Clinical trials: Congenital Anomaly
Erythropoietic Protoporphyria
Phase III Confirmatory Study in Erythropoietic Protoporphyria
This is a randomized placebo-controlled study to be conducted in two parallel study arms for a six month period (three doses). Between 75 and 100 eligible patients will be enrolled.
- Trial name
- NCT01605136
- Testing
- Afamelanotide
- Where
- 7 sites in United States
- Volunteers
- 93 people
- Sponsor
- Clinuvel Pharmaceuticals Limited
Thyroid Eye Disease
An Efficacy, Safety, and Tolerability Study of VRDN-003 in Participants With Active Thyroid Eye Disease (TED)
This is a clinical trial assessing the efficacy, safety, and tolerability of an investigational drug, VRDN-003, in participants with active Thyroid Eye Disease (TED).
- Trial name
- REVEAL-1 (NCT06625411)
- Testing
- VRDN-003
- Where
- 48 sites in United States, Germany and 3 more
- Volunteers
- 132 people
- Sponsor
- Viridian Therapeutics, Inc.
Severe Hemophilia A
Safety and Pharmacokinetics of Subcutaneous Injection of OCTA101 in Adult Patients With Severe Hemophilia A
This Phase 1/2 study will be a dose escalation study in adults in 5 cohorts (named cohorts 1, 2, 3, 5 and 6), with the main purpose to assess the safety of subcutaneous injection of OCTA101 (a human-cl rhFVIII and recombinant…
- Trial name
- NCT04046848
- Testing
- OCTA101
- Where
- Bulgaria
- Volunteers
- 36 people
- Sponsor
- Octapharma
Hemophilia A, Hemophilia B
Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors.
- Trial name
- MATCHBOX (NCT01921855)
- Testing
- BAY Factor VII (BAY86-6150)
- Where
- 4 sites in Poland, South Africa and 1 more
- Volunteers
- 16 people
- Sponsor
- Bayer
Hydrocephalus, Nervous System Diseases
Study of Shunt Flow Sensor Accuracy in Extra-ventricular Drains.
The study hypothesis is that an ultrasonic flow sensor can accurately measure flow in hydrocephalic shunts. The ultrasonic sensor will measure cerebrospinal fluid drainage in hydrocephalus patients with external ventriculostomies…
- Conditions
- Hydrocephalus, Nervous System Diseases, Infant, Newborn Diseases
- Trial name
- NCT01108965
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Transonic Systems Inc.
Cystic Fibrosis
Study of Denufosol Inhalation Solution in Patients With Mild to Moderate Cystic Fibrosis Lung Disease
The purpose of this trial is to evaluate the effects of a single dose of denufosol versus placebo on mucociliary clearance in patients with mild to moderate CF lung disease
- Trial name
- NCT00425165
- Testing
- denufosol tetrasodium (INS37217) Inhalation Solution
- Volunteers
- 6 people
- Sponsor
- Merck Sharp & Dohme LLC
ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum
Evaluation of Safety, Tolerability, and Efficacy of INZ-701 in Adults With ABCC6 Deficiency Causing PXE
The purpose of this study is to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics PD) of multiple ascending doses of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) recombinant…
- Conditions
- ATP-Binding Cassette Subfamily C Member 6 Deficiency, Pseudoxanthoma Elasticum, Generalized Arterial Calcification of Infancy
- Trial name
- NCT05030831
- Testing
- INZ-701
- Where
- 2 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Inozyme Pharma
Primary Hyperoxaluria, Primary Hyperoxaluria Type 1 (PH1)
A Study of Lumasiran in Infants and Young Children With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in infants and young children with confirmed primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-B (NCT03905694)
- Testing
- Lumasiran
- Where
- 9 sites in United States, France and 3 more
- Volunteers
- 18 people
- Sponsor
- Alnylam Pharmaceuticals
Rheumatoid Arthritis, Osteoarthritis
A Single Centre Study to Assess the Long-term Performance of the Pinnacle™ Cup With a Ceramic-on-ceramic Bearing in Primary Total Hip Replacement
The purpose of this study is to monitor the performance of the Pinnacle™ Cup with a ceramic-on-ceramic bearing combination in the treatment of patients with hip joint disease requiring a total hip replacement.
- Conditions
- Rheumatoid Arthritis, Osteoarthritis, Post-traumatic Arthritis, Collagen Disorders
- Trial name
- NCT00872222
- Testing
- Pinnacle™ Acetabular System
- Where
- Czechia
- Volunteers
- 98 people
- Sponsor
- DePuy International
Atopic Dermatitis
Safety, Tolerance and Pharmacokinetics Clinical Study of VC005 in Healthy Subjects and Patients with Mild to Moderate Atopic Dermatitis
This study is a single center, randomized, double-blind, Vehicle controlled,, single and multiple dose clinical study.
- Trial name
- NCT06009094
- Testing
- VC005
- Where
- China
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Jiangsu vcare pharmaceutical technology co., LTD
Alpha 1-Antitrypsin Deficiency, Emphysema
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
- Trial name
- ELEVAATE (NCT05856331)
- Testing
- SAR447537, Zemaira
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 99 people
- Sponsor
- Sanofi
Hypophosphatasia
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
- Trial name
- CHESTNUT (NCT06079372)
- Testing
- ALXN1850, asfotase alfa
- Where
- 21 sites in United States, Argentina and 5 more
- Volunteers
- 43 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Spina Bifida
Establishing the Preliminary Utility of a Novel Pediatric Manual Mobile Standing Wheelchair
To establish the preliminary utility of a novel pediatric manual mobile standing wheelchair (PedMMSWC).
- Trial name
- NCT07290556
- Testing
- PedMMSWC Wheelchair User Testing, PedMMSWC Simulated Home, School, Community Environment
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Optimal Mobility, Inc
Complication of Renal Dialysis, End Stage Renal Disease
A Study Testing the Use of a Perivascular Sirolimus Formulation (Sirogen) in ESRD Patients Undergoing AV Fistula Surgery
The primary study objective is to evaluate the benefit of the Sirolimus eluting Collagen implant (SeCI; Sirogen), a single dose prophylactic treatment delivered intraoperatively at the time of surgical creation of an…
- Conditions
- Complication of Renal Dialysis, End Stage Renal Disease, End Stage Kidney Disease, ESRD
- Trial name
- ACCESS2 (NCT05425056)
- Testing
- Sirolimus
- Where
- 22 sites in United States, United Kingdom
- Volunteers
- 136 people
- Sponsor
- Vascular Therapies, Inc.
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of Long-term Treatment With TEZ/IVA in CF Participants With an F508del CFTR Mutation
This study evaluates the long-term safety and tolerability of tezacaftor in combination with ivacaftor (TEZ/IVA) in participants with cystic fibrosis (CF) aged 6 years and older, homozygous or heterozygous for the F508del…
- Trial name
- NCT03537651
- Testing
- TEZ/IVA, IVA
- Where
- 55 sites in United States, Australia and 9 more
- Volunteers
- 130 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Arteriovenous Fistula
An Efficacy and Safety Study of SRM003 in the Treatment of Subjects Undergoing Creation of an Arteriovenous Fistula to Facilitate Hemodialysis Access
A study to evaluate the efficacy of SRM003 treatment versus participating sites' standard practice treatment in improving the rate of AVF maturation and use in subjects with end-stage renal disease undergoing surgery for creation…
- Trial name
- NCT01806545
- Testing
- SRM003, Participating Site's standard practice
- Where
- 47 sites in United States
- Volunteers
- 64 people
- Sponsor
- Shire
Retinitis Pigmentosa
Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis Pigmentosa
This study is a multicenter trial performed in Japan, consisting of a comparative study period and a continuous administration period.
- Trial name
- NCT01786395
- Testing
- UF-021
- Where
- 34 sites in Japan
- Volunteers
- 202 people
- Sponsor
- R-Tech Ueno, Ltd.
Cystic Fibrosis
Study to Evaluate the Safety and Efficacy of CTP-656 in Patients With Cystic Fibrosis With CFTR Gating Mutations
This study will evaluate the efficacy and safety of CTP-656 in patients with cystic fibrosis (CF) who have a cystic fibrosis transmembrane conductance regulator (CFTR) gating mutation.
- Trial name
- NCT02971839
- Testing
- VX-561, IVA
- Where
- 14 sites in United States
- Volunteers
- 11 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Gaucher Disease
Lentiviral Vector Gene Therapy - The Guard1 Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease
This was a multinational, open-label study to assess the safety and efficacy of AVR-RD-02 in approximately 8 to 16 subjects (male or female) who are ≥18 and ≤50 years of age and post pubertal at Screening with a confirmed…
- Trial name
- NCT04145037
- Testing
- AVR-RD-02
- Where
- 5 sites in United States, Canada
- Volunteers
- 8 people
- Sponsor
- AVROBIO
Limb-Girdle Muscular Dystrophies, Facioscapulohumeral Muscular Dystrophy
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
The purpose of this study is to assess the safety and biological activity of ATYR1940 in participants with LGMD2B and FSHD.
- Trial name
- FSHD (NCT02579239)
- Testing
- ATYR1940
- Where
- 6 sites in United States, Denmark and 1 more
- Volunteers
- 18 people
- Sponsor
- aTyr Pharma, Inc.
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