Airtable - Myeloid disorders

2 hidden fields

Filter

Grouped by 2 fields

Sort

Subtype

AML

Count4

VICTOR

RESPECT

AMADEUS

MoTD

A Study to Investigate APL-4098 Alone and/or in Combination With Azacitidine in R/R AML and High-Risk MDS

TIARA

Study to Evaluate Efficacy and Safety of Avapritinib (BLU-285)

APEX: A Study of CGT9486 in Patients with Advanced Systemic Mastocytosis

MoTD

RESPECT

SUMMIT (CGT9486-21-202)

AMADEUS

A Study of AG-946 in Participants With Anemia Due to Lower-Risk Myelodysplastic Syndromes (LR-MDS)

Ascelus-H

RESPECT

StarMS

Fedora

PACIFICA Phase 3

GRN163LMYF3001

KER050-MF-301

M20-178 Navitoclax for Relapsed/Refractory Myelofibrosis

PROMise

MoTD

VAC85135MPN1001: A Phase 1 Study of VAC85135, a Neoantigen Vaccine Regimen, Concurrently Administered with Ipilimumab for the Treatment of Myeloproliferative Neoplasm

RESPECT

NS-018 versus Best Available Therapy in Subjects with Primary Myelofibrosis

Commodore 1

MITHRIDATE Trial

AZURE - BLU-263-2101

Treatment stage

Multiple stages

Subtype

AML

MDS

MDS-exess blasts

Count1

Treatment stage

Relapsed / refractory

Subtype

Aplastic anaemia

Count1

Treatment stage

Multiple stages

Subtype

ASM

Count2

Treatment stage

Multiple stages

Subtype

CML

Count2

Treatment stage

Multiple stages

Subtype

ISM

Count1

Treatment stage

Multiple stages

Subtype

MDS

Count4

Treatment stage

Multiple stages

Subtype

Count1

Treatment stage

Relapsed / refractory

Subtype

Myelofibrosis

Count10

Treatment stage

First line

Treatment stage

Relapsed / refractory

Treatment stage

Multiple stages

Subtype

PNH

Count1

Treatment stage

Multiple stages

Subtype

Count1

Treatment stage

First line

Subtype

AHM

Count1

Treatment stage

Multiple stages

Summary

For adults, >= 60 years with CD33 positive AML

Phase 2 study: Venetoclax or intensive chemotherapy

0-2

Krishnamurthy, P

Dillon, R

Gupta, S

Rashid, S

Acute myeloid leukaemia (AML) is an aggressive blood cancer affecting 3000+ people per year in the UK. Patients who are relatively young and healthy are given potentially curative treatment with intensive chemotherapy (IC) which is fairly effective in inducing remission, and for some patients, long-term cure. IC has severe short-term side effects including decreasing white blood cell count (which can lead to potentially fatal infections), mouth ulcers, nausea, vomiting and hair loss. Long-term s

Diagnosis of CD33 positive Acute Myeloid Leukaemia

For adults undergoing HSCT

Phase 3 randomised trial: Rezafungin vs standard treatment to prevent IFI

Not specified

Mehra, V

The ReSPECT trial is a global, randomized, double-blind, controlled, pivotal Phase 3 trial of rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillus and Pneumocystis in subjects undergoing allogeneic BMT. Rezafungin, dosed once-weekly, will be compared to a daily regimen containing multiple drugs including fluconazole or posaconazole, and trimethoprim-sulfamethoxazole, also known as Bactrim, for 90 days, at which time fungal-free survi

≥18 years of age

Adult patients with AML or MDS, able to commence study therapy following allo-SCT

Phase 3 randomised study: Azacitidine vs placebo as maintenance after allo-STC

0-2

Potter, V

Treatment options for older adults with Acute Myeloid Leukaemia (AML) and Myelodysplasia (MDS) are limited. Although stem cell transplantation remains one of the most effective treatments it is associated with severe side effects which have until recently prevented its use in older adults. In the last decade the use of reduced intensity transplants has allowed the extension of the potentially curative effect of transplantation to older patients in whom it was previously precluded. Although a maj

Age > = 16 at the time of signing the informed consent form

For adults awaiting a planned allo-SCT

Phase 2 trial: Thymoglobulin vs. Calcineurin inhibitor or cyclophosphamide as GvHD prophylaxis

Not specified

Krishnamurthy, P

Stem cell transplantation from a matched sibling or unrelated donor (allo-SCT) is the only curative therapy for many children and adults with blood cancer. However allo-SCT remains associated with a number of life-threatening side effects, the most serious of which is ‘graftversus-host disease’ (GvHD). This complication occurs when donor immune cells (the ‘graft’) see the patient’s healthy skin, gut or liver (the ‘host’) as foreign and attacks them. Patients currently receive a combination of d

Availability of suitably matched unrelated donor (9/10 or 10/10)

For adults with relapsed or refractory acute myeloid leukemia, myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB).

Phase 1/2 study: APL-4098 alone or with azacitidine

0-2

Dillon, R

This is an open-label, Phase 1/2 study to determine the safety, tolerability, and efficacy of APL-4098 alone and/or in combination with azacitidine for the treatment of relapsed or refractory (R/R) acute myeloid leukemia (AML), myelodysplastic syndrome (MDS)/AML and MDS-excess blasts (EB). Participants with the MDS-EB subtype will be eligible for the Phase 1 part of the study only.

Inclusion:

For adults with acquired idiopathic AA, lacking donors or ineligible for MSD/HID/MUD HSCT

Phase 1 study: Production of expanded autologous regulatory T cells

Mufti, G

‘TIARA’ is a phase I clinical trial investigating the safety and tolerability of expanded autologous T regulatory cells (Tregs) to treat patients with Aplastic Anaemia (AA) who have failed, or are considered ineligible for IST / other treatments. Tregs (starting material) will be collected via a leukapheresis procedure at King’s College Hospital and manufactured in the GMP production facility at Guy’s Hospital. Two dose levels will be explored in a 3+3 dose escalation design. Expanded autologous

Acquired idiopathic AA

For adults with ASM

Phase 2 study: Avapritinib (BLU-285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor

0-3

Radia, D

An Open-label, Single Arm, Phase 2 Study to Evaluate Efficacy and Safety of Avapritinib (BLU285), A Selective KIT Mutation-targeted Tyrosine Kinase Inhibitor, in Patients with Advanced Systemic Mastocytosis

Patients who are ≥ 18 years of age.

Adult patients with Advanced Systemic Mastocytosis

Phase 2 open label study, single agent bezuclastinib

0-3

Deepti, R

The purpose of this study is to see how safe and effective the study drug, CGT9486, is as an inhibitor of the KIT gene in people that have advanced systemic mastocystosis (AdvSM). AdvSM is aggressive and life-threatening, disease progression is driven by the activation of the KIT receptor. Targeted therapies against KIT are emerging as potential treatments for AdvSM. There are 2 main parts to this study. Participants cannot take part in both parts. Part 1 will determine the optimal dose for pati

Diagnosed with 1 of the following advanced mastocytosis diagnoses based on WHO diagnostic criteria (Appendix A of the protocol):

For adults awaiting a planned allo-SCT

Phase 2 trial: Thymoglobulin vs. Calcineurin inhibitor or cyclophosphamide as GvHD prophylaxis

Not specified

Krishnamurthy, P

Availability of suitably matched unrelated donor (9/10 or 10/10)

For adults undergoing HSCT

Phase 3 randomised trial: Rezafungin vs standard treatment to prevent IFI

Not specified

Mehra, V

≥18 years of age

Adult patients with Nonadvanced Systemic Mastocytosis

Phase 2 randomised study: bezuclastinib vs placebo

0-2

Deepti, R

A Multi-Part, Randomized, Double-Blind, Placebo-Controlled Phase 2 Clinical Study of the Safety and Efficacy of CGT9486 in Subjects with Nonadvanced Systemic Mastocytosis

Diagnosed with 1 of the following diagnoses according to the 2016 WHO classification for systemic mastocytosis (SM):

Adult patients with AML or MDS, able to commence study therapy following allo-SCT

Phase 3 randomised study: Azacitidine vs placebo as maintenance after allo-STC

0-2

Potter, V

Age > = 16 at the time of signing the informed consent form

For adult patients with low-risk MDS

Phase 2a/2b study: AG-946 vs placebo

0-2

Kulasekararaj, A

This purpose of this study is to establish proof of concept of AG-946 in participants with LR-MDS in Phase 2a and to compare the effect of AG-946 versus placebo and to detect a dose response for erythroid response in participants with LR-MDS in Phase 2b.

Phase 2a:

For adults with stable CLL, Haemochromatosis, irron-deficient anaemia, MDS, MGUS, Smouldering myeloma, SCD, or Waldenstrom’s macroglobulinemia

Digital follow-up pathway monitoring long-term stable blood conditions

None

Benjamin, R.

This study has 5 arms: Arm 1 - Patient and carer co-design,collecting qualitative data on patient satisfaction and empowerment on follow-up pathways,via focus groups and individual interviews Arm 2 – Healthcare professionals co-design and feedback,collecting qualitative data on Healthcare professionals views and feedback of the digital follow-up pathways,via focus groups and interviews Arm 3 – Key Opinion Leader (KOL) interviews,investigating issues of implementation,scalability,and commercialis

Inclusion for Arm 1:

For adults undergoing HSCT

Phase 3 randomised trial: Rezafungin vs standard treatment to prevent IFI

Not specified

Mehra, V

≥18 years of age

For adults with relapsing MS

Randomised study: autologous HSCT versus alemtuzumab or ocrelizumab

Not specified

Kazmi, M

Multiple sclerosis (MS) is a chronic autoimmune inflammatory disease of the central nervous system (CNS) which leads to impairment in strength, sensation, balance, vision, cognition and sphincter function. For patients with highly active relapsing remitting MS (RRMS), disease modifying therapies (DMTs) are available however there is growing evidence that autologous haematopoietic stem cell transplantation (aHSCT) may be more effective in reducing relapse rates and improving disability and qualit

Diagnosis of MS using the 2017 McDonald criteria.

For adults with JAK2 V617F positive primary or secondary MF

Phase 2 study: fedratinib with ropeginterferon alfa-2b in patients with myelofibrosis

0-2

O'Sullivan, J

FEDORA is a prospective, multicentre, open label, phase II clinical trial for patients with myelofibrosis (MF). MF is a rare blood disorder that causes scarring of the bone marrow. More than 55% of people with MF have a change in a gene called JAK2. JAK2 inhibitors are drugs that slow or stop the growth of cancer cells. Although JAK2 inhibitors have shown promising results for some patients with MF, treatment with a JAK2 inhibitor does not usually cure the patient’s disease. Previous studies hav

Age 18 or over at trial entry

For adults with Primary Myelofibrosis, Post Polycythemia Vera Myelofibrosis, or Post Essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia

Phase 3 randomised study: Pacritinib vs physician’s choice

0-2

Harrison, C

PACIFICA is a multi-national, multi-centre, randomized, controlled phase 3 trial of pacritinib versus Physician Coice in adults with primary or secondary myelofibrosis with DIPSS intermediate- or high-risk disease, ECOG PS 0-2, platelet counts <50 x 109/L, limited ( up to 3 months Ruxolitinib treatment) or no prior JAK2 inhibitor treatment, and are not candidates for stem cell transplant.

PMF, PPV-MF, or PET-MF (as defined by Tefferi and Vardiman 2008).

For adults with JAK refractory intermediate-2 or high-risk MF

Phase 3 randomised study: Imetelstat vs best available therapy

0-2

Harrison, C

A Randomized Open-Label, Phase 3 Study to Evaluate Imetelstat (GRN163L) Versus Best Available Therapy (BAT) in Patients with Intermediate-2 or High-risk Myelofibrosis (MF) Relapsed / Refractory (R/R) to Janus Kinase (JAK) Inhibitor. To compare the OS of participants, treated with imetelstat versus best available therapy (BAT), with intermediate-2 or high-risk MF whose disease is relapsed / refractory to JAK inhibitor treatment. Trial Drug: Imetelstat

Diagnosis of PMF according to the revised WHO criteria ; or PET-MF or PPV-MF according to the IWG-MRT criteria

For adults with MF

Phase 2 study: KER-050 monotherapy or with Ruxolitinib

0-2

O'Sullivan, J

A phase 2 Open-label study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of KER-050 as monotherapy or in combination with Ruxolitinib in participants with MF. To evaluate the safety, tolerability, PK, PD, and efficacy of KER-050, administered with or without ruxolitinib, in participants with PMF, post-ET MF, or post-PV MF. Part 1 (Dose Escalation) will evaluate the safety and tolerability of KER-050 and identify the dose of KER-050 to be evaluated in Part 2 (Dose Expansio

Ability to understand the purpose and risks of the study and provide signed and dated informed consent and authorization to use protected health information in accordance with national and local study participant privacy regulations.

For adults with Myelofibrosis

Phase 3 randomised study: Navitoclax with Ruxolitinib vs best available therapy

0-2

Harrison, C

Myelofibrosis (MF) is a bone marrow illness that affects blood-forming tissues in the body. MF disturbs the body's normal production of blood cells, causing extensive scarring in the bone marrow. This leads to severe anemia, weakness, fatigue, and an enlarged spleen. The purpose of this study is to assess safety, tolerability, and change in spleen volume when navitoclax is given in combination with ruxolitinib, as compared to best available therapy, for adult participants with MF. Navitoclax is

Subject ≥ 18 years of age.

For adults with with intermediate-2 or high risk myelofibrosis not receiving an adequate response with ruxolitinib alone

PLX2853 with ruxolitinib for myelofibrosis

Not specified

Harrington, P

Myelofibrosis (MF) is a cancer of the bone marrow that disrupts the production of blood cells. Symptoms of MF include anaemia (low red cell levels), weakness, tiredness and often an enlarged spleen. Standard treatment can involve chemotherapy, radiotherapy, a stem cell transplant or treatment with a drug called ruxolitinib. Ruxolitinib targets the specific cells that play a part in the development of MF, and is the only drug licenced to treat MF. Previous research has shown that ruxolitinib is g

Age> = 16 years.

For adults awaiting a planned allo-SCT

Phase 2 trial: Thymoglobulin vs. Calcineurin inhibitor or cyclophosphamide as GvHD prophylaxis

Not specified

Krishnamurthy, P

Availability of suitably matched unrelated donor (9/10 or 10/10)

For adults with MPN with Type 1 or Type 2 CALR mutation

Phase 1 study: VAC85135 and Ipilumumab

0-2

Harrison, C

The primary objective is to evaluate the safety of VAC85135 administered with ipilimumab in participants with MPNs. Key secondary endpoints are to evaluate the immunogenicity and anti-tumor clinical activity. Trial Drug: VAC85135 and Ipilumumab

Essential thrombocythemia that is not very low risk per NCCN 2021 guidelines.

For adults undergoing HSCT

Phase 3 randomised trial: Rezafungin vs standard treatment to prevent IFI

Not specified

Mehra, V

≥18 years of age

For adults with myelofibrosis

Phase 2 open-label study: NS-018 vs best available therapy

0-2

de Lavallade, H

A Phase 2, Open-label, Multi-center Study to Assess the Efficacy and Safety of Orally Administered NS-018 Versus Best Available Therapy in Subjects with Primary Myelofibrosis, Post-polycythemia Vera Myelofibrosis, or Post-essential Thrombocythemia Myelofibrosis with Severe Thrombocytopenia (Platelet Count <50,000/μL). This is a Phase 2, open-label, randomized study in subjects with PMF, post-PV MF, or post-ET MF comparing the efficacy and safety of NS-018 versus Best-available therapy (BAT), as

Able to provide written informed consent prior to enrollment into the study.

For adults with PNH

Phase 3 randomised study: Soliris for PNH

Not specified

Kulasekararaj, A

This study is comparing a new medication against Soliris, a already approved medication, after treatment with complement inhibitors (medication that helps our body to not self attack but defend against harmful intruders) to evaluate which treatment is safer and more effective for patients with Paroxysmal Nocturnal Hemoblobinuria (PNH), a rare blood disease that causes the red blood cells to break apart, due to a missing protein on the patients blood cells surface.

[None listed]

For adults with high risk PV

Phase 3 randomised study: ruxolitinib with either hydroxycarbamide or interferon alpha

Not specified

Harrison, C

Polycythemia vera (PV) is a rare blood cancer characterised by a high-risk of thrombosis (clotting) and haemorrhage (bleeding). Average survival in high-risk patients receiving contemporary care is 10.9 years. Current treatments include aspirin, venesection (blood drawing) and selected use of standard therapies (hydroxycarbamide (HC) or interferon alpha (IFNa)). The strategies remain less than perfect, with on-going risks of developing blood clots, bleeding, dying early due to either these event

Patient > = 18 years of age

Adult patients with altered hematologic malignancies treated with 1 prior selective KIT inhibitor

Phase 1 / 2 open label study: Elenestinib monotherapy and with Azacytidine

0-3

Deepti, R

A Phase 1/2, open-label, 2-arm study evaluating BLU-263 as monotherapy and in combination with azacitidine, in patients with KIT altered hematologic malignancies

Patient is ≥ 18 years of age at the time of signing the informed consent.

Summary

29 records

Summary

Alert

Lorem ipsum

Okay