Clinical trials: Gene Therapy
Therapy Related Myelodysplastic Syndrome and Therapy Related Acute Myeloid Leukemia
SRSF2 Gene Mutation in Patients With t-MDS/AML
To detect SRSF2 gene mutation by polymerase chain reaction (PCR) in the two types of t-MDS/AML which recognized in the WHO classification.
- Trial name
- NCT03894852
- Testing
- PCR and cytogenetics
- Where
- 2 sites in Egypt
- Volunteers
- 139 people
- Sponsor
- Zeinab Albadry Mohammed Zahran
Cancer
Immunotherapy Treating GI Cancer
The primary objectives are to evaluate the safety and efficacy of infusion of autologous gastro-intestinal (GI) cancer antigen-specific engineered immune effector cells (EIE).
- Trial name
- NCT03614650
- Testing
- engineered immune cells
- Where
- China
- Volunteers
- 100 people
- Sponsor
- Shenzhen Geno-Immune Medical Institute
X-Linked Retinitis Pigmentosa
A Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Caused by RPGR Mutations (SKYLINE)
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
- NCT06333249
- Testing
- rAAV2tYF-GRK1-RPGR
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Beacon Therapeutics
Chronic Granulomatous Disease X-linked (X-CGD)
Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD
This is a phase II exploratory study conducted to evaluate the safety and efficacy of the combination of Ibuprofen, G-CSF and Plerixafor as stem cell mobilization regimen in patients affected by X-CGD.
- Trial name
- XCGD-MOBI (NCT03055247)
- Testing
- Ibuprofen, Myelostim, Mozobil
- Where
- 2 sites in Italy
- Volunteers
- 3 people
- Sponsor
- IRCCS San Raffaele
Obesity & Overweight, Obesity
Genetic, Microbiological and Behavioral Factors in Obesity
This study will assess the impact of genetic markers, microbiological (microbiome) and behavioral factors on tolerance, adherence and effectiveness of dual GLP1/GIP RA in the treatment of obesity.
- Conditions
- Obesity & Overweight, Obesity, Genetics
- Trial name
- OPTIMIZE-GEN (NCT07650149)
- Testing
- genetic polymorphism, gut microbiome
- Where
- 2 sites in Poland
- Volunteers
- 200 people
- Sponsor
- Jan Kochanowski University
DLBCL, MYD88 Gene Mutation
Ibrutinib Combined With Rituximab for Treatment of Relapsed Refractory MYD88 and CD79A/B (or CD79B Alone) DLBCL Who Have Received at Least Two Prior Therapies
The purpose of this study is to evaluate the efficacy and safety of Ibrutinib Combined With Rituximab in Relapsed Refractory MYD88 and CD79A/B (or CD79B Alone) DLBCL Who Have Received at Least Two Prior Therapies.
- Conditions
- DLBCL, MYD88 Gene Mutation, CD79A Gene Mutation, CD79B Gene Mutation
- Trial name
- NCT04994626
- Testing
- Ibrutinib Combined With Rituximab
- Volunteers
- 20 people
- Sponsor
- Chinese Academy of Medical Sciences
Stage I Breast Cancer, Stage II Breast Cancer
Niraparib + Dostarlimab In BRCA Mutated Breast Cancer
This research study involves pre-operative therapy that is specifically targeted for breast cancer in individuals with BRCA and PALB2 mutations.
- Conditions
- Stage I Breast Cancer, Stage II Breast Cancer, Stage III Breast Cancer, Breast Cancer
- Trial name
- NCT04584255
- Testing
- Niraparib, Dostarlimab
- Where
- 8 sites in United States
- Volunteers
- 66 people
- Sponsor
- Dana-Farber Cancer Institute
HIV Infection
Analysis of Genetic Factors Which May Influence the Course of HIV Infection
Patients with HIV an have a variable course of their infection. We are trying to identify factors which worsen or ameliorate the course of the disease.
- Trial name
- NCT00159068
- Where
- Israel
- Volunteers
- 100 people
- Sponsor
- Deborah Rund
Duchenne Muscular Dystrophy
Treatment of a Single Patient With CRD-TMH-001
The study is a single patient study intended to understand the effects of a gene-editing therapeutic to treat a rare mutation of Duchenne muscular dystrophy.
- Trial name
- NCT05514249
- Testing
- CRD-TMH-001
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Cure Rare Disease, Inc
Neovascular Age-Related Macular Degeneration (nAMD) Wet AMD
Ixoberogene Soroparvovec (Ixo-vec) Contralateral Dosing Study in Participants With Neovascular Age-related Macular Degeneration
The purpose of this study is to evaluate safety, effectiveness and durability of a gene therapy called Ixo-vec (Ixoberogene soroparvovec) when administered to the contralateral (second) eye of adult participants (≥ 50 years of…
- Trial name
- NCT07630649
- Testing
- Ixo-vec
- Where
- 12 sites in United States
- Volunteers
- 15 people
- Sponsor
- Adverum Biotechnologies, Inc.
Retinitis Pigmentosa
Promising ROd-cone DYstrophy Gene therapY
This is a two-step, multicenter, Phase I/II study including an open-label dose-escalation phase (Step 1) and a three-arm, controlled, double-masked, randomized extension phase (Step 2), in subjects with advanced RCD due to a…
- Trial name
- PRODYGY (NCT05748873)
- Testing
- SPVN06
- Where
- 6 sites in United States, France
- Volunteers
- 33 people
- Sponsor
- SparingVision
Coronary Arterial Disease (CAD)
The Switching Antiplatelet-9 (SWAP-9) Study
The purpose of this study is to compare the pharmacodynamic effects of ABCD-GENE guided vs. unguided de-escalation strategies among patients on dual antiplatelet therapy (DAPT) following percutaneous coronary intervention (PCI).
- Trial name
- SWAP-9 (NCT06588595)
- Testing
- Prasugrel/Ticagrelor monotherapy or aspirin plus clopidogrel, Aspirin plus clopidogrel
- Where
- United States
- Volunteers
- 90 people
- Sponsor
- University of Florida
Pancreatic Tubular Adenocarcinoma
Influence of Gemcitabine Treatment on Immunological and Serological Profile in Patients With Pancreatic Cancer
Observational study of influence of gemcitabine treatment on serological and immunological status and gene expression profile in patients with pancreatic tubular carcinoma after tumor resection.
- Trial name
- GEMSTHER (NCT00947102)
- Testing
- Observation
- Where
- Slovenia
- Sponsor
- Blood Transfusion Centre of Slovenia
Relapsed Neuroblastoma, Refractory Neuroblastoma
NKG2D.Zeta-NK Cell Conditioning With C7R.GD2.CAR-T Cells for Patients With Relapsed or Refractory Osteosarcoma or Neuroblastoma
The purpose of this study is to find the largest safe dose of i15.NKG2D.zeta-NK cells in combination with C7R.GD2.CAR-T cells, and additionally to evaluate how long they can be detected in patients' blood and what affect they…
- Conditions
- Relapsed Neuroblastoma, Refractory Neuroblastoma, Relapsed Osteosarcoma, Refractory Osteosarcoma
- Trial name
- NCT07211737
- Testing
- i15.NKG2D.zeta NK cells and C7R.GD2.CARTs cells
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- Baylor College of Medicine
Clear Cell RCC
A Clinical Gene Therapy Study With Hematopoietic Stem Cells for the Treatment, With Single Dose of Temferon, of Patients Suffering From Metastatic Renal Cell Carcinoma
This is an open label, single-centre phase 1/2 study involving a single dose of Temferon, an investigational Advanced Therapy Medicinal Product (ATMP), to treat patients with metastatic clear cell renal cell carcinoma (RCC) with…
- Trial name
- TEM-GU (NCT06716853)
- Testing
- Temferon, Pembrolizumab, Cabozantinib
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- Genenta Science
Myelofibrosis
Clinical Trail to Evaluate the Effect of Long-term Treatment With Gecacitinib on Myelofibrosis and Gene Mutation Levels
This study was an observational study, and about 40 patients with intermediate and high risk myelofibrosis who had participated in any previous clinical study of Gecacitinib hydrochloride and were still receiving Gecacitinib…
- Trial name
- NCT07342712
- Where
- China
- Volunteers
- 40 people
- Sponsor
- First Affiliated Hospital of Zhejiang University
Angelman Syndrome
A Phase 1/2 Study of the Safety and Efficacy of MVX-220 in Angelman Syndrome
The purpose of this study is to evaluate the safety and efficacy of MVX-220 gene therapy in children and adults with Angelman syndrome with UBE3A gene deletion, uniparental disomy, or imprinting center defect genotypes.
- Trial name
- ASCEND-AS (NCT07181837)
- Testing
- MVX-220
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- MavriX Bio, LLC
Gaucher Disease, Type 1
Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular…
- Trial name
- NCT00954460
- Testing
- velaglucerase alfa
- Where
- 32 sites in United States
- Sponsor
- Shire
Non Small Cell Lung Cancer, T790M
Osimertinib for Russian EGFR T790M Mutation-positive NSCLC Patients Who Progressed on or After EGFR TKI Therapy
The purpose of this study is to assess safety of Osimertinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with progression during or after therapy with a prior EGFR tyrosine kinase inhibitor…
- Conditions
- Non Small Cell Lung Cancer, T790M, EGFR Gene Mutation
- Trial name
- TRUST (NCT05314296)
- Where
- Russia
- Volunteers
- 70 people
- Sponsor
- N.N. Blokhin National Medical Research Center of Oncology
Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma
CD19.CAR Allogeneic NKT for Patients With Relapsed or Refractory B-Cell Malignancies (ANCHOR)
This study is for patients who have lymphoma or leukemia that has come back or has not gone away after treatment. Because there is no standard treatment for this cancer, patients are being asked to volunteer for a gene transfer…
- Conditions
- Refractory B-Cell Non-Hodgkin Lymphoma, Refractory B-Cell Small Lymphocytic Lymphoma, Relapsed Adult ALL, Relapsed CLL
- Trial name
- NCT03774654
- Testing
- CD19.CAR-aNKT cells
- Where
- 2 sites in United States
- Volunteers
- 13 people
- Sponsor
- Baylor College of Medicine