Clinical trials: Genetic Diseases
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies
Single Ascending Dose Study in Participants With LCA10
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound…
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease
- Trial name
- NCT03872479
- Testing
- EDIT-101
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Editas Medicine, Inc.
Primary Immune Deficiency
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
- Trial name
- NCT04640142
- Testing
- Newnorm
- Where
- 23 sites in United States, Germany and 5 more
- Volunteers
- 50 people
- Sponsor
- Octapharma
Genetic Disease
Developing Protocols for Modelling of Genetic Diseases Using Induced Pluripotent Stem Cells
Recent advances have shown that cells from human blood, skin and urine samples can be reprogrammed to become stem cells. These are called induced Pluripotent Stem Cells (iPSCs) and share many characteristics with embryonic stem…
- Trial name
- NCT03612310
- Testing
- Skin biopsy/Urine Collection/Blood Sample Collection
- Where
- United Kingdom
- Volunteers
- 3,000 people
- Sponsor
- Sapna Vyas
Pruritus, Prurigo Nodularis
Study of the Long Term Safety of Serlopitant for the Treatment of Pruritus (Itch)
Study of the long term safety of serlopitant for the treatment of pruritus in adults.
- Conditions
- Pruritus, Prurigo Nodularis, Atopic Dermatitis, Psoriasis
- Trial name
- NCT03540160
- Testing
- 5 mg Serlopitant Tablets
- Where
- 85 sites in United States, Austria and 2 more
- Volunteers
- 558 people
- Sponsor
- Vyne Therapeutics Inc.
Atopic Dermatitis
Long-term Follow-up Study to Evaluate the Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD Inj
A multi-center, open, long-term follow-up study to Evaluate the Safety and Explore the Efficacy of Multiple Doses of FURESTEM-AD inj. for Moderate to Severe Chronic Atopic Dermatitis: 5-year Results from the K0104 Extension Study
- Trial name
- NCT04730024
- Testing
- Not applicable(observational study)
- Where
- South Korea
- Volunteers
- 102 people
- Sponsor
- Kang Stem Biotech Co., Ltd.
Lysosomal Storage Diseases, Metachromatic Leukodystrophy
OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD)
OTL-200 is a cryopreserved dispersion for infusion containing autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human…
- Trial name
- NCT04283227
- Testing
- OTL-200
- Where
- Italy
- Volunteers
- 6 people
- Sponsor
- Orchard Therapeutics
Niemann-Pick Disease, Type C
Adrabetadex to Treat Niemann-Pick Type C1 (NPC1) Disease
Due to different study designs, the sponsor separated Part C into this separate registration (NCT04958642), leaving Parts A/B in NCT02534844.
- Trial name
- NCT04958642
- Testing
- Adrabetadex
- Where
- 30 sites in United States, Australia and 7 more
- Volunteers
- 66 people
- Sponsor
- Mandos LLC
Duchenne Muscular Dystrophy
AFFINITY BEYOND: Anti-AAV8 Antibody Assessment Study of Males With DMD
This is an observational screening study to evaluate the prevalence of anti-adeno-associated serotype 8 (AAV8) antibodies in participants with Duchenne muscular dystrophy (DMD).
- Trial name
- NCT05683379
- Testing
- AAV8 DetectCDx
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- REGENXBIO Inc.
Hereditary Angioedema
OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)
The purpose of this study was to evaluate the safety and efficacy of donidalorsen in participants with HAE and effect of donidalorsen on the quality and pattern of HAE attacks and their impact on quality of life (QoL).
- Trial name
- NCT05139810
- Testing
- Donidalorsen
- Where
- 51 sites in United States, Belgium and 12 more
- Volunteers
- 91 people
- Sponsor
- Ionis Pharmaceuticals, Inc.
Alzheimer s Disease, Mild Cognitive Impairment (MCI)
Crownlands Observing Progression With Neurons Study
The CROWN-I Study is an observational study to learn about molecular features of Alzheimer's disease (AD) and mild cognitive impairment (MCI).
- Trial name
- CROWN-I (NCT07611396)
- Where
- United States
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Crownlands
Cystitis / Pyelonephritis / Cystic Fibrosis / Anthrax
Special Drug Use Investigation of Ciproxan Injection in Pediatrics
The objective in this study is collecting post-marketing information on the safety and efficacy of Ciproxan injection under the routine clinical practice.
- Trial name
- NCT02555059
- Testing
- Cipro (Ciprofloxacin, BAYQ3939)
- Where
- Japan
- Volunteers
- 48 people
- Sponsor
- Bayer
Hereditary Cancer-Predisposing Syndrome
Inherited Risk Evaluation Tool (InheRET): Identifying Patients at Increased Risk for Hereditary Disease
This study will evaluate the impact InheRET™, an online family history gathering and risk assessment reporting tool, has on facilitating National Comprehensive Cancer Network(NCCN) guideline compliant referrals for cancer genetic…
- Trial name
- NCT04510896
- Testing
- InheRET, Patient Acceptance, Physician Acceptance Survey
- Where
- 4 sites in United States
- Volunteers
- 2,232 people, healthy volunteers welcome
- Sponsor
- InheRET, Inc
Cystic Fibrosis
Safety and Efficacy of Inhaled OligoG CF-5/20 for the Treatment Cystic Fibrosis
Cystic fibrosis (CF) is a disease caused by a mutation in the gene that makes the cystic fibrosis transmembrane regulator protein.
- Trial name
- NCT00970346
- Testing
- OligoG CF-5/20
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- AlgiPharma AS
Behcet's Disease
Special Investigation in Patients With Intestinal Behcet's Disease (All Case Investigation)
This investigation was conducted to obtain the following information regarding the use of Adalimumab (Humira®) 40mg Syringe 0.8mL for Subcutaneous Injection in patients with Intestinal Behcet's Disease. 1.
- Trial name
- NCT01960790
- Volunteers
- 473 people
- Sponsor
- AbbVie
X-Linked Retinitis Pigmentosa
Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations
This study will evaluate the safety and efficacy of a recombinant adeno-associated virus vector (rAAV2tYF-GRK1-RPGR) in patients with X-linked retinitis pigmentosa caused by RPGR mutations.
- Trial name
- HORIZON (NCT03316560)
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 4 sites in United States
- Volunteers
- 29 people
- Sponsor
- Beacon Therapeutics
Alpha 1-Antitrypsin Deficiency
Phase II/III Study of an Alpha-1 Proteinase Inhibitor (Kamada-API) in Individuals With Alpha-1 Antitrypsin Deficiency
The primary purpose of this Phase II/III study is to demonstrate that Kamada-API, a new API concentrate manufactured by Kamada Ltd., is comparable to a currently marketed API product.
- Trial name
- Kamada API (NCT00460096)
- Testing
- Kamada-API
- Where
- 3 sites in United States
- Volunteers
- 50 people
- Sponsor
- Kamada, Ltd.
Phenylketonuria
A Study of PTC923 in Participants With Phenylketonuria
The main purpose of this trial is to evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in participants with phenylketonuria as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6…
- Trial name
- NCT05099640
- Testing
- PTC923
- Where
- 43 sites in United States, Australia and 13 more
- Volunteers
- 157 people
- Sponsor
- PTC Therapeutics
Atopic Dermatitis
Study to Evaluate the Efficacy, Safety and Pharmacokinetics of ASB17061 Capsules in Adult Subjects With Atopic Dermatitis
The purpose of this research study is to gather scientific information about the effectiveness of the study drug, ASB17061 capsules, when compared to placebo in adult subjects with atopic dermatitis.
- Trial name
- NCT01756898
- Testing
- 5 mg ASB17061, 10 mg ASB17061, 20 mg ASB17061
- Where
- 40 sites in United States
- Volunteers
- 370 people
- Sponsor
- Daiichi Sankyo
Gastric Adenocarcinoma
Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma
The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e.
- Trial name
- FIDES-03 (NCT04604132)
- Testing
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 47 people
- Sponsor
- Basilea Pharmaceutica
Infertility, Hereditary Diseases
Embryo Health Study
This study intends to determine the patients' perception and motivation to obtain additional information on their preimplantation embryos' risks of polygenic disorders.
- Trial name
- EHS (NCT04528498)
- Testing
- Preimplantation Genetic Testing
- Where
- United States
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Genomic Prediction Inc.
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