Clinical trials: Natural History
Hepatitis C, Anemia
A Study to Evaluate the Erythropoietic Response in Hepatitis C Virus (HCV) Patients Receiving Combination Ribavirin (RBV)/Interferon (IFN) Therapy or RBV/PEG IFN (NATURAL HISTORY-HCV)
The purpose of this study was to describe the time course and extent of hemoglobin (Hb) changes and the erythropoietic response to PEG-IFN/RBV-induced anemia In HCV-infected subjects.
- Trial name
- NCT00328549
- Testing
- Pegylated Interferon and Ribavirin
- Volunteers
- 105 people
- Sponsor
- Ortho Biotech Products, L.P.
Infantile Neuroaxonal Dystrophy, INAD
Natural History of Infantile Neuroaxonal Dystrophy
This is a retrospective and cross-sectional review of the natural history of INAD.
- Trial name
- NCT03999814
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Biojiva LLC
Fucosidosis
A Long-Term Observational Study of Patients With Fucosidosis
The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time.
- Trial name
- NCT07615400
- Where
- 16 sites in United States, India and 4 more
- Volunteers
- 57 people
- Sponsor
- JCR Pharmaceuticals Co., Ltd.
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Spinocerebellar Ataxias
Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control
The purpose of this study is to leverage two sources of real-world data (RWD) to assess the effectiveness of troriluzole after three years of treatment in patients with SCA by comparison to an external control of untreated…
- Trial name
- NCT06529146
- Testing
- BHV-4157
- Where
- United States
- Volunteers
- 909 people
- Sponsor
- Biohaven Therapeutics Ltd.
Infections, Papillomavirus
Study of Natural History of Human Papillomavirus (HPV) Infections in Adult Women With Recurrent Conizations in Norway
This is a register-based, epidemiological study of HPV type distribution in women aged 18 years and above with recurrent conization following a primary conization for high grade lesions/ microinvasive carcinomas.
- Trial name
- NCT00924794
- Testing
- Cancer Registry of Norway (database)
- Where
- Norway
- Volunteers
- 410 people
- Sponsor
- GlaxoSmithKline
KCNT1-Related Epilepsy
A Natural History Study of Participants With Potassium Sodium-Activated Channel Subfamily T Member 1 (KCNT1)-Related Epilepsy
The primary objective of the study is to characterize seizures in participants with KCNT1-related epilepsy. The secondary objectives are to characterize head growth, symptom severity, neurocognitive and social functions, adaptive…
- Trial name
- K1Te (NCT04924153)
- Testing
- No Intervention
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Biogen
Fibrodysplasia Ossificans Progressiva
A Natural History Study of Fibrodysplasia Ossificans Progressiva (FOP)
Fibrodysplasia Ossificans Progressiva (FOP) is a rare, severely disabling disease characterized by painful, recurrent episodes of soft tissue swelling (flare-ups) that result in abnormal bone formation in muscles, tendons, and…
- Trial name
- NCT02322255
- Where
- 7 sites in United States, Argentina and 4 more
- Volunteers
- 114 people
- Sponsor
- Clementia Pharmaceuticals Inc.
Hepatocellular Cancer
A Longitudinal Observational Study of the Natural History and Management of Patients With HCC
TARGET-HCC is a longitudinal, observational study of patients being managed for HCC in usual clinical practice. TARGET-HCC will create a research registry of participants with HCC within academic and community real-world…
- Trial name
- NCT02954094
- Where
- 71 sites in United States, France and 3 more
- Volunteers
- 10,000 people
- Sponsor
- Target PharmaSolutions, Inc.
Amyloidosis, Leukocyte Chemotactic Factor 2 Amyloidosis
A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
The purpose of this study is to characterize the natural history of leukocyte chemotactic factor 2 amyloidotic (ALECT2) disease. In this observational study participants with ALECT2 disease will be enrolled.
- Trial name
- NCT03774784
- Where
- 31 sites in United States, Egypt and 4 more
- Volunteers
- 26 people
- Sponsor
- Alnylam Pharmaceuticals
GM1 Gangliosidosis, Sandhoff Disease
GM1 and GM2 Gangliosidosis PROspective Neurological Disease TrajectOry Study (PRONTO)
The study aims to characterize prospectively longitudinal progression of neurological domains in GM1 and GM2 Gangliosidosis patients with high-quality standards (GCP compliant).
- Conditions
- GM1 Gangliosidosis, Sandhoff Disease, Tay-Sachs Disease
- Trial name
- PRONTO (NCT05109793)
- Where
- 13 sites in United States, Brazil and 4 more
- Volunteers
- 31 people
- Sponsor
- Azafaros B.V.
Dry Age-related Macular Degeneration, Geographic Atrophy
Investigating Geographic Atrophy Insights (i-GAIN) Natural History Study
An observational study to investigate the natural history and evaluate biomarkers of participants with geographic atrophy secondary to age-related macular degeneration
- Conditions
- Dry Age-related Macular Degeneration, Geographic Atrophy, Retinal Disease, Macular Degeneration
- Trial name
- i-GAIN (NCT05797896)
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 234 people
- Sponsor
- Complement Therapeutics
Venous Thromboembolism
An Observational Study on Incidence, Natural History and Treatment Patterns of Venous Thromboembolism in Taiwan
The research question addressed by this study is primarily on the clinical epidemiology of VTE in Taiwan. The study aims to estimate the incidence of VTE and describe natural history of VTE, including long term clinical outcomes…
- Trial name
- NCT03799016
- Where
- Taiwan
- Volunteers
- 104,215 people
- Sponsor
- Bayer
Retinitis Pigmentosa, Eye Diseases
Natural History Study of Patients With EYS-Associated RP
This natural history study of patients with EYS mutations from Russia and former CIS (Commonwealth of Independent States) territories will accelerate the development of outcome measures for clinical trials.
- Trial name
- RUS_EYS (NCT07228793)
- Testing
- Whole exome/genome sequencing
- Where
- Russia
- Volunteers
- 45 people
- Sponsor
- Sensor Technology for Deafblind
Leber Congenital Amaurosis (LCA), Retinitis Pigmentosa (RP)
Natural History Study in Inherited Retinal Disease Subjects Caused by Mutations in RPE65 or LRAT
To evaluate the natural history of visual function in subjects with IRD phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by RPE65 or LRAT gene mutations.
- Trial name
- NCT02575430
- Testing
- No treatment: retrospective chart review
- Where
- 9 sites in United States, Canada and 5 more
- Volunteers
- 59 people
- Sponsor
- QLT Inc.
Sanfilippo Syndrome Type A
A Study of Patients With Sanfilippo Syndrome Type A (MPS IIIA)
The purpose is to evaluate the course of disease progression in MPS IIIA patients who are untreated to identify potential surrogate endpoints that may be utilized in future ERT trials of MPS IIIA via defined assessments including…
- Trial name
- NCT01047306
- Testing
- assessment
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Shire
Danon Disease
Natural History of Danon Disease
This is a multicenter, international, non-interventional, natural history study designed to collect longitudinal retrospective clinical information on patients with Danon disease (DD).
- Trial name
- NCT05548855
- Testing
- Heart Transplant, Cardiac Assistive Device
- Where
- 15 sites in Czechia, France and 3 more
- Volunteers
- 59 people
- Sponsor
- Rocket Pharmaceuticals Inc.
Hereditary Inclusion Body Myopathy, GNE Myopathy
GNE-Myopathy Disease Monitoring Program (GNEM-DMP): A Registry and Prospective Observational Natural History Study to Assess GNE Myopathy or Hereditary Inclusion Body Myopathy (HIBM)
HIBM is a severe progressive myopathy that typically presents in early adulthood as weakness in the distal muscles of the lower extremities and progresses proximally, leading to a loss of muscle strength and function, and…
- Conditions
- Hereditary Inclusion Body Myopathy, GNE Myopathy, Nonaka Disease, Quadriceps Sparing Myopathy (QSM)
- Trial name
- NCT01784679
- Where
- 5 sites in United States, Bulgaria and 3 more
- Volunteers
- 319 people
- Sponsor
- Ultragenyx Pharmaceutical Inc