A Phase 2 interventional study of HST5040 and Placebo in Methylmalonic Acidemia and Propionic Acidemia, sponsored by HemoShear Therapeutics. Terminated at 16 sites in 3 countries. Open to participants aged 2 Years and older. Per ClinicalTrials.gov, last updated 2024-01-05.
Sponsored by HemoShear Therapeutics · Phase 2, Interventional, and Treatment
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA). The study consists of 3 parts:
This study will determine whether HST5040 can improve levels of disease-associated toxins that accumulate in patients with PA and MMA.
26 studies on the registry are indexed under Methylmalonic acidemia; 7 are open to participants now.
This study's enrollment of 26 is above the median of 23 across 12 interventional studies indexed under Methylmalonic acidemia.
Browse Methylmalonic acidemia studies →HemoShear Therapeutics is the lead sponsor of 2 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Part B is the 6-month, randomized, double-blind (Subject/Investigator/Sponsor), placebo-controlled, 2-period crossover study consisting of 2 intervention periods of 12 weeks each to evaluate the safety and efficacy of the optimal dose of HST5040 in PA and MMA subjects ≥ 2 years old (N = minimum 12) in addition to SoC determined in Part A (within-subject dose escalation).
Drug: HST5040
Placebo in addition to standard of care.
Drug: Placebo
Liquid solution
Liquid solution
Change in plasma 2-methylcitric acid (MCA) levels
nmol/mL
Time frame: 6 months
Change in plasma propionyl-carnitine (3)
µmol/L
Time frame: 6 months
Change in C3 to acetyl-carnitine ratio (C3:C2)
µmol/L
Time frame: 6 months
Change in 3-OH propionate
g/mol
Time frame: 6 months
Change in Methylmalonic acid (in MMA subjects)
nmol/L
Time frame: 6 months
Change in NH3
nmol/L
Time frame: 6 months
Anion Gap
mEq/L
Time frame: 6 months
Pharmacokinetics parameters - Cmax
Maximum concentration (Cmax) after administration of HST5040
Time frame: 6 months
Pharmacokinetics parameters - Tmax
Time of maximum concentration (Tmax)
Time frame: 6 months
Pharmacokinetics parameters - AUC
Area under the concentration time curve (AUC)
Time frame: 6 months
Oral Intake
Food diary - change from baseline to end of each dose level interval in oral intake
Time frame: 6 months
Acute Metabolic Decompensations
Change in the total number of metabolic decompensation events requiring an emergency room (ER) visit of hospitalization
Time frame: 6 months
MetabQoL 1.0 - Health Related Quality of Life (HRQOL)
Score 0-100 Scale. Higher Score indicates better HRQOL
Time frame: 6 months
PedsQL 1.0 Family Impact Score - Health Related Quality of Life (HRQOL)
Score 0-100 Scale. Higher Score indicates better HRQOL
Time frame: 6 months
Plan to share: No
No publications or documents are linked to this record.
This study is terminated, as verified in Jan 2024. You cannot join it, but the record below documents what was studied.
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HemoShear Therapeutics