Clinical trials: Deficiency Disease
HIV Infections, Chickenpox
A Comparison of 882C87 Versus Acyclovir in the Treatment of Herpes Zoster in Patients With Weakened Immune Systems
To determine the efficacy of oral 882C87 compared with oral acyclovir in the treatment of localized herpes zoster in immunocompromised patients. To assess the safety and tolerance of oral 882C87 in immunocompromised patients.
- Trial name
- NCT00002315
- Testing
- Netivudine, Acyclovir
- Where
- 25 sites in United States
- Volunteers
- 400 people
- Sponsor
- Glaxo Wellcome
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.
Colorectal Cancer
Efficacy and Safety of Tislelizumab (BGB-A317) as Neo-Adjuvant Treatment in Participants With Colorectal Cancer
The main purpose of this study was to help meet the medical needs of people in China with certain types of solid tumors that have specific genetic changes called microsatellite instability-high (MSI-H) or deficient mismatch…
- Trial name
- NCT05116085
- Testing
- Tislelizumab
- Where
- 8 sites in China
- Volunteers
- 33 people
- Sponsor
- BeiGene
Methylmalonic Acidemia (MMA), Metabolism, Inborn Errors
Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia
This First-in-Human (FIH) Phase 1/2 study will evaluate mRNA-3704 in patients with methylmalonic acidemia/aciduria (MMA) due to methylmalonyl-coenzyme A mutase (MUT) deficiency between 1 to 18 years of age with elevated plasma…
- Trial name
- NCT03810690
- Testing
- mRNA-3704
- Sponsor
- ModernaTX, Inc.
HIV Infection, Infection, Human Immunodeficiency Virus
KALETRA Or LEXIVA With Ritonavir Combined With EPIVIR And Abacavir In Naive Subjects Over 48 Weeks
This study will compare the ability of fosamprenavir 700 mg with ritonavir 100 mg twice a day or lopinavir 400 mg with ritonavir 100 mg twice a day both combined with a fixed dose combination tablet of abacavir 600 mg and…
- Trial name
- NCT00085943
- Testing
- LEXIVA (GW433908), Ritonavir, KALETRA
- Where
- 119 sites in United States, Austria and 12 more
- Volunteers
- 866 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
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
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Advanced Solid Tumors, Breast Cancer Recurrent
First-in-Human Study of ATX-559, an Oral Inhibitor of DHX9, in Patients With Advanced or Metastatic Solid Tumors, and Molecularly Defined Cancers
The goal of this study is to identify a safe and tolerated dose of the orally administered DHX9 inhibitor ATX-559. In addition, this study will evaluate the pharmacokinetics, pharmacodynamics and preliminary antitumor activity of…
- Conditions
- Advanced Solid Tumors, Breast Cancer Recurrent, Colorectal Cancer Metastatic, Colon Cancer
- Trial name
- NCT06625515
- Testing
- ATX-559
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Accent Therapeutics
Hypogammaglobulinemia
Kinetics, Efficacy and Safety of IVIG-L in Hypogammaglobulinemia Patients
The kinetics, efficacy and safety of a liquid intravenous immunoglobulin product, IVIG-L, were studied in patients with hypogammaglobulinemia, who are regularly treated with intravenous immunoglobulin substitution therapy.
- Trial name
- NCT00138697
- Testing
- IVIG-L
- Where
- 6 sites in Netherlands
- Volunteers
- 15 people
- Sponsor
- Prothya Biosolutions
HIV Infections
A Phase I/II Clinical Study of Nystatin I.V. (Intravenous) in Patients With HIV Infection.
To evaluate the clinical toxicity, safety, and maximum tolerated dose (MTD) of intravenous nystatin in patients with HIV infection. To evaluate the potential anti-HIV activity and clinical pharmacology of intravenous nystatin.
- Trial name
- NCT00002097
- Testing
- Nystatin
- Where
- United States
- Sponsor
- Argus Pharmaceuticals
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
Adult Growth Hormone Deficiency
Extension Study on Safety of Long-Term Growth Hormone Replacement in Adult Patients With Growth Hormone Deficiency
To evaluate long-term safety of growth hormone replacement in adult patients with growth hormone deficiency
- Trial name
- NCT00191360
- Testing
- Somatropin
- Where
- 14 sites in Japan
- Volunteers
- 68 people
- Sponsor
- Eli Lilly and Company
HIV Infections, Kidney Failure, Chronic
Retrovir Capsules in the Treatment of HIV-Infected Patients in Renal Failure
To evaluate the safety, tolerance, and pharmacokinetics of Retrovir (AZT) administration in HIV-infected patients in renal failure receiving maintenance hemodialysis.
- Trial name
- NCT00002047
- Testing
- Zidovudine
- Where
- United States
- Sponsor
- Glaxo Wellcome
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
Colorectal Cancer (CRC), Endometrial Cancer
Pembrolizumab Combined With Itacitinib (INCB039110) and/or Pembrolizumab Combined With INCB050465 in Advanced Solid Tumors
This is an open-label, multicenter, Phase 1b platform study in subjects with advanced or metastatic solid tumors (Part 1a) and subjects with selected solid tumors (Part 1b and Part 2).
- Conditions
- Colorectal Cancer (CRC), Endometrial Cancer, Melanoma, Head and Neck Cancer
- Trial name
- NCT02646748
- Testing
- Pembrolizumab, itacitinib, INCB050465
- Where
- 17 sites in United States
- Volunteers
- 159 people
- Sponsor
- Incyte Corporation
HIV Infections
Pilot Study of High-Concentration Capsaicin Patches in the Treatment of Painful HIV-Associated Neuropathy
The purpose of the study is to gain initial information on the tolerability and feasibility of high-concentration capsaicin patches for the treatment of painful HIV-associated neuropathy, whether resulting from HIV disease and/or…
- Trial name
- NCT00061152
- Testing
- Capsaicin Dermal Patch
- Where
- 3 sites in United States
- Sponsor
- NeurogesX
Candidiasis, Mycoses
Non-Comparative Study of Fluconazole in Patients With Serious Mycoses and Who Cannot Be Treated With Conventional Antifungal Therapy
The primary purpose of this protocol is to provide fluconazole for the treatment of individual patients who require therapy for serious or life-threatening systemic fungal infection, who have failed on conventional antifungal…
- Conditions
- Candidiasis, Mycoses, HIV Infections
- Trial name
- NCT00002038
- Testing
- Fluconazole
- Where
- United States
- Sponsor
- Pfizer
Pyruvate Kinase Deficiency
Pyruvate Kinase Deficiency Global Longitudinal Registry
This study is an observational (ie, noninterventional), longitudinal, multicenter, global registry for patients with pyruvate kinase (PK) deficiency, a rare nonspherocytic hemolytic anemia.
- Trial name
- PEAK Registry (NCT03481738)
- Where
- 52 sites in United States, Canada and 14 more
- Volunteers
- 500 people
- Sponsor
- Agios Pharmaceuticals, Inc.
Alpha-Mannosidosis
Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis
The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years
- Trial name
- rhLaman-08 (NCT02998879)
- Testing
- Velmanase Alfa (e.g. Lamazym)
- Where
- 6 sites in Austria, Denmark and 3 more
- Volunteers
- 5 people
- Sponsor
- Chiesi Farmaceutici S.p.A.
Glycogen Storage Disease Type II
A Prospective, Observational Study in Patients With Late-Onset Pompe Disease
Pompe disease (also known as glycogen storage disease type II, "GSD-II") is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Trial name
- NCT00077662
- Where
- 5 sites in United States, France and 1 more
- Volunteers
- 61 people
- Sponsor
- Genzyme, a Sanofi Company
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