OHB-607 for Bronchopulmonary Dysplasia

Recruiting at 76 trial locations

Overseen By OHB Contact

Age: < 18

Sex: Any

Trial Phase: Phase 2

Sponsor: Shire

Disqualifiers: Congenital malformation, Chromosomal abnormality, Neurological disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Approximately 30 weeks

338 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial aims to determine if a new drug, OHB-607, can prevent Bronchopulmonary Dysplasia, a serious lung condition, in extremely premature infants. The study compares infants receiving OHB-607 to those receiving standard care for preemies. Ideal participants are babies born between 23 and 27 weeks of gestation. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, offering a chance to contribute to important medical advancements.

Will I have to stop taking my current medications?

The trial information does not specify whether participants must stop taking their current medications. It is best to discuss this with the trial organizers or your doctor.

Is there any evidence suggesting that OHB-607 is likely to be safe for humans?

Research shows that OHB-607 is under study to help prevent Bronchopulmonary Dysplasia (BPD), a serious lung condition in premature babies. Earlier studies have assessed the safety of this treatment for these tiny patients. Although these studies don't provide detailed safety data, the treatment's progression to a Phase 2 trial indicates it has passed some initial safety tests.

Treatments in this phase have typically been tested in smaller groups to ensure they don't cause harmful side effects. This doesn't guarantee the absence of risks, but it usually means no serious problems have been found so far. Participants and their families should discuss any concerns with healthcare providers to better understand the safety and potential benefits. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Most treatments for bronchopulmonary dysplasia (BPD) focus on providing supportive care to manage symptoms, like oxygen therapy and mechanical ventilation. However, OHB-607 stands out because it is delivered through continuous IV infusion from birth, potentially offering more direct and consistent therapeutic effects. Researchers are excited about OHB-607 because it could address the underlying issues of BPD, possibly improving outcomes for premature infants much earlier than traditional methods. This new approach could represent a significant advancement in neonatal care for BPD.

What evidence suggests that OHB-607 might be an effective treatment for Bronchopulmonary Dysplasia?

Research shows that OHB-607, which participants in this trial may receive, might help lower the risk of Bronchopulmonary Dysplasia (BPD), a serious lung condition in premature babies. Studies have found that OHB-607 could reduce the chances of severe BPD compared to usual newborn care. This treatment aids lung development, potentially leading to fewer long-term lung problems. Early results suggest that babies receiving OHB-607 have better lung health. Although more research is needed to confirm these findings, the initial data is promising for this new treatment. ^{1 2 3 4 5}

Who Is on the Research Team?

Study Director

Principal Investigator

Shire

Are You a Good Fit for This Trial?

This trial is for extremely premature infants born between 23 and 27 weeks of gestation. Parents must consent to the study, which excludes infants with significant neurological disease, major congenital malformations, genetic disorders, or those whose mothers had severe COVID-19 during pregnancy.

Inclusion Criteria

Written informed consents and/or assents must be signed and dated by the participant's parent(s) prior to any study related procedures. The informed consent and any assents for underage parents must be approved by the IRB/IEC (in accordance with local regulations).

Written informed consents and/or assents must be signed and dated by the participant's birth mother prior to providing study-related information related to birth mother medical history, pregnancy and the birth of the participant. The informed consent and any assents for underage birth mothers must be approved by the IRB/IEC (in accordance with local regulations).

Participants must be between 23 weeks and 27 weeks of pregnancy.

Exclusion Criteria

I have a genetic condition identified by my doctor.

My blood sugar is very low even after taking glucose.

Current or planned participation in a clinical study of another investigational study treatment, device, or procedure (participation in non-interventional studies is permitted on a case-by-case basis).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive continuous IV infusion of OHB-607 from birth up to PMA 29 weeks +6 days

Approximately 30 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, including neurodevelopmental outcomes and mortality rates

24 months

Long-term follow-up

Assessment of neurodevelopmental outcomes and chronic lung disease burden reduction

12 to 24 months

What Are the Treatments Tested in This Trial?

Interventions

• OHB-607
• SHP607

Trial Overview The study tests OHB-607's effectiveness in preventing chronic lung disease in extremely premature babies compared to standard care alone. It aims to see if this drug can reduce long-term respiratory issues these infants often face.

How Is the Trial Designed?

Group I: OHB-607 Experimental Treatment 1 Intervention

Participants will receive continuous IV infusion of OHB-607 through from birth up to PMA 29 weeks +6 days.

Group II: Standard Neonatal Care Active Control 1 Intervention

Standard neonatal care alone will be provided.

Who Is Running the Clinical Trial?

Shire

Lead Sponsor

Trials

457

Recruited

96,000+

Pierre S. Sayad

Shire

Chief Medical Officer

MD from Loma Linda University

Flemming Ornskov

Shire

Chief Executive Officer since 2013

PhD in Medicine from Aarhus University

OHB Neonatology Ltd.

Lead Sponsor

Trials

1

Recruited

340+

Oak Hill Bio Ltd

Lead Sponsor

Trials

2

Recruited

360+

Published Research Related to This Trial

Mecasermin rinfabate (iPLEX) is a once-daily subcutaneous injection that combines rhIGF-I with rhIGFBP-3 to potentially extend the drug's half-life and reduce side effects, showing similar efficacy to rhIGF-I in various conditions like diabetes and growth hormone insensitivity syndrome.

The safety profile of Mecasermin rinfabate appears comparable to that of rhIGF-I, but there is limited data on its pharmacokinetics in humans, indicating a need for further research in this area.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mecasermin rinfabate: rhIGF-I/rhIGFBP-3 complex: iPLEX. Williams, RM., McDonald, A., O'Savage, M., et al.[2019]

Infusing recombinant human IGF-1 bound to IGFBP-3 at a dosage of 1.5 mg/kg/day for 3 days in mechanically ventilated preterm lambs significantly improved pulmonary and cardiovascular outcomes without causing toxicity to vital organs.

This study establishes that maintaining physiological plasma levels of IGF-1 can positively influence lung development and function in preterm infants, suggesting a potential therapeutic approach for preventing bronchopulmonary dysplasia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Pilot dose-ranging of rhIGF-1/rhIGFBP-3 in a preterm lamb model of evolving bronchopulmonary dysplasia. Albertine, KH., Dahl, MJ., Rebentisch, A., et al.[2023]

Mecasermin rinfabate, a combination of IGF-I and its binding protein IGFBP-3, is FDA-approved for treating severe primary IGF deficiency and growth hormone gene deletion, effectively increasing growth velocity in affected children.

This treatment has a longer serum half-life compared to unbound IGF-I, which helps reduce the risk of hypoglycemia, a common adverse effect associated with unbound IGF-I administration.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Mecasermin rinfabate. Kemp, SF.[2017]

Recombinant human insulin-like growth factor (rhIGF-I) can effectively increase growth velocity in children with IGF deficiency, but it has been associated with adverse events like hypoglycemia, especially when unbound.

Mecasermin rinfabate (iPlex), a combination of IGF-I and its binding protein IGFBP-3, shows a longer half-life and fewer adverse events compared to unbound IGF-I, suggesting it may be a safer option for treating growth disorders.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Efficacy and safety of mecasermin rinfabate. Kemp, SF., Fowlkes, JL., Thrailkill, KM.[2019]

Postnatal treatment with recombinant human IGF-1 (rhIGF-1) and binding peptide 3 (BP3) significantly reduced lung injury and pulmonary fibrosis in models of bronchopulmonary dysplasia (BPD) caused by chorioamnionitis and hyperoxia, as evidenced by improved lung function and structure in newborn rats.

The treatment also prevented right ventricular hypertrophy (RVH) and inhibited epithelial-mesenchymal transition (EMT) in airway epithelial cells, suggesting that rhIGF-1/BP3 could be a promising therapeutic strategy for managing BPD.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

The role of rhIGF-1/BP3 in the prevention of pulmonary hypertension in bronchopulmonary dysplasia and its underlying mechanism. Qu, S., Shan, L., Chen, X., et al.[2023]

In a study involving 5 preterm infants, intravenous administration of rhIGF-I with its binding protein rhIGFBP-3 safely increased serum IGF-I levels, which are crucial for growth and development.

The treatment was well tolerated, with no adverse effects on blood glucose or other physiological safety measures, indicating that this approach could be a promising intervention for improving growth in preterm infants.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic and dosing study of intravenous insulin-like growth factor-I and IGF-binding protein-3 complex to preterm infants. Löfqvist, C., Niklasson, A., Engström, E., et al.[2022]

The investigational product rhIGF-1/rhIGFBP-3, delivered via continuous intravenous infusion to preterm infants, showed no degradation when mixed with caffeine citrate, indicating it is safe to co-administer with this medication.

However, furosemide was found to be physically incompatible with rhIGF-1/rhIGFBP-3, and mixing it with ampicillin resulted in lower recovery of the rhIGF-1/rhIGFBP-3 components, suggesting caution is needed when administering these medications together.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of Protein-Specific Analytical Methodologies to Evaluate Compatibility of Recombinant Human (rh)IGF-1/rhIGFBP-3 with Intravenous Medications Co-Administered to Neonates. Salamat-Miller, N., Qu, W., Chadwick, JS., et al.[2022]

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03253263

A Clinical Efficacy and Safety Study of OHB-607 in ... The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung disease in ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04506619

Study Details | NCT04506619 | Safety and Efficacy ... The purpose of this study is to evaluate long-term safety and efficacy outcomes following previously administered short-term exposure to SHP607, as ...

3. clinicaltrials.cedars-sinai.edu clinicaltrials.cedars-sinai.edu/view/OHB-607-202

OHB-607 Compared to Standard Neonatal Care for the ... A Phase 2b, Multicenter, Randomized, Open-label, Two-Arm Study to Evaluate the Clinical Efficacy and Safety of OHB-607 Compared to Standard Neonatal Care

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC8959440/

Pharmacotherapy in Bronchopulmonary Dysplasia: What Is ... Due to inconsistent results obtained with various studies regarding the benefit of retinol in the prevention of BPD, a study was conducted by the NICHD neonatal ...

5. clinicaltrialsregister.eu clinicaltrialsregister.eu/ctr-search/trial/2018-001393-16/NL

Clinical Trials Register To assess the effect of OHB-607 on reducing the burden of CLD, as indicated by a reduction in the incidence of Grade 2 and Grade 3 (severe) BPD ...