Clinical trials: Neonatal Outcome
Alagille Syndrome, Progressive Familial Intrahepatic Cholestasis
Validation of the Itch Reported Outcome (ItchRO) Diaries in Pediatric Cholestatic Liver Disease
The purpose of the study is to validate the ItchRO instrument (a clinical outcome assessment measure of itching) prior to the analysis of longitudinal treatment effect data being generated in ongoing clinical trials.
- Trial name
- NCT02131623
- Where
- 5 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Gaucher Disease
Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received.
- Trial name
- NCT03291223
- Where
- United States
- Volunteers
- 1,257 people
- Sponsor
- Shire
Hemophilia A, Prophylaxis of Bleeding
An Observational Study Called JOIHA to Learn More About How Well the Treatment With Jivi Works to Prevent Problems With Joints in Adults With Hemophilia A.
This is an observational study in which data from people with hemophilia A who decide on their own or by recommendation of their doctors to take Jivi are collected and studied.
- Trial name
- JOIHA (NCT05643560)
- Testing
- Damoctocog alfa pegol (Jivi, BAY94-9027)
- Where
- Italy
- Volunteers
- 33 people
- Sponsor
- Bayer
Hunter Syndrome
Hunter Outcome Survey (HOS)
The purpose of this study is to collect data that will increase understanding of Hunter syndrome. The data from HOS may provide guidance to healthcare professionals about disease treatment options.
- Trial name
- HOS (NCT03292887)
- Where
- United States
- Volunteers
- 1,443 people
- Sponsor
- Shire
Pregnancy, Neonates
Pregnancy & Neonatal Follow-up of Ongoing Pregnancies Established During Clinical Trial 38826 (Study 38827)(P06056)(COMPLETED)
The objective of this trial was to evaluate whether Org 36286 treatment for the induction of multifollicular growth in women undergoing controlled ovarian stimulation (COS) for in vitro fertilization (IVF) or intracytoplasmatic…
- Trial name
- NCT00702988
- Testing
- Org 36286, recFSH
- Volunteers
- 44 people
- Sponsor
- Organon and Co
Hemophilia A
GAS-Hem Feasibility Study
The purpose of this study is to investigate the feasibility and acceptability of the Standardized Goal Attainment Scaling menu for Hemophilia (GAS-Hem) as a patient reported outcome (PRO) measure to monitor clinical progress in…
- Trial name
- NCT02674997
- Testing
- Factor VIII
- Where
- 4 sites in United States, Canada
- Volunteers
- 44 people
- Sponsor
- Baxalta now part of Shire
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447980
- Testing
- NT-501
- Where
- 12 sites in United States
- Volunteers
- 73 people
- Sponsor
- Neurotech Pharmaceuticals
Fabry Disease
Fabry Outcome Survey (FOS)
The purpose of this study is to collect data that will increase understanding of Fabry disease history and progression, in treated and untreated patients with Fabry disease.
- Trial name
- FOS (NCT03289065)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Shire
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05714 (Care Program)(P05715)
The objective of this trial is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of multifollicular growth in women undergoing controlled ovarian stimulation (COS) prior to in vitro fertilization…
- Trial name
- Care (NCT00702234)
- Testing
- Corifollitropin alfa, GnRH antagonist, (rec)hCG
- Volunteers
- 268 people
- Sponsor
- Organon and Co
Prader-Willi Syndrome
PWS Outcomes Assessment Study
This is a longitudinal study during which qualitative interviews will be conducted with caregivers of Soleno C601/C602 study subjects.
- Trial name
- NCT04102839
- Testing
- Interview
- Where
- United States
- Volunteers
- 59 people, healthy volunteers welcome
- Sponsor
- Soleno Therapeutics, Inc.
Retinitis Pigmentosa, Eye Diseases, Hereditary
Natural History of PRPF31 Mutation-Associated Retinal Dystrophy
The purpose of this study is to characterize the natural history through temporal systemic evaluation of subjects identified with PRPF31 mutation-associated retinal dystrophy, also called retinitis pigmentosa type 11, or RP11.
- Conditions
- Retinitis Pigmentosa, Eye Diseases, Hereditary, Retinal Dystrophies, Retinal Dystrophy Rod
- Trial name
- NCT05573984
- Where
- 7 sites in United States, Australia
- Volunteers
- 50 people
- Sponsor
- PYC Therapeutics
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Canadian Medical Assessment of JINARC™ Outcomes Registry
This study is part of the Health Canada approval requirement for JINARC™ (tolvaptan) and is an observational, non-interventional study (NIS) describing the impact of tolvaptan on ADPKD-related burden of illness as measured with a…
- Trial name
- C-MAJOR (NCT02925221)
- Where
- 6 sites in Canada
- Volunteers
- 530 people
- Sponsor
- Otsuka Canada Pharmaceutical Inc.
COVID-19, Pregnant Women
Qatar PREgnancy Covid-19 OUtcome Study
The in-utero exposure of acute viral infection in some instances is proven to have short- and long-term neonatal effects during the postnatal and childhood period and the Zika virus, measles, mumps, and rubella are few examples.
- Trial name
- QPRECIOUS (NCT05349279)
- Where
- Qatar
- Volunteers
- 4,000 people, healthy volunteers welcome
- Sponsor
- Hamad Medical Corporation
Gaucher Disease
Survey Study for Velaglucerase Alfa (VPRIV) in Japan
The objective of this post-marketing survey study is to collect data to determine the safety and efficacy of velaglucerase alfa (VPRIV) in participants with Gaucher disease who are new to therapy or have been switched from…
- Trial name
- NCT03625882
- Where
- 35 sites in Japan
- Volunteers
- 63 people
- Sponsor
- Takeda
Preterm Delivery
Neonatal Outcome by Reason for Delivery
To determine the rate of Composite Neonatal Morbidity for very preterm babies delivered secondary to preterm labor (PTL) vs. prelabor rupture of membranes (PROM).
- Trial name
- NCT01818518
- Where
- 10 sites in United States
- Volunteers
- 995 people, healthy volunteers welcome
- Sponsor
- Obstetrix Medical Group
Retinitis Pigmentosa
A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis Pigmentosa
The purpose of this study is to look at the safety and effectiveness of CNTF implants on vision in persons with retinitis pigmentosa, Usher type II \& III, and Choroideremia.
- Trial name
- NCT00447993
- Testing
- Low Dose NT-501, High Dose NT-501
- Where
- 11 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurotech Pharmaceuticals
Facioscapulohumeral Muscular Dystrophy 1
Evaluation of Safety, Tolerability, and Changes in Biomarker and Clinical Outcome Assessments of Losmapimod for FSHD1 With Extension
This clinical trial is a study to evaluate the safety, tolerability, and changes in biomarker and clinical outcome assessments of Losmapimod for patients with Facioscapulohumeral Muscular Dystrophy 1 (FSHD1) with an open-label…
- Trial name
- FSHD (NCT04004000)
- Testing
- Losmapimod
- Where
- Netherlands
- Volunteers
- 14 people
- Sponsor
- Fulcrum Therapeutics
PolG, Primary Mitochondrial Myopathy
Mitochondrial Video Assessment Source Material Study
Source material collection of videos to develop the Mitochondrial Video Assessment (MVA) for patients with mitochondrial myopathies.
- Trial name
- NCT06180421
- Where
- United States
- Volunteers
- 28 people
- Sponsor
- The Emmes Company, LLC
Hemophilia
A Study to Learn About the Study Medicine (Called Marstacimab) in People With Hemophilia A or B
The purpose of this non-interventional, observational, real-world study is to find out what are the real-world experiences of people living with hemophilia A or B[OK1.1] who start the study medicine (called marstacimab) in…
- Trial name
- NCT07859527
- Testing
- Marstacimab
- Volunteers
- 100 people
- Sponsor
- Pfizer
Hereditary Tyrosinemia, Type I
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
This is a prospective, non-interventional, non-comparative, multicenter study to collect data on HT-1 patients in China treated with Nitisinone in a routine clinical setting. No tests or examinations are mandated in the study.
- Trial name
- NCT06227429
- Testing
- Nitisinone
- Where
- 4 sites in China
- Sponsor
- Swedish Orphan Biovitrum