Cell and Gene Therapy Catapult clinical trials database

Results: 60
Ionis Pharmaceuticals, Inc., Roche
A Randomized, Double-blind, Placebo-controlled Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Ascending Doses of Intrathecally Administered ISIS 443139 in Patients With Early Manifest Huntington's Disease
This study will test the safety, tolerability, pharmacokinetics and pharmacodynamics of multiple ascending doses of IONIS-HTTRx administered intrathecally to adult patients with early manifest Huntington's Disease.
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase I/II
Trial status:
Recruiting
Recruitment target:
36
Year trial started:
2015
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Neurology
Clinical indication:
Huntington’s disease
Clinical trial sites:
Cambridge University Hospital, University College London, University of Manchester, St. Mary's Hospital
Viral vectors used:
Single stranded antisense oligonucleotide (ASO)
Gene Therapy Study in Severe Haemophilia A Patients
A Phase 1/2, Dose-Escalation Safety, Tolerability and Efficacy Study of BMN 270, an Adenovirus- Associated Virus Vector-Mediated Gene Transfer of Human Factor VIII in Patients With Severe Haemophilia A.
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase I/II
Trial status:
Recruiting
Recruitment target:
12
Year trial started:
2015
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Blood
Clinical indication:
Haemophilia A
Clinical trial sites:
Hampshire Hospitals NHS Foundation Trust, Basingstoke Queen Elizabeth Hospital, Birmingham University Hospitals Bristol NHS Foundation Cambridge University Hospitals NHS Foundation, Greater Glasgow Health Board, Barts Health NHS Trust, London Guy's & St. Thomas' NHS Foundation Trust, London Imperial College Healthcare NHS Trust
Viral vectors used:
AAV
GenSight Biologics, GenSight Biologics
Randomized, Double-Masked, Sham- Controlled Clinical Trial to Evaluate the Efficacy of a Single Intravitreal Injection of GS010 in Subjects Affected for More Than 6 Months and To 12 Months by LHON Due to the G11778A Mutation in the ND4 Gene
The goal of this study is to assess the efficacy of GS010, a gene therpy, in improving the visual outcome in patients with LHON due to the G11778A ND4 mitochondrial mutation when vision loss is present for more than six months and up to one year (REVERSE).
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase III
Trial status:
Recruiting
Recruitment target:
36
Year trial started:
2016
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Eye
Clinical indication:
Leber Hereditary Optic Neuropathy (LHON)
Clinical trial sites:
Moorfields Eye Hospital NHS Foundation Trust
Viral vectors used:
GS010:recombinant adeno-associated viral vector srotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4)
GenSight Biologics, GenSight Biologics
A Randomized, double-masked, sham-controlled clinical trial to evaluate the efficacy of a single intravitreal injection of GS010 in subjects affected for 6 months or less by Leber Hereditary Optic Neuropathy (LHON) due to the G11778A mutation in the mitoc
The goal of this study is to assess the efficacy of GS010, a gene therpy, in improving the visual outcome in patients up to 6 months from onset of Leber Hereditary Optic Neuropathy (LHON) due to the ND4 mitochondrial mutation (RESCUE).
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase III
Trial status:
Recruiting
Recruitment target:
36
Year trial started:
2016
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Eye
Clinical indication:
Leber Hereditary Optic Neuropathy (LHON)
Clinical trial sites:
Moorfields Eye Hospital NHS Foundation Trust
Viral vectors used:
GS010: recombinant adeno-associated viral vector serotype 2 (rAAV2/2) containing the wild-type ND4 gene (rAAV2/2-ND4).
Oxford BioMedica, Henri Mondor Hospital Paris
A Multicentre, Open-label Study to Determine the Long Term Safety, Tolerability and Efficacy of ProSavin in Patients With Bilateral, Idiopathic Parkinson's Disease.
This study is designed to determine the long term (10 years) safety, tolerability and efficacy of ProSavin, a lentiviral based vector carrying three genes that encode the key enzymes for the synthesis of dopamine, in patients with bilateral, idiopathic Parkinson's disease who received the ProSavin in previous study (PS1/001/07).
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase I/II
Trial status:
In follow-up
Recruitment target:
15
Year trial started:
2011
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Neurology
Clinical indication:
Parkinson's disease
Clinical trial sites:
Addenbrookes Hospital Cambridge
Viral vectors used:
Lentiviral vector
University of Oxford , Oxford University Hospital
Gene Therapy for Blindness Caused by Choroideremia
An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1).
Cell type:
Not specified
Cell source:
Not specified
Trial phase:
Phase I
Trial status:
Recruiting
Recruitment target:
14
Year trial started:
2011
Autologous/allogeneic:
N/A
Gene modification/gene therapy:
Yes. In-vivo.
Disease area:
Eye
Clinical indication:
Choroideraemia
Clinical trial sites:
Oxford Eye Hospital
Viral vectors used:
rAAV2
Pfizer, UK, University College London
Phase 1, Open-label, Safety And Feasibility Study Of Implantation Of Pf-05206388 (Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (Rpe) Living Tissue Equivalent) In Subjects With Acute Wet Age Related Macular Degeneration and Recent Rapid Vis
A Study Of Implantation Of Retinal Pigment Epithelium In Subjects With Acute Wet Age Related Macular Degeneration.
Cell type:
Retinal cells
Cell source:
Human induced pluripotent stem cells (hiPSCs)
Trial phase:
Phase I
Trial status:
In follow-up
Recruitment target:
10
Year trial started:
2015
Autologous/allogeneic:
Allogeneic
Gene modification/gene therapy:
No
Disease area:
Eye
Clinical indication:
Acute wet age related macular degeneration
Clinical trial sites:
Moorfields Eye Hospital
St Georges University London, Orphan Technologies
Clinical development of erythrocyte encapsulated thymidine phosphorylase - a therapy for mitochondrial neurogastrointestinal encephalomyopathy
The aim of this trial is to evaluate erythrocyte encapsulated thymidine phosphorylase (EE-TP) in patients with mitochondrial neurogastrointestinal encephalomyopathy (MNGIE). Conducting a multi- centre (pan European), open-label, multiple ascending dose, Phase II trial in 10 patients with MNGIE, over 36 months.
Cell type:
Erythrocytes
Cell source:
Blood
Trial phase:
Phase II
Trial status:
In planning
Recruitment target:
10
Year trial started:
2016
Autologous/allogeneic:
Autologous
Gene modification/gene therapy:
No
Disease area:
Metabolic and endocrine
Clinical indication:
Mitochondrial neurogastrointestina l encephalomyopathy (MNGIE)
A phase 1, open label, non- comparative, monocenter study to evaluate the safety and the ability of UCART19 to induce molecular remission in paediatric patients with relapsed /refractory B acute lymphoblastic leukaemia (UCART19_PALL)
This study aims at evaluating the safety and efficacy of UCART19, an allogeneic CAR T-cell product for treatment of CD19-expressing hematological malignancies, gene edited with TALEN®, to induced molecular remission in pediatric patients with relapsed or refractory CD19-positive B-cell acute lymphoblastic leukemia (B-ALL) ahead of planned allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Cell type:
T cells
Cell source:
Bone marrow
Trial phase:
Phase I
Trial status:
Recruiting
Recruitment target:
10
Year trial started:
2016
Autologous/allogeneic:
Autologous
Gene modification/gene therapy:
Yes. Ex-vivo.
Disease area:
Cancer (haematology)
Clinical indication:
B-cell acute lymphoblastic leukemia
Clinical trial sites:
Great Ormond Street Hospital
Viral vectors used:
TALEN gene edited cells

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