Study of Weekly ALTU-238 Compared With Daily Nutropin AQ in Prepubertal Children With Growth Hormone Deficiency

Growth Hormone Deficiency Clinical Trial

Official title:

A Twelve Month, Phase II, Randomized, Open-Label, Multi-Center, Dose-Ranging Study of Weekly ALTU-238 (Somatropin) as Compared With Daily Nutropin AQ (Somatropin) in Prepubertal Children With Growth Hormone Deficiency

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT00837863
• Other study ID #: 0001194
• Status: Recruiting
• Phase: Phase 2
• First received: February 2, 2009
• Last updated: May 6, 2009
• Start date: March 2009

Study information

• Verified date: May 2009
• Source: Altus Pharmaceuticals
• Contact: Dr. Kenneth Attie, Medical Monitor
• Phone: 781-373-6481
• Email: kattie[@]altus.com
• Is FDA regulated: No
• Health authority: United States: Food and Drug Administration
• Study type: Interventional

Clinical Trial Summary

The purpose of the study is to evaluate the safety and effectiveness of ALTU-238 in the treatment of children with growth hormone deficiency who have not yet reached puberty who lack the normal ability to make growth hormone themselves. This study will also test if ALTU-238 works as a weekly treatment.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 36
• Est. primary completion date: September 2010
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 3 Years to 13 Years

Eligibility

Inclusion Criteria:

1. Assent of subject, if applicable, and written informed consent of parent or legal guardian

2. Diagnosis of GHD as defined by a maximum stimulated GH < 7 ng/mL (µg/L) on two stimulation tests (using any two distinct agents from the following list: arginine, L-dopa, clonidine, insulin, or glucagon); if two documented historical tests are not available,test(s) must be performed during Screening period

3. Available results from one or more historical CT or MRI scans of the head obtained at or following the diagnosis of GHD

4. Chronologic age at Screening of 3 to 13 years (inclusive) for boys and 3 to 12 years(inclusive) for girls

5. Bone age at Screening of = 11 years for boys and = 10 years for girls

6. Pre-pubertal at Screening (Tanner stage 1 for both breast/genitalia and pubic hair

7. For subjects with idiopathic GHD, a Screening height SDS = -2.0 (standardized for chronologic age and sex) there is no height SDS requirement if the subject has organic GHD (as defined by a CNS lesion or insult on a historical CT or MRI scan)

8. Pre-treatment annualized height velocity = median (50th percentile) for chronologic age and sex (based on values for delayed maturers provided in Appendix 4), utilizing Screening height and height obtained 52 ± 13 weeks (i.e. 39 to 65 weeks) prior to Screening

9. Screening IGF-1 SDS for chronologic age and sex < -1

10. If on thyroid hormone replacement therapy, the dose must be stable for at least 6 weeks prior to Screening and the free thyroxine level (T4), TSH, and cortisol must be within the normal range at the Screening visit

Exclusion Criteria:

1. History of any prior rhGH, rhIGF-1, or sex steroid treatment

2. History of treatment with any medications that may affect growth

3. Evidence of active intracranial neoplasm per recent serial CT or MRI scans of the head or other criteria

4. Surgery/chemotherapy/radiation therapy for intracranial neoplasm within the prior 52 weeks

5. Any history of non-intracranial neoplasm

6. History of or active benign intracranial hypertension

7. High-dose chronic systemic corticosteroid treatment (oral or injected) within prior 13 weeks

8. Acute or severe illness within prior 26 weeks

9. History of diabetes mellitus, anorexia nervosa, cystic fibrosis, chronic severe kidney or liver disease, chronic infectious disease, inborn errors of metabolism, chromosomal disorders, intrauterine growth retardation, or other childhood disease associated with growth failure

10. History of congenital syndromes associated with abnormal growth, including Turner syndrome, Noonan syndrome, Prader-Willi syndrome, etc.

11. History of severe associated pathology affecting growth, including malnutrition,malabsorption, or bone dysplasia

12. History of autoimmune disease

13. Serum ALT or AST = 1.5X ULN

14. Participation in another clinical trial or treatment with any investigational agent (drug or biologic) within 30 days prior to Baseline if the half-life of the agent is known to be = 6 days or within 6 weeks prior to Baseline if the half-life is > 6 days or not known

15. History of any allergic or abnormal reaction to any of the components of the study drugs

16. Any previous or ongoing clinically significant illness, PE findings, or laboratory abnormality that, in the opinion of the Investigator or the Medical Monitor, could prevent the subject from completing the protocol-specified requirements successfully

17. Poor likelihood, in the Investigator's opinion, that the subject will comply with protocol requirements (e.g., uncooperative attitude, inability to return for follow-up visits, history of medical noncompliance) and/or poor likelihood of completing the study

Study Design

Allocation: Randomized, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency

Intervention

Drug:
• Somatropin
ALTU-238 0.3 mg/kg daily
• Somatropin
ALTU-238 0.6 mg/kg daily
• Somatropin
ALTU-238 0.9 mg/kg daily
• Somatropin
Nutropin AQ 0.043 mg/kg daily

Locations

Country	Name	City	State
United States	Children's Hospital Medical Centre	Cincinnati	Ohio
United States	Cook Children's Hospital	Ft. Worth	Texas
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Morristown Memorial Hospital	Morristown	New Jersey
United States	Schneider Children's Hospital	New Hyde Park	New York
United States	Nemours Children's Clinic	Orlando	Florida
United States	Seattle Children's Hospital	Seattle	Washington
United States	Swedish Medical Center	Seattle	Washington
United States	Baystate Medical Centre	Springfield	Massachusetts
United States	UMass Memorial Medical Center	Worcester	Massachusetts

Sponsors (1)

Lead Sponsor	Collaborator
Altus Pharmaceuticals

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Mean change in annualized height velocity from pre-treatment to the first 26 weeks of treatment		26 Weeks	No