Clinical trials: Deficiency Disease
Hypophosphatasia (HPP)
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.
- Trial name
- NCT02104219
- Where
- 19 sites in United States, Australia and 6 more
- Volunteers
- 32 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hereditary Angioedema
A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE)
This is a multicenter, randomized, placebo-controlled, parallel-arm, phase 2 study to investigate the clinical efficacy, pharmacokinetics, and safety of CSL312 as prophylaxis to prevent attacks in subjects with HAE.
- Trial name
- NCT03712228
- Testing
- Factor XIIa antagonist monoclonal antibody
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 44 people
- Sponsor
- CSL Behring
Fanconi Anemia
Whole Blood Biospecimen Collection for Subjects With Fanconi Anemia
The primary study objective is to collect biospecimen samples (e.g., blood) from participants diagnosed with Fanconi Anemia. The biospecimens will be used to create a biorepository that can be used to identify disease associated…
- Trial name
- NCT05910853
- Testing
- Specimen Donation
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Sanguine Biosciences
Growth Hormone Deficiency
Safety and Efficacy of Y-shape Pegylated Somatropin in Growth Hormone Deficiency Children
This is a multicenter, randomized, open-labeled, positive controlled phase 2\&3 combined study to evaluate the safety and efficacy of weekly Y-shape pegylated somatropin, compared to daily somatropin (Norditropin®), in…
- Trial name
- NCT04513171
- Testing
- Y-shape pegylated somatropin, Norditropin®, Norditropin
- Where
- China
- Volunteers
- 434 people
- Sponsor
- Xiamen Amoytop Biotech Co., Ltd.
Pompe Disease
Screening Protocol to Evaluate Acid Alpha-Glucosidase (GAA) Activity and GAA Gene Mutations in Patients With Late Onset Pompe Disease
Pompe disease (also known as glycogen storage disease type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA).
- Trial name
- NCT00113035
- Where
- 5 sites in United States
- Volunteers
- 60 people
- Sponsor
- Genzyme, a Sanofi Company
HIV Infections
A Study of an Oral Entry Inhibitor, SP01A, in Treatment-Experienced HIV-Infected Patients
One measurement of an HIV infected person's risk of progressing to AIDS is the number of viral particles of HIV in their blood (called a "viral load").
- Trial name
- NCT00113412
- Testing
- SP01A
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- Samaritan Pharmaceuticals, Inc
HIV Infections
A Trial of PEHRG214 in HIV-Infected Patients
HRG2 is a Phase 2 randomized, controlled, open-label, multi-dose trial to determine the efficacy, safety, immunogenicity, and pharmacokinetic profile of PEHRG214 in HIV-infected patients, treated three times weekly for up to 16…
- Trial name
- NCT00385567
- Testing
- PEHRG214
- Where
- 7 sites in United States, Puerto Rico
- Volunteers
- 70 people
- Sponsor
- Virionyx Corporation Limited
X-linked Hypophosphatemic Rickets/Osteomalacia
A Study of KRN23 in Subjects With X-linked Hypophosphatemic Rickets/Osteomalacia
The objective of this study is to assess the safety and tolerability of KRN23 after a single subcutaneous (SC) administration in subjects with X-linked hypophosphatemic rickets/osteomalacia (XLH) in Japan or Korea.
- Trial name
- NCT02181764
- Testing
- KRN23
- Where
- 2 sites in Japan, South Korea
- Volunteers
- 15 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Hereditary Angioedema (HAE)
A Study of Navenibart in Participants With Hereditary Angioedema
This is a Phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2…
- Trial name
- ALPHA-ORBIT (NCT06842823)
- Testing
- navenibart
- Where
- 86 sites in United States, Australia and 22 more
- Volunteers
- 157 people
- Sponsor
- Astria Therapeutics, Inc.
HIV Infections
Naïve HIV POC Monotherapy Trial
The primary objective of the trial is: to evaluate the change from baseline in plasma viral load with placebo and one of up to 4 dose regimens of RDEA806.
- Trial name
- NCT00617526
- Testing
- RDEA806 400 mg, RDEA806 1000 mg, RDEA806 600 mg
- Where
- 2 sites in Germany, United Kingdom
- Volunteers
- 48 people
- Sponsor
- Ardea Biosciences, Inc.
HIV Infections
Double Blind Study of Thymopentin Effects on HIV-1 Infectivity of Blood Mononuclear Cells and Semen in HIV Infected Patients
Examine the ability of Timunox (thymopentin) to reduce the amount and/or frequency of virus isolation. Examine the ability of thymopentin to stimulate the immune system and alter the clinical findings of patients infected with…
- Trial name
- NCT00002049
- Testing
- Thymopentin
- Where
- United States
- Sponsor
- Immunobiology Research Institute
Growth Hormone Deficiency (GHD)
Phase 3 Study of LUM-201 in Children With Growth Hormone Deficiency
The OraGrowtH Phase 3 Trial is a multi-national trial. The goals of the trial are to study LUM-201 as a treatment for Pediatric Growth Hormone Deficiency (PGHD) in naive to treatment children and validate the LUM-201 predictive…
- Trial name
- NCT06948214
- Testing
- LUM-201
- Where
- 55 sites in United States, Argentina and 6 more
- Volunteers
- 150 people
- Sponsor
- Lumos Pharma
HIV Infections
A Dose Response and Safety Study of Procaine HCl in HIV-Infected Patients
This a Phase I/II non-randomized, open-label clinical study of 8 weeks duration using SP01A in HIV positive patients on a stable antiretroviral regimen.
- Trial name
- NCT00299338
- Testing
- SP01A
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Samaritan Pharmaceuticals, Inc
Menkes Disease, Occipital Horn Syndrome
Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency
Menkes disease and occipital horn syndrome are two forms of copper deficiency that must be diagnosed and treated very early in life to prevent serious developmental problems.
- Conditions
- Menkes Disease, Occipital Horn Syndrome, Unexplained Copper Deficiency
- Trial name
- NCT00811785
- Testing
- Copper Histidine
- Where
- United States
- Volunteers
- 93 people
- Sponsor
- Cyprium Therapeutics, Inc.
Lymphoma, Non-Hodgkin, HIV Infections
A Study of Mitoguazone Dihydrochloride in Patients With AIDS-Related Non-Hodgkin's Lymphoma
To estimate the response rate, response duration, clinical benefit, and toxicity of mitoguazone dihydrochloride (MGBG) in patients with AIDS-related refractory or relapsing non-Hodgkin's lymphoma (NHL).
- Trial name
- NCT00002348
- Testing
- Mitoguazone
- Where
- United States
- Sponsor
- Genzyme, a Sanofi Company
Growth Failure, Growth Hormone Deficiency
r-hGH Liquid Multidose Versus Freeze-dried Multidose Bioequivalence Trial
The primary objective of the trial was to assess the bioequivalence for two concentrations (5.83 mg/mL and 8 mg/mL) of the new r-hGH liquid multidose formulation using the r hGH freeze-dried multidose formulation (Saizen® 8 mg…
- Trial name
- NCT01034735
- Testing
- r-hGH liquid (Saizen), r-hGH freeze-dried
- Where
- Germany
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- EMD Serono
HIV Infections
BLQ Study: A Study of a Protease Inhibitor With Fuzeon (Enfuvirtide) in Treatment-Experienced Patients With HIV-1.
This single arm study will evaluate the efficacy, safety and tolerability of a new investigational protease inhibitor (PI) plus background antiretrovirals plus Fuzeon (90mg sc bid) in HIV-1 infected, triple-class…
- Trial name
- NCT00326963
- Testing
- Background ARVs, PI, enfuvirtide [Fuzeon]
- Where
- 38 sites in United States, Australia
- Volunteers
- 142 people
- Sponsor
- Hoffmann-La Roche
HIV Infections, HIV Seronegativity
A Phase I, Observer-Blind, Placebo-Controlled Study of the Chiron Vaccine HIV p24/MF59 Administered to Healthy HIV-Seronegative Adults
To evaluate the safety and tolerability of the Chiron vaccine human immunodeficiency virus (HIV) p24(25 or 50 micrograms)/MF59 in healthy HIV-1 seronegative adults.
- Trial name
- NCT00002204
- Testing
- HIV p24/MF59 Vaccine
- Where
- United States
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- Chiron Corporation
Familial Chylomicronemia Syndrome
Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
The purpose of the Expanded Access Program is to provide pre-approval access of olezarsen to eligible patients with Familial Chylomicronemia Syndrome (FCS).
- Trial name
- NCT06360237
- Testing
- Olezarsen
- Where
- United States
- Sponsor
- Ionis Pharmaceuticals, Inc.
Ataxia Telangiectasia
Comparative Bioavailability of Betamethasone Oral Solution Metered Spray (GTX-102) in Healthy Subjects
A Randomized, Open-label, Crossover Study to Evaluate the Comparative Bioavailability, Pharmacokinetics, and Safety of GTX-102 Administered as an Oral Spray Compared to Intramuscular Injection - betamethasone and an Oral Solution…
- Trial name
- NCT05531890
- Testing
- GTX-102 medium dose fast Period 1 and Period 2, GTX-102 medium dose slow Period 1 and Period 2, GTX-102 high dose fast Period 1 and Period 2
- Where
- Canada
- Volunteers
- 48 people, healthy volunteers welcome
- Sponsor
- Grace Therapeutics Inc.
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