Clinical trials: Protein Deficiency
Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency
Fatty Acid Oxidation Defects and Insulin Sensitivity
The purpose of this study is to learn more about what causes insulin resistance. It has been suggested that proper breakdown of fat into energy (oxidation) in the body is important to allow insulin to keep blood sugar in the…
- Conditions
- Very Long-chain Acyl-CoA Dehydrogenase Deficiency, Trifunctional Protein Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Medium-chain Acyl-CoA Dehydrogenase Deficiency
- Trial name
- NCT02517307
- Testing
- Intralipid/Heparin, Glycerol/Saline, Hyperinsulinemic euglycemic clamp
- Where
- United States
- Volunteers
- 41 people, healthy volunteers welcome
- Sponsor
- Oregon Health and Science University
Kidney Transplant, Myeloma
Study Evaluating SARS-CoV-2 (COVID-19) Humoral Response After BNT162b2 Vaccine in Immunocompromised Adults Compared to Healthy Adults
The primary endpoint of this study is to compare the humoral response (titre and neutralizing capacity of induced antibodies) against SARS-CoV-2 following vaccination with BNT162b2 (Pfizer BioNTech) in immunocompromised persons…
- Conditions
- Kidney Transplant, Myeloma, Cancer, Hematologic Malignancy
- Trial name
- EREVA (NCT04952766)
- Testing
- Biological samples
- Where
- France
- Volunteers
- 196 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Régional d'Orléans
Factor VII Deficiency
A Phase II Exploratory Study of SR604 Injection Evaluating the Safety and Efficacy in the Treatment of Congenital Coagulation Factor VII Deficiency
This is an open-label, multicenter, exploratory Phase II clinical trial designed to evaluate the efficacy, safety, and pharmacokinetic characteristics of SR604 Injection in patients with congenital coagulation Factor VII…
- Trial name
- NCT07711158
- Testing
- SR604
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency
A Study of UX007 (Triheptanoin) in Participants With Long-Chain Fatty Acid Oxidation Disorders (LC-FAOD)
The primary objective of the study was to evaluate the impact of UX007 on acute clinical pathophysiology associated with LC-FAOD following 24 weeks of treatment.
- Conditions
- Long-chain Fatty Acid Oxidation Disorders (LC-FAOD), Carnitine Palmitoyltransferase (CPT II) Deficiency, Very Long Chain Acyl-CoA Dehydrogenase (VLCAD) Deficiency, Longchain 3-hydroxy-acyl-CoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- NCT01886378
- Testing
- UX007
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 29 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency
Study of Triheptanoin for Treatment of Long-Chain Fatty Acid Oxidation Disorder
Humans eat long-chain fat in their diet and use it for energy during exercise and during periods of fasting. Patients with long-chain fatty acid oxidation disorders cannot use dietary fat for energy.
- Conditions
- Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase 2 (CPT2) Deficiency, Mitochondrial Trifunctional Protein (TFP) Deficiency, Long-chain 3 hydroxyacylCoA Dehydrogenase (LCHAD) Deficiency
- Trial name
- Triheptanoin (NCT01379625)
- Testing
- Triheptanoin
- Where
- 2 sites in United States
- Volunteers
- 32 people
- Sponsor
- Oregon Health and Science University
Anogenital Warts, HIV Infection
Effectivity of Protein Purified Derivative Treatment for Anogenital Warts in HIV Patient
Warts are a common viral infection of the skin and are prevalent throughout the world, with an overall prevalence in the United States estimated at 2-20%.
- Conditions
- Anogenital Warts, HIV Infection, Tuberculin Purified Protein Derivative
- Trial name
- NCT06587542
- Testing
- Injection of protein purified derivative
- Volunteers
- 30 people
- Sponsor
- Universitas Padjadjaran
The Immune Dysregulation Polyendocrinopathy Enteropathy X-linked Syndrome is a Primary Immunodeficiency Caused by Pathogenic Variants in Forkhead Box Protein 3
Study Evaluating a Gene Therapy for IPEX Syndrome Through the Expression of FOXP3 on Deficient T Cells to Produce Tregs-like.
The purpose of this study is to evaluate the safety and efficacy of FOXP3-T4 (an autologous gene therapy) alone or in combination with low-dose IL-2 for the treatment of IPEX syndrome.
- Trial name
- THERIPEX (NCT07697118)
- Testing
- FOXP3-T4 drug product, ILT-101
- Where
- France
- Volunteers
- 5 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
HIV Infections
Evaluating the Safety and Immunogenicity of Env (A,B,C,A/E)/Gag (C) DNA and gp120 (A,B,C,A/E) Protein/GLA-SE HIV Vaccines, Given Individually or Co-administered, in Healthy, HIV-1-Uninfected Adults
The purpose of this study is to evaluate the safety, tolerability, and immunogenicity of env (A,B,C,A/E)/gag (C) DNA and gp120 (A,B,C,A/E) protein/GLA-SE HIV-1 vaccines (PDPHV-201401) as a prime-boost regimen or co-administered…
- Trial name
- NCT03409276
- Testing
- env (A,B,C,A/E)/gag (C) DNA Vaccine, gp120 (A,B,C,A/E) Protein Vaccine, GLA-SE adjuvant
- Where
- 6 sites in United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Chronic Myeloproliferative Disorders, Fanconi Anemia
Research Study in Healthy Volunteers of Patients With Fanconi Anemia, Myeloproliferative Disorders, or Myeloma
RATIONALE: Analyzing tissue and blood samples from healthy volunteers or patients with Fanconi anemia, myelodysplasia, myeloproliferative disorders, or myeloma in the laboratory may help doctors learn more about the causes of…
- Conditions
- Chronic Myeloproliferative Disorders, Fanconi Anemia, Multiple Myeloma and Plasma Cell Neoplasm, Myelodysplastic/Myeloproliferative Neoplasms
- Trial name
- NCT00900055
- Testing
- microarray analysis, polyacrylamide gel electrophoresis, polymerase chain reaction
- Where
- United States
- Volunteers
- 213 people, healthy volunteers welcome
- Sponsor
- OHSU Knight Cancer Institute
Acquired Immunodeficiency Syndrome, HIV Infections
Prospective Study of HIV Infection in Hemophiliacs
To examine mechanisms of individual differences in the progression of HIV infection in hemophiliacs.
- Conditions
- Acquired Immunodeficiency Syndrome, HIV Infections, Blood Disease, Hemophilia A
- Trial name
- NCT00005309
- Sponsor
- Bloodworks
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
Testing Nivolumab as a Potential Targeted Treatment in Cancers With Mismatch Repair Deficiency (MATCH-Subprotocol Z1D)
This phase II MATCH treatment trial identifies the effects of nivolumab in patients whose cancer has a genetic change called mismatch repair deficiency.
- Conditions
- Advanced Lymphoma, Advanced Malignant Solid Neoplasm, Hematopoietic and Lymphoid Cell Neoplasm, Refractory Lymphoma
- Trial name
- NCT04439214
- Testing
- Nivolumab
- Where
- United States
- Volunteers
- 47 people
- Sponsor
- National Cancer Institute (NCI)
Protein Deficiency
Comparison of Meal Replacements Versus Protein Supplements and Utilizing the Protein Digestibility Corrected Amino Acid Score (PDCAAS) Method
The purpose of this research study is to compare the varying qualities of protein supplements utilizing the Protein Digestibility Corrected Amino Acid Score (PDCAAS) method and evaluating the clinical outcome related to protein…
- Trial name
- PDCAAS (NCT01399333)
- Testing
- Group 1 Meal Replacement, Group 2 Protein Supplement, Group 3
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Southeast Bariatrics, PA
Nutritional Deficiency, Ileostomy; Functional Disturbance
Absorption of Peptides, Fluid, and Electrolytes in Patients With an Ileostomy
Clinical trial, active comparator, cross over, randomised. In total, 12 adults with an ileostomy will be randomised to a sequential 4 weeks intervention with different qualities and sources of protein wish 2-week washout periods.
- Conditions
- Nutritional Deficiency, Ileostomy; Functional Disturbance, Short Bowel Syndrome, Absorption; Disorder, Protein
- Trial name
- ABSOLYT (NCT04141826)
- Testing
- Hydrolysed whey, Intact whey, Caseinate
- Where
- Denmark
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Christian Hvas
HIV Infections
A Phase I Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Safety and Immunogenicity of HIV-1 Recombinant Envelope Glycoprotein gp160
To determine the reactivity and safety of HIV-1 recombinant envelope glycoprotein gp160. To determine the immunogenicity of gp160.
- Trial name
- NCT00000745
- Testing
- Hepatitis B Vaccine (Recombinant), gp160 Vaccine (MicroGeneSys)
- Where
- 2 sites in United States
- Volunteers
- 72 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Immune Deficiency Disorders, Severe Combined Immunodeficiency
Immune Disorder HSCT Protocol
This study hypothesizes that a reduced intensity immunosuppressive preparative regimen will establish engraftment of donor hematopoietic cells with acceptable early and delayed toxicity in patients with immune function disorders.
- Conditions
- Immune Deficiency Disorders, Severe Combined Immunodeficiency, Chronic Granulomatous Disease, X-linked Agammaglobulinemia
- Trial name
- NCT01821781
- Testing
- Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- Washington University School of Medicine
Hemophilia A
Pharmacokinetics, Efficacy and Safety Study of IMMUNATE SD (Human Plasma-Derived Coagulation Factor VIII Concentrate) in Hemophilia A Patients
The purpose of this study is to evaluate whether IMMUNATE S/D is effective and safe in the treatment of hemophilia A patients. The study consists of 3 parts: Part 1 is a pharmacokinetic comparison of IMMUNATE S/D and its…
- Trial name
- NCT00162019
- Testing
- Human Plasma-Derived Coagulation Factor VIII Concentrate (Virus Inactivated by Polysorbate 80 Treatment and Vapor Heat Treatment)
- Where
- 5 sites in Bulgaria, Czechia and 2 more
- Volunteers
- 56 people
- Sponsor
- Baxalta now part of Shire
Acquired Antithrombin Deficiency
Phase 3 Study on the Efficacy and Safety of Human Plasma Derived Antithrombin (Atenativ) in Heparin-Resistant Patients Scheduled to Undergo Cardiac Surgery Necessitating Cardiopulmonary Bypass
The primary objective of this study is to evaluate the efficacy of two different doses of Atenativ, versus placebo, in restoring and maintaining heparin responsiveness in adult patients undergoing cardiac surgery necessitating…
- Trial name
- NCT06096116
- Testing
- Human plasma derived antithrombin
- Where
- 27 sites in United States, Austria and 8 more
- Volunteers
- 120 people
- Sponsor
- Octapharma
Overweight, Nutritional Deficiency
The PROTEIN Project
New advances in information computer technology and artificial intelligence (AI) offer the possibility to create a personalized tool and support system for healthy living and eating, and this is the principal objective of the…
- Conditions
- Overweight, Nutritional Deficiency, Poor Nutrition, Iron-deficiency
- Trial name
- PROTEIN (NCT04829851)
- Testing
- PROTEIN Mobile App, Expert Dashboard
- Where
- United Kingdom
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- University of Surrey
Neoplasm Metastasis
Study of PF-07265807 in Participants With Metastatic Solid Tumors.
A First-in-Human Pharmacokinetic, Safety, and Tolerability Study of PF-07265807 as Monotherapy and in Combination in Participants with Advanced or Metastatic Solid Tumors
- Trial name
- NCT04458259
- Testing
- PF-07265807, Sasanlimab, Axitinib
- Where
- 33 sites in United States, Canada and 3 more
- Volunteers
- 88 people
- Sponsor
- Pfizer