Clinical trials: Multiple Congenital Anomaly
Huntington's Disease
Study to Measure Cerebrospinal Fluid Mutant Huntingtin Protein in Participants With Early Manifest Stage I or Stage II Huntington's Disease
The study is designed as a multi-site, prospective, 15-month longitudinal, cohort study measuring CSF mHTT in participants with early manifest Stage I or Stage II Huntington's Disease (HD).
- Trial name
- NCT03664804
- Testing
- No Study Drug was Administered in this Study
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 95 people
- Sponsor
- Hoffmann-La Roche
Advanced Nonhaematologic Malignancies
Safety, Efficacy, and Tolerability of BOS172738 in Patients With Advanced Rearranged During Transfection (RET) Gene-Altered Tumors
This study will be conducted to assess the safety and tolerability of BOS172738 when administered to patients with advanced solid tumors with rearranged during transfection (RET) gene alterations and also to establish the maximum…
- Trial name
- NCT03780517
- Testing
- BOS172738
- Where
- 21 sites in United States, Belgium and 5 more
- Volunteers
- 117 people
- Sponsor
- Boston Pharmaceuticals
Huntington Disease
An Exploratory Clinical Trial in Early Stage Huntington's Disease Patients With SEN0014196
The primary objective of this study is to provide biological samples from patients with Huntington's disease to allow characterisation of the pharmacological mechanism of action of SEN0014196.
- Trial name
- PADDINGTON (NCT01485952)
- Testing
- SEN0014196 (Low Dose), SEN0014196 (High Dose)
- Where
- Germany
- Volunteers
- 55 people
- Sponsor
- Siena Biotech S.p.A.
Alpha 1-Antitrypsin Deficiency
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver…
- Trial name
- NCT03946449
- Testing
- ARO-AAT
- Where
- 4 sites in Austria, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Arrowhead Pharmaceuticals
Atopic Dermatitis
Study Comparing Product 0405 to a Vehicle Control in the Treatment of Mild to Moderate Atopic Dermatitis in Pediatric Subjects
The purpose of this study is to evaluate the safety and efficacy of product 0405 in pediatric subjects with Mild to Moderate Atopic Dermatitis.
- Trial name
- NCT02176538
- Testing
- Product 0405
- Sponsor
- Fougera Pharmaceuticals Inc.
Basal Cell Carcinoma, Gorlin Syndrome
Efficacy, Safety and Pharmacokinetics of Oral LDE225 in Treatment of Patients With Nevoid Basal Cell Carcinoma Syndrome (NBCCS)
This was a phase II, double-blind, randomized, proof-of-concept, dose-ranging trial evaluating the efficacy, safety and pharmacokinetics of oral LDE225 in treatment of adult patients with NBCCS.
- Conditions
- Basal Cell Carcinoma, Gorlin Syndrome, Nevoid Basal Cell Carcinoma Syndrome
- Trial name
- NCT01350115
- Testing
- LDE225
- Where
- 6 sites in Austria, Belgium and 2 more
- Volunteers
- 10 people
- Sponsor
- Novartis Pharmaceuticals
Atopic Dermatitis
Dupilumab in Japanese Patients With Atopic Dermatitis
Primary Objective: To evaluate the efficacy of dupilumab administered concomitantly with topical corticosteroids (TCS) Secondary Objective: To evaluate the efficacy of dupilumab administered concomitantly with TCS.
- Trial name
- NCT04678882
- Testing
- Dupilumab SAR231893
- Where
- 19 sites in Japan
- Volunteers
- 62 people
- Sponsor
- Sanofi
Adenocarcinoma, Adenocystic Carcinoma
The Registry of Oncology Outcomes Associated With Testing and Treatment
This study is to collect and validate regulatory-grade real-world data (RWD) in oncology using the novel, Master Observational Trial construct. This data can be then used in real-world evidence (RWE) generation.
- Conditions
- Adenocarcinoma, Adenocystic Carcinoma, Anal Cancer, Appendix Cancer
- Trial name
- ROOT (NCT04028479)
- Testing
- Biomarker Testing (L), Systemic Treatment (T), Patient Reported Outcomes (P)
- Where
- 2 sites in United States
- Volunteers
- 167 people
- Sponsor
- Taproot Health
Healthy Volunteers, Down Syndrome
Leucettinib-21 First-in-Human Phase 1 in Healthy Volunteers and Subjects With Down Syndrome and Alzheimer's Disease
Leucettinib-21 First-in-Human Phase 1 Study in 6 Parts: Single (Part 1 and 5) and Multiple (Part 3 and 6) Ascending Doses, and Food-Effect (Part 2) in Healthy Subjects, and Single Dose (Part 4) in People with Down Syndrome (DS)…
- Conditions
- Healthy Volunteers, Down Syndrome, Alzheimer's Disease
- Trial name
- LEUCETTA (NCT06206824)
- Testing
- Leucettinib-21
- Where
- France
- Volunteers
- 152 people, healthy volunteers welcome
- Sponsor
- Perha Pharmaceuticals
Cytomegalovirus Infections, HIV Infections
A Phase I Pharmacokinetic Study in HIV-Positive Subjects of Oral Ganciclovir and Concomitant Antiretroviral Zidovudine and Didanosine
To determine whether there is a pharmacokinetic drug interaction between oral ganciclovir and oral zidovudine (AZT) and between oral ganciclovir and oral didanosine (ddI).
- Trial name
- NCT00002096
- Testing
- Zidovudine, Didanosine, Ganciclovir
- Where
- 3 sites in United States
- Volunteers
- 24 people
- Sponsor
- Hoffmann-La Roche
Glanzmann Thrombasthenia
A Phase 1/2 Study to Investigate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Glanzmann Thrombasthenia
The goal of this clinical trial is to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of HMB-001 in Participants with Glanzmann Thrombasthenia.
- Trial name
- NCT06211634
- Testing
- HMB-001
- Where
- 17 sites in United States, Belgium and 4 more
- Volunteers
- 57 people
- Sponsor
- Hemab ApS
Cystic Fibrosis (CF)
Safety, Tolerability and Efficacy of MP-376 Given for 28 Days to Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- NCT00677365
- Testing
- MP-376
- Where
- 50 sites in United States, Germany and 1 more
- Volunteers
- 151 people
- Sponsor
- Amgen
Congenital Bleeding Disorder, Haemophilia B
Safety and Efficacy of NNC-0156-0000-0009 in Haemophilia B Patients
This trial is conducted in Africa, Asia, Europe, Japan and North America. The aim of this trial is to evaluate the safety and efficacy, including pharmacokinetics (the rate at which the body eliminates the trial drug), of…
- Trial name
- paradigm™ 2 (NCT01333111)
- Testing
- nonacog beta pegol
- Where
- 43 sites in United States, Canada and 13 more
- Volunteers
- 74 people
- Sponsor
- Novo Nordisk A/S
Kidney Disease
A Study to Evaluate the Safety, Tolerability, and Effects on Blood and Urine Markers of Single Ascending Dose of GSK4771261 in Healthy Participants and Participants With Autosomal Dominant Polycystic Kidney Disease
This is a study where a new drug, called GSK4771261 is being tested. Neither the study doctors, study staff or participants will be aware of what treatment is being given.
- Trial name
- NCT06734234
- Testing
- GSK4771261
- Where
- 23 sites in United States, Belgium and 6 more
- Volunteers
- 86 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
MELAS Syndrome, Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like Episodes (MELAS)
A Study to Assess TTI-0102 vs Placebo in MELAS Patients
This is a randomized, double-blind, placebo-controlled study. Prior to treatment, patients will undergo a screening visit. If eligible, each subject will return for a Day 1 visit and will receive their first dose of…
- Trial name
- NCT06644534
- Testing
- TTI-0102
- Where
- 2 sites in France, Netherlands
- Volunteers
- 9 people
- Sponsor
- Thiogenesis Therapeutics, Inc.
Cardiomyopathy, Hypertrophic, Cardiomyopathy, Hypertrophic, Familial
Open-Label Study of Perhexiline in Patients With Hypertrophic Cardiomyopathy and Moderate to Severe Heart Failure
The purpose of this study is to evaluate the effect of perhexiline on exercise performance (efficacy) and safety in patients with hypertrophic cardiomyopathy and moderate-to-severe heart failure following dosing for 16 weeks.
- Trial name
- NCT02862600
- Testing
- Perhexiline, Use of bioanalytical assay to monitor plasma levels of perhexiline
- Where
- 11 sites in United States
- Volunteers
- 35 people
- Sponsor
- Heart Metabolics Limited
Cystic Fibrosis
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
This is a Phase 2 open-label, dose-escalation study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 with and without ivacaftor in patients with CF with at least one G542X allele.
- Trial name
- NCT04135495
- Testing
- ELX-02, Ivacaftor
- Where
- 10 sites in United States, Canada
- Volunteers
- 17 people
- Sponsor
- Eloxx Pharmaceuticals, Inc.
Fabry Disease
Safety and Efficacy Study of Several Replagal Dosing Regimens on Cardiac Function in Adults With Fabry Disease
The purpose of this study is to compare the safety and effectiveness of various doses of Replagal in patients with cardiomyopathy due to Fabry disease.
- Trial name
- NCT00864851
- Testing
- Replagal
- Where
- 12 sites in United States, Australia and 6 more
- Volunteers
- 44 people
- Sponsor
- Shire
Acute Intermittent Porphyria
A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)
The purpose of this study is to evaluate the safety and tolerability of givosiran (ALN-AS1) in AIP patients as well as to characterize pharmacokinetics (PK) and pharmacodynamics (PD) of ALN-AS1 in AIP patients.
- Trial name
- NCT02452372
- Testing
- givosiran (ALN-AS1), Sterile Normal Saline (0.9% NaCl)
- Where
- 6 sites in United States, Sweden and 1 more
- Volunteers
- 40 people
- Sponsor
- Alnylam Pharmaceuticals
Lax Skin
Renuvion APR Device to Improve the Appearance of Lax Tissue in the Neck and Submental Region
This is a prospective, multi-center, multi-phase, evaluator-blinded study of subjects undergoing a procedure with the Renuvion APR Device to improve the appearance of lax tissue in the neck and submental region.
- Trial name
- NCT04146467
- Testing
- Renuvion APR Device
- Where
- 6 sites in United States
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Apyx Medical