Phase 2 Study on the Pharmacokinetics and Safety of RLYB212 in Pregnant Women at Higher Risk for HPA-1a Alloimmunization

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Rallybio Ipa LLC

Participant type

Patients

Age range

18-64 years

Gender

Female

Therapeutic area

Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Trial duration

20 Nov 2024 → 1 Oct 2025

Decision date (initial)

2024-10-01

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Rallybio IPA, LLC

External identifiers

EU CT number

2024-512651-20-00

ClinicalTrials.gov

NCT06435845

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic

To evaluate the PK profile of RLYB212 during pregnancy following repeat SC administration and to assess maternal and fetal safety of RLYB212 during pregnancy

Secondary objectives 6

1. To evaluate RLYB212 exposure in the neonate at delivery
2. To assess the safety of RLYB212 in the HPA-1a positive neonate
3. To assess the immunogenicity of RLYB212
4. To assess pregnancy and neonatal outcomes following antenatal RLYB212 administration
5. To assess the occurrence of neonatal thrombocytopenia following antenatal RLYB212 administration
6. To assess the occurrence of HPA-1a alloimmunization

Conditions and MedDRA coding

Fetal and Neonatal Alloimmune Thrombocytopenia due to Human Platelet Antigen 1 incompatibility

Regulatory references

Scientific advice from competent authorities

European Medicines Agency

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Pregnant women who present at Gestational Week 6 or after and confirmed to be: HPA-1b/b (HPA-1a negative), HLA-DRB3*01:01 positive, Anti-HPA-1a alloantibody negative, Carrying an HPA-1a/b (HPA-1a positive) fetus
2. 18-45 years of age

Exclusion criteria 5

1. Prior history of HPA-1a related fetal and neonatal alloimmune thrombocytopenia
2. Multiple pregnancy (more than 1 fetus)
3. Prior history of platelet transfusion or other blood transfusions
4. Known sensitivity and/or immediate hypersensitivity to any components of RLYB212 or its formulation
5. Any co-morbid medical or obstetric condition(s), laboratory abnormality, concomitant treatment, or other reason that, in the investigator’s opinion, could adversely affect the safety of the participant and/or fetus, impair the assessment of study results, or preclude compliance with the study

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. Maternal PK parameters: ○ half-life of RLYB212 (t½); ○ max RLYB212 concentration (Cmax); ○ time to maximum RLYB212 concentration (tmax); ○ apparent clearance (CL/F) of RLYB212; ○ apparent volume of distribution (Vd); ○ area under the RLYB212 concentration versus time curve (AUC)
2. • Type, seriousness, and incidence of AEs; • Physical examination findings; • Vital signs; • Maternal clinical laboratory values; • ECG; • Obstetric/fetal doppler ultrasound

Secondary endpoints 6

1. Concentration of RLYB212 in cord blood
2. • Type, seriousness, and incidence of AEs including fetal/neonatal AEs; • Physical examination findings, including APGAR score; • Vital signs
3. Development of ADAs (anti-drug antibody)
4. Number of: Spontaneous abortions: non-deliberate fetal death before gestational wk (GW) 20; Elective abortions: deliberate termination of pregnancy at any stage; Stillbirths: non-deliberate fetal death on or after GW 19 wks; Premature births: live birth prior to 37 completed GW; Live births (37 or more completed GW); Health/development of infants up to 4-6 wks of age
5. Neonatal thrombocytopenia and severe neonatal thrombocytopenia, as defined by a platelet count < 150 × 10^9/L and < 50 × 10^9/L respectively, within 72 hours of parturition
6. Presence of maternal anti-HPA-1a alloantibodies at Week 10 post pregnancy

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Rallybio Ipa LLC

Sponsor organisation

Rallybio Ipa LLC

Address

234 Church Street Suite 1020

City

New Haven

Postcode

06510-1869

Country

United States

Scientific contact point

Organisation

Rallybio Ipa LLC

Contact name

Head of Regulatory/Quality

Public contact point

Organisation

Rallybio Ipa LLC

Contact name

Head of Regulatory/Quality

Third parties 5

Locations

4 EU/EEA countries · 6 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 4 · Art. 38 CTR

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 36 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

5 submissions — initial application plus substantial / non-substantial modifications