RECRUITING

A Phase 2 Basket Study of Vosoritide in Children with Turner Syndrome, SHOX Deficiency and Noonan Syndrome with an Inadequate Response to Human Growth Hormone

Conditions

Short Stature Homeobox- Containing Gene SHOX Deficiency Noonan Syndrome Turner Syndrome Short Stature Musculoskeletal Diseases Bone Diseases Developmental Endocrine System Diseases Natriuretic Peptide, C-Type

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide versus hGH on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

Official Title

A Phase 2, Randomized, Human Growth Hormone-Controlled, Multicenter, Basket Study of Vosoritide in Children with Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome with an Inadequate Response to Human Growth Hormone

Quick Facts

Study Start:2024-11-22

Study Completion:2041-09

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06668805

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:3 Years to 11 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion Criteria
1. Participants must be ≥ 3 years old, and \< 10 years old (females) or \< 11 years old (males),at the time of signing the informed consent form 2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome. 3. A height assessment corresponding to a height Z score of ≤ -2.00 SDs in reference to the general population of the same age and sex. 4. Tanner Stage 1, at time of signing the ICF. 5. Have been receiving continuous hGH for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment. 6. Are willing to continue on hGH for the Baseline Growth Phase, and for 2 years post randomization if randomized to the hGH arm. 7. Inadequate response to prior hGH treatment.	1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia. 2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. 3. Bone age advanced beyond chronological age by more than 2 years. 4. Congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension, 5. Have an unstable condition likely to require surgical intervention during the study. 6. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays. 7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period. 8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Inclusion Criteria

Exclusion Criteria

1. Participants must be ≥ 3 years old, and \< 10 years old (females) or \< 11 years old (males),at the time of signing the informed consent form
2. A genetically confirmed diagnosis of Turner syndrome, SHOX deficiency or Noonan syndrome.
3. A height assessment corresponding to a height Z score of ≤ -2.00 SDs in reference to the general population of the same age and sex.
4. Tanner Stage 1, at time of signing the ICF.
5. Have been receiving continuous hGH for the treatment of short stature associated with their condition for a minimum of 1 year immediately prior to enrollment.
6. Are willing to continue on hGH for the Baseline Growth Phase, and for 2 years post randomization if randomized to the hGH arm.
7. Inadequate response to prior hGH treatment.

1. Participants with Turner syndrome known to have Y-chromosome material unless they have undergone gonadectomy and have fully external female genitalia.
2. Diagnosis of systemic disease or condition that may cause short stature other than Turner syndrome, SHOX deficiency, or Noonan syndrome, eg, renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.
3. Bone age advanced beyond chronological age by more than 2 years.
4. Congenital heart disease which places the participant at increased risk of an adverse cardiac outcome in the setting of hypotension,
5. Have an unstable condition likely to require surgical intervention during the study.
6. Evidence of decreased growth velocity (AGV \< 1.5 cm/year) as assessed over a period of at least 6 months and growth plate closure assessed using bilateral lower extremity X-rays.
7. Previous limb-lengthening surgery, or planned or expected to have limb lengthening surgery during the study period.
8. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period.

Contacts and Locations

Study Contact

Trial Specialist

CONTACT

1-800-983-4587

medinfo@bmrn.com

Study Manager

CONTACT

1-800-983-4587

Medinfo@bmrn.com

Principal Investigator

Medical Director, MD, PhD

STUDY_DIRECTOR

BioMarin Pharmaceutical

Study Locations (Sites)

Children's Hospital Colorado

Aurora, Colorado, 80045

United States

Nemours Children's Hospital, Delaware (Alfred I. Dupont Hospital for Children)

Wilmington, Delaware, 19803

United States

St. Luke's Children's Endocrinology and Diabetes

Boise, Idaho, 83712

United States

Kentucky Children's Hospital

Lexington, Kentucky, 40506

United States

Children's Hospital at Montefiore

Bronx, New York, 10467

United States

Collaborators and Investigators

Sponsor: BioMarin Pharmaceutical

Medical Director, MD, PhD, STUDY_DIRECTOR, BioMarin Pharmaceutical

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-11-22

Study Completion Date2041-09

Study Record Updates

Study Start Date2024-11-22

Study Completion Date2041-09

Terms related to this study

Keywords Provided by Researchers

Short Stature
Musculoskeletal Diseases
Bone Diseases
Developmental Endocrine System Diseases Natriuretic Peptide, C-Type

Additional Relevant MeSH Terms

Short Stature Homeobox- Containing Gene SHOX Deficiency
Noonan Syndrome
Turner Syndrome