Clinical trials: Progressive Disease
- Condition Progressive Disease
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Progressive Familial Intrahepatic Cholestasis (PFIC)
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
The purpose of this study is to determine whether the investigational treatment (maralixibat) is safe and effective in pediatric participants with Progressive Familial Intrahepatic Cholestasis (PFIC).
- Trial name
- MARCH-PFIC (NCT03905330)
- Testing
- Maralixibat
- Where
- 31 sites in United States, Argentina and 15 more
- Volunteers
- 93 people
- Sponsor
- Mirum Pharmaceuticals, Inc.
Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Observational Study in Patients With Autosomal Dominant Polycystic Kidney Disease
To collect characteristics of patients with ADPKD across a broad population, over time to better understand disease progression (signs, symptoms and outcomes).
- Trial name
- OVERTURE (NCT01430494)
- Where
- 266 sites in United States, Argentina and 18 more
- Volunteers
- 3,409 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Myopia, Progressive, Myopia
Clinical Evaluation of a Myopia Control Lens in Slowing Myopia Progression
This is a monocenter, randomized, controlled, parallel-group, double-masked clinical trial to evaluate the effectiveness of test lens in slowing myopia progression with respect to axial length elongation at baseline compared to a…
- Trial name
- NCT05331378
- Testing
- Test myopia control lenses, Single vision lenses
- Where
- Singapore
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Essilor International
Myopia, Myopia, Progressive
Study to Evaluate the Efficacy of Alleance® (Atropine Sulfate 0.01%) as a Treatment to Delay Myopia and Axial Ocular Elongation in Children.
Phase III clinical study to evaluate the efficacy of Alleance® (atropine sulfate 0.01%) ophthalmic solution through the incidence of unexpected adverse events, changes in Best Corrected Visual Acuity (BCVA), changes in…
- Trial name
- NCT06389110
- Testing
- Alleance®
- Where
- Mexico
- Volunteers
- 123 people, healthy volunteers welcome
- Sponsor
- Laboratorios Sophia S.A de C.V.
Brain Tumor, Recurrent, Brain Tumor, Pediatric
RRx-001 Given With Irinotecan and Temozolomide for Pediatric Patients With Recurrent or Progressive Malignant Solid and Central Nervous System Tumors
The PIRATE study tests the experimental drug RRx-001 in combination with 2 chemotherapy drugs that are commonly used in patients with cancer. RRx-001 has been used alone and with other anti-cancer medicines in adults.
- Conditions
- Brain Tumor, Recurrent, Brain Tumor, Pediatric, Central Nervous System Neoplasms, Unspecified Childhood Solid Tumor, Protocol Specific
- Trial name
- PIRATE (NCT04525014)
- Testing
- RRx-001, Temozolomide, Irinotecan
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- EpicentRx, Inc.
Myopia
Progressive Myopia Treatment Evaluation for NaturalVue Multifocal Contact Lens Trial
This is a multi-center, randomized, double-masked clinical trial. All study devices are market approved/cleared in the localities where the study is conducted.
- Trial name
- PROTECT (NCT05159765)
- Testing
- Single vision contact lenses, Multifocal contact lenses
- Where
- 8 sites in United States, Canada and 2 more
- Volunteers
- 144 people, healthy volunteers welcome
- Sponsor
- Visioneering Technologies, Inc
Keratoconus, Ectasia Corneal
Comparison of Standard vs. Accelerated Corneal Crosslinking
The study objective is to compare accelerated and standard corneal crosslinking for treatment of progressive keratoconus or corneal ectasia.
- Trial name
- NCT03922542
- Testing
- riboflavin 0.1%
- Where
- United States
- Volunteers
- 508 people
- Sponsor
- Price Vision Group
Pulmonary Arterial Hypertension
A Study to Assess Whether Macitentan Delays Disease Progression in Children With Pulmonary Arterial Hypertension (PAH)
This is a prospective, multicenter, open-label, randomized, controlled, parallel Phase 3 study with an open-label single-arm extension period to evaluate pharmacokinetics (PK), safety and efficacy of macitentan in children with…
- Trial name
- TOMORROW (NCT02932410)
- Testing
- Macitentan, Standard-of-care
- Where
- 85 sites in United States, Australia and 21 more
- Volunteers
- 165 people
- Sponsor
- Actelion
Inherited Mitochondrial Disease
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical…
- Trial name
- NCT05218655
- Testing
- Vatiquinone
- Where
- 26 sites in United States, France and 5 more
- Volunteers
- 101 people
- Sponsor
- PTC Therapeutics
Myopia, Progressive
Slow Myopia Progression With Different Irradiance Light
It is a prospective clinical control study on red light control myopia with specail design spectacles for 75 children in 3 groups. Study Groups with two different powers of 0.6 mW and 1.2 mW at wavelength of 650nm.
- Trial name
- NCT05881655
- Testing
- Airdoc Red Lighting Device, Special Spectacles to Control Myopia
- Where
- China
- Volunteers
- 75 people
- Sponsor
- Beijing Airdoc Technology Co., Ltd.
Myopia, Progressive
Myopia Preventing With Distance-image Screen
This study is for testing the efficacy and safety of distance-image screens for preventing myopia in children for 12 month. Each subjects would be asked to paired test the ocular parameters between before and after 12-month…
- Trial name
- DIS (NCT06088225)
- Testing
- distance-image screen
- Where
- China
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Beijing Airdoc Technology Co., Ltd.
Relapsing-remitting Multiple Sclerosis, Active Secondary Progressive Multiple Sclerosis
Special Drug-use Surveillance for Kesimpta for s.c. Injection 20 mg Pen
This study was an uncontrolled, central registration system, open-label, multicenter observational study in patients using Kesimpta for the labeled indication.
- Trial name
- NCT04940065
- Testing
- Kesimpta
- Where
- 103 sites in Japan
- Volunteers
- 367 people
- Sponsor
- Novartis Pharmaceuticals
Spinal Muscular Atrophy
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
This is an interventional study to evaluate safety and efficacy of AAV-hSMN1 in spinal muscular atrophy patients.
- Trial name
- NCT06288230
- Testing
- vesemnogene lantuparvovec
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Lantu Biopharma
Myopia
To Evaluate the Effectiveness of New Specially Designed Fitlens Soft Lens.
Myopia is a common disorder, affecting approximately one-third of the US population and over 90% of the population in some East Asian countries.
- Trial name
- NCT05433792
- Testing
- Myolens CN (Central-Near) soft contact lens, MiSight® soft contact lens
- Where
- Israel
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- Fitlens Ltd.
PFIC1, PFIC2
This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
Double blind, randomized, placebo controlled, Phase 3 study to investigate the efficacy and safety of low doses and high doses of A4250 compared to placebo in children with progressive familial intrahepatic cholestasis (PFIC)…
- Trial name
- PEDFIC 1 (NCT03566238)
- Testing
- A4250 (odevixibat)
- Where
- 45 sites in United States, Australia and 13 more
- Volunteers
- 62 people
- Sponsor
- Albireo
Myopia, Progressive
Myopia Control Combined PBM With Myopic Defocus Lens in Children
To study the efficacy of myopi control with two methods: low lever red light and peripheral defocus spectacles with four groups and design with prospective, double-blind, randimized, and with control for 6 month.
- Trial name
- NCT05756959
- Testing
- PBM, Peripheral defocus spectacles, Single vision spectacles
- Where
- China
- Volunteers
- 304 people
- Sponsor
- Beijing Airdoc Technology Co., Ltd.
HIV Infections, Leukoencephalopathy, Progressive Multifocal
A Study of ddI in Patients With AIDS Who Become Sicker While Taking Zidovudine
The objective of this open-label study regimen is to make didanosine (ddI) available to patients with AIDS who are clinically deteriorating on zidovudine (AZT) and cannot enter the Phase II ddI programs due to protocol exclusion…
- Trial name
- NCT00002274
- Testing
- Didanosine
- Where
- United States
- Sponsor
- Bristol-Myers Squibb
Cystic Fibrosis, Pseudomonas Aeruginosa Airway Infection
Aztreonam Lysine for Inhalation in Patients With Cystic Fibrosis and Pseudomonas Aeruginosa Airway Infection
The primary objective of this program is to provide expanded access to aztreonam lysine for inhalation (AZLI) 75 mg prior to its commercial availability to patients with cystic fibrosis (CF) and chronic P.
- Trial name
- NCT00499720
- Testing
- Aztreonam Lysine for Inhalation
- Where
- 65 sites in United States, Puerto Rico
- Sponsor
- Gilead Sciences
Keratoconus, Post-Refractive Ectasia
Corneal Crosslinking in Patients With Keratoconus and Post-Refractive Ectasia
The purpose of this study is to evaluate the effectiveness of using riboflavin and UV light to treat progressive keratoconus or post-refractive corneal ectasia.
- Trial name
- NCT01143389
- Testing
- Riboflavin, UVX light
- Where
- United States
- Volunteers
- 510 people
- Sponsor
- Price Vision Group
Mitochondrial Diseases, Drug Resistant Epilepsy
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
This is a parallel-arm, double-blind, placebo-controlled study with a screening phase that includes a 28-day run-in phase to establish baseline seizure frequency, followed by a 24-week, randomized, placebo-controlled phase.
- Conditions
- Mitochondrial Diseases, Drug Resistant Epilepsy, Leigh Disease, Leigh Syndrome
- Trial name
- MIT-E (NCT04378075)
- Testing
- Vatiquinone
- Where
- 27 sites in United States, Canada and 7 more
- Volunteers
- 68 people
- Sponsor
- PTC Therapeutics