Clinical trials: Newborn Diseases
Mitochondrial Diseases
EPI-743 for Mitochondrial Respiratory Chain Diseases
This study will evaluate the safety and efficacy of EPI-743 in participants with severe mitochondrial respiratory chain diseases who are considered to be within 90 days of end-of-life care.
- Trial name
- NCT01370447
- Testing
- EPI-743
- Where
- 16 sites in United States
- Sponsor
- PTC Therapeutics
Friedreich's Ataxia
Safety and Pharmacology Study of VP 20629 in Adults With Friedreich's Ataxia
The objectives of the study are: To evaluate the safety and tolerability of single and multiple oral doses of VP 20629 in subjects with Friedreich's ataxia (FA).
- Trial name
- NCT01898884
- Testing
- VP 20629
- Where
- 5 sites in United States
- Volunteers
- 46 people
- Sponsor
- Shire
Mucopolysaccharidosis II
A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome
This is a multicenter, multiregional, open-label study to assess the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of tividenofusp alfa (DNL310), an investigational central nervous system (CNS)-penetrant enzyme…
- Trial name
- NCT04251026
- Testing
- tividenofusp alfa
- Where
- 7 sites in United States, Canada and 2 more
- Volunteers
- 47 people
- Sponsor
- Denali Therapeutics Inc.
Sickle Cell Disease
Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy
This is a multi-center, long-term safety and efficacy follow-up study for subjects with sickle cell disease who have been treated with ex vivo gene therapy drug product in bluebird bio-sponsored clinical studies.
- Trial name
- NCT04628585
- Testing
- Safety and efficacy assessments
- Where
- 16 sites in United States, France
- Volunteers
- 85 people
- Sponsor
- Genetix Biotherapeutics Inc.
Atopic Dermatitis
Tralokinumab Monotherapy for Moderate to Severe Atopic Dermatitis - ECZTRA 2 (ECZema TRAlokinumab Trial no. 2)
Primary objective: To evaluate the efficacy of tralokinumab compared with placebo in treating moderate to severe atopic dermatitis (AD).
- Trial name
- ECZTRA 2 (NCT03160885)
- Testing
- Tralokinumab
- Where
- 115 sites in United States, Australia and 7 more
- Volunteers
- 794 people
- Sponsor
- LEO Pharma
Skin Laxity, Wrinkles
Feasibility Study: Evaluation of Ulthera Versus Thermage for Treating the Face and Neck
Up to 20 subjects will be enrolled and randomly assigned to one of two treatment groups. Group A will receive Ultherapy™ on the right side of the face and neck and Thermage on the left side.
- Trial name
- NCT01713985
- Testing
- Ulthera System Treatment, Thermage
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Ulthera, Inc
PKU, Tyrosinemias
GMP Case Studies of Tolerance, Safety and Acceptability in PKU and TYR
Three new protein substitutes have been developed to help with compliance in PKU and TYR patients, which are powdered protein substitutes, low in Phe and, low in Phe and Tyr respectively, with a mixed balance of glycomacropeptide…
- Trial name
- NCT05062226
- Testing
- PKU/TYR GMP Protein Substitute
- Where
- United Kingdom
- Volunteers
- 45 people
- Sponsor
- Nutricia UK Ltd
Hypertriglyceridemia, Hyperlipoproteinemia Type IV
Effect of Rosuvastatin on Triglyceride Levels in Mexican Hypertriglyceridemic Patients
The primary purpose of this trial is to determine if the treatment with rosuvastatin 10 and 20mg/day during 8 weeks in hypertriglyceridemic patients will reduce their triglyceride levels.
- Conditions
- Hypertriglyceridemia, Hyperlipoproteinemia Type IV, Hyperlipoproteinemia Type V, Hyperlipoproteinemia Type IIb
- Trial name
- NCT00473655
- Testing
- rosuvastatin
- Where
- 3 sites in Mexico
- Volunteers
- 334 people
- Sponsor
- AstraZeneca
Friedreich's Ataxia (FA)
A Study of SGT-212 Gene Therapy in Friedreich's Ataxia
This is a phase 1b, first in-human, open-label, dose-finding study investigating the safety and tolerability of SGT-212 in participants with Friedreich's ataxia (FA).
- Trial name
- FALCON (NCT07180355)
- Testing
- SGT-212
- Where
- 3 sites in United States
- Volunteers
- 10 people
- Sponsor
- Solid Biosciences Inc.
Hemophilia B
A Safety and Efficacy Study of a Recombinant Factor IX in Patients With Severe Hemophilia B
This study will examine the safety and efficacy of a Recombinant Coagulation Factor IX Albumin Fusion Protein (rIX-FP) for the control and prevention of bleeding episodes in subjects who have previously received factor…
- Trial name
- NCT01361126
- Testing
- Recombinant Coagulation Factor IX Albumin Fusion Protein
- Where
- 2 sites in Bulgaria, Israel
- Volunteers
- 17 people
- Sponsor
- CSL Behring
Retinitis Pigmentosa, Retinitis
Non-Interventional Long Term Follow-up Study of Participants Previously Enrolled in the RESTORE Study
This study will be conducted following Good Clinical Practice (GCP) and International Conference on Harmonization (ICH) guidelines.
- Conditions
- Retinitis Pigmentosa, Retinitis, Retinal Diseases, Eye Diseases
- Trial name
- REMAIN (NCT06162585)
- Testing
- Gene Therapy product-MCO-010
- Where
- 6 sites in United States, Puerto Rico
- Volunteers
- 18 people
- Sponsor
- Nanoscope Therapeutics Inc.
Leber's Hereditary Optic Neuropathy
Study With Idebenone in Patients With Chronic Vision Loss Due to Leber's Hereditary Optic Neuropathy (LHON)
The objective of the study is to determine whether administration of idebenone can shorten the time to improvement of visual acuity in patients with chronic vision loss due to LHON.
- Trial name
- NCT01495715
- Testing
- Idebenone
- Sponsor
- Santhera Pharmaceuticals
Hemophilia A
A Trial of the Safety and Efficacy of Single-Dose Administration of ANB-010 in Subjects With Hemophilia A
The goal of this multicenter, two-stage, open-label study is to investigate the safety, immunogenicity, and efficacy of ANB-010 in subjects with hemophilia A.
- Trial name
- EDELWEISS (NCT06185335)
- Testing
- ANB-010, dose 1, ANB-010, dose 2, ANB-010, dose 3
- Where
- 17 sites in Russia
- Volunteers
- 50 people
- Sponsor
- Biocad
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Junctional
INM-755 (Cannabinol) Cream for Treatment of Epidermolysis Bullosa
The purpose of this study is to evaluate the safety of INM-755 (cannabinol) cream and obtain preliminary evidence of efficacy in treating symptoms and healing wounds over a 28-day period in patients with epidermolysis bullosa…
- Conditions
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa, Junctional, Epidermolysis Bullosa Dystrophica, Kindler Syndrome
- Trial name
- NCT04908215
- Testing
- INM-755 (cannabinol) cream, Vehicle Cream
- Where
- 7 sites in France, Germany and 3 more
- Volunteers
- 19 people
- Sponsor
- InMed Pharmaceuticals Inc.
Hypochondroplasia
An Interventional Study of Infigratinib in Children With Hypochondroplasia
ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib at one of two doses…
- Trial name
- HCH (NCT06873035)
- Testing
- infigratinib 0.128 mg/kg/day, infigratinib 0.25 mg/kg/day
- Where
- 24 sites in United States, Australia and 8 more
- Volunteers
- 24 people
- Sponsor
- QED Therapeutics, a BridgeBio company
Cystic Fibrosis
A Phase 2 Study Evaluating Safety and Tolerability of RCT2100 (CFTR mRNA) in Healthy Participants and in Participants With CF
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
- Trial name
- NCT06237335
- Testing
- RCT2100, Ivacaftor
- Where
- 23 sites in United States, France and 3 more
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- ReCode Therapeutics
Conjunctivitis, Atopic Dermatitis
Observational Study of Conjunctivitis in the Setting of DUPIXENT® Treatment for Atopic Dermatitis
The primary objective of the study is to characterize the clinical phenotype(s) of DUPIXENT®-associated conjunctivitis events. The secondary objectives of the study are to characterize the course of conjunctivitis events during…
- Trial name
- NCT04287608
- Testing
- dupilumab
- Where
- 15 sites in United States
- Volunteers
- 50 people
- Sponsor
- Regeneron Pharmaceuticals
Atopic Dermatitis
A Study of KT-621 Administered Orally to Participants With Moderate to Severe Atopic Dermatitis
This phase 2b study is designed to evaluate the safety and efficacy of KT-621 in adult and adolescent participants with moderate-to-severe atopic dermatitis (AD), a common form of eczema.
- Trial name
- BROADEN2 (NCT07217015)
- Testing
- KT-621
- Where
- 61 sites in United States, Australia and 6 more
- Volunteers
- 200 people
- Sponsor
- Kymera Therapeutics, Inc.
Niemann-Pick Type C Disease
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease (NPC)
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of Niemann-Pick type C disease
- Trial name
- NCT07082725
- Testing
- Nizubaglustat
- Where
- 34 sites in United States, Argentina and 13 more
- Volunteers
- 72 people
- Sponsor
- Azafaros B.V.
Cystic Fibrosis
Phase 1, QT/QTC Interval Study in Healthy Subjects
This study is designed to evaluate the effect of multiple doses of lumacaftor in combination with ivacaftor on cardiac repolarization, as detected by QT/QTc interval corrected for heart rate in healthy subjects.
- Trial name
- NCT01910415
- Testing
- Lumacaftor, Ivacaftor, moxifloxacin hydrochloride
- Where
- Netherlands
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
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