Clinical trials: Genetic Diseases, Inborn
Duchenne Muscular Dystrophy
Efficacy and Safety Study of WVE-210201 (Suvodirsen) With Open-label Extension in Ambulatory Patients With Duchenne Muscular Dystrophy
This is a Phase 2/3, multicenter, randomized, double-blind, placebo-controlled study with an open-label extension period to evaluate the safety and efficacy of WVE-210201 (suvodirsen) in ambulatory male pediatric patients with…
- Trial name
- DYSTANCE 51 (NCT03907072)
- Testing
- WVE-210201 (suvodirsen)
- Where
- 22 sites in United States, Belgium and 6 more
- Volunteers
- 6 people
- Sponsor
- Wave Life Sciences USA, Inc.
Cystic Fibrosis
Absorption and Safety With Sustained Use of RELiZORB Evaluation (ASSURE) Study
Protocol 0000498: Multicenter, open label study to evaluate the effect of sustained RELiZORB (immobilized lipase) cartridge use during enteral feeding on fat absorption, as well as safety and tolerability of sustained RELiZORB…
- Trial name
- ASSURE (NCT02750501)
- Testing
- RELiZORB (immobilized lipase) cartridge, Impact Peptide 1.5
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Alcresta Therapeutics, Inc.
Beta-Thalassemia
A Multiple Ascending Dose Study of 9MW3011 in Patients With Non-transfusion-dependent β-thalassemia
This is a phase Ib, randomized, double-blind, placebo-controlled, multiple ascending dose study . The objectives of the study are to evaluate the safety , tolerability, pharmacokinetics(PK), pharmacodynamics(PD), and…
- Trial name
- NCT06772766
- Testing
- 9MW3011
- Where
- 2 sites in China
- Volunteers
- 40 people
- Sponsor
- Mabwell (Shanghai) Bioscience Co., Ltd.
Autosomal Dominant Polycystic Kidney Disease
Short-term Renal Hemodynamic Effects of Tolvaptan in Subjects With Autosomal Dominant Polycystic Kidney Disease (ADPKD)
The purpose of the trial was to determine the short-term effects of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD) at various levels of renal function.
- Trial name
- NCT01336972
- Testing
- Tolvaptan
- Where
- Netherlands
- Volunteers
- 29 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
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
Dermatitis, Atopic
Intervention Study to Evaluate a Probiotic in Mild Atopic Dermatitis Young Patients
The aim of the study is to evaluate the beneficial effect of a probiotic preparation with an antiinflammatory and modulating activity on immunological processes, with positive results on different inflammatory and atopic…
- Trial name
- NCT02585986
- Testing
- Probiotic
- Where
- Spain
- Volunteers
- 51 people, healthy volunteers welcome
- Sponsor
- Biopolis S.L.
Epilepsy, Dravet Syndrome
An Open Label Extension Study of Cannabidiol (GWP42003-P) in Children and Adults With Dravet or Lennox-Gastaut Syndromes
To investigate the potential antiepileptic effects of cannabidiol (GWP42003-P) in children and adults with Dravet or Lennox-Gastaut syndromes.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- GWPCARE5 (NCT02224573)
- Testing
- GWP42003-P
- Volunteers
- 681 people
- Sponsor
- GW Research Ltd
Atopic Dermatitis
A Study to Determine the Efficacy of ZPL-3893787 in Subjects With Atopic Dermatitis
This was a randomized, double blind, placebo controlled, parallel group study in approximately 90 subjects with moderate-severe AD Eczema Area and Severity Index (EASI) ≥12 and ≤ 48 (0-72 scale).
- Trial name
- NCT02424253
- Testing
- ZPL-3893787
- Where
- 17 sites in Belgium, Germany and 2 more
- Volunteers
- 98 people
- Sponsor
- Ziarco Pharma Ltd
Cystic Fibrosis
Study of Dose Escalation of Liposomal Amikacin for Inhalation (ARIKAYCE™) - Extension Phase
A major factor in the respiratory health of cystic fibrosis (CF) patients is acquisition of chronic Pseudomonas (P.) aeruginosa infections. The infection rate with P.
- Trial name
- NCT03905642
- Testing
- Arikayce™
- Where
- 11 sites in Belgium, Hungary and 5 more
- Volunteers
- 49 people
- Sponsor
- Insmed Incorporated
Gaucher Disease, Type 1, Gaucher Disease, Type 3
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by deficient activity of the enzyme acid β-glucosidase, causing glucosylceramide to accumulate within macrophages and leading to hepatosplenomegaly, anemia…
- Trial name
- NCT05447494
- Testing
- Low-dose CAN103, High-dose CAN103
- Where
- China
- Volunteers
- 40 people
- Sponsor
- CANbridge (Suzhou) Bio-pharma Co., Ltd.
Hemophilia A
Study of Pharmacokinetics, Efficacy and Safety of a Recombinant and Protein-Free Factor VIII (rAHF-PFM) in Hemophilia A Patients - A Continuation of Clinical Study 069901
The purpose of this study is to evaluate whether rAHF-PFM is safe and effective in the treatment of hemophilia A subjects. The study consists of 2 parts: Part 1 is a pharmacokinetic and safety study, and Part 2 is an evaluation…
- Trial name
- NCT00157053
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 24 sites in United States, Austria and 7 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Inherited Epidermolysis Bullosa
Oleogel-S10 in Wound Healing of Inherited Epidermolysis Bullosa (BEB-10)
The purpose of this study was to compare intra-individually the reepithelialization of skin lesion(s) in inherited Epidermolysis bullosa (either 1 wound ≥10 cm2 and ≤200 cm2 in size divided in 2 equal halves or 2 comparable…
- Trial name
- NCT01294241
- Testing
- Oleogel-S10, Mepilex® soft silicone faced polyurethane foam dressing
- Where
- Germany
- Volunteers
- 10 people
- Sponsor
- Birken AG
Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema
Phase 2 Randomized Double-blind Study to Assess Topical Zabalafin Hydrogel Vs Vehicle in Mild to Moderate AD.
A Phase 2b study investigating the efficacy of zabalafin in people with mild to moderate atopic dermatitis (eczema).
- Conditions
- Atopic Dermatitis (Eczema), Atopic Dermatitis Eczema, Atopic Dermatitis, Atopic Dermatitis (AD)
- Trial name
- CLEAR-AD1 (NCT06855745)
- Testing
- zabalafin
- Where
- 12 sites in Australia
- Volunteers
- 72 people
- Sponsor
- Alphyn Biologics
Sickle Cell Disease
Study of Intravenous GMI-1070 in Adults With Sickle Cell Disease
This Phase 1/Phase 2 study will evaluate GMI-1070, a pan-selectin inhibitor, in adults with stable sickle cell disease. The study will assess safety, pharmacokinetics, and microvascular effects of intravenous GMI-1070 in the…
- Trial name
- NCT00911495
- Testing
- GMI-1070
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- GlycoMimetics Incorporated
Atopic Dermatitis
Study to Investigate the Effect of Rocatinlimab (AMG 451) on the Pharmacokinetics of Multiple Cytochrome P450 (CYP450) Substrates in Participants With Moderate to Severe Atopic Dermatitis
The primary objective of the study is to evaluate the pharmacokinetics (PK) of multiple cytochrome P450 (CYP450) substrates alone and in combination with rocatinlimab in participants with moderate to severe atopic dermatitis (AD).
- Trial name
- NCT05891119
- Testing
- Caffeine, Metoprolol, Midazolam
- Where
- 7 sites in United States
- Volunteers
- 21 people
- Sponsor
- Amgen
Hemophilia a, Hemophilia B
The Long-term Safety and Efficacy of SerpinPC in Subjects with Hemophilia Who Completed a Sponsored SerpinPC Clinical Trial
The purpose of the study is to evaluate the efficacy, safety, tolerability and pharmacokinetic (PK) profile of prophylactic SerpinPC in participants with hemophilia
- Conditions
- Hemophilia a, Hemophilia B, Hemophilia a with Inhibitor, Hemophilia B with Inhibitor
- Trial name
- PRESent-6 (NCT06568302)
- Testing
- SerpinPC
- Where
- 2 sites in Georgia, Moldova
- Volunteers
- 15 people
- Sponsor
- ApcinteX Ltd
ADPKD (Autosomal Dominant Polycystic Kidney Disease)
A Study to Investigate the Effects of JMKX003142 on QTc Interval in Healthy Adults
The primary objective of this study is to assess the effect of a therapeutic and supratherapeutic oral dose of JMKX003142 on the corrected cardiac QT interval relative to a placebo in healthy participants
- Trial name
- NCT07470177
- Testing
- JMKX003142 tablets
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Jemincare
Huntington Disease
A Randomized Study of SPK-10001 Gene Therapy in Participants With Huntington's Disease
The main goal of this study is to evaluate the safety, tolerability, and preliminary efficacy of SPK-10001 in participants with Huntington's Disease.
- Trial name
- NCT06826612
- Testing
- SPK-10001
- Where
- 5 sites in United States
- Volunteers
- 53 people
- Sponsor
- Hoffmann-La Roche
Solid Tumors
A Safety, Pharmacokinetic and Pharmacodynamic Study of Kevetrin in Patients With Advanced Solid Tumors
In the laboratory, Kevetrin activates p53, a tumor suppressor protein that has an important role in protecting the body. p53 functions by activating proteins that repair DNA and kill cells that have genetic mutations such as in…
- Trial name
- NCT01664000
- Testing
- thioureidobutyronitrile
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Cellceutix Corporation
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