A Phase I Pilot Study of Abaloparatide + Bevacizumab in Myelodysplastic Syndromes

What Was Tested

This registered phase 1 trial tested Abaloparatide.

The primary objective of this study is to determine the safety and tolerability of combined abaloparatide and bevacizumab in patients with Myelodysplastic Syndromes (MDS).

A secondary objective is to determine the response to treatment (based on bone marrow and peripheral blood findings).

A tertiary objective is to determine the impact of therapy on health-related quality of life (HRQOL) and patient-reported outcomes (PRO).

It had 1 arm (groups): abaloparatide and bevacizumab treatment (experimental).

The registry classes the interventions as drug.

More on the Plan

The main objective of this study is to determine the safety and tolerability of combined abaloparatide + bevacizumab in MDS patients.

This will be a single center, single arm, phase 1 trial in MDS patients.

This will enable the investigator to monitor for any potential toxicities of single-agent abaloparatide in the MDS patient population.

Following cycle 1, patients will have a bone marrow aspirate to determine the impact of abaloparatide treatment on bone marrow stromal cell and hematopoietic cell populations.

Who Took Part

Enrollment: 20 actual participants took part.

It was open to people of any sex aged from 18 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Age equal to or greater than 18. Patients must have a documented diagnosis of MDS or non-proliferative chronic myelomonocytic leukemia (CMML) (WBC \< 12,000/mcL) according to World Health Organization (WHO) criteria (27). Patients can be treatment-naïve or have received prior MDS-directed chemotherapy. MDS patients previously treated with disease-modifying chemotherapy (i.e. azacitidine, decitabine, lenalidomide, intensive chemotherapy, and/or an investigational agent) are eligible irrespective of IPSS-R score. Patients must be off all non-transfusion therapy for MDS for 28 days prior to initiation of study treatment, including all types of growth factors.

The full record lists 21 inclusion and 31 exclusion criteria; among the exclusions: patients actively receiving either abaloparatide, teriparatide or bisphosphonate therapy for other indications; cumulative prior use of abaloparatide and/or any other parathyroid hormone analogs for \> 20 months; history of allogeneic stem cell transplant.

How the Study Was Run

Design: single-group and open-label.

Being open-label, the Abaloparatide study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2019-02-14 with completion listed for 2022-04-01.

It ran at 1 site in United States.

Its stated primary purpose is treatment.

What Was Measured

Primary outcome: Proportion of patients who experience a therapy-limiting Toxicity (TLT) (time frame: 7 months).

It also tracked 1 secondary outcome, for example Proportion of patients who responded to therapy.

What This Means in Plain Language

Put simply, this is a phase 1 trial of Abaloparatide in 20 people.

Its size is small, so on its own it can only give an early look.

The trial is marked completed; no results are posted to the registry yet, so nothing here describes outcomes.

More context: the Abaloparatide compound page, all human trials.