- Cabotegravir sodium (Oral Lead In), Cabotegravir 400 mg/mL, Cabotegravir 200 mg/mL
- Where
- 5 sites in United States, New Zealand
- Volunteers
- 138 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
RPS19 Deficient Diamond-Blackfan Anemia
Assessing the Safety, Tolerability, and Efficacy of APR-2020 in Pediatric and Adolescent Subjects With RPS19 Deficient Diamond-Blackfan Anemia
Brief summary The goal of this clinical trial is to learn if APR-2020 is safe and can help treat Diamond-Blackfan Anemia (DBA) in adolescents and children.
- Trial name
- NCT07476183
- Testing
- APR-2020
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Apriligen, Inc.
Meningitis, Cryptococcal, HIV Infections
A Study of Fluconazole in the Treatment of Cryptococcal Meningitis in Patients With AIDS
To compare the safety and effectiveness of fluconazole with that of placebo as maintenance treatment for preventing the relapse of cryptococcal meningitis in patients with AIDS.
- Trial name
- NCT00002294
- Testing
- Fluconazole
- Where
- 5 sites in United States
- Sponsor
- Pfizer
Human Immunodeficiency Virus Type 1
A Roll-over Study With Rilpivirine for Human Immunodeficiency Virus Type 1 (HIV-1) Infected Participants Who Participated in Rilpivirine Pediatric Studies
The purpose of this study is to provide continued access to rilpivirine (RPV) for participants who were treated with RPV in a clinical development pediatric study with rilpivirine and who, at the time of roll-over, experience and…
- Trial name
- NCT02494986
- Testing
- Rilpivirine
- Where
- 8 sites in Portugal, South Africa and 3 more
- Volunteers
- 48 people
- Sponsor
- Janssen Sciences Ireland UC
Eyes Dry Chronic, Chronic Conjunctivitis of Both Eyes
CDL Validation Study
Ocular surface photography is significantly limited in standardization and reproducibility. This reduces its applicability for clinical monitoring of acute or chronic disease.
- Conditions
- Eyes Dry Chronic, Chronic Conjunctivitis of Both Eyes, Limbal Stem-cell Deficiency, Eye Lesion
- Trial name
- NCT05461469
- Where
- Austria
- Volunteers
- 104 people
- Sponsor
- Occyo GmbH
HIV Infections
A Randomized Phase II Efficacy, Activity and Safety Study of GLQ223 Alone and in Combination With Zidovudine in Symptomatic HIV-Infected Patients Without Prior Treatment With GLQ223 or Trichosanthin
This study involves GLQ223 administration to patients who have not previously been exposed to it, but who have received at least 9 months of zidovudine therapy.
- Trial name
- NCT00001989
- Testing
- Trichosanthin, Zidovudine
- Where
- 8 sites in United States
- Sponsor
- Genelabs Technologies
HIV Infections
A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of a New Formulation of Cabotegravir Long-Acting Administered Intramuscularly in a 4-month Dosing Interval (Q4M)
This study will evaluate the pharmacokinetics (PK), safety, and tolerability of a new formulation of Cabotegravir (CAB) dosed every 4-months (Q4M) for pre-exposure prophylaxis (PrEP) in participants at risk of HIV-1 acquisition.
- Trial name
- NCT06741397
- Testing
- CAB LA, New formulation of CAB LA
- Where
- 27 sites in United States, Puerto Rico
- Volunteers
- 229 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
HIV Infections
Tipranavir and Ritonavir vs. Saquinavir and Ritonavir Used With Two Nucleoside Reverse Transcriptase Inhibitors in Single Protease Inhibitor-experienced HIV-1 Patients
Objectives of the study were to evaluate the efficacy and safety of two different doses of tipranavir (TPV) in combination with ritonavir (TPV/r) compared with a standard dual PI combination of saquinavir (SQV) and ritonavir…
- Trial name
- NCT02239835
- Testing
- Tipranavir (TPV) low dose, Tipranavir (TPV) high dose, Ritonavir low dose
- Volunteers
- 79 people
- Sponsor
- Boehringer Ingelheim
SARS-CoV 2, COVID
In-vitro Diagnostic Test to Predict COVID-19 Mortality and Disease Severity
The COVID-19 Androgen Sensitivity Test is a non-invasive In-Vitro Diagnostic device that utilizes Next Generation Sequencing Technology (NGS).
- Conditions
- SARS-CoV 2, COVID, COVID-19, Androgenetic Alopecia
- Trial name
- NCT04368897
- Testing
- CAG length <22, CAG length >=22
- Where
- Spain
- Sponsor
- Applied Biology, Inc.
Ataxia Telangiectasia
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
This is a multinational, multicenter, open-label, rater-blinded prospective Phase II study which will assess the safety and efficacy of N-Acetyl-L-Leucine (IB1001) for the treatment of Ataxia-Telangiectasia (A-T).
- Trial name
- NCT03759678
- Testing
- IB1001
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 17 people
- Sponsor
- IntraBio Inc
HIV Infections
Single-Blind, Controlled Safety and Immunogenicity Study of Recombinant MVA Virus to Treat HIV Infection
At the end of 2004 there were more than 40 million people infected Worldwide with HIV, with an estimated 16,000 new infections every day (UNAIDS, 2004).
- Trial name
- NCT00390078
- Testing
- MVA-mBN32, IMVAMUNE
- Where
- Germany
- Volunteers
- 30 people
- Sponsor
- Bavarian Nordic
Thymidine Kinase 2 (TK2)
A RETROspective Study of Patients With TK2d
This is a medical chart review study to collect information for patients who have been taking pyrimidine nucleosides for treatment of TK2 deficiency.
- Trial name
- RETRO (NCT03701568)
- Testing
- dC/dT
- Where
- 8 sites in United States, Israel and 1 more
- Volunteers
- 38 people
- Sponsor
- Zogenix MDS, Inc.
HIV Infections
Low-dose Fostemsavir Extended Release Relative Bioavailability Study
This is a two-part study. Part 1 will evaluate relative bioavailability of temsavir (TMR) following single dose administration of the reference fostemsavir (FTR) compared to two low-dose ER tablet formulations of FTR.
- Trial name
- NCT04757974
- Testing
- Fostemsavir 600 mg, Fostemsavir 200 mg
- Where
- United States
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- ViiV Healthcare
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