Clinical trials: Deficiency Disease
- Condition Deficiency Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Primary Immune Deficiency
Safety Study of Subcutaneous Ig NextGen 16% in Patients With Primary Immunodeficiency
The study aims to assess the safety and tolerability of subcutaneous Ig NextGen 16% in patients with Primary Immune Deficiency who require Immunoglobulin (Ig) G replacement therapy.
- Trial name
- NCT00680446
- Testing
- Immunoglobulin G (Ig NextGen 16%)
- Where
- 9 sites in Australia, New Zealand
- Volunteers
- 41 people
- Sponsor
- CSL Limited
Pneumonia, Pneumocystis Carinii, HIV Infections
A Compassionate Treatment Protocol for the Use of Trimetrexate Glucuronate With Leucovorin Protection for Patients With Pneumocystis Carinii Pneumonia.
To provide trimetrexate glucuronate to immunosuppressed patients with Pneumocystis carinii pneumonia (PCP) for whom this investigational compound could provide significant medical benefit.
- Trial name
- NCT00002102
- Testing
- Trimetrexate glucuronate, Leucovorin calcium
- Where
- United States
- Sponsor
- U.S. Bioscience
HIV Infection
An Expanded Access Study of Oral Liquid Efavirenz in the Treatment of Children With HIV Infection: Canada
This study is being conducted to assess the safety and tolerability of an oral liquid solution of Sustiva for antiretroviral therapy-naive or therapy-experienced HIV-1 infected children between the ages of 3-16 who are failing or…
- Trial name
- NCT00162188
- Testing
- Efavirenz
- Where
- 4 sites in Canada
- Sponsor
- Bristol-Myers Squibb
Essential Fatty Acid Deficiency (EFAD)
Pediatric Study to Evaluate Risk of Developing Essential Fatty Acid Deficiency When Receiving Clinolipid or Standard-of-Care Lipid Emulsion
This will be a descriptive study designed to evaluate the propensity for hospitalized pediatric patients treated adequately with Clinolipid or standard of care for up to 90 days to develop Essential Fatty Acid Deficiency (EFAD).
- Trial name
- NCT02476994
- Testing
- Clinolipid, Intralipid
- Where
- 4 sites in United States
- Volunteers
- 1 people
- Sponsor
- Baxter Healthcare Corporation
HIV Infections
The Safety and Effectiveness of Two Forms of Saquinavir Combined With Other Anti-HIV Drugs in HIV-Infected Infants and Children
To determine the pharmacokinetic properties, tolerance and safety of 2 formulations of saquinavir, given in combination with 2 nucleoside antiretroviral drugs (part 1) or in combination with nelfinavir and nucleoside…
- Trial name
- NCT00002380
- Testing
- Nelfinavir mesylate, Saquinavir
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Hoffmann-La Roche
Intestinal Failure-associated Liver Disease
Prevalence of Liver Disease in Patients Dependent on Parenteral Nutrition
This is a multi-center prospective cross-sectional observational study that will assess the prevalence of liver disease in patients dependent on parenteral nutrition (PN) for 4 or more days per week.
- Trial name
- THRIVE-1 (NCT05011370)
- Where
- 15 sites in United States, Denmark and 2 more
- Volunteers
- 80 people
- Sponsor
- Protara Therapeutics
Primary Immunodeficiency (PID)
Subcutaneous Ig NextGen 16% in PID Patients
This study aims to assess the safety, tolerability, efficacy and pharmacokinetics of Ig NextGen 16% in people with antibody deficiency currently being treated with IntragamP.
- Trial name
- NCT00391131
- Testing
- IgNextGen 16%
- Where
- 12 sites in Australia, New Zealand
- Volunteers
- 35 people
- Sponsor
- CSL Limited
HIV Infections
Safety and Effectiveness of Giving Adefovir Dipivoxil Plus Abacavir Plus Efavirenz Plus Amprenavir to HIV-Infected Patients Who Have Failed to Respond to Previous Protease Inhibitor Treatment
The purpose of this study is to see if it is safe and effective to give adefovir dipivoxil plus abacavir (ABC) plus efavirenz (EFV) plus amprenavir (APV) to HIV-infected patients who have failed to respond to previous treatment…
- Trial name
- NCT00002419
- Testing
- Abacavir sulfate, Amprenavir, Efavirenz
- Where
- 5 sites in United States
- Volunteers
- 25 people
- Sponsor
- Gilead Sciences
Growth Hormone Deficiency
A Long-Term Safety Study of Somavaratan in Japanese Children With Growth Hormone Deficiency
This study is a multi-center, open-label safety study assessing long-term somavaratan administration.
- Trial name
- NCT03145831
- Testing
- Somavaratan
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Versartis Inc.
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.
HIV Infections
A Study of Amprenavir in HIV-Infected Patients
The purpose of this study is to see if it is safe to give amprenavir (APV) to HIV-infected patients. This study also examines the effect APV has on the level of HIV in the blood.
- Trial name
- NCT00002405
- Testing
- Amprenavir
- Where
- United States
- Sponsor
- Glaxo Wellcome
Hereditary Angioedema
C1 Esterase Inhibitor in Hereditary Angioedema (HAE)(Extension Study)
Hereditary angioedema (HAE) is a rare disorder characterized by congenital lack of functional C1 esterase inhibitor. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities…
- Trial name
- NCT00292981
- Testing
- C1 Esterase Inhibitor
- Where
- 14 sites in United States, Canada
- Volunteers
- 57 people
- Sponsor
- CSL Behring
Growth Hormone Disorder, Pituitary Dwarfism
Somatropin (Norditropin) in Insulin-like Growth Factor (IGF) Deficient Children
This trial is conducted in the United States of America (USA). This is a 12 month study to determine if Norditropin is safe and effective in children with IGF deficiency.
- Trial name
- NCT00102817
- Testing
- somatropin
- Where
- 27 sites in United States
- Volunteers
- 149 people
- Sponsor
- Novo Nordisk A/S
Pneumonia, Pneumocystis Carinii, HIV Infections
A Comparison of Atovaquone and Pentamidine in the Prevention of Pneumocystis Carinii Pneumonia in HIV-Infected Patients Who Cannot Take TMP/SMX
To assess whether high dose or low dose atovaquone suspension is more effective than aerosolized pentamidine as prophylaxis against Pneumocystis carinii pneumonia (PCP) in high-risk HIV-infected patients.
- Trial name
- NCT00002340
- Testing
- Atovaquone, Pentamidine isethionate
- Where
- 5 sites in United States
- Volunteers
- 615 people
- Sponsor
- Glaxo Wellcome
HIV Infections
A Study of Ritonavir/Indinavir Combination in HIV-Infected Patients
The purpose of this study is to determine how many HIV-infected patients continue taking ritonavir/indinavir combination after having taken indinavir three times a day as part of their anti-HIV drug therapy.
- Trial name
- NCT00002223
- Testing
- Indinavir sulfate, Ritonavir
- Where
- 6 sites in United States
- Sponsor
- Abbott
Meningitis, Cryptococcal, HIV Infections
A Study of Fluconazole in the Treatment of Cryptococcal Meningitis in Patients Who Have Not Had Success With Amphotericin B
To evaluate the safety and effectiveness of fluconazole as treatment for acute cryptococcal meningitis in patients who have had an unsatisfactory response to or have experienced unacceptable toxicity with amphotericin B.
- Trial name
- NCT00002306
- Testing
- Fluconazole
- Where
- 14 sites in United States
- Sponsor
- Pfizer
Hereditary Angioedema, Hereditary Angioedema Type I
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand…
- Conditions
- Hereditary Angioedema, Hereditary Angioedema Type I, Hereditary Angioedema Type II, Hereditary Angioedema Types I and II
- Trial name
- RAPIDe-3 (NCT06343779)
- Where
- 76 sites in United States, Argentina and 27 more
- Volunteers
- 134 people
- Sponsor
- Pharvaris Netherlands B.V.
HIV Infections
An Open-Label Safety Program for the Use of Zalcitabine (Dideoxycytidine; ddC) in Pediatric Patients With Symptomatic HIV Infection Who Have Failed or Are Intolerant to AZT Monotherapy, or Who Have Completed Other ddC Protocols, or Are Ineligible for Other Ongoing Clinical Studies
To allow, on a compassionate use basis, zalcitabine (ddC) for pediatric patients with symptomatic HIV disease who have failed treatment or who are intolerant to zidovudine (AZT), or who have completed other ddC protocols, or who…
- Trial name
- NCT00002118
- Testing
- Zalcitabine
- Where
- United States
- Volunteers
- 600 people
- Sponsor
- Hoffmann-La Roche
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
HIV Infections
Clinical Performance of Reveal G4 Rapid HIV-1 Antibody Test
To determine the efficacy of an improved rapid diagnostic test using venous whole blood and fingerstick whole blood. The clinical performance of Reveal G4 Rapid HIV-1 Antibody Test will be determined by comparing the results with…
- Trial name
- NCT02190578
- Testing
- Reveal G4
- Where
- 4 sites in United States
- Volunteers
- 1,649 people, healthy volunteers welcome
- Sponsor
- MedMira Laboratories Inc.